Author Interviews, Diabetes, NEJM, OBGYNE, Technology / 19.08.2016

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. (more…)
Author Interviews, Brigham & Women's - Harvard, JAMA, Mental Health Research, OBGYNE / 19.08.2016

MedicalResearch.com Interview with: Krista F. Huybrechts, M.S., Ph.D. Assistant Professor of Medicine Harvard Medical School Epidemiologist Division of Pharmacoepidemiology and Pharmacoeconomics Brigham and Women’s Hospital Boston, MA 02120 MedicalResearch.com: What is the background for this study? Response: The use of antipsychotic medications during pregnancy has doubled in the last decade. Yet, information on the safety of antipsychotic medication use during pregnancy for the developing fetus is very limited: existing studies tend to be small (the largest study available to date includes 570 exposed women) and findings have been inconsistent. Concerns have been raised about a potential association with congenital malformations. The objective of our study was to examine the risk of congenital malformations overall, as well as cardiac malformations given findings from earlier studies, in a large cohort of pregnant women. We used a nationwide sample of 1.3 mln pregnant women insured through Medicaid between 2000-2010, of which 9,258 used an atypical antipsychotic and 733 used a typical antipsychotic during the first trimester, the etiologically relevant period for organogenesis. We also examined the risks associated with the most commonly used individual medications. (more…)
Author Interviews, JAMA, Prostate Cancer / 18.08.2016

MedicalResearch.com Interview with: Dr. Ahmedin Jemal, DVM, PhD Vice President, Surveillance and Health Services Research American Cancer Society MedicalResearch.com: What is the background for this study? What are the main findings? Response: We previously showed large decrease in early stage prostate cancer incidence rates from 2011 to 2012 in men 50 years and older following the US Preventive services Task Force recommendation against routine prostate-specific antigen testing in 2011. In this paper, we examined whether the decrease in early stage incidence persisted through 2013. We found that early stage prostate cancer incidence rates in men age 50 and older decreased from 2012 to 2013, although the decrease (6%) was lower compared to the decrease from 2011-2012 (19%). In contrast, rates for distant stage disease between 2012 and 2013 remained unchanged. (more…)
Author Interviews, Colon Cancer, Genetic Research, Journal Clinical Oncology, MD Anderson / 18.08.2016

MedicalResearch.com Interview with: Y. Nancy You, MD, MHSc Associate Professor Section of Colorectal Surgery Department of Surgical Oncology Medical Director Familial High-risk Gastrointestinal Cancer Clinic University of Texas MD Anderson Cancer Center MedicalResearch.com: What is the background for this study? What are the main findings? Response: Despite the progress in the treatment of many cancers, colorectal cancer (CRC) remains the third most common and lethal cancer in the US. Over 130,000 people are expected to be diagnosed and over 50,000 patients will die from CRC this year. In the recent years, the most exciting development has been our understanding of the molecular complexity of CRC. Currently, four major molecular subtypes of colorectal cancer are recognized. Our study focuses on the Consensus Molecular Subtype 1, which accounts for up to 15% of CRCs, and is characterized by a deficiency in DNA mismatch repair (dMMR), a high level of mutations (i.e. hypermutated), by instability in parts of the genome called microsatellites, and by strong immune activation. Prior to our study, patients with rectal cancer that belong to this molecular subtype have represented an unknown, in terms of their prognosis, and how the tumors respond to current treatments. More importantly, this molecular subtype harbor a genetic condition that can be transmitted within the family called “Lynch Syndrome”. So we designed our study to fill these gaps in our understanding that exist in this subtype of CRCs and to highlight key clinical care issues related to the caring for patients with a genetic syndrome. The main findings are that rectal cancers belonging to this molecular subtype have favorable prognosis, respond well to standard chemoradiation, and are linked to Lynch Syndrome and should be treated at centers with expertise in hereditary cancer syndromes. (more…)
Accidents & Violence, Author Interviews, Pediatrics / 18.08.2016

MedicalResearch.com Interview with: Kristi Roberts, MS, MPH Research associate Center for Injury Research and Policy Nationwide Children’s Hospital. MedicalResearch.com: What is the background for this study? Response: Strollers (such as these luxury strollers) and carriers are used regularly by caregivers and are intended to provide a safe and secure way to transport young children during everyday activities. However, parents and caregivers should be aware that injuries do occur while using these products. (more…)
Author Interviews, Dermatology / 18.08.2016

MedicalResearch.com Interview with: Thomas Bold President and CEO RenovaCare Inc. MedicalResearch.com: What is the background for the CellMist™ System? How are the stem cells harvested and processed? Response: Our flagship CellMist™ System makes use of a patient’s own stem cells, which are sprayed onto wounds using our novel SkinGun™ device. For patients suffering severe burns and other wounds, the prospect of a quick-healing, gentle spray containing their own stem cells will be a promising alternative to conventional skin graft surgery, which can be painful, prone to complications like infections, and slow-to-heal. Based on preliminary case studies, CellMist™ System patients can be treated within 90 minutes of arriving in an emergency room; a patient’s stem cells are isolated, processed, and sprayed on to the wound sites for rapid healing. The CellMist™ Solution is a liquid suspension containing a patient’s own regenerative skin stem cells. A small sample (as small as a square inch) of the patient’s skin is quickly processed to liberate the stem cells from surrounding tissue. The resulting product is referred to as the ‘CellMist™ Solution’. The CellMist™ Solution is placed in the SkinGun™ and gently sprayed onto the patient’s wound. (more…)
Author Interviews, Heart Disease, JAMA, Lipids, Nutrition, Omega-3 Fatty Acids / 18.08.2016

MedicalResearch.com Interview with: David Iggman, MD, PhD Unit for Clinical Nutrition and Metabolism Department of Public Health and Caring Sciences Uppsala University, Uppsala Center for Clinical Research Dalarna Falun, Sweden MedicalResearch.com: What is the background for this study? What are the main findings? Response: There is some controversy regarding which dietary fats are preferable and in what amounts, not least regarding the polyunsaturated fats. It is also challenging to adequately assess peoples intakes of dietary fats. The main findings of this study was that among fatty acids in the body (reflecting the intake during the last year or so), linoleic acid (omega-6) was associated with lower mortality in 71-year-old men with 15 years follow-up. (more…)
Author Interviews, Biomarkers, Heart Disease, Microbiome / 18.08.2016

MedicalResearch.com Interview with: Lemin Zheng, Ph.D. Professor, Lab Director, and Principal Investigator The Institute of Cardiovascular Sciences and Institute of Systems Biomedicine Peking University Health Science Center Beijing  China MedicalResearch.com: What is the background for this study? Response: Optical coherence tomography (OCT) has been considered as an ideal tool to characterize accurately atherosclerotic plaques and has potential to detect plaque rupture due to high-resolution (10-20 μm) cross-sectional images of tissue with near infrared light (1-3). Trimethylamine-N-oxide (TMAO) is a gut microbiota-dependent-generated metabolite which is associated with cardiovascular risk by a pathway involving dietary ingestion of nutrients containing trimethylamine, including phosphatidylcholine, choline, and L-carnitine (4-6). In the gut, choline, betaine and carnitine can be metabolized to trimethylamine (TMA) by gut flora microorganism. And TMA could be further oxidized to a proatherogenic species, TMAO, in the liver by flavin monooxygenases 3 (FMO3)4-6. These risk associations have been repeatedly shown in large observational trials (7-10). (more…)
Author Interviews, Breast Cancer, Cancer Research, Cost of Health Care, Sloan Kettering, Surgical Research / 18.08.2016

MedicalResearch.com Interview with: Monica Morrow, MD, FACS Chief, Breast Service Department of Surgery Anne Burnett Windfohr Chair of Clinical Oncology Memorial Sloan-Kettering Cancer Center MedicalResearch.com: What is the background for this study? Response: DCIS, ductal carcinoma in situ, intraductal cancer or Stage 0 cancer refers to what some people call the earliest form of cancer we can find and others term “precancerous”. This difference in terms is due to the fact that DCIS lacks the ability to spread to other parts of the body, a fundamental characteristic of cancer. The goal of treatment in DCIS is to prevent progression to invasive cancer which has the ability to spread. DCIS accounted for only 2-3 % of breast cancers seen in the pre-screening mammography era, but it comprises 25-30% of the malignancies detected in screening mammography programs. For this reason it is uncommon in women under age 40, and more commonly seen in women over 50 years of age. Approximately 70% of the women in the US diagnosed with DCIS are treated with lumpectomy (removal of the DCIS and a margin of surrounding normal breast tissue), and additional surgeries to obtain clear, or more widely clear, margins are done in approximately 30% of women. For this reason, the Society of Surgical Oncology, the American Society for Therapeutic Radiation Oncology, and the American Society of Clinical Oncology undertook the development of an evidence based guideline to determine the optimal clear margin for women with DCIS treated with lumpectomy and whole breast radiotherapy. (more…)
Asthma, Author Interviews, Brigham & Women's - Harvard, NEJM, Pediatrics / 18.08.2016

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 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? (more…)
Author Interviews, Technology, Weight Research / 18.08.2016

MedicalResearch.com Interview with: Katherine Tweden Ph.D. Vice President, Clinical and Regulatory EnteroMedics® Inc St. Paul, Minnesota 55113 MedicalResearch.com: What is the background for this study? What are the main findings? Response: This report summarizes 2 year outcomes of the pivotal study that evaluated the safety and efficacy of EnteroMedics’ vagal blockade (vBloc Therapy) delivered by our Maestro Rechargeable System neuromodulation device for the treatment of obesity. The study demonstrates that the device continues to have a promising safety profile and that the weight loss achieved through 2 years is clinically meaningful as shown by the positive impact of vBloc Therapy on participant’s co-morbid conditions, quality of life, and eating behaviors. Specifically, the study showed 21% excess weight loss and approximately 50% reduction in pre-diabetes and metabolic syndrome for those participants who presented with the syndrome at baseline. In addition, cardiovascular parameters improved in those at risk with approximately 10 mmHg drop in systolic and diastolic blood pressure compared to baseline in participants with elevated blood pressure and 16 and 46 mg/dL drop in LDL and triglycerides, respectively, in participants with elevated lipids compared to baseline. In addition, metabolic parameters improved with a reduction in hemoglobin A1c of 0.3 percentage points. Participants’ quality of life improved by 20 units compared to baseline and their control over their eating behavior, such as hunger and the ability to control the amount they eat, was significantly improved by approximately 50% compared to baseline. (more…)
Author Interviews, PAD, Surgical Research / 18.08.2016

MedicalResearch.com Interview with: George Adams, M.D., M.H.S., F.A.C.C., F.S.C.A.I. Director of Cardiovascular and Peripheral Vascular Research Rex Hospital Raleigh, North Carolina Summary: The early findings of a novel all-comers PAD study (LIBERTY 360°) suggest that ‘watchful waiting’ in Rutherford class 2-3 and ‘primary amputation’ in Rutherford class 6 may not be necessary. Peripheral vascular intervention can be successful in these patient populations as well. MedicalResearch.com: What is the background for this study?  Response: Approximately 18 million Americans have peripheral artery disease (PAD), and 2 million of these patients suffer from critical limb ischemia (CLI)1,2, the end stage of PAD3. CLI is highly prevalent in older patients with diabetes and/or end-stage renal disease4, and is associated with high risk of amputation and mortality5. Briefly, the LIBERTY 360° study6 is a prospective, observational, multi-center study with liberal inclusion criteria and few exclusions, meant to evaluate procedural and long-term clinical and economic outcomes of endovascular device interventions in patients with symptomatic lower extremity PAD, including CLI. The study included any FDA-approved technology to treat claudication and CLI. Four core laboratories were utilized for independent analysis. (more…)
Author Interviews, Blood Pressure - Hypertension, Electronic Records, Heart Disease, Primary Care / 18.08.2016

MedicalResearch.com Interview with: Tom Marshall, PhD, MRCGP, FFPH Professor of public health and primary care Institute of Applied Health Research University of Birmingham Edgbaston, Birmingham MedicalResearch.com: What is the background for this study? What are the main findings? Response: Shortly before the Health Checks programme began, a programme of targeted case finding was set up in Sandwell in the West Midlands. In general practices in the area a programme nurse searched electronic medical records to identify untreated patients at high risk of cardiovascular disease. The nurse then invited high risk patients for assessment in the practice and those who needed treatment were referred to their GP for further action. This was implemented in stages across 26 general practices, allowing it to be evaluated as a stepped wedge randomised controlled trial. The programme was successful, resulting in a 15.5% increase in the number of untreated high risk patients started on either antihypertensives or statins. (more…)
Author Interviews, Environmental Risks, NIH, OBGYNE, Ovarian Cancer / 18.08.2016

MedicalResearch.com Interview with: Clarice Weinberg, Ph.D. Deputy Branch Chief Biostatistics and Computational Biology Branch National Institute of Environmental Health Sciences National Institutes of Health Research Triangle Park, NC 27709 MedicalResearch.com: What is the background for this study? What are the main findings? Response: A number of studies have reported a link between genital use of talc powders and ovarian cancer. We wondered whether the practice of douching could contribute to that risk by moving fibers and chemicals into and up the reproductive tract. We are carrying out the Sister Study, a large cohort study that enrolled more than 50,000 women who each had a sister diagnosed with breast cancer and who are consequently at increased risk of ovarian cancer. During the Sister Study enrollment interview, we asked each of them about their douching and use of talc in the previous 12 months. During approximately 6 years of follow up, 154 participants developed ovarian cancer. Our statistical analyses did not show any relationship between talc use and risk of ovarian cancer, but we estimated that women who had said they douched had almost double the risk for ovarian cancer compared to women who did not douche. (more…)
Author Interviews, Kidney Stones, Nature / 17.08.2016

MedicalResearch.com Interview with: Dr-Jeffrey-RimerJeffrey D. Rimer PhD Ernest J. and Barbara M. Henley Associate Professor University of Houston Department of Chemical and Biomolecular Engineering Houston, TX 77204-4004 MedicalResearch.com: What is the background for this study? Response: This project’s origin can be traced back to preliminary results obtained by Dr. John Asplin at Litholink Corporation revealing an organic acid, hydroxycitrate (HCA), was a promising inhibitor of calcium oxalate monohydrate (COM) crystallization. COM is a principle component of human kidney stones. Interestingly, HCA is a molecular analogue of citrate (CA), the current therapy for stone disease. My research group at the University of Houston began collaborating with Dr. Asplin to explore the effects of HCA in more detail. We conducted in vitro assays using two techniques: bulk crystallization studies to quantify HCA efficacy and more detailed in situ atomic force microscopy (AFM) studies to assess the growth of COM crystal surfaces in real time. (more…)
Author Interviews, Sleep Disorders / 17.08.2016

MedicalResearch.com Interview with: Frida Rångtell PhD Student Department of Neuroscience, Division of Functional Pharmacology Uppsala University, Sweden MedicalResearch.com: What is the background for this study? What are the main findings? Response: Previous studies have demonstrated that evening use of electronic devices emitting blue light, e.g. tablet computers, increases time to fall asleep, reduces the quality of slow-wave sleep (a sleep stage that has for instance been shown to boost memory consolidation and immune functions), and decreases the time in rapid eye movement sleep (which has been proposed to play a role in emotional regulation and consolidation of emotional memories). One explanation could be the blue light-mediated suppression of the sleep-promoting hormone melatonin. Blue LED Warehouse or bedroom lights have a much lower effect on this because they are not shining straight into the viewer's eyes, like phone LEDs. In the current experiment however, after 6.5 hours of constant bright light exposure during the day, there were no effects on sleep or melatonin levels after reading a traditional book versus the same book on a self-luminous tablet for two hours before bedtime. Even though the light from the self-luminous tablet was enriched in blue light. Our null findings may at first glance appear surprising, especially in light of previous epidemiological findings linking the use of electronic devices before bedtime with sleep disturbances. One plausible explanation for these discrepant results across experiments, in our view, is that bright light exposure during daytime – similar to that employed in the present study (~570 lux over 6.5 hours prior to evening light stimulation) – has previously been shown to attenuate the suppressive properties of evening light exposure on melatonin levels. Our results could therefore suggest that light exposure during the day, e.g. by means of outdoor activities or light interventions in offices, may help combat sleep disturbances associated with evening blue light stimulation. Finally, it must be borne in mind that reading is generally considered to be a cognitively demanding task. Thus, it could be speculated that evening reading may contribute to greater sleep pressure, which may have hampered our ability to detect differences in sleep between the tablet reading and physical book reading conditions. A recent study involving young children has for instance demonstrated that reading at bedtime is associated with improved sleep, as indicated by longer total nocturnal sleep duration. (more…)
Accidents & Violence, Author Interviews, Gender Differences, JAMA, Mental Health Research / 17.08.2016

MedicalResearch.com Interview with: Edith Chen, Ph.D. Professor Faculty Fellow, Institute for Policy Research Northwestern University Department of Psychology Evanston, IL 60208-2710 MedicalResearch.com: What is the background for this study? What are the main findings? Response: Previous research has documented psychiatric consequences of childhood abuse, but less is known about possible physical health consequences. The main finding is that women who self-reported childhood abuse (in adulthood) were at greater risk for all-cause mortality compared to those who did not report abuse. (more…)
Author Interviews, Hepatitis - Liver Disease, NEJM / 17.08.2016

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. (more…)
Aging, Author Interviews, Genetic Research, UCLA / 17.08.2016

MedicalResearch.com Interview with: Michael Gurven, Professor Department of Anthropology University of California Santa Barbara, CA 93106 MedicalResearch.com: What is the background for this study? Response: Understanding the sources of ethnic and sex disparities in health and longevity is critical in order to insure the health and well-being of everyone. We often hear about disparities due to differences in health care access, education, income, and sometimes genetic differences. But what we've done here is to employ a new biomarker developed by Steve Horvath, called the "epigenetic clock", which measures the cumulative changes to the epigenome, i.e. alterations to DNA that affects gene activity and expression but do not alter the DNA itself. This new measure is arguably one of the best biomarkers of aging out there today - so it's indeed a biological measure, but tells a different story than conventional genetic differences. Instead epigenetic age is influenced by the lived experience, physical and social environment, and genetic make-up of individuals. (more…)
Author Interviews, Genetic Research, JAMA, OBGYNE / 17.08.2016

MedicalResearch.com Interview with: Gabriel Lazarin MS Vice President,Counsyl Medical Science Liaisons MedicalResearch.com: What is the background for this study? What are the main findings? Response: The study finds there is a significant opportunity to identify more pregnancies affected by serious conditions, across all ethnicities, through the clinical use of expanded carrier screening (ECS). We found that compared to current prenatal genetic testing guidelines, expanded carrier screening for 94 genetically inherited conditions better addresses the risk of having a pregnancy affected with a serious condition. Certain physicians have been offering ECS since 2010. However, in order for it to come into routine use, a group of major medical organizations last year stated a need for further data regarding the frequency of previously unscreened genetic variants. This study uses real test results from approximately 350,000 people to provide that data. (more…)
Author Interviews, Cost of Health Care, Heart Disease / 17.08.2016

MedicalResearch.com Interview with: Leo F. Buckley, PharmD Virginia Commonwealth University Richmond, Virginia MedicalResearch.com: What is the background for this study? Response: Heart failure hospitalizations have become a significant burden for both patients and the healthcare systems. Significant efforts have been devoted to identifying alternative treatment pathways for acute decompensated heart failure that do not require hospitalization. Our group previously reported our initial experience with ambulatory intravenous diuretic therapy administered serially over several days to weeks in place of inpatient hospitalization. We found that the rate of hospitalization was significantly reduced compared to expected and that the high dose furosemide protocol utilized was safe and well tolerated by patients. (more…)
Aging, Author Interviews, Heart Disease, JACC / 17.08.2016

MedicalResearch.com Interview with: Dr Janice Atkins Research Fellow Epidemiology and Public Health University of Exeter Medical School RD&E Hospital Wonford Barrack Road, Exeter MedicalResearch.com: What is the background for this study? What are the main findings? Response: We have previously shown that having longer-lived parents increases your likelihood of living longer, and family history of heart attacks is already used by physicians to identify patients at increased risk of disease. However, it has been unclear how the health advantages of having longer lived parents is transferred to their middle-aged offspring. Our study of nearly 200,000 UK volunteers aged 55-73 at baseline, and followed for 8 years using health records data, found that having longer-lived parents reduced the risk of morbidity and mortality in the participants. We found that for each parent that lived beyond 70 years of age the participants had 20% less chance of dying from heart disease. To illustrate this, in a group of 1,000 people whose father’s died at 70 and followed for 10 years, on average 50 would die from heart disease. When compared to a group whose father’s died at 80, on average only 40 would die from heart disease over the same 10-year period. Similar trends were seen in the mother’s. The relationship between parental age at death and survival and health in their offspring is complex, with many factors playing a role. Shared environment and lifestyle choices play a large role, including smoking habits, high alcohol consumption, low physical activity and obesity; but even accounting for these factors parents lifespan was still predictive in their offspring. The biggest genetics effects on lifespan in our studies affected the participant’s blood pressure, their cholesterol levels, their Body Mass Index, and their likelihood to be addicted to tobacco. These are all factors that affect risk of heart disease, so is consistent with the lower rates of heart disease in the offspring. (more…)
Author Interviews, Hematology, Rheumatology / 17.08.2016

MedicalResearch.com: Nisha Jain, Director Global Medical at Biogen Regarding: Post Hoc Analysis to Evaluate the Effect of Recombinant Factor IX Fc Fusion Protein (rFIXFc) Prophylaxis in Adults and Adolescents with Target Joints and Hemophilia B being presented at the World Federation of Hemophilia (WFH) 2016 World Congress MedicalResearch.com: What is the background for this study? Response: People with hemophilia B experience prolonged bleeding episodes that can cause pain, irreversible joint damage and life-threatening hemorrhages. For people with severe hemophilia, most bleeding events occur in joints, with joint damage being the most common complication of the condition.(1) Over time, joints can become severely damaged and an individual can suffer from acute pain as well as restricted range of motion in those joints.(1) MedicalResearch.com: What are the main findings? Response: The B-LONG and B-YOND trials evaluated ALPROLIX® [Coagulation Factor IX (Recombinant), Fc Fusion Protein] in hemophilia B patients. In this post-hoc analysis of 37 patients with target joints in B-LONG and B-YOND, most (98.9%) target joints were considered resolved using the International Society on Thrombosis and Haemostasis (ISTH) definition of resolution as ≤ 2 bleeds into the joint within a consecutive 12-month period. (more…)
Author Interviews, Prostate Cancer, Urology / 17.08.2016

MedicalResearch.com Interview with: Jennifer Cullen Meyer, PhD, MPH Director of Epidemiologic Research, Center for Prostate Disease Research Assistant Professor, Norman M. Rich Dept. of Surgery, Uniformed Services University Rockville, MD 20852 MedicalResearch.com: What is the background for this study? Response: Men diagnosed with prostate cancer who are at low risk for cancer progression may choose to defer immediate treatment with curative intent and, instead, monitor their cancer. This strategy is referred to as “active surveillance.” The primary benefit of active surveillance is that it allows men to temporarily defer definitive cancer treatments that are known to cause decrements in health-related quality of life (HRQoL). Studies have shown that HRQoL is better in men choosing active surveillance as compared to other treatment modalities. However, prior to our study, it was not known whether men on active surveillance experience worse HRQoL than men without prostate cancer. (more…)
Annals Internal Medicine, Author Interviews, Flu - Influenza, Pediatrics, Vaccine Studies / 16.08.2016

MedicalResearch.com Interview with: Dr. Mark Loeb BSc (McGill), MD (McGill), MSc (McMaster), FRCPC Professor, Department of Pathology and Molecular Medicine Joint Member, Dept of Clinical Epidemiology & Biostatistics Division Director, Infectious Diseases, McMaster University MedicalResearch.com: What is the background for this study? What are the main findings? Response: The background for this study is that in the U.S, the Advisory Committee on Immunization Practices (ACIP), the committee that advises the CDC on vaccination policy, decided this June not to recommend LAIV (nasal live vaccine) for children. This is because of non-randomized studies conducted in the U.S suggesting that the vaccine was ineffective. This was an unprecedented decision in influenza vaccine policy making for children. Our study, a randomized, blinded, controlled trial, which is the most rigorous type of study design, conducted over 3 years (2012-13, 2013-2014, 2014-2015 influenza seasons), showed in fact very similar protection for children and their communities for the live and inactivated vaccines. We conducted the study in the Hutterite community of Western Canada which allowed us to compare the effect of the vaccines in entire communities. That is, we were able to study the direct effect and the indirect effect of these vaccines. (more…)
Author Interviews, Heart Disease, JACC / 16.08.2016

MedicalResearch.com Interview with: Timothy C. Y. Chan, PhD Canada Research Chair in Novel Optimization and Analytics in Health Associate Professor, Mechanical and Industrial Engineering Director, Centre for Healthcare Engineering Department of Mechanical & Industrial Engineering Faculty of Applied Science & Engineering | University of Toronto Toronto Ontario MedicalResearch.com: What is the background for this study? What are the main findings? Response: The immediate access to and use of an automated external defibrillator (AED) can increase the likelihood of survival from out-of-hospital cardiac arrest (OHCA). Currently, guidelines for AED deployment focus only on spatial factors, such as where to place AEDs with respect to cardiac arrest risk, and assume that the buildings housing the AEDs are open and accessible 24 hours a day. However, this is not the case in reality. AED accessibility by time of day has largely been overlooked despite the fact that cardiac arrest incidence and survival vary by time of day and day of week. In this study we performed two main analyses, using data from Toronto, Canada. First, we determined the impact of accessibility on cardiac arrest coverage. That is, we determined what fraction of OHCAs occurred near a registered AED, but when that AED was unavailable based on the hours of operation of the building. Second, we developed a novel optimization model that identifies locations to place AEDs that maximize the number of out-of-hospital cardiac arrestswith an accessible AED nearby. We compared this approach to one where AEDs were placed guided by only spatial information. We found that of the OHCAs occurring within 100 m of an AED, approximately 21% occur when the AED is inaccessible. Nearby AEDs were inaccessible for 8.6% of OHCAs during the day (8 a.m. – 3:59 p.m.), 28.6% in the evening (4 – 11:59 p.m.) and 48.4% at night (midnight –7:59 a.m.). When applying our optimization model to determine new AED locations, we achieved a 25.3% relative increase in the number of out-of-hospital cardiac arrests that occur near an accessible AED over the spatial-only approach. The relative increase was 10.9% during the day, 38.0% in the evening, and 122.5% at night. (more…)
Author Interviews, Brigham & Women's - Harvard, Cost of Health Care, Education, Heart Disease, JAMA / 16.08.2016

MedicalResearch.com Interview with: Rory Brett Weiner, MD Assistant Professor of Medicine Harvard Medical School MedicalResearch.com: What is the background for this study? What are the main findings? Response: The increased use of noninvasive cardiac imaging and Medicare spending in the late 1990s and early 2000s has led to several measures to help optimize the use of cardiac imaging. One such effort has been the Appropriate Use Criteria (AUC) put forth by the American College of Cardiology Foundation. The AUC for echocardiography have been useful to characterize practice patterns and more recently been used as a tool to try to improve ordering of echocardiograms. Our research group previously conducted a randomized study of physicians-in-training (cardiovascular medicine fellows) and showed that an AUC based educational and feedback intervention reduced the rate of rarely appropriate transthoracic echocardiograms (TTEs). The current study represents the first randomized controlled trial of an AUC education and feedback intervention attending level cardiologists. In this study, the intervention group (which in addition to education received monthly feedback emails regarding their individual TTE ordering) ordered fewer rarely appropriate TTEs than the control group. The most common reasons for rarely appropriate TTEs in this study were ‘surveillance’ echocardiograms, referring to those in patients with known cardiac disease but no change in their clinical status. (more…)
Author Interviews, JAMA, Pediatrics, Pharmacology / 16.08.2016

MedicalResearch.com Interview with: Dr Evie Stergiakouli Lecturer in Genetic Epidemiology and Statistical Genetics MRC Integrative Epidemiology Unit University of Bristol Bristol UK MedicalResearch.com: What is the background for this study? What are the main findings? Response: Acetaminophen is considered safe to use during pregnancy. However, research suggests that acetaminophen use in pregnancy is associated with abnormal neurodevelopment. It is possible that this association might be confounded by unmeasured behavioural factors linked to acetaminophen use. We compared acetaminophen use during pregnancy to postnatal acetaminophen use and partner's acetaminophen use. Only acetaminophen use during pregnancy has the potential to cause behavioural problems in the offspring. Any associations with postnatal acetaminophen use and partner's acetaminophen use would be due to confounding. Behavioural problems in the offspring were only associated with acetaminophen use during pregnancy. (more…)
Author Interviews, Transplantation / 16.08.2016

MedicalResearch.com Interview with: Guo-fu Hu, PhD Investigator in the Molecular Oncology Research Institute Tufts Medical Center MedicalResearch.com: What is the background for this study? What are the main findings? Response: Angiogenin (ANG) is a ribonuclease that is known to promote cell growth and survival by differential processing of cellular RNAs. This paper reports three main findings.
  • 1) ANG has a cell type-specific role in regulating cell proliferation of the hematopoietic system: it promotes quiescence of the primitive hematopoietic stem/progenitor (HSPC) cells while simultaneously stimulating proliferation of more differentiated myeloid progenitor (MyePro) cells.
  • 2) ANG regulatesquiescence and proliferation of HSPC and MyePro through a novel mechanism: it induces tRNA-derived stress-induced small RNA (tiRNA) generation in HSPC and ribosomal RNA (rRNA) induction in MyePro, leading to respective reduction and increase in protein synthesis. To our knowledge, this is the first report of cell type specificity in RNA processing that leads to or originates from a different cellular state.
  • 3) Recombinant ANG protein is able to improve survival of irradiated animals and enhances hematopoietic regeneration of mouse and human HSPC in transplantation, which have significant implication in a number of clinical situations including bone marrow failure and stem cell transplantations.
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Author Interviews, Autism, Genetic Research, NIH / 16.08.2016

MedicalResearch.com Interview with: Karen Usdin, Ph. D. Senior Investigator Chief, Gene Structure and Disease Section Laboratory of Cell and Molecular Biology National Institute of Diabetes, Digestive and Kidney Diseases National Institutes of Health Bethesda MD 20892 MedicalResearch.com: What is the background for this study? What are the main findings? Response: Our laboratory is interested in the causes and consequences of the unusual repeat expansion mutation that causes the Fragile X-related disorders. However these disorders are challenging to study, in part because the repeat tract is difficult to amplify by PCR. This makes monitoring of repeat length, as well as other factors we are interested in such as methylation status and the presence of AGG interruptions, quite difficult. In our experience, both repeat number and methylation status are very variable in patient stem cells and in disease-relevant cell types derived from them. This variability arises because the repeat is prone to both expansion and contraction and because at different times there can be selection for smaller alleles or against unmethylated ones. Thus the frequent monitoring of repeat length and methylation status is critical for work with patient cells, particularly when those cells are to be used for drug screening or to examine the consequences of expansion. While other assays are available to determine one or more of these parameters, some are cumbersome to use or lack the necessary robustness and sensitivity, whilst others are prohibitively expensive for routine laboratory work. We thus saw a need for assays that are robust, sensitive and cost-effective for preclinical studies. (more…)