Fully Magnetized HeartMate 3 LVAD Eliminates Pump Clots

MedicalResearch.com Interview with:
Mandeep R. Mehra, MD, FACC, FESC, FHFSA, FRCP Medical Director, Brigham and Women’s Hospital Heart and Vascular Center Executive Director, Center for Advanced Heart Disease Professor of Medicine, Harvard Medical School Editor in Chief, The Journal of Heart and Lung Transplantation Brigham and Women's Hospital Boston, MA Mandeep R. Mehra, MD, FACC, FESC, FHFSA, FRCP
Medical Director, Brigham and Women’s Hospital Heart and Vascular Center
Executive Director, Center for Advanced Heart Disease
Professor of Medicine, Harvard Medical School
Editor in Chief, The Journal of Heart and Lung Transplantation
Brigham and Women’s Hospital
Boston, MA


MedicalResearch.com: What is the background for this study?

Response: 10% of patients with heart failure and a reduced ejection fraction transition into Advanced Stages of disease where they become unresponsive to life prolonging traditional medications. Such patients typically require intravenous inotropic therapy to preserve cardiac function but most remain profoundly limited in their quality of life. In such cases a heart transplant is desirable but this is an option for only a few patients.

Left Ventricular Assist Devices (LVADs) have become the mainstay for treating such patients either while they await a transplant or as a permanent option. However, there are challenges leading to infections, strokes, bleeding and most importantly pump malfunction due to thrombosis of the LVAD itself. The HeartMate 3 LVAD is a centrifugal pump that is designed to overcome the problem of pump thrombosis by virtue of 3 engineering attributes:

(a) A frictionless rotor that is based on a fully magnetically levitated platform
(b) wide blood flow passages that reduce red cell destruction and
(c) an artificial intrinsic pulse that prevents stasis of blood within the pump.

Continue reading

Phase1 Trial Supports PCSK9-Inhibitor Inclisiran For Reducing LDL Cholesterol

MedicalResearch.com Interview with:

Kevin Fitzgerald, Ph.D. Alnylam Pharmaceuticals Cambridge, MA 02142

Dr. Kevin Fitzgerald

Kevin Fitzgerald, Ph.D.
Alnylam Pharmaceuticals
Cambridge, MA 02142

MedicalResearch.com: What is the background for this study?

Response: Inclisiran (ALN-PCSsc) is a subcutaneously administered RNAi therapeutic targeting PCSK9 in development for the treatment of hypercholesterolemia. The Phase 1 trial of inclisiran was conducted in the U.K. as a randomized, single-blind, placebo controlled, single ascending-and multi-dose, subcutaneous dose-escalation study in 69 volunteer subjects with elevated baseline LDL-C (≥ 100 mg/dL). The primary objective of the study was to evaluate the safety, side effect profile, and pharmacodynamics effects of inclisiran.

Continue reading

Patient-Reported Outcomes after Monitoring, Surgery, or Radiotherapy for Localized Prostate Cancer

MedicalResearch.com Interview with:

Professor Jenny Donovan OBE FMedSci NIHR-SI AcSS FFPHM Director, NIHR CLAHRC West (National Institute for Health Research Collaboration for Leadership in Applied Health Research and Care West) at University Hospitals Bristol NHS Trust Lewins Mead, Bristol Professor of Social Medicine School of Social and Community Medicine University of Bristol

Prof. Jenny Donovan

Professor Jenny Donovan
OBE FMedSci NIHR-SI AcSS FFPHM
Director, NIHR CLAHRC West
(National Institute for Health Research Collaboration for
Leadership in Applied Health Research and Care West)
at University Hospitals Bristol NHS Trust
Lewins Mead, Bristol
Professor of Social Medicine
School of Social and Community Medicine
University of Bristol 

MedicalResearch.com: What is the background for this study? What are the main findings?

Response: PSA testing identifies many men with prostate cancer, but they do not all benefit from treatment. Surgery, radiation therapy and various programs of active monitoring/surveillance can be given as treatments for fit men with clinically localized prostate cancer. Previous studies have not compared the most commonly used treatments in terms of mortality, disease progression and patient-reported outcomes. In the ProtecT study, we used a comprehensive set of validated measures, completed by the men at baseline (before diagnosis), at six and 12 months and then annually for six years.

The main finding is that each treatment has a particular pattern of side-effects and recovery which needs to be balanced against the findings from the paper reporting the clinical outcomes (Hamdy et al).

Continue reading

Detailed Map of Malaria Mortality Offers Hope of Better Prevention and Control

MedicalResearch.com Interview with:

Simon I. Hay, BSc, DPhil, DSc Professor of Global Health University of Washington Director of Geospatial Science Institute for Health Metrics and Evaluation (IHME).

Prof. Simon Hay

Simon I. Hay, BSc, DPhil, DSc
Professor of Global Health
University of Washington
Director of Geospatial Science
Institute for Health Metrics and Evaluation (IHME).

MedicalResearch.com: Why did you undertake this study?

Response: As malaria control has not been routinely informed by subnational variation of malaria burden, we undertook the study to highlight the potential for high-resolution maps of disease burden to better understand the epidemiology of malaria as well as the contribution of recent control efforts as well as to better inform future malaria control efforts.

Continue reading

Romosozumab Has Potential To Reduce New Vertebral Fractures at 12 Months

MedicalResearch.com Interview with:

MedicalResearch.com Interview with: Felicia Cosman, M.D.

Dr. Felicia Cosman

Felicia Cosman, M.D.
Medical Director of the Clinical Research Center
Helen Hayes Hospital
Professor of Medicine
Columbia University College of Physician and Surgeons
New York
Editor-in-Chief, Osteoporosis International

MedicalResearch.com: What is the background for this study? What are the main findings?

Response: Amgen and UCB presented detailed data from the Phase 3 FRAME study in an oral session at ASBMR 2016, and the data were also published in the New England Journal of Medicine. Additionally, the FRAME abstract has been awarded the 2016 ASBMR Most Outstanding Clinical Abstract Award. The FRAME data show significant reductions in both new vertebral and clinical fractures in postmenopausal women with osteoporosis.

Patients receiving a monthly subcutaneous 210 mg dose of romosozumab experienced a statistically significant 73 percent reduction in the relative risk of a vertebral (spine) fracture through 12 months, the co-primary endpoint, compared to those receiving placebo (fracture incidence 0.5 percent vs. 1.8 percent, respectively [p<0.001]). By six months, new vertebral fractures occurred in 14 romosozumab and 26 placebo patients; between six to 12 months, fractures occurred in two versus 33 additional patients in each group, respectively.

Patients receiving romosozumab experienced a statistically significant 36 percent reduction in the relative risk of a clinical fracture, a secondary endpoint, through 12 months compared to those receiving placebo (fracture incidence 1.6 percent vs. 2.5 percent, respectively [p=0.008]).

In patients who received romosozumab in year one, fracture risk reduction continued through month 24 after both groups transitioned to denosumab treatment through the second year of the study: there was a statistically significant 75 percent reduction in the risk of vertebral fracture at month 24 (the other co-primary endpoint) in patients who received romosozumab followed by denosumab vs. placebo followed by denosumab (fracture incidence 0.6 percent vs. 2.5 percent, respectively [p<0.001]).

Clinical fractures encompass all symptomatic fractures (both non-vertebral and painful vertebral fractures; all clinical fractures assessed in the FRAME study were symptomatic fragility fractures. A 33 percent reduction in relative risk of clinical fracture was observed through 24 months after patients transitioned from romosozumab to denosumab compared to patients transitioning from placebo to denosumab (nominal p=0.002, adjusted p=0.096).

Continue reading

Cures of Multidrug Resistant Tuberculosis Probably Underestimated Under Current WHO Guidelines

MedicalResearch.com Interview with:
Dr. Heinke Kunst, M.D.
Queen Mary University Hospital, London, United Kingdom

MedicalResearch.com: What is the background for this study? What are the main findings?

Response: Multidrug resistant tuberculosis (MDR-TB) has been on the increase worldwide over the past decade. Many patients who have been identified with MDR-TB live in the European region. Despite treatment with expensive second-line drug regimens, curing MDR TB remains a challenge and cure rates were thought to be very low. As part of the EU Commission funded TB-PANNET project 380 patients with MDR-TB were observed at 23 TB centers in countries of high, intermediate and low TB burden in Europe over a period of 5 years. Observation started from the time of diagnosis and lasted until one year after the end of the treatment.

The TBNET proposed new definitions for “cure” and “failure” of MDR-TB treatment based on the sputum culture status at 6 month after the initiation of therapy and whether patients were free from disease recurrence one year after the end of therapy. The researchers found that the WHO criterion for “cure” could not be applied in the majority of patients, simply because most patients who were being treated successfully were not able to produce sputum after 8 months of therapy. The TB-PANNET study showed much higher cure rates using a new definition of cure and failure of treatment for MDR TB in the European region. (61% cure rates compared to only 31% when using WHO criteria.)

The study also demonstrates that the results for “cure” from MDR-TB correlate very well with the level of drug resistance and the time to culture conversion that means the time when TB bacilli are no longer detectable in sputum. The new definitions are also independent of the total duration of treatment and can be applied to the standard 20 months MDR-TB regimen as well as to the 9-12 months shorter course MDR-TB treatment that was recently proposed by the WHO.

Continue reading

Localized Prostate Cancer: Patient-Reported Outcomes after Monitoring, Surgery, or Radiotherapy

MedicalResearch.com Interview with:

Professor Jenny Donovan  PhD   OBE FMedSci NIHR-SI AcSS FFPHM Director, NIHR CLAHRC West (National Institute for Health Research Collaboration for  Leadership in Applied Health Research and Care West) at University Hospitals Bristol NHS Trust Bristol, UK

Prof. Jenny Donovan

Professor Jenny Donovan  PhD
OBE FMedSci NIHR-SI AcSS FFPHM
Director, NIHR CLAHRC West
(National Institute for Health Research Collaboration for
Leadership in Applied Health Research and Care West)
at University Hospitals Bristol NHS Trust
Bristol, UK

MedicalResearch.com: What is the background for this study? What are the main findings?

Response: PSA testing identifies many men with prostate cancer, but they do not all benefit from treatment. Surgery, radiation therapy and various programs of active monitoring/surveillance can be given as treatments for fit men with clinically localized prostate cancer. Previous studies have not compared the most commonly used treatments in terms of mortality, disease progression and patient-reported outcomes. In the ProtecT study, we used a comprehensive set of validated measures, completed by the men at baseline (before diagnosis), at six and 12 months and then annually for six years.

The main finding is that each treatment has a particular pattern of side-effects and recovery which needs to be balanced against the findings from the paper reporting the clinical outcomes (Hamdy et al).

Continue reading

Cord Blood Transplant in Leukemia With Minimal Residual Disease

MedicalResearch.com Interview with:

Dr. Filippo Milano, MD, PhD Assistant Member, Clinical Research Division Associate Director Cord Blood Transplantation Cord Blood Program Assistant Professor, University of Washington Fred Hutchinson Cancer Research Center

Dr. Filippo Milano

Dr. Filippo Milano, MD, PhD
Assistant Member, Clinical Research Division
Associate Director Cord Blood Transplantation
Cord Blood Program
Assistant Professor, University of Washington
Fred Hutchinson Cancer Research Center

MedicalResearch.com: What is the background for this study?

Response: When first introduced, cord blood (CB) graft was used only as a last resort when no suitable conventional donor could be identified, largely due to the limiting cell doses available in a cord blood graft. A CB graft, however, is attractive due to the increased level of HLA disparity that can be tolerated, without increased risk of graft versus host disease, allowing nearly all patients to find such a donor.

The main intent of the study was to evaluate whether or not, at our Institution, cord blood SHOULD STILL BE considered only AS an alternative DONOR or IF instead outcomes were comparable to those obtained with more “conventional” types of transplants from matched and mismatched unrelated donors.

Continue reading

Real World Trial Show Efficacy of Fluticasone Furoate–Vilanterol for COPD in Clinical Practice

MedicalResearch.com Interview with:
Jørgen Vestbo DMSc FRCP FERS

Professor of  Respiratory Medicine
Division of Infection, Immunity and Respiratory Medicine
University of Manchester
Education and Research Centre
University Hospital of South Manchester
Manchester

MedicalResearch.com: What is the background for this study? What are the main findings?

Response: Efficacy studies are limited in their usefulness to clinicians as there are often restricted inclusion criteria, with many exclusion criteria and patients are followed closely with high adherence to study treatment. They therefore show what the drugs can do but not necessarily what they do do in the real world.

Randomised studies in everyday practice, not limiting the entry (effectiveness trials) are therefore needed.

In our study we showed that it is feasible to do randomised studies in the “real world”.

Our study showed that a simple treatment with a once-daily combination of an inhaled corticosteroid and a long-acting beta-agonist (Breo/Relvar) was superior to the usual care chose by the patients’ general practitioners to manage their COPD.

Continue reading

Long-Acting Beta-Agonists-Steroid Combination in Pediatric Asthma

MedicalResearch.com Interview with:
David A Stempel, MD
Medical Affairs Lead
US Medical Affairs
GlaxoSmithKline

MedicalResearch.com: What is the background for this study? What are the main findings?

Response: Long-acting beta-agonists (LABAs) have been shown to increase the risk of asthma-related death among adults and the risk of asthma-related hospitalization among children.

It is unknown whether the concomitant use of inhaled glucocorticoids
with LABAs mitigates those risks. This trial prospectively evaluated the safety of the LABA salmeterol, added to fluticasone propionate, in a fixed-dose combination in children.

Continue reading

Drug-Eluting Stents Reduce Need for Revascularization after PCI


MedicalResearch.com Interview with:
Dr. Kaare Harald Bønaa

Principal investigator
University of Tromsø, Norway

MedicalResearch.com: What is the background for this study?

Response: The NORSTENT study was designed shortly after the “Barcelona fire storm” in 2006 that raised severe safety concerns against drug-eluting stents (DES). At that time there was evidence for increased risk of stent thrombosis with DES. How this could influence long term results compared to PCI with bare metal stents (MMS) was not known.

Accordingly, we designed the NORSTENT study with the primary composite endpoint of all-cause mortality and non-fatal spontaneous myocardial infarction at a medial of 5 years of follow-up.

Continue reading

Study Finds CPAP Improved Quality of Life But Did Not Reduce Cardiovascular Risk

MedicalResearch.com Interview with:
Prof. Craig Anderson, PhD
Professor of Stroke Medicine and Clinical Neuroscience
Medicine, The George Institute for Global Health
University of Sydney

MedicalResearch.com: What is the background for this study? What are the main findings?

Response: We wished to prove whether treatment of obstructive sleep apnea with continuous positive airway pressure (CPAP ) can modify the risk of cardiovascular disease. The is a lot of association data from epidemiological and clinical studies but no large scale international clinical trials assessing the effects of CPAP on the prevention of serious cardiovascular events like heart attack and stroke.

Our study in nearly 3000 adults with prior heart attack or stroke and moderate to severe obstructive sleep apnea showed that CPAP treatment did not prevent recurrent cardiovascular events or major cardiovascular risk factors. However CPAP did improve wearers’ sense of wellbeing, mood and work productivity.

Continue reading

70-Gene Signature Can Help Identify Early-Stage Breast Cancer Patients Who Do Not Need Chemotherapy

MedicalResearch.com Interview with:

Prof. Laura van ’t Veer, PhD Leader, Breast Oncology Program, and Director, Applied Genomics, UCSF Helen Diller Family Comprehensive Cancer Center Angela and Shu Kai Chan Endowed Chair in Cancer Research UCSF Helen Diller Family Comprehensive Cancer Center

Dr. Laura Van’t Leer

Prof. Laura van ’t Veer, PhD
Leader, Breast Oncology Program, and Director, Applied Genomics, UCSF Helen Diller Family Comprehensive Cancer Center
Angela and Shu Kai Chan Endowed Chair in Cancer Research
UCSF Helen Diller Family Comprehensive Cancer Center

MedicalResearch.com: What is the background for this study? What are the main findings?

Response: MINDACT was designed to involve only patients with node negative and 1 to 3 positive lymph node breast cancer. Node negative breast cancer is a cancer that has not spread to the surrounding lymph nodes and therefore has a lower risk of recurrence. Scientists have also demonstrated that breast cancer which has spread to 1 to 3 lymph nodes may behave like node negative breast cancer. Patients with either node negative cancer or with a cancer that involves 1-3 lymph nodes are often prescribed chemotherapy, although physicians believe that approximately 15% of them do not require such treatment.

MINDACT provides the highest level of evidence to show that using MammaPrint® can substantially reduce the use of chemotherapy in patients with node-negative and 1-to-3 node positive breast cancer – in other words, it can identify patients with these types of breast cancer who can safely be spared a treatment that may cause significant side effects, and will offer no to very little benefit.

Continue reading

Closed-Loop Insulin Delivery During Pregnancy Improves Glucose Control

MedicalResearch.com Interview with:
Professor Helen Murphy
and
Dr Zoe Stewart
Institute of Metabolic Science
University of Cambridge

MedicalResearch.com: What is the background for this study?

Response: Controlling blood glucose levels is a daily challenge for people with Type 1 diabetes and is particularly crucial during pregnancy. Previous research shows that women with type 1 diabetes spend only 12 hours per day within the recommended glucose target levels, leading to increased rates of complications including preterm delivery and large for gestational age infants. National surveys show that one in two babies suffer complications related to type 1 diabetes in the mother.

The hormonal changes that occur in pregnancy make it difficult for women to predict the best insulin doses for every meal and overnight. Too much insulin causes low glucose levels harmful for the mother and too little causes problems for the developing baby. The artificial pancreas automates the insulin delivery giving better glucose control than we can achieve with currently available treatments. Previous studies show that the closed-loop system also known as artificial pancreas can be used safely in children and adults and our study aimed to investigate whether or not it was helpful for women with type1 diabetes during pregnancy.

Continue reading

No Difference In Asthma Exacerbations Between Acetaminophen and Ibuprofen in Young Children

MedicalResearch.com Interview with:

Wanda Phipatanakul, MD, MS Associate Professor of Pediatrics Harvard Medical School Director, Asthma Clinical Research Center Boston Children's Hospital Asthma, Allergy and Immunology Boston, MA 02115

Dr. Wanda Phipatanakul

Wanda Phipatanakul, MD, MS
Associate Professor of Pediatrics
Harvard Medical School
Director, Asthma Clinical Research Center
Boston Children’s Hospital
Asthma, Allergy and Immunology
Boston, MA 02115

MedicalResearch.com: What is the background for this study? What are the main findings?

Response:  Acetaminophen (e.g., Tylenol, Panadol) and ibuprofen (e.g., Advil, Motrin) are the only available treatments for pain and fever in toddlers and the most commonly utilized medications worldwide. Recently there has been controversy and even alarm with suggestive observational data that acetaminophen makes asthma worse. This has led some experts to recommend the avoidance of acetaminophen in children with asthma. We sought to find the answer to this burning question through the first prospective, double-blind, randomized clinical trial comparing acetaminophen versus ibuprofen head to head for use when clinically indicated for fever or pain. Is there a difference in asthma morbidity (exacerbations) in young children between the age of 12-59 months, who have asthma?

Continue reading

POISE Data Support New Medication Ocaliva For Primary Biliary Cholangitis

MedicalResearch.com Interview with:

Prof. Dr. F. Nevens, MD, PhD Professor of Medicine Hepatology and liver transplantation University Hospitals KU Leuven, Belgium

Prof. Dr. F. Nevens, MD, PhD
Professor of Medicine
Hepatology and liver transplantation
University Hospitals KU Leuven, Belgium

MedicalResearch.com: What is the background for this study?

Response: Primary biliary cholangitis (PBC) is a rare, autoimmune cholestatic liver disease that puts patients at risk for life-threatening complications. PBC is primarily a disease of women, affecting approximately one in 1,000 women over the age of 40. If left untreated, survival of PBC patients is significantly worse than the general population.

The POISE trial evaluated the safety and efficacy of once-daily treatment with Ocaliva® (obeticholic acid) in PBC patients with an inadequate therapeutic response to, or who are unable to tolerate, ursodeoxycholic acid (UDCA), the current standard of care. Ocaliva is the first PBC therapy that targets the farnesoid X receptor (FXR), a key regulator of bile acid, inflammatory, fibrotic and metabolic pathways.

The trial’s primary endpoint was an alkaline phosphatase (ALP) level of less than 1.67 times the upper limit of the normal range, with a reduction of at least 15% from baseline, and a total bilirubin level at or below the upper limit of the normal range after 12 months of obeticholic acid therapy. These liver biomarkers have been shown to predict progression to liver failure and resulting liver transplant or premature death in patients with PBC.

Continue reading

Thymectomy A Rational Choice For Some Patients With Myasthenia Gravis

MedicalResearch.com Interview with:

Photograph: Douglas Levere

Dr. Gil Wolfe

Gil I. Wolfe, MD, FAAN
Irvin and Rosemary Smith Professor and Chair
Dept. of Neurology/Jacobs Neurological Institute
Univ. at Buffalo Jacobs School of Medicine and Biomedical Sciences/SUNY
Buffalo General Medical Center
Buffalo, NY 14203-1126

MedicalResearch.com: What is the background for this study? What are the main findings?

Response: Thymectomy has been used in myasthenia gravis (MG), in particular those patients who do not have a tumor of the thymus gland, known as a thymoma, for over 75 years without randomized data to support its use. A practice parameter in 2000 on behalf of the American Academy of Neurology stated that the benefits of thymectomy in this population of non-thymomatou smyasthenia gravis patients remained uncertain, classified thymectomy as a treatment option in this group, and called for rigorous, randomized studies.

What we found is that compared to an identical prednisone protocol alone, that extended transsternal thymectomy confers significant benefits to non-thymomatous MG patients over a period of three years after the procedure. The benefits include better disease status, reduced prednisone requirements, fewer hospitalizations to manage  myasthenia gravis worsenings, and a lower symptom profile related to side effects from medications used to control the disease state.

Continue reading

Sickle Cell Trait Not Associated With Increased Mortality in Military Population

MedicalResearch.com Interview with:
D. Alan Nelson, MPAS, PhD
Postdoctoral research fellow
Stanford Medicine

MedicalResearch.com: What is the background for this study?

Response: The study was inspired by the uncertainty surrounding sickle cell trait (SCT) and its association with serious exertional collapse events and mortality in active populations. I conducted initial, exploratory analyses on these topics in 2014-15 while examining a range of military readiness predictors and outcomes. The early work indicated that the risk of mortality, rhabdomyolysis and other exertional events arising from SCT might be substantially lower than that suggested by prior work in the research literature.

Dr. Lianne Kurina and I decided to conduct further, focused study at the Stanford University School of Medicine to confirm or refute these findings. In considering best approaches, we noted that there was an absence of prior research in which the  sickle cell trait status of an entire, large, physically-active study population was known. This limitation could introduce bias to inflate the apparent impact of a theorized predictive factor.

Aside from the challenges in studying the impact of SCT on exertional outcomes, with respect to prevention, a further concern is that  sickle cell trait is a non-modifiable trait. If it were a serious risk factor for rhabdomyolysis and/or mortality, despite careful exertional injury precautions such as those employed by the Army, this might present great challenges for prevention efforts. To maximize the potential for new research to provide actionable prevention information, our interests included examining a range of modifiable risk factors for rhabdomyolysis.

Dr. Kurina and I have employed large, longitudinal military datasets for about five years to examine critical military health outcomes, making this study a natural progression of our joint work. The research proceeded with the support of the Uniformed Services University of the Health Sciences, and in cooperation with a distinguished group of experts who co-authored the paper and advised the project. The study was conducted using de-identified records of all SCT-tested African American US Army soldiers on active duty during 2011 – 2014 (N = 47,944).

Continue reading

Innate Immune Activation Protects Amish Children From Asthma

MedicalResearch.com Interview with:

Donata Vercelli, MD Professor of Cellular and Molecular Medicine, University of Arizona Director, Arizona Center for the Biology of Complex Diseases Associate Director, Asthma and Airway Disease Research Center The BIO5 Institute, Rm. 339 Tucson, AZ 85721

Dr. Donata Vercelli

Donata Vercelli, MD
Professor of Cellular and Molecular Medicine, University of Arizona
Director, Arizona Center for the Biology of Complex Diseases
Associate Director, Asthma and Airway Disease Research Center
The BIO5 Institute
Tucson, AZ 85721

MedicalResearch.com: What is the background for this study?

Response: By probing the differences between two farming communities—the Amish of Indiana and the Hutterites of South Dakota—our interdisciplinary team (which included, among others, Erika von Mutius from Ludwig-Maximilians University in Munich, Carole Ober and Anne Sperling from the University of Chicago, and myself) found that substances in the house dust from Amish, but not Hutterite, homes shape the innate immune system in ways that may prevent the development of allergic asthma.

Growing up in a microbe-rich farm environment has been known to protect against asthma. Our current study extends these findings by showing that in both humans and mice protection requires engagement of the innate immune system.

The Amish and Hutterite farming communities in the United States, founded by immigrants from Central Europe in the 18th and 19th centuries, provide textbook opportunities for comparative studies. The Amish and the Hutterites have similar genetic ancestry and share lifestyles (e.g., family size, diet, lack of exposure to indoor pets) known to affect asthma risk. However, their farming practices differ. The Amish have retained traditional methods, live on single-family dairy farms and rely on horses for fieldwork and transportation. In contrast, the Hutterites live on large communal farms and use modern, industrialized farm machinery. This distances young Hutterite children from the constant daily exposure to farm animals.

Continue reading

Hormone Overcomes Genetic Cause of Morbid Obesity

MedicalResearch.com Interview with:
Dr. Peter Kühnen MD
Institute for Experimental Pediatric Endocrinology
Charité–Universitätsmedizin Berlin
Berlin, Germany

MedicalResearch.com: What is the background for this study? What are the main findings?

Dr. Kühnen: The patients, which were included in this study, are suffering from a genetic defect in a gene called POMC. This gene is cleaved into different hormones as e.g. MSH (melanocyte stimulating hormone). MSH is very important for the regulation of satiety by activation of the MC-4 receptor. For this reason these patients are persistent hyperphagic due to the lack of MSH and they gain weight very fast in the first months of their life.
Setmelanotide activates the MC-4 receptor, which is important for the activation of satiety. By restoring the lost function Setmelanotide leads to a reduction of hyperphagia and to a reduction of body weight in this POMC deficient patients.

Continue reading

Meningococcal B Vaccine Tested During University Outbreak of Meningitis

MedicalResearch.com Interview with:

Nicole E. Basta, PhD MPhil Assistant Professor Division of Epidemiology and Community Health School of Public Health University of Minnesota

Dr. Nicole Basta

Nicole E. Basta, PhD MPhil
Assistant Professor
Division of Epidemiology and Community Health
School of Public Health
University of Minnesota

MedicalResearch.com: What is the background for this study?

Response: Meningococcal disease is a serious and often life-threatening condition.

In the past several years, multiple outbreaks caused by meningococcal serogroup B (MenB) have occurred on college campuses in the US. Recently, a new meningococcal B vaccine known as 4CMenB or Bexsero was developed. The FDA granted special approval to use the vaccine to control an outbreak at a University in New Jersey prior to its licensure.

We took advantage of this unique opportunity to investigate the impact of Bexsero during the outbreak. In doing so, we conducted the first clinical study of Bexsero among teens and young adults in the US.

Continue reading

NEJM Study Calculates Cost Of Training Residents in Teaching Health Centers

MedicalResearch.com Interview with:

Marsha Regenstein, Ph.D, Professor From the Department of Health Policy and Management Milken Institute School of Public Health George Washington University Washington, DC

Prof. Marsha Regenstein

Marsha Regenstein, Ph.D, Professor
From the Department of Health Policy and Management
Milken Institute School of Public Health
George Washington University
Washington, DC

MedicalResearch.com: What is the background for this study?

Response: Despite the billions of dollars in public spending on graduate medical education (GME) in the United States, little is known about the true cost of training a resident, with the few studies that exist showing wide variation in their methods and results. At the same time, the U.S. appears to be producing too few primary care physicians to meet the health care needs of the population, and especially those who live in underserved areas with high health care needs and shortages of health professionals. The Teaching Health Center (THC) Graduate Medical Education funding program was established under the Affordable Care Act to increase the number of medical and dental residents training in six primary care specialties in underserved areas. The Teaching Health Center funding supports community-based residency training in settings such as Federally-qualified health centers, rural clinics, mental health clinics and other non-profit community-based organizations. Hospitals commonly serve as training partners, but THC funding goes directly to the community-based partner, bringing funding and training closer to the communities where underserved patients live. The Health Resources and Services Administration (HRSA), which manages and funds the program, set an interim payment of $150,000 per resident; currently, 59 THCs are training 690 residents in 27 states and the District of Columbia. The interim payment rate was based on the best available information at the time and was meant to cover the full cost of training a resident.

Continue reading

Multikinase Inhibitor Midostaurin Improved Symptoms and Survival in Most Advanced Forms of Blood Cancer Mastocytosis

MedicalResearch.com Interview with:

Jason R. Gotlib, MD The Clinical Investigator Pathway Hematology Division at Stanford University Medical Cent

Dr. Jason R. Gotlib

Jason R. Gotlib, MD
The Clinical Investigator Pathway
Hematology Division
Stanford University Medical Center

MedicalResearch.com: What is the background for this study? What are the main findings?

Response: The background is that advanced forms of systemic mastocytosis, which are blood cancers characterized by accumulation of abnormal mast cells in the bone marrow and additional organs, represent a group of orphan diseases with a large unmet need. Approximately 90% of patients harbor the acquired KIT D816V mutation, a mutated receptor tyrosine kinase on the surface of mast cells which a primary driver of disease pathogenesis. Only 1 drug is approved for patients with one form of advanced systemic mastocytosis, termed ‘aggressive systemic mastocytosis, or ‘ASM’. This therapy is imatinib (Gleevec), but it is only approved for patients without the KIT D816V mutation, or with KIT mutation status unknown because the KIT D816V mutation is resistant to imatinib. Therefore, this drug may only be useful for approximately 10% of patients. Other drugs that have been used off-label for systemic mastocytosis (but are not approved for this indication) include interferon-alpha or cladribine, which show some activity, but their evaluation to date has been primarily limited to small case series which are usually retrospective in nature, and include mixed populations of systemic mastocytosis patients who have both early stage disease without organ damage (e.g. indolent systemic mastocytosis) and and advanced stage patients, as included in this trial, who have one or more findings of organ damage. Also, those trials employed differing response criteria and no central adjudication of eligibility and response assessments was undertaken.

Midostaurin is a multikinase inhibitor with activity against both wild-type KIT, but most importantly, KIT D816V (in contrast to imatinib). Prior work demonstrated that cell lines transformed with the KIT D816V mutation can be inhibited at relatively low concentrations of midostaurin. These concentrations could also be achieved in vivo (e.g. at concentrations achievable in the blood of patients). Cell lines transformed by KIT D816V could not be inhibited by imatinib.

Continue reading

This Generation of Malaria Vaccine Not Perfect But Can Still Save Lives

MedicalResearch.com Interview with:

Philip Bejon, Ph.D. Professor of Tropical Medicine, Director of the Wellcome-KEMRI-Oxford Collaborative Research Programme, Group Head / PI, Consultant Physician and Unit Director Kilifi, Kenya

Dr. Philip Bejon

Philip Bejon, Ph.D.
Professor of Tropical Medicine, Director of the Wellcome-KEMRI-Oxford Collaborative Research Programme, Group Head / PI, Consultant Physician and Unit Director
Kilifi, Kenya

MedicalResearch.com: What is the background for this study?

Response: According to the latest World Health Organisation (WHO) estimates more than 400,000 people died from malaria in 2015, with over 90% of these deaths occurring in sub-Saharan Africa. The vast majority who die are children under 5, and almost all cases are caused by the P. falciparum strain of malaria transmitted by female Anopheles mosquitoes.

RTS,S, which protects only against P. falciparum, was developed by GlaxoSmithKline with support from the PATH Malaria Vaccine Initiative (MVI) and with grant funds from the Bill & Melinda Gates Foundation to MVI. In July 2015, it received a positive opinion from the European Medicines Agency.

Earlier this year, the WHO recommended further evaluation of the four-dose regimen of RTS,S in a pilot implementation programme in sub-Saharan Africa, to address several knowledge gaps before the vaccine might be rolled out more widely.

Continue reading

Requests for Abortions in South American Rise Dramatically Since Zika, Especially in Brazil

MedicalResearch.com Interview with:

Abigail R.A. Aiken, MD, MPH, PhD Assistant Professor LBJ School of Public Affairs University of Texas at Austin Austin, TX, 78713

Dr. Abigail Aiken

Abigail R.A. Aiken, MD, MPH, PhD
Assistant Professor
LBJ School of Public Affairs
University of Texas at Austin
Austin, TX, 78713

MedicalResearch.com: What is the background for this study?

Response: As Zika began to emerge as an epidemic in Latin America and its links with microcephaly began to be realized, we were aware that women in the region who were already pregnant or who would become pregnant would have a very limited set of reproductive options. Research and media attention about the possible biological effects of Zika in pregnancy began to appear rapidly. But much less attention was been paid to the impacts of Zika on women. We followed the responses of governments and health organizations and when they began to issue advisories warning women to avoid pregnancy, we knew it would be important to investigate the impacts of those advisories. A country-wide policy that is impossible to follow if you are pregnant or cannot avoid pregnancy is an unusual and important public issue. Accurate data on abortion are very difficult to obtain in Latin America because in most countries, abortion is highly restricted. We wanted to provide a window on the issue of how women were responding to the risks of Zika and its associated advisories, so we worked with Women on Web (WoW), an online non-profit telemedicine initiative that provides safe medical abortion to women in countries where safe, legal abortion is not universally available.

Continue reading

Opioid-Restricting Laws Have Not Reduced Overdoses in Disabled Workers

MedicalResearch.com Interview with:

Professor Ellen Meara, PhD Professor The Dartmouth Institute for Health Policy and Clinical Practice

Prof. Ellen Meara

Professor Ellen Meara, PhD
Professor
The Dartmouth Institute for Health Policy and Clinical Practice

MedicalResearch.com: What is the background for this study?

Response: Responding to a fourfold rise in death rates, between 2006 and 2012, states collectively enacted 81 laws restricting prescribing and dispensing of prescription opioids. Jill Horwitz, PhD, JD, said “states hoped passing a range of laws might help. So they are enacting small fixes — forbidding patients from “doctor-shopping,” and requiring doctors to use tamper-resistant prescription forms. They are also implementing major efforts such as prescription drug monitoring programs (PDMPs) — online databases that allow law enforcement and clinicians to monitor prescriptions.”

Continue reading

PET-CT Scan Can Guide Treatment in Advanced Hodgkin’s Lymphoma

MedicalResearch.com Interview with:

Peter Johnson MA, MD, FRCP Professor of Medical Oncology Cancer Research UK Centre Southampton General Hospital Southampton

Prof. Peter Johnson

Peter Johnson MA, MD, FRCP
Professor of Medical Oncology
Cancer Research UK Centre
Southampton General Hospital
Southampton

MedicalResearch.com: What is the background for this study? What are the main findings?

Prof. Johnson: Based upon retrospective series looking at the ability of interim PET to predict the outcomes of treatment, we aimed to test the idea of modulating treatment in response to an early assessment of the response to ABVD: could we safely reduce the amount of treatment by omitting bleomycin in the group who had responded well? Although the risk of severe toxicity from bleomycin is generally low, for the small number of patients who experience it, it can be life-changing or even fatal. We also wanted to test whether it might be possible to reduce the use of consolidation radiotherapy by comparison to our previous trials, and this seems to have worked too: we used radiotherapy in less than 10% of patients in RATHL, as compared to around half in our previous trials. We have seen better survival figures than in our previous studies with less treatment overall, so it feels as though we are on the right track.

Continue reading

Over Half Sudden Cardiac Deaths in Young are Male; Over 25% Have Identifiable Genetic Cause

MedicalResearch.com Interview with:

Professor Chris Semsarian MBBS PhD MPH FRACP FAHMS FAHA FHRS FCSANZ Professor of Medicine, University of Sydney Cardiologist, Royal Prince Alfred Hospital NHMRC Practitioner Fellow Head, Molecular Cardiology Program Centenary Institute, Newtown NSW Australia

Prof. Chris Semsarian

Professor Chris Semsarian
MBBS PhD MPH FRACP FAHMS FAHA FHRS FCSANZ
Professor of Medicine, University of Sydney
Cardiologist, Royal Prince Alfred Hospital
NHMRC Practitioner Fellow
Head, Molecular Cardiology Program
Centenary Institute,
Newtown NSW Australia

MedicalResearch.com: What is the background for this study?

Response: Sudden cardiac death is a tragic and devastating event at all ages, and especially in the young (aged under 35 years). Understanding the causes and circumstances of SCD in the young is critical if we are to develop strategies to prevent SCD in the young. Our study represents the first prospective, population-based study of SCD in the young across two nations, Australia and New Zealand.

Continue reading

No Clinical Benefit With Intensive Blood-Pressure Lowering in Acute Cerebral Hemorrhage

MedicalResearch.com Interview with:
Adnan I. Qureshi, M.D
Zeenat Qureshi Stroke Research Center
University of Minnesota
Minneapolis, MN

MedicalResearch.com: What is the background for this study? What are the main findings?

Dr. Qureshi: An acute hypertensive response in patients with intracerebral hemorrhage is common and may be associated with hematoma expansion and increased mortality. The Antihypertensive Treatment of Acute Cerebral Hemorrhage II (ATACH-2) trial was designed to determine the efficacy of rapidly lowering systolic blood pressure in patients in an earlier time window after symptom onset than evaluated in previous trials. The trial was based on evidence that hematoma expansion and subsequent death or disability might be reduced with very early and more aggressive reduction in systolic blood pressure in those at higher risk due to presence of high systolic blood pressure at presentation. The trial randomized eligible subjects with intracerebral hemorrhage to test the superiority of intensive (goal 110-140 mmHg) over standard (goal 140-180 mmHg) systolic blood pressure reduction using intravenous nicardipine within 4.5 hours of symptom onset. Of a total of 1000 subjects that were recruited with a mean (standard deviation) baseline systolic blood pressure of 200.6 (27.0) mmHg, 500 were assigned to intensive-treatment and 500 to standard-treatment. Enrollment was stopped following a pre-specified interim analysis because of futility.

The primary endpoint of death or disability at 3 months post-randomization was observed in 38.7% (186/481) of subjects receiving intensive treatment and 37.7% (181/480) subjects receiving standard treatment (relative risk: 1.03; 95% confidence interval: 0.85 to 1.27), adjusted for age, initial Glasgow Coma scale, and presence or absence of intraventricular hemorrhage. The rate of renal adverse events within 7 days of randomization was significantly higher among subjects randomized to intensive treatment. Compared to a target systolic blood pressure of 140-180 mmHg, treating subjects with intracerebral hemorrhage to a target systolic blood pressure of 110-140 mmHg did not lower the rate of death or disability.

Continue reading

Chewing Gum Test Unmasks Jaw Claudication of Giant Cell Arteritis

MedicalResearch.com Interview with:
Chih-Hung Kuo, M.B., B.S.
Peter McCluskey, M.D.
Clare L. Fraser, M.B., B.S.
University of Sydney
Sydney, NSW, Australia

MedicalResearch.com: What is the background for this study? What are the main findings?

Response: Giant cell arteritis is a life and sight threatening systemic inflammatory condition, which remains difficult to diagnose. Jaw claudication (cramping of muscle from ischemia) is a highly specific symptom with significant diagnostic and prognostic (risk of permanent blindness) values. The reporting of jaw symptoms may be affected by many factors, such as diet. There remains no standardized clinical test available for clinicians. We study the use of chewing gum as a standardized test (like a stress test for angina pain) to better characterize this critical symptom.

The pilot study of two cases with abnormal results were published in the New England Journal of Medicine. Chewing gum at a rate of 1 chew/second can reproduce the jaw claudication symptom around 2-3 minutes. In one case, the jaw claudication was unmasked by the test with a subsequent positive biopsy result. The test result became negative after corticosteroid treatment.

Continue reading

National Program Reduces Urinary Tract Infections in Hospitalized Patients

Sanjay Saint, MD, MPH Chief of Medicine, VA Ann Arbor Healthcare System George Dock Professor of Internal Medicine & Senior Associate Chair - Department of Internal Medicine University of Michigan Medical School

Dr. Sanjay Saint

MedicalResearch.com Interview with:
Sanjay Saint, MD, MPH
Chief of Medicine
VA Ann Arbor Healthcare System
George Dock Professor of Internal Medicine & Senior Associate Chair –
Department of Internal Medicine
University of Michigan Medical School

MedicalResearch.com: What is the background for this study? What are the main findings?

Dr. Saint: Catheter-associated urinary tract infection (CAUTI) is a common, costly, and morbid complication of hospitalization. Urinary tract infection (UTI) is one of the most common device-related infections in the United States. CAUTI rates rose nationally between 2009 and 2013.

We put in place a national program to reduce CAUTI. Specifically, we enrolled 926 intensive care unit (ICU) and non-ICU hospital units in 603 hospitals spread over 32 states, the District of Columbia and Puerto Rico between March 2011 and November 2013.

By the end of the 18-month program, UTI rates among hospital patients in general wards had dropped by a third. Specifically:
• The rate of CAUTIs dropped from 2.40 per 1000 days of catheter use to 2.05 (a ~14 percent overall drop).
• Nearly all of the decrease in CAUTI rates was due to changes in infection rates in non-ICUs, which went from 2.28 to 1.54 infections per 1,000 catheter-days – a drop of 32 percent. In non-ICUs, the overall use of catheters decreased by 7%.
• ICUs didn’t see a substantial change in either CAUTI or catheter use, likely because the nature of patients treated in ICUs means more frequent urine output monitoring and culturing of urine, so UTIs are more likely to be spotted.

Continue reading

Genome Sequencing Identifies Some Forms of Potentially Treatable Intellectual Disabilities

MedicalResearch.com Interview with:

Dr. Clara van Karnebeek PhD Certified Pediatrician and Biochemical Geneticist at the BC Children’s Hospital Principal Investigator, University of British Columbia

Dr. Clara van Karnebeek

Dr. Clara van Karnebeek PhD
Certified Pediatrician and Biochemical Geneticist at the BC Children’s Hospital
Principal Investigator, University of British Columbia

MedicalResearch.com: What is the background for this study?

Dr. van Karnebeek: The goal of the study was to diagnose patients with genetic conditions and discover and describe new diseases with potential for treatment. The study included patients with neurodevelopmental conditions that doctors suspected were genetic or metabolic in origin but had not been diagnosed using conventional methods. Our team tested the children and their parents using a combination of metabolomic (large scale chemical) analysis and a type of genomic sequencing called whole exome sequencing. With this state-of-the-art technique, experts analyze and interpret the portion of DNA called genes that hold the codes for proteins.

Some people’s intellectual disability is due to rare genetic conditions that interfere with the processes the body uses to break down food. Because of these metabolic dysfunctions, there is an energy deficit and build-up of toxic substances in the brain and body leading to symptoms such as developmental and cognitive delays, epilepsy, and organ dysfunction. Some of these rare diseases respond to treatments targeting the metabolic dysfunction at the cellular level and range from simple interventions like dietary modifications, vitamin supplements and medications to more invasive procedures like bone marrow transplants. Because the right treatment can improve cognitive functioning or slow or stop irreversible brain damage, early intervention can improve lifelong outcomes for affected children and their families.

Continue reading

Many Caregivers Report High Levels of Depression and Loss of Control Over Their Lives

MedicalResearch.com Interview with:

MedicalResearch.com Interview with: Jill Cameron, PhD Canadian Institutes of Health Research New Investigator Associate Professor, Department of Occupational Science and Occupational Therapy Rehabilitation Sciences Institute Faculty of Medicine, University of Toronto     MedicalResearch.com: What is the background for this study? What are the main findings?  Dr. Cameron: In the world of critical illness, a lot of research has focused on helping people to survive – and now that more people are surviving, we need to ask ourselves, what does quality of life and wellbeing look like afterwards for both patients and caregivers? The aim of our research was to identify factors associated with family caregiver health and wellbeing during the first year after patients were discharged from the Intensive Care Unit. We examined factors related to the patient and their functional wellbeing, the caregiving situation including the impact it has on caregivers everyday lives, and caregiver including their sense of control over their lives and available social support. We used Pearlin’s Caregiving Stress Process model to guide this research.   From 2007-2014, caregivers of patients who received seven or more days of mechanical ventilation in an ICU across 10 Canadian university-affiliated hospitals were given self-administered questionnaires to assess caregiver and patient characteristics, caregiver depression symptoms, psychological wellbeing, and health-related quality of life. Assessments occurred seven days and three, six and 12-months after ICU discharge.  The study found that most caregivers reported high levels of depression symptoms, which commonly persisted up to one year and did not improve in some. Caregiver sense of control, impact on caregivers’ everyday lives, and social support had the largest relationships with the outcomes. Caregivers’ experienced better health outcomes when they were older, caring for a spouse, had higher income, better social support, sense of control, and caregiving had less of a negative impact on their everyday lives. No patient characteristics or indicators of illness severity were associated with caregiver outcomes.   Poor caregiver outcomes may compromise patients’ rehabilitation potential and sustainability of home care. Identifying risk factors for caregiver distress is an important first step to prevent more suffering and allow ICU survivors and caregivers to regain active and fulfilling lives.  MedicalResearch.com: What should readers take away from your report?  Dr. Cameron: Our findings suggest that family caregiver health and wellbeing outcomes are more closely related to characteristics of the caregiver and caregiving situation than patient characteristics including functional abilities and neuropsychological wellbeing. This suggests that when determining which caregivers are in need of support, we can't base this decision on the level of sickness of the patient. We need to screen the caregivers themselves to identify those in need of care and support. Our findings suggest caregivers with low levels of social support, poor sense of control over their situation, and whose caregiving is more likely to impact their everyday lives are more likely to experience poor outcomes and are in need of support from the health care system.  MedicalResearch.com: What recommendations do you have for future research as a result of this study?  Dr. Cameron: Future research should continue to be theoretically driven and follow caregivers longitudinally. Qualitative research involving in depth interviews will enhance our understanding of ways to assist, support, and care for family caregivers across the illness trajectory. Interventions and models of care that target those caregivers in need of support should be developed and tested and ultimately implemented into the health care system.   MedicalResearch.com: Is there anything else you would like to add?  Dr. Cameron: Ultimately, adopting a family centered model of care has the potential to improve the health and wellbeing of family caregivers and their care recipients. One example of adopting this approach concerns the transition of the patient back home. Patients and their caregivers should be assessed for their readiness to go home and provided with education and training to optimize this transition. Once home, families should continue to be monitored and provided with additional supports as needed as they adjust to life in the community. A family centered approach can be incorporated across the care continuum to optimize caregiver and patient outcomes.   Citation:  One-Year Outcomes in Caregivers of Critically Ill Patients Jill I. Cameron, Ph.D., Leslie M. Chu, B.Sc., Andrea Matte, B.Sc., George Tomlinson, Ph.D., Linda Chan, B.A.Sc., Claire Thomas, R.N., Jan O. Friedrich, M.D., D.Phil., Sangeeta Mehta, M.D., Francois Lamontagne, M.D., Melanie Levasseur, M.D., Niall D. Ferguson, M.D., Neill K.J. Adhikari, M.D., Jill C. Rudkowski, M.D., Hilary Meggison, M.D., Yoanna Skrobik, M.D., John Flannery, M.D., Mark Bayley, M.D., Jane Batt, M.D., Claudia dos Santos, M.D., Susan E. Abbey, M.D., Adrienne Tan, M.D., Vincent Lo, P.T., B.Sc., Sunita Mathur, P.T., Ph.D., Matteo Parotto, M.D., Denise Morris, R.N., Linda Flockhart, R.N., Eddy Fan, M.D., Ph.D., Christie M. Lee, M.D., M. Elizabeth Wilcox, M.D., Najib Ayas, M.D., Karen Choong, M.D., Robert Fowler, M.D., Damon C. Scales, M.D., Tasnim Sinuff, M.D., Brian H. Cuthbertson, M.D., Louise Rose, R.N., Ph.D., Priscila Robles, P.T., Ph.D., Stacey Burns, R.N., Marcelo Cypel, M.D., Lianne Singer, M.D., Cecilia Chaparro, M.D., Chung-Wai Chow, M.D., Shaf Keshavjee, M.D., Laurent Brochard, M.D., Paul Hébert, M.D., Arthur S. Slutsky, M.D., John C. Marshall, M.D., Deborah Cook, M.D., and Margaret S. Herridge, M.D., M.P.H., for the RECOVER Program Investigators (Phase 1: towards RECOVER) and the Canadian Critical Care Trials Group N Engl J Med 2016; 374:1831-1841May 12, 2016DOI: 10.1056/NEJMoa1511160   Note: Content is Not intended as medical advice. Please consult your health care provider regarding your specific medical condition and questions. More Medical Research Interviews on MedicalResearch.com

Dr. Jill Cameron

Jill Cameron, PhD
Canadian Institutes of Health Research New Investigator
Associate Professor,
Department of Occupational Science and Occupational Therapy
Rehabilitation Sciences Institute
Faculty of Medicine,
University of Toronto

MedicalResearch.com: What is the background for this study? What are the main findings?

Dr. Cameron: In the world of critical illness, a lot of research has focused on helping people to survive – and now that more people are surviving, we need to ask ourselves, what does quality of life and wellbeing look like afterwards for both patients and caregivers? The aim of our research was to identify factors associated with family caregiver health and wellbeing during the first year after patients were discharged from the Intensive Care Unit. We examined factors related to the patient and their functional wellbeing, the caregiving situation including the impact it has on caregivers everyday lives, and caregiver including their sense of control over their lives and available social support. We used Pearlin’s Caregiving Stress Process model to guide this research.

From 2007-2014, caregivers of patients who received seven or more days of mechanical ventilation in an ICU across 10 Canadian university-affiliated hospitals were given self-administered questionnaires to assess caregiver and patient characteristics, caregiver depression symptoms, psychological wellbeing, and health-related quality of life. Assessments occurred seven days and three, six and 12-months after ICU discharge.

The study found that most caregivers reported high levels of depression symptoms, which commonly persisted up to one year and did not improve in some. Caregiver sense of control, impact on caregivers’ everyday lives, and social support had the largest relationships with the outcomes. Caregivers’ experienced better health outcomes when they were older, caring for a spouse, had higher income, better social support, sense of control, and caregiving had less of a negative impact on their everyday lives. No patient characteristics or indicators of illness severity were associated with caregiver outcomes.

Poor caregiver outcomes may compromise patients’ rehabilitation potential and sustainability of home care. Identifying risk factors for caregiver distress is an important first step to prevent more suffering and allow ICU survivors and caregivers to regain active and fulfilling lives.

Continue reading

Persistent Childhood Asthma Linked to Long-Term Lung Function Deficits

MedicalResearch.com Interview with:

Dr. James P. Kiley Ph.D National Institutes of Health Bethesda Maryland

Dr. James Kiley

Dr. James P. Kiley Ph.D
National Institutes of Health Bethesda
Maryland 

MedicalResearch.com: What is the background for this study? What are the main findings?
Dr. Kiley: While a higher proportion of children have asthma compared to adults, the disease is limited to childhood for many individuals who appear to be unaffected as adults. Regardless of whether asthma continues into adulthood or reoccurs during adulthood, the impact of childhood asthma on lung function later in life is unclear. This study demonstrated that in children with chronic persistent asthma at the age of 5-12 years who continued to be followed through their early twenties, 75% of them had some abnormality in the pattern of their lung growth. The study examined the trajectory of lung growth, and the decline from maximum growth, in a large cohort of persons who had persistent, mild-to-moderate asthma in childhood and determined the demographic and clinical factors associated with abnormal patterns of lung growth and decline.
Continue reading

Children With Persistent Asthma At Risk For Future Lung Disease

MedicalResearch.com Interview with:

Michael McGeachie, PhD Instructor in Medicine Harvard Medical School Channing Division of Network Medicine Brigham and Women's Hospital

Dr. Michael McGeachie

Michael McGeachie, PhD
Instructor in Medicine
Harvard Medical School
Channing Division of Network Medicine
Brigham and Women’s Hospital

MedicalResearch.com: What is the background for this study?

Dr. McGeachie: In asthma, and in general but particularly in asthma, a person’s level of lung function has a big impact on his or her quality of life, level of respiratory symptoms and complications, and general morbidity. In asthma, low lung function leads to greater severity and frequency of asthma symptoms. Asthma is a common childhood illness, affecting 9-10% of children. Many children grow out of asthma as they become adults, but other asthmatics remain effected through adulthood, which can lead to a lifetime of respiratory symptoms and chronic airway obstruction, including chronic obstructive pulmonary disease (COPD).

If you consider lung function longitudinally, throughout development, plateau, and decline, different people and different asthmatics tend to exhibit different patterns of lung function. Healthy, non-asthmatic people tend to have a period of rapid lung function increase in adolescence, a plateau of lung function level in their late teens and early 20s, and starting around 25 or so a slow, gradual decline of lung function that continues throughout old age. We call this Normal Growth of lung function. However, some people exhibit Reduced Growth, where they don’t reach their expected maximum lung function for a person of the same age, sex, height, and race. Others can show Early Decline, who might reach a normal maximum but then begin to decline immediately without a plateau or with a truncated plateau. We hypothesized that these patterns, Reduced Growth and Early Decline, might have different baseline indicators, precursors, outcomes, and risk of developing COPD.

Continue reading

Lower Dose of tPA Found Safer For Patients With Acute Ischemic Stroke

MedicalResearch.com Interview with:

Professor Craig Anderson Professor of Stroke Medicine and Clinical Neuroscience Sydney Medical School at the University of Sydney Institute of Neurosciences of Royal Prince Alfred Hospital

Prof. Craig Anderson

Professor Craig Anderson
Professor of Stroke Medicine and Clinical Neuroscience
Sydney Medical School at the University of Sydney
Institute of Neurosciences of Royal Prince Alfred Hospital

MedicalResearch.com: What is the background for this study?

Prof. Anderson: Intravenous use of the clot-busting drug, alteplase (or rtPA), at a dose of 0.9 mg/kg body weight is the only proven medical treatment of acute ischemic stroke.  However, a  major drawback to the treatment is an increased risk of major bleeding in the brain, or intracerebral hemorrhage (ICH), that occurs in about 5% of cases, and can be fatal.  This balance of effectiveness (recovery from disability) and risks (ICH, and bleeding elsewhere and uncommon drug allergic reactions) has led to much of the controversy over the net benefit of the drug.  The optimal dose of the drug has never been established, but the Japanese drug safety regulatory authority, has approved a lower dose (0.6mg/kg) on the basis of a small, non-randomized, open study which showed comparable outcomes and lower risk of ICH than historical controls.  This ‘east-west’ divide over the approved dose of alteplase has led to much variation in the dose of alteplase used in clinical practice in Asia – according to a doctor’s perceived risk of ICH in individual patients and the affordability of this relatively expensive treatment in low resource settings.  Data from the Get-with-the Guidelines Quality Registry in the United States suggests Asian patients are at higher risk of ICH after standard-dose alteplase than non-Asians.

Our research aimed to resolve this uncertainty over the optimal dose of alteplase, as an international, active-comparator, open-label, blinded outcome assessed, clinical trial of low-dose (0.6 mg/kg) versus standard-dose (0.9mg/kg) in 3310 patients recruited from over 100 hospitals in 13 countries between 2012 and 2015.

Continue reading

Pembrolizumab Immunotherapy Benefits Some Merkel Cell Carcinoma Patients

MedicalResearch.com Interview with:

Paul Nghiem, MD, PhD Professor & Head, University of Washington Dermatology George F. Odland Endowed Chair Affiliate Investigator, Fred Hutchinson Cancer Research Center Professor, Adjunct, of Pathology and Oral Health Sciences Clinical Director, Skin Oncology, Seattle Cancer Care Alliance UW Medical Center at Lake Union Seattle WA 98109

Dr. Paul Nghiem

Paul Nghiem, MD, PhD
Professor & Head, University of Washington Dermatology
George F. Odland Endowed Chair
Affiliate Investigator, Fred Hutchinson Cancer Research Center
Professor, Adjunct, of Pathology and Oral Health Sciences
Clinical Director, Skin Oncology, Seattle Cancer Care Alliance
UW Medical Center at Lake Union
Seattle WA 98109

 

MedicalResearch.com: What is the background for this study? What are the main findings?

Dr. Nghiem: Merkel cell carcinoma (MCC) is about 30 times less common than malignant melanoma, but about 3 times more likely to kill a patient than a melanoma. There is no FDA-approved therapy for this cancer & chemotherapy typically only provides about 90 days prior to the cancer progressing. Because of the strong links between MCC and the immune system, including the fact that most MCCs are caused by a virus, there was interest in trying to use immune checkpoint therapy to treat advanced Merkel cell carcinoma. The response to immune stimulation with anti-PD1 therapy was about as frequent as to chemotherapy (56% of patients responded) but importantly, among the responders, 86% remained in ongoing responses at a median of 7.6 months.  While still early, this appears to be strikingly more durable than responses to chemotherapy.

Continue reading

Adding Lumbar Fusion to Laminectomy Improves Long-Term Quality of Life

MedicalResearch.com Interview with:

Zoher Ghogawala MD FACS Department of Neurosurgery Lahey Hospital and Medical Center Burlington, MA 01805

Dr. Zoher Ghogawala

Zoher Ghogawala MD FACS
Department of Neurosurgery
Lahey Hospital and Medical Center
Burlington, MA 01805

MedicalResearch.com: What is the background for this study?

Dr. Ghogawala: There is enormous practice variation around the utilization of lumbar spinal fusion in the United States and across the world.  In the United States, lumbar spinal fusion utilization has increased to 465,000 hospital-based procedures in 2011 according to a report from the AHRQ (published in 2014).  Spinal fusion accounts now for the highest aggregate hospital cost (12.8 billion dollars in 2011) of any surgical procedure performed in US hospitals.  What is problematic is that there are no top tier studies that address the question of whether or not adding a lumbar spinal fusion when performing a simple decompression is necessary or helpful.  The question is whether we perform too many fusions in the United States.

The SLIP study is the first class I study that demonstrates that the addition of a lumbar fusion when performing a lumbar laminectomy to decompress spinal nerves improves health-related quality of life for patients suffering from low back pain and sciatica from lumbar stenosis with spondylolisthesis – a very common cause of low back pain caused by nerve compression associated with one spinal bone being slightly out of alignment. 

MedicalResearch.com: What are the main findings?

Dr. Ghogawala:

1)  Adding a lumbar fusion when performing a lumbar laminectomy results in superior health-related quality of life at 2,3, and 4 years after surgery.

2)  Patients with fusion obtained durable results but 14% required re-operation for problems adjacent to their fusion over the 4 year study period.

3)  Lumbar laminectomy alone provided good results for 70% of patients.  There was less blood loss and faster recovery for these patients.  On the other hand, the outcomes were less durable.  One in three patients who underwent a lumbar laminectomy alone required re-operation within 4 years because their back became unstable.  These patients underwent fusion and their health-related quality of life improved.

Continue reading

Extended Release Naltrexone Helps Prevent Opioid Relapse in Criminal Justice Offenders

MedicalResearch.com Interview with:

Joshua D. Lee MD, MSc Associate Professor in Medicine and Psychiatry NYU Langone Medical Center

Dr. Joshua Lee

Joshua D. Lee MD, MSc
Associate Professor in Medicine and Psychiatry
NYU Langone Medical Center

MedicalResearch.com: What is the background for this study?

Dr. Lee: Opioid use disorders, both from prescription pain medication and heroin use, and related death rates are increasing annually in the US.  Many states, counties, and cities that have previously not had great experience with heroin addiction are now overwhelmed.  This presents unprecedented challenges to affected families and communities, and also health providers and criminal justice systems that have historically not provided high rates of evidence-based treatment for opioid addictions.  Left untreated or inadequately treated, opioid use disorders are chronic, destructive, and often fatal. Extended-release naltrexone, an opioid receptor blocker, is a promising relapse prevention medication intervention, but had not been evaluated in a US criminal justice system (CJS) setting or under real-world conditions.

This effectiveness study recruited 308 adults with US criminal justice system involvement (i.e., recent jail or prison incarceration, on parole or probation) and a history of opioid dependence (addiction), who were not currently accessing methadone or buprenorphine maintenance treatment, and were interested in treatment with extended-release naltrexone (XR-naltrexone).  All participants were off opioids (detoxed or recently abstinent) at the time of study start (randomization).  Participants randomized to an open-label, non-blinded evaluation of XR-naltrexone versus treatment-as-usual for six months of treatment.  Long-term follow-up occurred at 12 months and 18 months (6 and 12 months post-treatment).  We estimated rates of opioid relapse and opioid use between the two arms over the course of treatment.  We also tracked other drug and alcohol use, re-incarceration rates, and overdose rates throughout the study.

Continue reading

Study Finds No Increased Risk of Heart Failure From Incretin-Based Diabetes Drugs

MedicalResearch.com Interview with:

Kristian B. Filion, Ph.D., FAHA Assistant Professor of Medicine, Division of Clinical Epidemiology, McGill University Principal Investigator, Center for Clinical Epidemiology, Lady Davis Institute, Jewish General Hospital Associate Member, Department of Epidemiology, Biostatistics, and Occupational Health, McGill University

Dr. Kristian Filion

Kristian B. Filion, Ph.D., FAHA
Assistant Professor of Medicine, Division of Clinical Epidemiology, McGill University
Principal Investigator, Center for Clinical Epidemiology, Lady Davis Institute, Jewish General Hospital
Associate Member, Department of Epidemiology, Biostatistics, and Occupational Health, McGill University

MedicalResearch.com: What is the background for this study? What are the main findings?

Dr. Filion:  Concerns regarding the cardiovascular safety of the antidiabetic incretin-based drugs arose following the unexpected finding of an increased risk of heart failure in the SAVOR-TIMI 53 trial of the DPP-4 inhibitor saxagliptin.  Although subsequent trials did not find an increased risk of heart failure with these drugs, concerns regarding their heart failure effects remained due to the conflicting available evidence.  Furthermore, with their highly-selected patient populations, the applicability of the results of these clinical trials to patients seen in a real world setting is unclear.

We therefore examined the risk of hospitalization for heart failure associated with incretin-based drugs in patients with type 2 diabetes in a real world setting using the health records of over 1.4 million patients from six jurisdictions (four Canadian provinces, the United Kingdom, and the United States). We used a common protocol in each database and statistically combined results across databases to obtain one overall estimate.  We found that there was no evidence of an increased risk, a finding that was consistent in separate analyses for patients with and without a history of heart failure.

MedicalResearch.com: What should clinicians and patients take away from your report?

Dr. Filion:  This study provides reassurance regarding concerns about the potential risk of heart failure with the use of incretin-based drugs compared with the use of combinations of oral antidiabetic drugs such as metformin and sulfonylureas.

MedicalResearch.com: What recommendations do you have for future research as a result of this study?

Dr. Filion:  It would be helpful to do additional analyses by individual incretin-based drug (i.e., by molecule), which could explain some of the heterogeneity observed in some of the clinical trials in this area.

In addition, many of the patients included in our study had a relatively short history of diabetes. In secondary analyses, we checked if the association differed by duration of treated diabetes and, although we found that it did not, it would be helpful to revisit this question in a few years as additional information becomes available.

MedicalResearch.com: Is there anything else you would like to add?

Dr. Filion:  This study was conducted by the Canadian Network for Observational Drug Effect Studies (CNODES), a pan-Canadian network funded by Health Canada and the Canadian Institutes of Health Research to study emerging drug safety issues.  CNODES has the ability to analyze a vast amount of de-identified data to efficiently respond to drug safety issues that arise after a drug is already on the market.

In this case, we were able to analyze the health records of over 1.4 million patients who were prescribed an antidiabetic medication in Canada, the United Kingdom, and the United States.

MedicalResearch.com: Thank you for your contribution to the MedicalResearch.com community.

Citation:

A Multicenter Observational Study of Incretin-based Drugs and Heart Failure

Kristian B. Filion, Ph.D., Laurent Azoulay, Ph.D., Robert W. Platt, Ph.D., Matthew Dahl, B.Sc., Colin R. Dormuth, Sc.D., Kristin K. Clemens, M.D., Nianping Hu, M.D., Ph.D., J. Michael Paterson, M.Sc., Laura Targownik, M.D., M.S.H.S., Tanvir C. Turin, M.D., Ph.D., Jacob A. Udell, M.D., M.P.H., and Pierre Ernst, M.D., for the CNODES Investigators*

N Engl J Med 2016; 374:1145-1154
March 24, 2016 DOI: 10.1056/NEJMoa1506115

Note: Content is Not intended as medical advice. Please consult your health care provider regarding your specific medical condition and questions.

More Medical Research Interviews on MedicalResearch.com

Kristian B. Filion, Ph.D., FAHA (2016). Study Finds No Increased Risk of Heart Failure From Incretin-Based Diabetes Drugs MedicalResearch.com

Informatics and Financial Incentives Can Lead To Safer Drug Prescribing

MedicalResearch.com Interview with:

Prof. Bruce Guthrie Primary Care Medicine and Honorary Consultant NHS Fife University of Dundee Dundee, Scotland

Prof. Bruce Guthrie

Prof. Bruce Guthrie
Primary Care Medicine and Honorary Consultant NHS Fife
University of Dundee
Dundee, Scotland

MedicalResearch.com: What is the background for this study? What are the main findings?

Dr. Guthrie: Most drug-related harm is caused by commonly prescribed drugs with moderate risk. This prescribing is not always inappropriate, because risk of harm may be outweighed by benefit in an individual, but we have previously shown that high-risk prescribing like this is common and highly variable between primary care practices, consistent with it being improvable. We therefore developed a complex intervention combining education, informatics to make it easy to identify and review patients, and a small financial incentive to review. We evaluated this intervention in a cluster-randomised trial in 33 Scottish primary care practices, targeting nine measures of high-risk non-steroidal anti-inflammatory drug (NSAID) and antiplatelet prescribing (for example, prescription of an NSAID to someone with chronic kidney disease; prescription of an antiplatelet to someone taking an anticoagulant without also prescribing a gastroprotective drug).

The intervention reduced the targeted prescribing by just over one third, and this reduction was sustained in the year after the intervention (including the payment to review) ceased. We also observed reductions in related hospital admissions with gastrointestinal bleeding and heart failure, although not acute kidney injury which was reduced but not statistically significantly.

Continue reading

Study Finds Aspirin Safe For People Waiting To Have Coronary Artery Surgery

MedicalResearch.com Interview with:

Professor Paul Myles MBBS, MPH, MD, FCARCSI, FANZCA, FRCA Director, Dept of Anaesthesia and Perioperative Medicine Alfred Hospital and Monash University, Melbourne, Australia

Prof. Paul Myles

Professor Paul Myles MBBS, MPH, MD, FCARCSI, FANZCA, FRCA
Director, Dept of Anaesthesia and Perioperative Medicine
Alfred Hospital and Monash University, Melbourne, Australia

Medical Research: What is the background for this study? What are the main findings?

Dr. Myles: When we set up this study 10 years ago there was marked variation in practice for  people taking aspirin waiting for coronary artery bypass surgery.  About half were being told that they must stop their aspirin 5-7 days before surgery, and the other half were told that they should stay on their aspirin. This variation existed across different countries, different cities, and even within a single hospital. Doctors had varied opinions because reliable medical research was sparse; the evidence was contradictory. We thus designed a definitive clinical trial in which half the patients were randomly assigned to receive aspirin and the other half received a placebo. Our study has shown that aspirin is safe (i.e. it does not increase the bleeding risk). We also found that there does not appear to be a benefit during and after surgery, but in view of the clear benefits that exist in daily life, including the preoperative waiting period, we recommend that people should stay on their aspirin if they are having coronary artery surgery.

Continue reading

Antiretroviral Vaginal Ring May Become HIV Prevention Tool For Women

MedicalResearch.com Interview with:

Jared Baeten, MD PhD Vice Chair, Department of Global Health Professor, Departments of Global Health, Medicine, and Epidemiology Co-Director, International Clinical Research Center University of Washington

Dr. Jaret Baeten

Jared Baeten, MD PhD
Vice Chair, Department of Global Health
Professor, Departments of Global Health, Medicine, and Epidemiology
Co-Director, International Clinical Research Center
University of Washington

MedicalResearch.com: What is the background for this studies?

Dr. Baeten: Women account for nearly 60 percent of adults with HIV in sub-Saharan Africa, where unprotected heterosexual sex is the primary driver of the epidemic. While several studies have shown that antiretroviral medications (ARVs) are highly effective in preventing HIV, other studies – such as VOICE and FACTS 001 – suggest that for young, at-risk women in Africa, ARVs delivered as a vaginal gel or as a tablet may not be acceptable. Products must be used to be effective, and that was not the case for most of the participants in previous studies.

Medical Research: What was the aim of ASPIRE and The Ring Study?

Dr. Baeten: As Phase III clinical trials, ASPIRE and The Ring Study were designed to determine whether a vaginal ring containing an antiretroviral (ARV) drug called dapivirine is safe and effective in protecting women against HIV when used for a month at a time. These trials also sought to determine whether women find the vaginal ring practical and easy to use. As sister studies, ASPIRE and The Ring Study were designed as the centerpiece of a broader licensure program to provide the strength of evidence to support potential licensure of the dapivirine vaginal ring for preventing HIV in women. Because at least two Phase III efficacy trials are usually needed for a product to be considered for regulatory approval, ASPIRE and The Ring Study were conducted in parallel to accelerate the timeline to the ring’s potential approval.

Continue reading

Outcomes of Collaborative US-European Treatment of Ebola Cases Reported

MedicalResearch.com Interview with:

Tim Uyeki MD, MPH, MPP Influenza Division National Center for Immunization and Respiratory Diseases Centers for Disease Control and Prevention and Associate Clinical Professor of Pediatrics Department of Pediatrics San Francisco General Hospital

Dr. Tim Uyeki

Tim Uyeki MD, MPH, MPP
Influenza Division
National Center for Immunization and Respiratory Diseases
Centers for Disease Control and Prevention and
Associate Clinical Professor of Pediatrics
Department of Pediatrics
San Francisco General Hospital

Medical Research: What is the background for this study?

Dr. Uyeki: During 2014-2015, 27 patients with Ebola virus disease (EVD) were hospitalized in the United States and Europe. Frequent international teleconferences were convened among U.S. and European clinicians caring for EVD patients, often on a weekly basis, to share detailed information and suggestions on clinical management of these patients. We collected clinical, epidemiologic, laboratory, and virologic data on all of these patients and performed descriptive data analyses. We summarized our findings in this article.

Medical Research: What are the main findings?

Dr. Uyeki: Of the 27 patients with Ebola virus disease cared for in 15 hospitals in nine countries, the median age was 36 years; 19 (70%) were male; 9 of 26 (35%) had underlying medical conditions; and 22 (81%) were healthcare personnel, including 17 of 22 (77%) who had worked in an Ebola treatment unit in West Africa. Of the 27 patients, 20 (74%) were medically evacuated from West Africa, 4 (15%) were imported cases, and 3 (11%) were healthcare personnel who acquired Ebola virus infection while caring for EVD patients in the U.S. or Europe. At illness onset, the signs and symptoms of EVD were non-specific; the most common symptom reported was fatigue. At admission to a hospital in the U.S. or Europe, most patients had fever, weakness, and gastrointestinal symptoms. The median time from illness onset to hospitalization was four days.

During hospitalization, all patients had diarrhea, often profuse watery diarrhea; and most experienced electrolyte abnormalities such as hyponatremia, hypokalemia, hypocalcemia, and hypomagnesemia, as well as hypoalbuminemia. One third of patients experienced renal abnormalities such as oliguria or anuria, nearly 60% were clinically diagnosed with systemic inflammatory response syndrome, and one third were clinically diagnosed with encephalopathy or encephalitis. Although minor bleeding abnormalities were reported in some patients, only two patients had any gross hemorrhage. Leukopenia was observed during the first week of illness, with increases in white blood cell count during the second week. Thrombocytopenia was common, and aminotransferase levels peaked in the second week of illness. Creatine kinase and lactate levels were elevated in most of the patients who were tested. Ebola virus levels in blood peaked on the seventh day of illness, and critical illness occurred at the end of the first week and during the second week after illness onset. All patients received intravenous fluids; most were treated empirically with antibiotics; and 85% received an investigational therapy, including 70% who received at least two experimental therapies. Eleven (41%) patients were critically ill, including seven who required invasive mechanical ventilation and five who received continuous renal replacement therapy. Five (18.5%) patients died (81.5% survival).

Continue reading

Asymptomatic Carotid Stenosis: Stenting and Surgery Equally Effective

MedicalResearch.com Interview with:

Dr. William A Gray, MD Chief of the Division of Cardiovascular Disease Main Line Health President of Main Line Health’s Lankenau Heart Institute

Dr. William A Gray

Dr. William A Gray, MD
Chief of the Division of Cardiovascular Disease
Main Line Health
President of Main Line Health’s Lankenau Heart Institute 

Medical Research: What is the background for this study? What are the main findings?

Dr. Gray: The basis for this study was two-fold: the ACST-1 trial had shown, in asymptomatic patients with severe carotid disease, that immediate Carotid Endarterectomy reduced subsequent stroke as compared to deferred Carotid Endarterectomy—so the next logical question was, could Carotid Artery Stenting (CAS) compare as an equal alternative to Carotid Endarterectomy (CEA) in this same, standard risk, population with severe carotid stenosis.

The CREST trial, as originally constructed and at the time ACT 1 was conceived did not include this population (although it later expanded to encompass asymptomatic patients as well), so it was an open question. The second reason had to do with Abbott Vascular, the study sponsor, achieving FDA regulatory approval for their stent system in this population—as well as in the symptomatic population being studied n CREST (which they were also the device sponsor).

The main findings were that the primary endpoint of death/stroke and MI at 30 days plus ipsilateral stroke out to 1 and 5 years was not different between CAS and CEA in asymptomatic patients with severe carotid stenosis on good medical secondary prevention therapy.

Continue reading

Dementia Incidence Dropping In Those With At Least High School Education

MedicalResearch.com Interview with:

Claudia L. Satizabal, PhD Instructor in Neurology Boston University School of Medicine The Framingham Heart Study Boston, MA 02118-2526

Dr. Claudia Satizabal

Claudia L. Satizabal, PhD
Instructor in Neurology
Boston University School of Medicine
The Framingham Heart Study
Boston, MA 02118-2526

MedicalResearch: What is the background for this study? What are the main findings?

 

Dr. Satizabal: Our societies are expected to face an increasing burden of dementia in the next decades due to increasing life expectancies and the aging of a big proportion of the population, the so called “baby boomers”. However, some studies conducted in high-income countries have suggested a decline in the total number of cases (prevalence) as well as new cases (incidence) of dementia at any given age. Yet the findings of these studies were not seen as definitive, either because results were of borderline significance or because they were based on survey data, and stronger evidence was lacking.

We used information collected since 1975 in the Framingham Heart Study to estimate the trends in dementia incidence. One of the strengths of this study is that investigators have been careful to use the same diagnostic criteria for over the past 30 decades, which allows us to provide more robust evidence of dementia trends over time.

We found that there has been a progressive decline in the incidence of dementia at any given age over the past 30 decades. Compared to the late 1970s, we observed a decline of 22% in the late 1980s, 38% in the 1990s and 44% in the 2000s. This beneficial trend was only seen among persons with at least a high school diploma. We also explored trends in vascular risk factors such as blood pressure, smoking, diabetes, and others; however, these trends did not completely explain the decline in dementia incidence. One interesting finding was that the risk of dementia associated with cardiovascular diseases, such as stroke or atrial fibrillation, decreased dramatically over the course of time from the late 1970s to the 2000s.

Continue reading

Nanobody is Potentially First Targeted Therapy for TTP

MedicalResearch.com Interview with:
Dr. Filip Callewaert PhD
Senior Clinical Scientist
Clinical Development, Ablynx
Zwijnaarde, Belgium

Medical Research: What is the background for this study? What are the main findings?

Dr. Callewaert: Acquired thrombotic thrombocytopenic purpura (TTP) is a rare and life-threatening coagulation disorder, in which accumulation of ultra-large von Willebrand factor (ULvWF) multimers is implicated, leading to an increased risk of thrombus formation in small blood vessels due to excessive platelet aggregation. There are no approved pharmacological therapies for acquired TTP. Despite treatment with the current standard of care (plasma exchange and immunosuppressive therapy), mortality remains at 10-20% and there is significant neurological, cardiac, and renal morbidity.

Caplacizumab is a bivalent Nanobody that binds to the A1 domain of vWF thereby preventing vWF-mediated platelet aggregation. The clinical effects of caplacizumab were demonstrated in the phase II randomised, placebo-controlled TITAN study in 75 patients with acquired TTP. Compared to placebo, there was a nearly 40% reduction in median time to platelet count normalisation in the caplacizumab group (p = 0.005). Treatment with caplacizumab reduced the use of daily plasma exchange and prevented further consumption of platelets in microthrombi and small blood vessel occlusion. In addition, there were fewer recurrences of TTP requiring re-initiation of daily plasma exchange during treatment with caplacizumab (N=3) vs. placebo (N=11). The safety profile of caplacizumab was favorable, with a slightly higher tendency of mostly mild bleeding events.  Continue reading

Belacept Found Superior to Cyclosporin For Kidney Transplantation

MedicalResearch.com Interview with:

Dr. Flavio Vincenti

Dr. Flavio Vincenti

Dr. Flavio Vincenti, M.D
Clinical Professor of Medicine and Surgery
Departments of Medicine and Surgery
Endowed Chair in Kidney Transplantation
University of California, San Francisco
San Francisco, CA 94143

Medical Research: What is the background for this study? What are the main findings?
Dr. Vincenti: This is a phase 3 study of belacept immunosuppression as compared to cyclosporine based immunosuppression in renal transplant recipients randomly assigned to 2 treatments arms of belatacept and a controlled arm consisting of cyclosporine. The main finding of this study is that Belatacept, a fusion receptor protein that blocks co-stimulation and is administered intravenously on the maintenance of a 4 weekly maintenance therapy, had superior outcomes at 5 and 7 years as compared to patients on a CsA-based regimen. The 7 year data show that patients on either arm of belatacept had a 43 percent risk reduction of deaths or grafts loss as compared to patients treated with cyclosporine. In addition, belatcept patients had significantly better preservation of renal function throughout the 7 years of follow up and had lower incidence of donor specific antibodies. Nephrotoxicity from cyclosporine and donor specific antibodies are important causes of late graft loss.
Continue reading

Prescription Opioids And Heroin Crisis Overlap in Important Ways

Dr. Wilson Compton

Dr. Wilson Compton

For more on Opioids on MedicalResearch.com please click here.

MedicalResearch.com Interview with:
Wilson M. Compton, M.D., M.P.E.
Deputy Director
National Institute on Drug Abuse

Medical Research: What is the background for this study? What are the main findings?

Dr. Compton: Deaths related to opioids (from both prescription pain killers and street drugs, like heroin) have dramatically increased in the past 15 years.  How these different types of opioids are related to each other is important because the pain killers ultimately are derived from prescriptions written by health care providers and street drugs, like heroin, are from illegal sources.  The different types of opioids vary in there source but are quite similar in their effects in the brain.  Given the different sources, interventions to reduce availability vary across the two categories. There is also a concern that interventions to reduce the availability of prescription opioids may be encouraging people to switch to heroin.  That’s the main question addressed in this review.

Continue reading

Lost-Cost Mesh Surgery for Groin Hernia Successful in Low Resource Setting

Dr. Jenny Löfgren Surgery and Perioperative Sciences, Faculty of Medicine, University Hospital of Umeå Umeå Sweden

Dr. Jenny Löfgren

MedicalResearch.com Interview with:
Dr. Jenny Löfgren
Surgery and Perioperative Sciences
Faculty of Medicine,
University Hospital of Umeå
Umeå Sweden 

Medical Research: What is the background for this study? What are the main findings?

Response: There are an estimated 220 million groin hernia patients in the World. 20 million are operated on annually making it one of the worlds most commonly performed surgeries. The surgical repair rate in low income settings is very low. Also, the quality of the surgery is lower than in high income settings. The superior technique that uses a synthetic mesh to reinforce the abdominal wall at the site of the hernia is not affordable due to the high cost of that mesh. Mosquito mesh, which is very similar to the expensive mesh, is already used in several settings but its safety and effectiveness had not previously been investigated in a randomized trial of sufficient size with follow up for as long as one year.  

Medical Research: What are the main findings?

Response: The most important finding of the study is that it was not able to detect any differences in terms of safety, effectiveness and patient satisfaction when outcomes in the group receiving the low-cost (mosquito) mesh with the group receiving a commonly used commercial mesh. The study also shows that high quality surgery, on par with standards in high income settings, can be provided for an underserved population in rural Uganda, at an affordable cost. Finally, the study shows that it is possible to conduct high quality surgical (clinical) research with high follow up rates also in settings such as rural Uganda. This should encourage us and others to conduct other trials in the future

Continue reading