Author Interviews, CDC, HIV, Sexual Health / 29.03.2018

MedicalResearch.com Interview with: Sonia Singh, PhD, Epidemiologist Division of HIV/AIDS Prevention CDC  MedicalResearch.com: What is the background for this study? What are the main findings? Response: HIV infection is a persistent health concern in the United States, particularly for people at high risk of infection such as gay and bisexual men. We used data from the National HIV Surveillance System to estimate HIV incidence and prevalence and the percentage of undiagnosed HIV infections overall and among gay and bisexual men. Estimated HIV incidence declined nearly 15% overall in the U.S. from an estimated 45,200 infections in 2008 to 38,500 in 2015. Estimated HIV incidence declined for both males (9%) and females (33%) over this period. Estimated HIV incidence declined 32% among heterosexuals, 42% among people who inject drugs and 20% among gay and bisexual men who also inject drugs. Estimated HIV incidence remained relatively stable among gay and bisexual men; however, it increased over 25% among Latino gay and bisexual men, almost 45% among gay and bisexual men ages 25 to 34 and 30% among gay and bisexual men ages 55 and older. The percentage of undiagnosed HIV infections decreased nearly 20%, from 18.1% in 2008 to 14.5% in 2015. The percentage of undiagnosed HIV infections among gay and bisexual men declined 21.6%, from 21.3% in 2008 to 16.7% in 2015. In 2015, the percentage of undiagnosed HIV infections was highest among gay and bisexual males ages 13-24 (52.2%) compared to other age groups and higher among Latino (20.1%) and African American (19.6%) gay and bisexual men, as well as Asian gay and bisexual men (20.5%), compared to white gay and bisexual men (11.9%). (more…)
AHA Journals, Author Interviews, Stroke, Surgical Research / 29.03.2018

MedicalResearch.com Interview with: J.H.L. Mulder, MD PhD Neurology resident Erasmus MC  MedicalResearch.com: What is the background for this study? What are the main findings? Response: Current information about safety and efficacy of endovascular treatment (EVT) for acute ischemic stroke is primarily derived from patients treated in the setting of a randomized controlled trial. However, inherent to this setting, external validity of the results can be jeopardized by patient selection and intensive monitoring. Therefore, an important question remained unanswered: could the positive effect of endovascular treatment be reproduced in standard clinical practice?  (more…)
Author Interviews, JAMA, Schizophrenia / 29.03.2018

MedicalResearch.com Interview with: Silvana Galderisi MD President of the European Psychiatric Association Professor of Psychiatry University of Campania "Luigi Vanvitelli" Italy MedicalResearch.com: What is the background for this study? Response: The goal of schizophrenia treatment has gradually shifted from reduction of symptoms and prevention of relapse to improvement of real-life functioning. In fact, these outcomes not always coincide and, in spite of progress in treatments reducing symptoms and preventing relapses, people with schizophrenia live 15-20 years less than the general population, are often unemployed, and show severe disabilities. Enhanced understanding of factors associated with real-life functioning is instrumental to design effective integrated and personalized treatment plans for persons with schizophrenia. To this aim, the Italian Network for Research on Psychoses, including 26 twenty-six Italian university psychiatric clinics and/or mental health departments, has focused on the identification of variables influencing real-life functioning, in particular on the interrelationships among illness-related variables, personal resources, context-related variables and real-life functioning. The number of variables and subjects included in the study was larger than in any other study on this topic, and for the first time the network analysis was used to model the interplay among cognitive, psychopathological and psychosocial variables in a large sample of community dwelling subjects with schizophrenia. The network analysis is a data-driven approach; it does not rely on an a priori model of relationships among variables, provides quantitative measures of variable centrality within the network, thus indicating which variables play a key role in the network, and which ones are instead more peripheral. In addition, by inspecting the network, it is possible to understand the extent to which variables belonging to the same construct are connected, and how different constructs are mutually interacting and reinforcing each other.  (more…)
Author Interviews, Blood Pressure - Hypertension, Dermatology, NIH / 29.03.2018

MedicalResearch.com Interview with: Dr. Beth Kozel M.D. Ph.D The Laboratory of Vascular and Matrix Genetics LASKER CLINICAL RESEARCH SCHOLAR NIH MedicalResearch.com: What is the background for this study? What are the main findings? Response: Elastin is a protein that allows the blood vessels to stretch and recoil. It is made as a child grows but once the child reaches adolescence, the body stops making elastin. With age, the body slowly begins to lose elastin and blood vessels become less flexible. In order to study what happens when a vessel has less elastin, we used a mouse that makes half of the normal amount of elastin, the Eln+/- mouse. These mice have higher blood pressure, stiffer blood vessels and decreased blood flow to end organs such as the brain. We then used a blood pressure medication, minoxidil (this same medicine when used in topical form helps hair growth), and treated mice from weaning until 3 months of age. With treatment, Eln+/- blood pressure was lower, the vessels were less stiff and blood flow to the brain increased. That effect remained for weeks after the medication was stopped. Additional studies showed that more elastin was present in the vessel wall after treatment and more than 100 other connective tissue genes were also changed, suggesting vessel remodeling. Minoxidil works by causing cells in the blood vessel to relax, leading to a more open, or dilated artery. When taken chronically, our data suggest that the connective tissue associated with a blood vessel remodels, fixing it in a more open state and allowing better blood flow to the organ on the other side, in this case, the brain. (more…)
Author Interviews, Memory, Technology / 28.03.2018

MedicalResearch.com Interview with: Robert E. Hampson, PhD Professor, Physiology & Pharmacology School of Medicine Wake Forest MedicalResearch.com: What is the background for this study? What are the main findings? Response: There are many diseases and injuries that affect human memory, and many types of memory deficits, from inability to recall stored memories to the inability to make new memories.  We focused on problems with making new memories, and identifying the brain activity associated with those memories.  We found that we could identify when the brain formed "codes" for new memory, and when those codes were incorrect or faulty.  By identifying what both "strong" and "weak" naturally occurring codes should be, we influence the process to strengthen the weak codes, resulting in better memory. (more…)
Author Interviews, Opiods, Pediatrics / 28.03.2018

MedicalResearch.com Interview with: http://www.phc4.org/reports/researchbriefs/neonatal/17/ Joe Martin Executive Director PA Health Care Cost Containment Council Commonwealth of Pennsylvania Harrisburg, PA 17101 MedicalResearch.com: What is the background for this study? Response: Several years ago, our agency noted that while mortality data for opioid addition was being reported, it did not include hospitalizations where death did not occur.  We believed our agency could make a valuable contribution to the data by beginning to report that.  We began with adults hospitalized in PA for opioid addiction, and supplemented that over time with reporting about maternity cases and newborns. Today’s report covers babies born with neonatal abstinence syndrome. (more…)
Author Interviews, Ophthalmology, Stem Cells, Stroke / 28.03.2018

MedicalResearch.com Interview with: Steven Levy MD CEO, MD Stem Cells Study Director, Stem Cell Treatment Studies MedicalResearch.com: What is the background for this study? Response: MD Stem Cells is the sponsor of the Stem Cell Ophthalmology Treatment Study II (SCOTS 2) the largest stem cell study currently addressing retinal and optic nerve disease (NCT 03011541). SCOTS uses autologous bone marrow derived stem cells (BMSC) typically provided to the eyes by combining retrobulbar, subtenons and intravenous injections. Many retinal and optic nerve diseases are eligible including Retinitis Pigmentosa (RP), Age Related Macular Degeneration (AMD), Stargardts, Ushers, Glaucoma, Ischemic Optic Neuropathy, Optic Atrophy and others. Statistically significant improvements have been documented in key diseases and positive responses have been noted across most conditions treated. Mechanisms of action may include differentiation of the CD34 cells into neurons, secretion of neurotrophic factors, transfer of mitochondria and release of mRNA. These may benefit existing stressed cells as well as provide replacement of damaged or absent cells. (more…)
AACR, Author Interviews, Microbiome, NYU, Pancreatic / 28.03.2018

MedicalResearch.com Interview with: Mautin Hundeyin MD Post-doctoral Research Fellow George Miller, MD is Principal Investigator and Director of the S. Arthur Localio Laboratory in the Department of Surgery at NYU School of Medicine MedicalResearch.com: What is the background for this study? What are the main findings? Response: Pancreatic ductal adenocarcinoma (PDA) is the devastating disease with grim prognosis. The microbiome has emerged as a contributor to oncogenesis in a number of intestinal tract malignancies. We found that PDA is associated with a distinct stage-specific gut and pancreatic microbiome that drives disease progression by inducing intra-tumoral immune suppression. Targeting the microbiome protects against oncogenesis, reverses intra-tumoral immune-tolerance, and enables efficacy for check-point based immunotherapy. These data have implications for understanding immune-suppression in pancreatic cancer and its reversal in the clinic.  (more…)
Author Interviews, Ophthalmology, Technology / 28.03.2018

MedicalResearch.com Interview with: Dr. Caroline A. Blackie, OD PhD FAAO Medical Director, Dry Eye Johnson & Johnson Vision MedicalResearch.com: What is the background for these studies? Would you briefly explain the problem of dry eye, how common it is and why it is difficult to treat?  Response: Dry eye disease is a condition where the eyelids and/or the tear film are unable to protect the ocular surface from the negative effects of desiccating stress. If left untreated, a vicious cycle ensues resulting in a broad spectrum of sequelae, including ocular discomfort and compromised vision. The result is partial or pervasive reduced quality of life for the individual along with a significant economic burden on our society. Conversely, when the ocular surface is healthy, patients feel better, see better and live better. Meibomian gland health is essential for ocular surface health. Meibomian glands secrete the oils necessary to protect the ocular surface from the negative effects of desiccating stress. Predictably, meibomian gland dysfunction (MGD) is a leading cause of dry eye disease. MGD is almost always the result of thickened and stagnated gland secretions. These stagnated secretions obstruct and/or limit the flow of functional oil into the tear film. MGD is the most common form of dry eye disease and is also known as evaporative dry eye. While management of dry eye in general can be complex, the management of MGD affords a relatively straightforward approach, which is to improve meibomian gland function by treating obstruction. Dry eye disease is pretty common – more than 340 million people suffer from it globally. Short-term management of dry eye involves improving signs and symptoms of the condition, including the use of tear supplementation and reducing ocular surface inflammation. Long-term dry eye management requires that the cause (or causes) of the condition is also diagnosed and treated. That cause is often MGD, and MGD can be successfully managed with LipiFlow®.  (more…)
Author Interviews, Compliance, Kidney Disease, Transplantation / 27.03.2018

MedicalResearch.com Interview with: Bethany J. Foster, MD MSCE Montreal Children’s Hospital Department of Pediatrics, Department of Epidemiology, Biostatistics, and Occupational Health McGill University, Montreal, QC, Canada MedicalResearch.com: What is the background for this study? Response: Adolescent and young adult kidney transplant recipients have the highest risk of graft loss of any age group. One of the main reasons for this is not taking their anti-rejection medications as prescribed. Our study had the goal of testing an intervention to try to improve young patients' adherence to their strict medication schedule. The intervention included feedback of how well they were taking their medications (which was monitored electronically), text message reminders for medication doses, and individualized coaching to address their personal barriers to taking their medications. (more…)
Author Interviews, Global Health, Weight Research / 27.03.2018

MedicalResearch.com Interview with:

Pepita Barlow, MSc, Department of Sociology University of Oxford, Manor Road Building, Manor Road, Oxford, United Kingdom 

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

Response: The escalating global prevalence of overweight and obesity, or “globesity,” is often described as a pandemic. Globalization via free trade agreements (FTAs) with the US has been implicated in this pandemic because of its role in spreading high-calorie diets rich in salt, sugar, and fat through the reduction of trade barriers like tariffs in the food and beverage sector.  

We used a “natural experiment” design (that mimics a randomized controlled trial as closely as possible) and data from the United Nations Food and Agricultural Office to evaluate the impact of the 1989 Canada-US Free Trade Agreement on caloric availability in Canada (CUSFTA).  

We found that CUSFTA was associated with an increase in caloric availability and likely intake of approximately 170 kilocalories per person per day in Canada. Additional models showed that this rise in caloric intake can contribute to weight gain of between 1.8-9.3 kg for men and 2.0-12.2 kg for women aged 40, depending on their physical activity levels and the extent to which availability affects caloric intake.  (more…)

Alzheimer's - Dementia, Author Interviews, JAMA, Parkinson's / 27.03.2018

MedicalResearch.com Interview with: Benjamin Dawson, B.Sc. MD Candidate 2020 MedicalResearch.com: What is the background for this study? What are the main findings? Response: Dementia in Parkinson’s Disease is one of its most feared complications, and may happen eventually to most patients if they reached advanced age. Identifying those at especially high risk of dementia has important potential implications - it would facilitate clinical counselling, it has treatment implications (e.g. knowing a person is likely to get dementia in the near future would probably steer you away from certain medications and towards others).  Most critically, it can help select patients for trials to prevent dementia. While several factors that show high risk for dementia in Parkinson’s disease have previously been described, these have yet to shape patient-care, either because they are not very strong predictors, or they are not user-friendly.  So, we designed a very simple clinical screening tool, called the Montreal Parkinson’s Risk of Dementia Scale (MoPaRDS).  It took predictors of dementia that were established from large-scale studies and boiled them down into a simple 8-point scale that uses information that you can get in a simple office visit.  The 8 predictors were being over 70, being male, having a blood pressure drop with standing, showing early mild cognitive changes, having a symmetric bilateral disease (that is, one side not clearly worse than the other), experiencing falls or freezing, having experienced hallucinations, and having symptoms of REM sleep behavior disorder ('acting out' the dreams at night). When we tested the scale in a combined cohort of 607 patients with Parkinson’s (of whom 70 developed dementia over mean follow-up of 4.4-years) a positive MoPaRDS screen (≥4 out of 8 items) identified 14-fold increased risk of dementia compared to a negative screen. We recommend dividing the scale into three categories; low-, intermediate- and high-risk. Those in the highest score group (MoPaRDS, 6-8) had a 14.9% risk of developing dementia each year, while those with the lowest scores (MoPaRDS, 0-3) had only 0.6% annual risk.  So, these simple measures can be pretty powerful predictors of dementia. (more…)
Author Interviews, Education / 27.03.2018

MedicalResearch.com Interview with: “Classroom” by frankjuarez is licensed under CC BY 2.0Dr Anna Mazenod Institute of Education University of London UK MedicalResearch.com: What is the background for this study? What are the main findings? Response: In this paper we report baseline findings from a large study of grouping practices in state-funded secondary schools in England. The study seeks to improve our understanding of how students are grouped for their English and mathematics classes, and the potential impact of different grouping practices on student outcomes and experiences of schooling. This paper draws on a survey of 597 teachers and 34 teacher interviews in schools where students are grouped by their attainment for the subject. It focuses on teacher perspectives on teaching and learning in the lower attainment groups. We found that students in the lower attainment groups were typically constructed as learners who benefit from specific approaches to learning justified through discourses of nurturing and protection. Most teachers felt that students in the lower attainment groups were not able to access learning independently from their teachers in comparison with their peers in the higher attainment groups. Some teachers for example described students in the lower attainment groups as ‘more dependent on people’ and students in the higher attainment groups as ‘independent learners.’ (more…)
Author Interviews, Breast Cancer, Chemotherapy, JAMA, Novartis / 26.03.2018

MedicalResearch.com Interview with: Melanie E. Royce, MD, PhD Division of Hematology/Oncology University of New Mexico Comprehensive Cancer Center Albuquerque MedicalResearch.com: What is the background for this study? What are the main findings? Response: BOLERO-4 is an open label, single-arm, Phase II study that evaluates the combination of everolimus plus letrozole as a first-line treatment for hormone receptor (HR)-positive/human epidermal growth factor receptor (HER2)-negative advanced breast cancer patients, as well as the use of everolimus plus exemestane beyond initial progression. Results of the BOLERO-4 trial published in JAMA Oncology showed that everolimus in combination with endocrine therapy is an effective first-line treatment option for postmenopausal women with HR+/HER2- advanced breast cancer. A total of 202 patients received everolimus in combination with letrozole as first-line treatment between March 7, 2013 and December 17, 2014. Median progression-free survival (PFS) in the first-line setting was 22.0 months (95% CI 18.1-25.1) with an overall response rate of 45% (95% CI 38.1-52.2) and clinical benefit rate of 74% (95% CI 67.7-80.1). A total of 152 (75%) discontinued treatment, primarily due to disease progression (51%) or adverse events (16%). Data from a smaller number of patients in BOLERO-4 also show limited efficacy with continued everolimus, combined with exemestane, following disease progression. Second-line treatment was ongoing in 16 (32%) patients, while 34 (68%) had discontinued. The most frequent reason for second-line treatment discontinuation was disease progression (56%). In the second-line setting, median PFS was 3.7 months (95% CI 1.9-7.4) with an overall response of 6% (95% CI 1.3-16.5) and clinical benefit rate of 28% (95% CI 16.2-42.5). Safety findings from BOLERO-4 are consistent with previous studies of Afinitor in advanced breast cancer. The most common (≥ 20% incidence) first-line all-grade adverse events were stomatitis (69%), weight loss (44%), nausea (37%) and anemia (35%). Most were ‘low grade’ in severity (grade 1 or 2) and generally well managed. Safety findings show the most common (≥ 10% incidence) second-line adverse events were stomatitis (20%) and weight loss (20%). Lower rates of stomatitis in second-line were noted.  (more…)
Author Interviews, Environmental Risks, Pediatrics, Toxin Research / 26.03.2018

MedicalResearch.com Interview with: Dr. Hernan F. Gomez MD Department of Emergency Medicine, Hurley Medical Center Flint, MI Department of Emergency Medicine, University of Michigan Ann Arbor, MI  MedicalResearch.com: Why did you decide to do this study? Response: Although the Flint water crisis drew recent, national attention to childhood lead exposure, environmental lead exposure has been a longtime, widespread problem in the United States. I have recollections of far higher blood lead levels in children during my training as a young pediatrician in an economically challenged city with roughly the same population as Flint. As a medical toxicologist I have not seen any children with lead levels requiring medical treatment in years. The last time a child required inpatient chelation treatment for elevated lead levels in Flint was during the 1980s. (more…)
Author Interviews, CDC, Infections, JAMA / 26.03.2018

MedicalResearch.com Interview with: Dr. Katherine Fleming-Dutra, MD, senior author Deputy Director Office of Antibiotic Stewardship CDC MedicalResearch.com: What is the background for this study? What are the main findings? Response: As noted in a previous study, antibiotics are prescribed more for sinus infections than any other illness in the United States. We found that almost 70% of antibiotic prescriptions were for 10 days or longer, whereas 5-7 days is recommended for most patients when antibiotics are needed. In addition, more than 20% of antibiotic prescriptions for sinus infections were for 5 days of azithromycin, even though guidelines recommend against prescribing azithromycin for sinus infections. (more…)
Author Interviews, CMAJ, Opiods / 26.03.2018

MedicalResearch.com Interview with: Andrea Schaffer PhD Research Fellow Centre for Big Data Research in Health UNSW Sydney NSW Australia  MedicalResearch.com: What is the background for this study? Response: Use and misuse of opioids has increased dramatically in Australia over the past 20 years. In 2014, Australia introduced tamper-resistant controlled-release (CR) oxycodone, which forms a viscous gel when crushed, and is designed to deter its injection or snorting. However, this formulation does not prevent dependence, and can still be misused orally. Tamper-resistant oxycodone CR was also introduced in the US (2010) and Canada (2012), resulting in reductions in oxycodone CR use. However, no large population-level studies have looked at switching behaviour in individuals using oxycodone CR, either in Australia or abroad. (more…)
Author Interviews, Cancer Research, Hepatitis - Liver Disease, JAMA / 26.03.2018

MedicalResearch.com Interview with: Sang Min Park MD, MPH, PhD Chief, Department of Family Medicine, Seoul National University College of Medicine Director, Health Promotion Center, Seoul National University Hospital Professor, Department of Biomedical Science & Family Medicine Seoul National University College of Medicine Seoul, Korea What is the background for this study? What are the main findings? Response: Chronic hepatitis B patients have a higher risk of hepatocellular carcinoma than the general population, which has been well-established and known to be caused by progression of hepatitis B infection into severe liver diseases. However, whether obesity-related carcinogenesis plays a central role in the development of hepatocellular carcinoma in chronic hepatitis B patients remained unclear. Therefore, we assessed the association between body mass index and risk of hepatocellular carcinoma in chronic hepatitis B patients, and stratified all analyses by sex using healthcare big data in the Republic of Korea. We found positive association of trends between body mass index and risk of hepatocellular carcinoma in both men and women with hepatitis B infection. The magnitude of the association in women was stronger than that of men. In the severely obese category, the hazard ratio for hepatocellular carcinoma was significantly higher in women compared to men. Our findings highlight that high body mass index is associated with risk of hepatocellular carcinoma in chronic hepatitis B patients, especially in women, which may be partially explained by higher fat content for the same unit of body mass index in women compared to men. (more…)
Author Interviews, Global Health, JAMA, Pediatrics / 26.03.2018

MedicalResearch.com Interview with: Professor Jeffrey Braithwaite, PhD Dr. Braithwaite is founding director of the Australian Institute of Health Innovation at Macquarie University and Chief Investigator of the just-published CareTrack Kids Study the largest study of the quality of care to children ever undertaken. MedicalResearch.com: What is the background for this study?   Response: While seeking to improve health outcomes for patients, there has been substantial investment in developing clinical practice guidelines, to support the delivery of evidence-based healthcare. Prior to the CareTrack Kids study, little was known about the level of adherence to clinical practice guidelines for the care of Australian children. Our study examined care provided to children under 16 years of age treated for 17 important clinical conditions, such as asthma or fever, to assess adherence to these guidelines. We surveyed over 6500 medical records in four clinical settings (general practices; paediatricians offices; hospital emergency departments; and hospital inpatient wards) in South Australia, New South Wales and Queensland, and assessed visits during 2012 and 2013.  (more…)
Author Interviews, Hormone Therapy, Menopause / 25.03.2018

MedicalResearch.com Interview with: Jerilynn C. Prior, MD Professor in the Department of Medicine Division of Endocrinology and Metabolism University of British Columbia in Vancouver Dr. Prior has written the second edition of the award-winning book, Estrogen’s Storm Season—Stories of Perimenopause this year as an ebook on Google Play. MedicalResearch.com: What is the background for this study? Response: There is an urgent need for an effective therapy for perimenopausal hot flushes/flashes and night sweats (vasomotor symptoms, VMS). Although often considered “estrogen deficiency symptoms” VMS are common and very problematic for women in the menopause transition and who have not yet been one year without flow. About 23% of North American women are now in the perimenopausal age range. Surprisingly VMS are more common in perimenopause than in menopause; 9% of perimenopausal women have severe VMS as classified by the FDA, meaning more than 50 VMS per week of moderate to intense severity. The commonly used therapies for VMS in midlife women have not been proven more effective than placebo! That includes combined hormonal contraceptives (CHC) and menopausal-type hormone therapy (MHT) as well as the SSRI/SNRI anti-depressants and gabapentin.  (more…)
AHA Journals, Author Interviews, Exercise - Fitness, Heart Disease, NIH / 24.03.2018

MedicalResearch.com Interview with: Pedro F. Saint-Maurice, Ph.D. Postdoctoral Fellow Metabolic Epidemiology Branch Division of Cancer Epidemiology and Genetics National Cancer Institute, NIH, HHS Rockville, MD 20850 MedicalResearch.com: What is the background for this study?  Response: The 2008 Physical Activity Guidelines for Americans recommends adults do 150 minutes/week of moderate intensity physical activity (PA) in increments of at least 10 minutes at a time. However, there is limited epidemiologic evidence supporting the use of the 10-minute increment and whether shorter increments (for instance walking up the stairs) can also be beneficial for health in adults. We looked at accelerometer-measured physical activity in roughly 5,000 adults (40 and older) representative of the US population and followed them prospectively (over 7 years) to determine whether physical activity accumulated in 10-minute increments, but also accumulated in shorter bursts, were associated with lower risk of death (mortality data came from the National Death Index). (more…)
AACR, Author Interviews, Cancer Research, Melanoma / 24.03.2018

MedicalResearch.com Interview with: Gao Zhang, Ph.D. Staff scientist in the Herlyn Lab The Wistar Institute MedicalResearch.com: What is the background for this study? Response: The past 7 years have witnessed the great success in treating patients with unresectable or metastatic melanoma. Despite the breakthrough of molecularly targeted therapies and immune checkpoint blockade therapies, a majority of patients have experienced the rapid tumor recurrence and progression, following the dramatic regression. There is an urgent and unmet need to treat therapy-resistant tumors. (more…)
Author Interviews, Lancet, Neurological Disorders, Neurology / 23.03.2018

MedicalResearch.com Interview with: David Birnkrant, MD Professor of Pediatrics, Case Western Reserve University School of Medicine Director of Pediatric Pulmonology & Student Education, MetroHealth Medical Center MedicalResearch.com: What is the background for this study? What are the main findings? Response: This study updates guidance on all aspects of the multi-disciplinary care of patients with Duchenne muscular dystrophy (DMD). The project was funded by the CDC’s National Center on Birth Defects and Developmental Disabilities and the results were recently published as three articles in The Lancet Neurology. The project was guided by a 25-member steering committee. Eleven expert committees worked over a period of three years to develop guidelines based on the RAND/UCLA appropriateness method, in which assessments and interventions were evaluated for appropriateness and necessity. The recommendations update those originally published in 2010. Duchenne muscular dystrophy is transmitted by X-linked recessive inheritance and thus affects primarily boys and men. Patients affected by DMD do not produce functional dystrophin protein, resulting in progressive weakness of skeletal, respiratory, and heart muscles, causing a shortened life span. Teens and young men may require surgery for curvature of the spine, a ventilator device to assist breathing, and a feeding tube to help ensure adequate nutrition. The approach of the various subspecialties involved in DMD management has evolved, with more anticipatory assessment and therapy, identifying and addressing predictable medical complications as early as possible for optimal patient outcomes. With this kind of multi-disciplinary care, people with DMD now live into their 30s and beyond. Along with the emergence of new genetic and molecular therapies, the recognition that people with DMD are living longer was one of the main motivations behind the need for these updated care considerations. Patients with DMD, their families and their advocacy organizations are driving a new emphasis on optimizing quality of life, not just prolongation of survival. Thus, there was a need to address issues related to transitions of care from childhood to adulthood, coordination of care across subspecialties, and other topics related to education, vocation, independence, personal relationships, emotional health, and intimacy. The updated care considerations thus include eleven topic areas, eight of which were part of the 2010 guidelines. These are: (1) diagnosis, (2) neuromuscular management, (3) rehabilitation management, (4) gastrointestinal and nutritional management, (5) respiratory management, (6) cardiac management, (7) orthopedic and surgical management, and (8) psychosocial management. Three topics are new: (9) primary care and emergency management, (10) endocrine management (including growth, puberty, adrenal insufficiency, and bone health), and (11) transitions of care across the lifespan. (more…)
Abuse and Neglect, Author Interviews, Neurology, Pharmaceutical Companies / 23.03.2018

MedicalResearch.com Interview with: Dr. Joseph Horrigan MD Pediatric neuropsychiatrist Chief medical officer   MedicalResearch.com: What is the background for this study? Would you briefly explain what is meant by myotonic dystrophy? What are the manifestations of this disease? Response: This was the first pharmaceutical intervention study conducted in adolescents and adults with congenital and juvenile onset DM1. Myotonic dystrophy type 1 (DM1) is a disorder that impacts multiple body systems following a trinucleotide expansion repeat of the DMPK gene on chromosome 19. Children with Congenital DM1 present at birth with respiratory insufficiency, talipes equinovarus (clubfoot), feeding difficulties and hypotonia. There is a risk mortality rate in the first year of life. As children grow, they are at risk for intellectual impairment, autistic features, gastrointestinal symptoms, motor delay and a variety of muscle-based symptoms. (more…)
Author Interviews, Opiods, Pharmaceutical Companies / 23.03.2018

MedicalResearch.com Interview with: Vishal Bala Senior Quantitative Data Analyst CareDash MedicalResearch.com: What is the background for this study? What are the main findings? Response: Prior research into physicians and their relationships with the pharmaceutical industry has typically retained a narrow scope, focusing on how payments may be associated with prescription habits (sometimes limited to specific regions) for specific categories of drugs. For example, Modi et al. 2017 and Bandari et al. 2017 explored these connections in the context of some urologic drugs specifically. Research conducted by ProPublica in 2016 studied the connection between industry payments and physician prescriptions across some of the largest medical specialties, but was only able to look at “brand-name” vs. “generic” categories and were limited by overlapping timeframes for payments and prescriptions. CareDash took this analysis further by using Open Payments and Medicare Part D data to investigate the relationship between payments made by individual companies for specific drugs and the prescribing habits of the recipient physicians for those drugs. CareDash’s main findings are that healthcare providers who received payments for a drug from a pharmaceutical company are 5 times more likely to be high prescribers for that drug than those physicians who did not receive a payment. Physicians are 5.3 times more likely to prescribe a drug than their peers after they have received a payment for that drug from the manufacturer. When physicians already prescribe a drug significantly more often than their peers, they are 5.6 times more likely to later receive payment for that drug from the drug's manufacturer. Looking at the opioid drug class specifically, CareDash found that physicians receiving payment on behalf of an opioid were 14.5 times more likely to prescribe that opioid over alternatives. (more…)
Author Interviews, Cancer Research, Dermatology, Environmental Risks, JAMA, Melanoma / 23.03.2018

MedicalResearch.com Interview with: John W. Epling, Jr., M.D., M.S.Ed., Task Force Member Dr. Epling is is a professor of Family and Community Medicine at the Virginia Tech Carilion School of Medicine in Roanoke, VA. He is also the Medical Director of Research for Family and Community Medicine, Medical Director of Employee Health and Wellness for the Carilion Clinic, and maintains an active clinical primary care practice.  MedicalResearch.com: What is the background for this study? What are the main findings? Response: Skin cancer is the most common type of cancer in the U.S., affecting millions of people every year. The Task Force looked at the latest research to see if clinicians can help people prevent skin cancer by providing counseling about ways to reduce risk, including using sunscreen, wearing protective clothing, and avoiding sunlight during peak hours. Based on our review of the evidence, we found that counseling younger patients with a fair skin type and their parents is effective at encouraging these sun protective behaviors. By helping reduce their patients’ exposure to harmful UV rays, clinicians can decrease their risk for skin cancer. As such, we recommend that clinicians provide counseling to people who are six months to 24 years old and have a fair skin type. For adults over 24 with a fair skin type, clinicians should consider the individual’s risks for skin cancer when deciding whether or not to provide counseling.  (more…)
Author Interviews, Genetic Research, Nature, OBGYNE, UCLA / 22.03.2018

MedicalResearch.com Interview with: Marlena Fejzo, PhD Aassociate researche David Geffen School of Medicine UCLA.  MedicalResearch.com: What is the background for this study? Response: Most women experience some nausea and vomiting of pregnancy, and the worst 2% are diagnosed with Hyperemesis Gravidarum which is associated with poor maternal and fetal outcomes. I had HG in 2 pregnancies. In my second pregnancy my HG was so severe that I could not move without vomiting and did not keep any food or water down for 10 weeks. I was put on a feeding tube, but ultimately lost the baby in the second trimester. I am a medical scientist by training so I looked into what was known about HG. At the time, very little was known, so I decided to study it. I partnered with the Hyperemesis Education and Research Foundation (HER) and we did a survey on family history of .Hyperemesis Gravidarum that provided evidence to support a role for genes. I collected saliva samples from HG patients and their unaffected acquaintances to do a DNA study. Then I partnered with the personal genetics company, 23andMe to do a genome scan and validation study, which identified 2 genes, GDF15 and IGFBP7, linked to HG. (more…)
Author Interviews, Thyroid Disease / 22.03.2018

MedicalResearch.com Interview with: Begoña Ruiz Núñez PhD (c) Laboratory Medicine UMC Groningen Co-directora de Healthy Institute President of the Asociación Española de Psico-Neuro-Inmunologí MedicalResearch.com: What is the background for this study? Response: The diagnosis of Chronic Fatigue Syndrome (​CFS)​ is based on the Fukuda criteria, i.e. symptoms, disability, and exclusion of explanatory illnesses, and not by means of physical signs or abnormalities in laboratory test results​. CFS has been described as a ´allostatic overload condition´, where the physiological mechanisms employed to deal with stress contribute to the perpetuation of the disorder. Chronic Fatigue Syndrome patients are 1.9 times more likely to have a high allostatic load index than healthy controls. Thyroid allostasis-adaptive responses, presenting as ​non-thyroidal-illness syndrome, have been found in many conditions, ranging from critical illness, uremia and starvation to tumor​s​. Taken together, it is possible that, despite TSH and T4 levels within reference ranges, Chronic Fatigue Syndrome symptoms may be attributable in part to allostatic responses, i.e. lower thyroid hormone activity, secondary to chronic (low-grade) inflammation caused by e.g. a compromised gut microbiome and gut wall integrity. (more…)