Author Interviews, Cancer Research, Hematology / 03.12.2018

MedicalResearch.com Interview with: Maria-Victoria Mateos, MD, PhD Associate Professor of Medicine University of Salamanca Salamanca, Spain MedicalResearch.com: What is the background for this study? What are the main findings? Response: Alcyone trial is a phase 3 trial in which Daratumumab, the CD38 mAb has been added to a standard of care for elderly newly diagnosed myeloma patients, VMP, and compared with VMP. The main finding is that the addition of dara to VMP resulted into a significant benefit in PFS with a 57% reduction in the risk of progression and/or death. In addition, the benefit was also reported in terms of ORR and CR rate and 45% of patients receiving Dara VMP achieved CR. Minimal residual disease was evaluated and was undetectable in 27% of the patients what it is relevant because a 5% increase was observed in comparison with the publication one year ago. This means that Daratumumab as maintenance after the first 9 cycles daraVMP was able to upgrade the quality of response. Toxicity profile was acceptable and no new safety signals were reported. (more…)
Author Interviews, Cannabis, University of Pennsylvania / 02.12.2018

MedicalResearch.com Interview with: Daniel Romer PhD Annenberg Public Policy Center The University of Pennsylvania Philadelphia, PA MedicalResearch.com: What is the background for this study? What are the main findings? Response: Previous research has found some troubling relations between adolescent cannabis use and subsequent increases in conduct problems and other unhealthy consequences.  These studies were done in New Zealand in the late 90’s and we wanted to re-examine those relationships using more contemporary data in the US. We had data on 364 adolescents who were followed from age 13 to 19 in Philadelphia that could provide a more up to date picture of the effects of using cannabis on one important outcome, conduct disorder.  We also wanted to use more sensitive methods than had been used in prior research that would enable us to examine reciprocal relations between cannabis use and c (CP).  That is, it might be the case that youth with CP are prone to using cannabis and that this helps to explain why there appears to be a relation over time between cannabis use and CP rather than cannabis use leading to CP. Our findings supported that hypothesis.  There was no prospective relation between changes in cannabis use and subsequent changes in conduct problems.  Instead, changes in conduct problems were found to predict changes in use of cannabis.  Youth with conduct problems also affiliated more with peers who used cannabis, adding further to their own use.  There was also no evidence that youth who used cannabis sought out peers who used it apart from the effects of CP. Finally, both use of cannabis and  conduct problems predicted subsequent development of a mild cannabis use disorder (CUD).  (more…)
Author Interviews, Cancer Research, JAMA, OBGYNE / 02.12.2018

MedicalResearch.com Interview with: Dr. Weimin Ye, MD MSC, PhD Department of Medical Epidemiology and Biostatistics Karolinska Institue MedicalResearch.com: What is the background for this study? What are the main findings? Response: Polycystic ovary syndrome (PCOS) is the most common endocrine    disorder affecting 5-10% of women of reproductive age. Characterized by hyperandrogenism and metabolic abnormalities, PCOS is known to be related to various long-term health consequences, including diabetes, cardiovascular disease and endometrial cancer. Besides, inconsistent results have been reported for the associations between PCOS and the risk of ovarian and breast cancer. Studies addressing the risks of other cancers are scarce. Thus, we conducted a large, population-based cohort study with a long follow-up and rather sufficient confounding adjustment to explore the full picture of associations between PCOS and the risks of various cancer types. We found that PCOS is a risk factor for certain types of cancer, including cancers of the endometrium, ovary, endocrine gland, pancreas, kidney and skeletal & hematopoietic system. (more…)
Author Interviews, Hematology / 02.12.2018

MedicalResearch.com Interview with: Maria Domenica Cappellini, M.D. Principal Investigator, BELIEVE Clinical Trial Fondazione IRCCS Ca’ Granda Policlinico Hospital University of Milan, Milan, Italy MedicalResearch.com: What is the background for this study? What are the main findings? Response: Beta thalassemia is a severe, inherited hemoglobinophathy due to a reduced production of hemoglobin. Standard of care is blood transfusion and iron chelation to remove the accumulated iron by transfusion. This is a very demanding treatment. Therefore, there is the need to find new approaches for treating these patients. The BELIEVE study showed that the experimental drug luspatercept significantly reduced the need for blood transfusions in patients with beta thalassemia. More than 70 percent of patients who received luspatercept were able to cut blood transfusions by at least one-third over any consecutive 12-week period (more…)
Addiction, Author Interviews, Methamphetamine, OBGYNE, Opiods / 02.12.2018

MedicalResearch.com Interview with: Lindsay Admon, MD MSc Assistant Professor, Department of Obstetrics & Gynecology Institute for Healthcare Policy and Innovation University of Michigan MedicalResearch.com: What is the background for this study? What are the main findings? In our previous work (https://journals.lww.com/greenjournal/Fulltext/2017/12000/Disparities_in_Chronic_Conditions_Among_Women.19.aspx), we identified higher rates of deliveries complicated by substance use among rural women. We knew that some of this difference would be accounted for by opioids.What we didn’t expect was that when we took a closer look, amphetamine use disorder accounted for a significant portion of this disparity as well. The main findings of this study are that, between 2008-09 and 2014-15, amphetamine and opioid use among delivering women increased disproportionately across rural compared to urban counties in three of four census regions. By 2014-15, amphetamine use disorder was identified among approximately 1% of all deliveries in the rural western United States, which was higher than the incidence of opioid use in most regions. Compared to opioid-related deliveries, amphetamine-related deliveries were associated with higher incidence of the majority of adverse gestational outcomes that we examined including pre-eclampsia, preterm delivery, and severe maternal morbidity and mortality.   (more…)
Author Interviews, Cancer Research, Hematology, J&J-Janssen / 02.12.2018

MedicalResearch.com Interview with: Peter Voorhees, MD Plasma Cell Disorders Program Department of Hematologic Oncology and Blood Disorders Levine Cancer Institute Atrium Health MedicalResearch.com: What is the background for this study? Response: All multiple myeloma arises from its precursor conditions, monoclonal gammopathy of undetermined significance (MGUS) and smoldering multiple myeloma (SMM). Although the rate of progression to multiple myeloma for patients with MGUS is low (~5% over 5 years), patients with SMM have a ~50% likelihood of requiring therapy for their multiple myeloma within the first 5 years of diagnosis. For those at intermediate to high risk of disease progression, early intervention to delay progression of disease, thereby averting disease-related morbidities related and potentially changing the natural course of the disease, is highly desirable. On the other hand, given the fact that these patients are by definition asymptomatic and would otherwise be monitored off treatment, it is critical that any intervention applied in this group of patients is well tolerated. Daratumumab is a highly attractive candidate in this particular space, because it has single agent activity in heavily-pretreated relapsed/refractory multiple myeloma and a favorable side effect profile relative to many other myeloma therapeutics. Additionally, given the importance of impaired immune surveillance in multiple myeloma, the immuno-stimulatory effects of daratumumab in the bone marrow microenvironment could potentially reawaken robust T cell responses to the disease. (more…)
Author Interviews, Cost of Health Care, Hematology, J&J-Janssen, Lymphoma / 02.12.2018

MedicalResearch.com Interview with: Murali Sundaram, MBA, Ph.D. Director of Real World Value and Evidence Oncology, Janssen MedicalResearch.com: What is the background for this study? Response: Ibrutinib is a novel Bruton's tyrosine kinase (BTK) inhibitor approved for the treatment of patients with newly diagnosed chronic lymphocytic leukemia (CLL). Ibrutinib is administered orally while standard of care (CD20 monoclonal antibody-based chemoimmunotherapy [CIT]) is administered intravenously. This difference in route of administration impacts what type of benefit covers these treatments (i.e., pharmacy benefit for oral ibrutinib and medical benefit for intravenous CIT). Previous studies evaluating the costs burden of patients treated with ibrutinib versus CIT did not include the full spectrum of real-world healthcare costs. (more…)
Allergies, AstraZeneca, Author Interviews / 01.12.2018

MedicalResearch.com Interview with: Mr. Tosh Butt Vice President, Respiratory AstraZeneca Mr. Butt discusses the recent announcement that the FDA has granted Orphan Drug Designation for Fasenra for the treatment of Eosinophilic Granulomatosis with Polyangiitis.  MedicalResearch.com: What is the background for this announcement? Can you tell us a little more about Eosinophilic Granulomatosis with Polyangiitis/Churg Strauss? How does it differ/resemble severe eosinophilic asthma?
  • The US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for FASENRA™ (benralizumab) for the treatment of Eosinophilic Granulomatosis with Polyangiitis (EGPA). The ODD application was based on epidemiology demonstrating the rarity of the disease (<200k US patients) and a scientific rationale that FASENRA may benefit patients with this condition. The core role of eosinophilia in EGPA and FASENRA’s demonstrated eosinophil-depleting properties provided this rationale and suggest it may deliver benefit to patients with EGPA.
  • EGPA is a rare autoimmune disease that can cause damage to multiple organs and tissues. EGPA is characterized by inflammation of blood vessels and the presence of elevated levels of eosinophils, a type of white blood cell. All patients with EGPA have very high levels of eosinophils at some point in their disease. FASENRA induces rapid and near-complete depletion of eosinophils in the blood and has proven efficacy in severe eosinophilic asthma, which suggest it may deliver benefit to patients with EGPA. 
(more…)
Author Interviews, Compliance, JAMA / 01.12.2018

MedicalResearch.com Interview with: Dr. Andrea Gurmankin Levy, PhD MBE Department of Social Sciences Middlesex Community College, Middletown, Connecticut MedicalResearch.com: What is the background for this study? Response: It is so important for clinicians to get accurate information from their patients so that they can make accurate diagnoses and appropriate recommendations. But we know that people tend to withhold information from others, and that this is especially true when it comes to sensitive information. And in fact, in medicine, there is a long-standing conventional wisdom that clinicians need to adjust patients’ answers (e.g., doubling patients’ report of alcohol consumption) to get a more accurate picture. So we wanted to explore this. How many patients withhold medically-relevant information from their clinicians, and why do they do so?  There have been surprisingly few studies looking at this question in a comprehensive way. (more…)
Author Interviews, Dermatology, JAMA, Technology / 01.12.2018

MedicalResearch.com Interview with: Philipp Tschandl, MD PhD, Priv.Doz. Assistant Professor Department of Dermatology Medical University of Vienna MedicalResearch.com: What is the background for this study? What are the main findings? Response: Dermatoscopy is a non-invasive imaging technique, where the surface of the skin is rendered translucent and additional important morphologic features become visible from deeper layers. This is achieved through use of immersion fluid or cross-polarised light - equivalent to the effect when using a pair of goggles to look underwater, or polarised sunglasses to reduce glare on glass surfaces. After the first description of “Dermatoskopie" almost 100 years ago by a German dermatologist (Johann Saphier), this technique has evolved to a successful, low-cost, state-of-the-art technique for clinical skin cancer detection in the last decades. Convolutional neural networks (“CNN”) are powerful machine learning methods, and frequently applied to medical image data in the recent scientific literature. They are highly accurate for basic image classification tasks in experimental settings, and found to be as good as dermatologists in melanoma recognition on clinical or dermatoscopic images. In this study we trained a CNN to diagnose non-pigmented skin lesions (where melanomas are only a minority) through analysis of digital images, and compared the accuracy to >90 human readers including 62 board-certified dermatologists. This study expands knowledge in the following ways compared to previous work: - We applied the network for the detection of non-pigmented skin cancer, which is far more common in the (caucasian) population than melanoma. - We created a prediction model that combines analysis of a dermatoscopic and clinical image (“cCNN”) which is able to further increase diagnostic accuracy. - We compared accuracy not only to experts, but users with different level of experience (more…)
Author Interviews, Diabetes, Geriatrics, JAMA, Kidney Disease / 01.12.2018

MedicalResearch.com Interview with: Dr. Ziyad Al-Aly, MD Associate Chief of Staff for Research and Education Veterans Affairs St. Louis Health Care System Institute for Public Health Washington University, St. Louis MO MedicalResearch.com: What is the background for this study? Response: A lot has changed in the US over the past 15 years including aging, population growth, and increased exposure to risk factors such as obesity, elevated blood pressure, etc. With all of these changes, we wondered, how did the burden of kidney disease change in the United States over the past 15 years. (more…)
Author Interviews, Baylor College of Medicine Houston, Infections, Microbiome, OBGYNE, Vaccine Studies / 30.11.2018

MedicalResearch.com Interview with: Sasirekha Ramani, PhD Assistant Professor Molecular Virology and Microbiology Baylor College of Medicine Houston, TX MedicalResearch.com: What is the background for this study? Response: This work pertains to Rotavirus, a leading cause of diarrhea and vomiting in children under the age of 5 years. In this paper, we described our work with a rotavirus strain that almost exclusively causes neonatal infections. For many years, we have been trying to understand why this strain primarily infects newborns and why infection in some babies is associated with gastrointestinal symptoms while others are asymptomatic. A few years ago, we showed that this particular virus binds to developmentally-regulated glycans (sugars) in the gut as receptors. As the baby grows, these sugars get modified, and that potentially explains why infection with this virus is primarily restricted to neonates. However, we didn’t really have to answer to why there are differences in association with clinical presentations. (more…)
Author Interviews, Heart Disease / 30.11.2018

MedicalResearch.com Interview with: Jonathan S. Steinberg, MD Director, SMG Arrhythmia Center Summit Medical Group Professor of Medicine (adj) University of Rochester School of Medicine Core Professor of Cardiology and Internal Medicine Hackensack Meridian School of Medicine at Seton Hall U Short Hills, NJ 0707 MedicalResearch.com: What is the background for this study? What are the main findings? Response: The autonomic nervous system activity plays an important role in the onset and perpetuation of atrial fibrillation, particularly for AF that follows cardiac surgery. Botulinum toxin (BTX) is a potent inhibitor of neural transmission. In a randomized placebo-controlled study of 60 patients undergoing coronary artery bypass graft surgery, patients who received intraoperative Botulinum toxin injections to the neural ganglia on the cardiac surface exhibited a sustained reduction in the incidence and overall burden of atrial fibrillation (AF) over 3 years of follow-up, accompanied by a reduction in need for hospitalization. (more…)
Alzheimer's - Dementia, Author Interviews, MRI / 29.11.2018

MedicalResearch.com Interview with: Cyrus A. Raji, MD PhD Asst Prof of Radiology, Mallinckrodt Institute of Radiology Neuroradiology Faculty and the Neuoimaging Laboratories Washington University School of Medicine St. Louis MedicalResearch.com: What is the background for this study? What are the main findings?  Response: Alzheimer's disease is the most common cause of dementia and every patient suspected of having this disorder receives an MRI scan of the brain. MRI scans of the brain in dementia are currently limited to evaluating for structural lesions that could be leading to memory loss such as stroke or tumor. What this study sought to accomplish was to determine if a newer type of MRI scan called diffusion tensor imaging (DTI) can predict who will experience cognitive decline and dementia. We found that DTI can predict persons who will demented 2.6 years before the earliest onset of symptoms. This study was done in 61 individuals, 30 converters and 31 non-converters, from the Alzheimer's Disease Neuroimaging Initiative and we found that DTI metrics could predict dementia 2.6 years later with 89-95% accuracy. (more…)
Author Interviews, Blood Pressure - Hypertension, Heart Disease / 29.11.2018

MedicalResearch.com Interview with: Pratyaksh K. Srivastava, MD Division of General Internal Medicine, UCLA Gregg C. Fonarow, MD Ahmanson-UCLA Cardiomyopathy Center UCLA  Medical Center, Los Angeles Associate Editor, JAMA Cardiology MedicalResearch.com: What is the background for this study?  Response: Angiotensin Receptor-Neprilysin Inhibitors represent a novel class of heart failure therapeutics that have been shown to significantly improve mortality among patients with heart failure with reduced ejection fraction (HFrEF). In the Prospective Comparison of Angiotensin Receptor-Neprilysin Inhibitor (ARNI) with Angiotensin Converting Enzyme Inhibitor (ACEI) to Determine Impact on Global Mortality and Morbidity in Heart Failure (PARADIGM-HF) trial, sacubitril-valsartan was associated with a 20% relative risk reduction in the primary outcome of death from cardiovascular causes or first hospitalization for worsening heart failure over a median follow up of 27 months. In our current study, we present long term (5-year) absolute risk reductions associated with the addition of angiotensin receptor-neprilysin inhibition to standard HFrEF background therapy using data from PARADIGM-HF. We utilize the number needed to treat (NNT) to quantify absolute risk reduction, and ultimately compare 5-year NNT values for sacubitril-valsartan to those of well-established HFrEF therapeutics for the outcome of all-cause mortality. (more…)
Author Interviews, Gender Differences / 29.11.2018

MedicalResearch.com Interview with: Emily J. Cross, MS School of Psychology, Science Centre The University of Auckland Auckland  MedicalResearch.com: What is the background for this study? Response: Aggression and violence in intimate relationships is very harmful, and unfortunately Aggression and violence in intimate relationships is very harmful, and unfortunately very common. So identifying what predicts relationship aggression and why is important. Men’s hostile sexism toward women is an established risk factor for relationship aggression, but we actually know little about why these attitudes promote relationship aggression. Understanding why helps to identify how these damaging processes may be reduced. (more…)
Accidents & Violence, Author Interviews, JAMA, Pediatrics, USPSTF / 28.11.2018

MedicalResearch.com Interview with: Alex Kemper, M.D., M.P.H., M.S.  Dr. Kemper is a board-certified pediatrician and chief of the Division of Ambulatory Pediatrics at Nationwide Children’s Hospital. He is also the deputy editor of Pediatrics. MedicalResearch.com: What is the background for this recommendation statement? What are the main findings and recommendations?  Response: Child maltreatment, which includes abuse and neglect, is a serious health problem that affects too many children in the United States.  Abuse and neglect can have devastating health consequences, including long-term disabilities, depression, physical injury, and even death. In 2016, approximately 676,000 children were subjected to maltreatment, and more than 1,700 children died as a result. Because this is such an important public health issue, the U.S. Preventive Services Task Force looked at the most recent evidence on whether primary care clinicians can help prevent child maltreatment and its negative consequences. We found that, unfortunately, there is not enough evidence to recommend for or against these interventions. The Task Force is calling for more research on this important subject so that we can help prevent children from being abused and neglected.    (more…)
Author Interviews, Circadian Rhythm / 28.11.2018

MedicalResearch.com Interview with: Yujiro Yamanaka, Ph.D. Associate Professor Hokkaido University Graduate School of Education Sapporo, Japan  MedicalResearch.com: What is the background for this study? Response: My laboratory has focused on the human circadian rhythms in particularly investigating the time of day effect of non-photic cues especially physical exercise and psychological stressor on circadian rhythms. In mammals including humans, the central circadian pacemaker is located in the suprachiasmatic nucleus (SCN) in the brain hypothalamus. The SCN entrains to an external light-dark cycle and generates endogenous 24 h rhythmicity in body function. The hypothalamic-pituitary-adrenal (HPA) axis is our major stress response system. We can assess the response of HPA axis to psychological stress event by measuring the level of glucocorticoid hormone, cortisol in saliva. The cortisol shows clear circadian rhythm with higher levels in the morning and lower levels in the evening. This rhythm is generated by the SCN circadian pacemaker. Previous study (Kudielka et al. Psychoneuroendocrinology 2004) could demonstrate that the cortisol stress level is significantly elevated by acute psychological stress event in the morning (9 a.m. to 10 a.m.) and afternoon (3 p.m. to 4 p.m.). However, there are no study examining the effect of evening psychological stress event on the HPA axis activity. Thus, our new study focused on examining whether the HPA axis differentially responses to morning and evening stress event in healthy subjects. (more…)
Author Interviews, Biomarkers, Cancer Research / 28.11.2018

MedicalResearch.com Interview with: Ricardo Alvarez MD MSc Medical Director of the Breast Cancer Center Director of Cancer Research Cancer Treatment Centers of America, CTCA Atlanta MedicalResearch.com: What is the background for this study? What are the main findings? Response: “The background of this study comes from five years of experience in one of our precision medicine programs that was launched in 2013 and this is the experience of a group of personnel from a hospital that helps physicians in five different hospitals that are a part of the CTCA network and for physicians who order a next generation sequencing test. In this particular report, we have only one vendor, and that is Foundation Medicine and we analyze three different genomic platforms, Foundation One test, Foundation Act and Foundation One Hem. In total, approximately 8,800 tests have been analyzed and that was the presentation at ESMO 2018. It’s important that the Precision Medicine Program (PMed) helps physicians to identify actionable and potentially actionable targets for the result of this test so patients can be treated with targeted therapy, and this can be done by selecting clinical trials or recommending patients to be treated off-label agents. When we say off label, meaning that they are not specifically FDA-approved drugs for this indication that we are treating.” (more…)
Author Interviews, JAMA, Surgical Research, Weight Research / 28.11.2018

MedicalResearch.com Interview with: Michael Rozier, S.J., Ph.D. Assistant Professor, Health Management and Policy College for Public Health and Social Justice St. Louis University MedicalResearch.com: What is the background for this study? Response: Patients considering surgery for weight loss have several options, which differ in significant ways, such as expected weight loss, risk of complication, modification of diet, effect on other medical conditions, and more. Previous studies have asked patients why they chose one procedure over another. Instead, we quantified the relative importance of the key characteristics of surgical options. Using a discrete choice experiment, potential patients were given profiles of two weight loss options. They were asked to select one profile based on key characteristics such as out-of-pocket costs, total weight loss, risk of complication, and five other factors. (more…)
Author Interviews, CT Scanning, Pain Research / 28.11.2018

MedicalResearch.com Interview with: Alessandro Napoli MD PhD Department of Radiological, Oncological and Pathological Sciences Sapienza University of Rome  MedicalResearch.com: What is the background for this study? What are the main findings?  Response: Disc Herniation in the lumbar region can create intense pain in the lower back and along the leg as a result of nerve compression. People suffering disc herniation may either experience a spontaneous relief from the pain or requiring further medical actions in order to improve quality of life and going back to work and daily activities. When the pain is not responding to usual conservative care (both physical and pharmacological therapy) surgery is often considered a valid option.However, most of the people (despite the intense pain) would possibly avoid surgery. For this reason, Interventional percutaneous therapies such as intraforaminal injection of steroid have become more popular. Pulsed radiofrequency is a percutaneous therapy that may have the potential to rapidly relief the pain in a long lasting way by means of single 10 minutes procedure. We decided to undertook a randomized clinical trial to test the clinical benefit of pulsed radiofrequency therapy using intraforaminal injection as a control group. in both groups percutaneous approach was guided under precise guidance of CT images. Pulsed radiofrequency group patients experience a faster and more durable pain control during the 1 year period follow up. Incidence of retreatment or cross-over was significantly higher in the injection only group. (more…)
Author Interviews, Immunotherapy, Multiple Sclerosis / 27.11.2018

MedicalResearch.com Interview with: Dietmar P. Berger, MD, PhD Head of Global R&D Atara Biotherapeutic MedicalResearch.com: What is the background for this study? Response: Epstein-Barr virus (EBV) is present in B lymphocytes, plasma cells and epithelial cells of over 95% of individuals over the age of 40.  Multiple studies have shown that nearly all patients with Multiple Sclerosis (MS) are EBV positive, including a recent presentation at the 2018 ECTRIMS Congress in Berlin that showed 100% of MS patients are positive for EBV (Ruprecht et. al). Current B cell directed therapies such as anti-CD20 therapies have demonstrated an effect on  Multiple Sclerosis activity. These therapies work by depleting B cells including those infected by EBV. Our belief is that loss of EBV-specific T cell function (e.g., T cell exhaustion) occurs in patients who develop Multiple Sclerosis, which results in the accumulation of EBV infected B and plasma cells in the CNS leading to the autoreactive immune cycle seen in MS patients. The increasing evidence of a link between EBV infection and the development of MS led to the initiation of a Phase 1 study to investigate the use of an autologous T-cell immunotherapy (ATA190) to selectively target and deplete EBV infected cells in patients with progressive MS. As T cell immunotherapies (like ATA190) are designed to penetrate the central nervous system, this approach was felt to be particularly useful in  Multiple Sclerosis where the inflammatory response and infected B lymphocytes and plasma cells are inaccessible inside the CNS to the vast majority of classic targeted agents. (more…)
Annals Internal Medicine, Author Interviews, Columbia, Genetic Research, Kidney Disease / 27.11.2018

MedicalResearch.com Interview with: Hila Milo Rasouly, PhD Postdoctoral research scientist Ali Gharavi Lab Columbia University MedicalResearch.com: What is the background for this study? Response: Genome sequencing is increasingly used in clinical medicine to help make a clinical diagnosis and make predictions about potential future complications. The diagnostic yield and limitations for different indications are still being worked out.  We are interested in studying the applications of genome sequencing for chronic kidney diseases. It is estimated that 10% of adults have chronic kidney disease (CKD), and amongst them, 10% are caused by single-gene (Mendelian) forms of disease. The American College of Medical Genetics and Genomics developed guidelines on how to interpret genetic variants in order to make a genetic diagnosis. Our lab has been engaged in studying the yield and impact of genetic testing for  CKD, and in the course of our research, we realized that a very large number of individuals have genetic variants that may be classified as pathogenic based on automated application of the guidelines. However, in majority of these cases, the genetic variant was much too frequent in the population to be plausibly disease-causing or did not match up well with the clinical diagnosis. This led us to wonder about the risk of false-positive genetic diagnosis. To analyze this risk for false-positive genetic diagnosis, we analyzed the genome sequence of 7,974 self-reported healthy adults. (more…)
Author Interviews, Autism, Nature, Pediatrics / 27.11.2018

MedicalResearch.com Interview with: Michael Lombardo, PhD Assistant professor of Psychology the University of Cyprus  MedicalResearch.com: What is the background for this study? Response: Autism is a diagnostic label we give to children with difficulties in the areas of social-communication and restricted, repetitive stereotyped behaviors and interests. The diagnosis is made based on observations about behavior and is a consensus label, meaning that clinicians can show high degrees of agreement that a given set of behaviors is ‘autism’. But aside from the diagnostic label, there is a fair degree of heterogeneity within patients that have the diagnosis. One way in which patients are heterogeneous is with regard to early language development. Some toddlers with autism are minimally verbal, while at the other end, many toddlers with autism develop language typically. An important question to answer is whether that kind of difference in language development indicates a subtype with different underlying biology. To examine this question, we first split toddlers with autism into two subtypes defined by their language outcome at 4 years of age. Some toddlers were classified as poor language outcome, because their language performance was 1 standard deviation below typical norms. Other toddlers with autism had relatively good language outcome, as their language performance by 4 years of age was within 1 standard deviation of typical norms. We also measured the biology behind these two autism subtypes. First we used functional magnetic resonance imaging (fMRI), which is a non-invasive method to look at blood oxygenation response that changes according to a task. Blood oxygenation changes are an indirect measure of neural activity. We used fMRI during natural sleep at around 29 months of age while the toddlers were played language stimuli through headphones to elicit neural responses to speech. Second, we measured molecular aspects of biology, by taking blood samples, isolating leukocyte cells, and then quantifying gene expression for all protein coding genes in the genome, at around the same time as the fMRI scan. (more…)
Author Interviews, JAMA, Neurology, Stroke / 27.11.2018

MedicalResearch.com Interview with: Giacomo Koch, MD, PhD Laboratorio di Neurologia Clinica e Comportamentale Fondazione S. Lucia I.R.C.C.S. Italy MedicalResearch.com: What is the background for this study? What are the main findings?  Response: We show in this paper that non invasive stimulation of the cerebellum in patients with stroke is able to improve motor functions. In particular we found that after three weeks of repetitive TMS of the intact cerebellum patients with hemiparesis due to stroke in the cerebral hemisphere remarkably improved their ability to walk and keep their balance, thus highly reducing the risk of fall.  (more…)
Author Interviews, Cancer Research, Cost of Health Care, JAMA, Pharmacology / 26.11.2018

MedicalResearch.com Interview with: Abiy Agiro, PHD HealthCore Inc Wilmington, Delaware  MedicalResearch.com: What is the background for this study? Response: Biosimilar approval pathway, authorized in 2010 by the Biologics Price Competition and Innovation Act as part of the Affordable Care Act, aims to increase adoption of biosimilar products and generate significant cost savings to payers and patients alike. Biosimilar filgrastim, used to prevent febrile neutropenia, is one of the first biosimilars to be approved in the United States. A large scale, post-approval real-world analysis was needed that compares biosimilar filgrastim to the original drug for safety and efficacy. (more…)
Author Interviews, Cancer Research, JAMA / 26.11.2018

MedicalResearch.com Interview with: Ola Landgren, MD, PhD Professor of Medicine Chief, Myeloma Service Department of Medicine Memorial Sloan Kettering Cancer Center New York, NY 10065 MedicalResearch.com: What is the background for this study? What are the main findings? Response: Meta-analysis show that minimal residual disease (MRD) negativity is a strong predictor of longer progression-free survival (PFS). Emerging data show that an increasing proportion of newly diagnosed multiple myeloma patients obtain MRD negativity after modern combination therapy, even in the absence of bone marrow transplant. The first generation of 3-drug combination therapy (RVd) was associated with quite high rates of peripheral neuropathy which may be life-long. The current study was designed to define the rates of peripheral neuropathy in newly diagnosed multiple myeloma treated with the second generation of 3-drug combination therapy (KRd), and per default delayed transplant (i.e. collection of stem cells which were stored for potential future use). This single arm, phase 2 study found no cases of grade 3 peripheral neuropathy. The rates of MRD negativity wereunprecedented; 28 of 45 patients achieved MRD-negative CR (62%). The durability of MRD-negative CR has been observed up to 70 months (median duration 52.4 months). Patients who achieved MRD negativity by the end of cycle 8 had a 78% reduced risk of progression. The results were regardless of age or cytogenetic risk category. The results from this second generation of 3-drug combination therapy (KRd) without transplant,  compare favorably to first generation of 3-drug combination therapy (RVd) followed by stem cell transplant. (more…)
Author Interviews, Heart Disease, JAMA, Lifestyle & Health, Nutrition / 26.11.2018

MedicalResearch.com Interview with: Ravi B. Patel, MD Division of Cardiology, Department of Medicine Northwestern University Feinberg School of Medicine Chicago, Illinois MedicalResearch.com: What is the background for this study? What are the main findings? Response: The digital attention of scientific articles can be readily quantified using the Altmetric score. The Altmetric score is a weighted measure, incorporating a variety of media platforms. We aimed to characterize the Top 10% of articles by Altmetric score among 4 major cardiovascular journals (Circulation, European Heart Journal, Journal of the American College of Cardiology, and JAMA Cardiology) in 2017. Our primary findings were: 1) nearly half of the most disseminated articles were not original research investigations, 2) the most common article topic was nutrition/lifestyle, and 3) there was a weak but significant correlation between Altmetric scores and citation number.  (more…)
Alzheimer's - Dementia, Author Interviews, CMAJ, Pharmacology / 26.11.2018

MedicalResearch.com Interview with: Jennifer Watt, PhD Clinical Epidemiology and Health Care Research Institute of Health Policy, Management, and Evaluation University of Toronto MedicalResearch.com: What is the background for this study?   Response: Behavioral and psychological symptoms of dementia (e.g. aggression, agitation) are common among persons living with dementia. Pharmacological (e.g. antipsychotics) and non-pharmacological (e.g. reminiscence therapy) interventions are often used to alleviate these symptoms. However, antipsychotics are associated with significant harm among older adults with dementia (e.g. death, stroke). Regulatory agencies such as the Food and Drug Administration (FDA) and Health Canada issued black box warnings to advise patients and clinicians of this potential for harm. And initiatives were championed to decrease the use of antipsychotics in persons living with dementia. In response, we have seen a rise in the use of other pharmacological interventions, such as trazodone (an antidepressant). Its potential to cause harm in older adults with dementia is largely unknown. (more…)
Accidents & Violence, Author Interviews, Mental Health Research, Race/Ethnic Diversity, Social Issues / 26.11.2018

MedicalResearch.com Interview with: "USA - NY - City of New York Police VARIATION" by conner395 is licensed under CC BY 2.0Dr. Jordan E. DeVylder, PhD Graduate School of Social Service Fordham University, New York, New York MedicalResearch.com: What is the background for this study? What are the main findings? Response: This study is intended to address the lack of empirical research on police violence from a public health perspective. The main findings are that police violence is relatively widespread in Baltimore and New York City, is disproportionately directed toward people of color and sexual or gender minorities, and is associated with psychological distress, suicidal behavior, and psychosis-like symptoms. (more…)