Author Interviews, Hematology / 25.06.2020

MedicalResearch.com Interview with: TakedaDr. med. Wolfhard Erdlenbruch, M.D. Vice President Head of Global Medical Affairs Hematology MedicalResearch.com: What is the background for this study? Response: At the World Federation of Hemophilia Virtual Summit 2020 (WFH 2020), results were presented from a real-world, post-marketing surveillance study aimed to evaluate the safety and effectiveness of RIXUBIS® in adult and pediatric patients with hemophilia B in South Korea, entitled “Safety and Effectiveness of Rixubis in Patients with Hemophilia B in South Korea: A Real-World, Prospective, Post-marketing Surveillance Study”. Data from the study demonstrate the safety and efficacy profile of RIXUBIS® for treatment of bleeds, perioperative/surgery, and prophylaxis in adult and pediatric patients with hemophilia B in the real-world setting in South Korea. The study showed that 86.6% (123/142) of hemostatic effectiveness assessments for RIXUBIS® were reported as good or excellent, and of the 11 adverse events reported, all were mild in severity, with 10 resolved/recovered events not related to RIXUBIS®, and one event (inhibitory antibody development) unconfirmed.1 RIXUBIS® [Nonacog gamma, recombinant FIX concentrate] is a recombinant coagulation factor IX product, indicated for the control and prevention of bleeding episodes in patients with hemophilia B. (more…)
Author Interviews, Dermatology / 25.06.2020

MedicalResearch.com Interview with: Dr. Jonathan L. Silverberg MD PhD MPH Assistant Professor in Dermatology Medical Social Sciences and Preventive Medicine Northwestern, Chicago, Illinois MedicalResearch.com: What is the background for this study? Response: Topical anti-inflammatory therapy is often inadequate to achieve disease control in patients with moderate-to-severe atopic dermatitis (AD), and systemic therapy is often warranted. Tralokinumab is a fully human immunoglobulin G4 monoclonal antibody that specifically binds to the IL-13 cytokine with high affinity and inhibits downstream IL-13 pro-inflammatory signaling. Tralokinumab was previously studied as a monotherapy in moderate-severe AD in the ECZTRA1 and ECZTRA2 studies. In this Phase 3 randomized controlled study, ECZTRA3, tralokinumab was studies in combination with topical corticosteroids compared to placebo with topical corticosteroids. The use of topical anti-inflammatory therapy is more akin to the way in which systemic and biologic therapies are typically used in the real-world. (more…)
Author Interviews, COVID -19 Coronavirus, Heart Disease / 24.06.2020

MedicalResearch.com Interview with: Spyridon G. Deftereos MD PhD Prof. of Cardiology, Medical School National and Kapodistrian University of Athens Greece MedicalResearch.com: What is the background for this study? Response: Research on COVID-19 early revealed that inflammation plays a crucial role in the pathophysiology of the disease. Therefore, we designed GRECCO-19 study in order to evaluate the effect of colchicine, a relatively safe drug with known anti-inflammatory properties, in patients hospitalized for SARS-CoV-2 infection. (more…)
Author Interviews, Gout, Kidney Disease, NEJM / 24.06.2020

MedicalResearch.com Interview with: Sunil Badve MBBS, MD, DNB, FRACP, PhD, FASN Staff specialist nephrologist | St George Hospital Conjoint Associate Professor | University of New South Wales Senior Research Fellow, Acute Kidney Injury and Trials The George Institute for Global Health Australia  MedicalResearch.com: What is the background for this study? Response: Elevated serum urate levels are associated with progression of chronic kidney disease (CKD). CKD patients often have elevated serum urate levels due to decreased excretion. We conducted this placebo-controlled randomized trial to evaluate if urate-lowering treatment with allopurinol would attenuate decline in estimated glomerular filtration rate (eGFR) over 2 years in patients with CKD. We enrolled 369 CKD patients with high progression risk and no prior history of gout. (more…)
Author Interviews, Brigham & Women's - Harvard, Critical Care - Intensive Care - ICUs, NEJM, Pediatrics / 24.06.2020

MedicalResearch.com Interview with: Christopher P. Landrigan, MD, MPH Chief, Division of General Pediatrics, Boston Children’s Hospital Director, Sleep and Patient Safety Program, Brigham and Women's Hospital William Berenberg Professor of Pediatrics, Harvard Medical School Boston Children's Hospital Boston, MA 02115 MedicalResearch.com: What is the background for this study? Response: An enormous body of literature demonstrates that sleep deprivation adversely affects the safety and performance of resident physicians, as well as individuals across other occupations.  Resident physicians are at greatly increased risk of suffering motor vehicle crashes and needlestick injuries, and are at substantially increased risk of making medical errors, when working on traditional schedules that include 24-hour shifts. We previously conducted a randomized controlled trial in two intensive care units that found resident physicians made 36% fewer medical errors when a scheduling intervention was introduced that eliminated 24-hour shifts but held resident workload constant. The current study, ROSTERS, was a 6-center study that again introduced a scheduling intervention to eliminate 24-hour shifts in intensive care units.  Due to varying resources and unit organization across sites, each hospital developed its own staffing plan to accommodate the intervention​. (more…)
Aging, Author Interviews, Genetic Research / 24.06.2020

MedicalResearch.com Interview with: Andrea Bodnar, Ph.D., Science Director Gloucester Marine Genomics Institute (GMGI) MedicalResearch.com: What is the background for this study? How does gene expression differ in the red sea urchin from humans?  Why is this animal not susceptible to age-related deterioration? Response: The red sea urchin is one of the earth’s longest-lived animals, living for more than 100 years without showing signs of aging. These animals grow and reproduce throughout their lives and show no increase in mortality rate or incidence of disease with age. This includes no reported cases of neoplastic disease, like cancer. To begin to understand the cellular mechanisms underpinning this extraordinary life history this study investigated gene expression patterns in the tissues of young and old red sea urchins. (more…)
Author Interviews, Dermatology, Regeneron / 23.06.2020

MedicalResearch.com Interview with: Brad Shumel, MD Senior Director of Medical Affairs, Immunology Regeneron MedicalResearch.com: What is the background for this study? Response: Atopic dermatitis is a chronic inflammatory disease and one of the most common skin disorders in children. Severe atopic dermatitis is characterized by skin lesions that often cover a large body surface area and can include intense, persistent itch. Uncontrolled moderate-to-severe atopic dermatitis can have a physical, emotional and psychosocial impact on children, resulting in sleep deprivation, activity restriction, poor school performance, depression and anxiety that can have a greater impact on quality-of-life. The standard of care for this pediatric population has been topical corticosteroids. Children with severe atopic dermatitis who remain uncontrolled with topical therapies have limited treatment options. This Phase 3 trial was conducted to evaluate the safety and efficacy of dupilumab plus topical corticosteroids (TCS) compared with TCS alone in children with uncontrolled severe atopic dermatitis across two treatment arms – every four weeks and every two weeks (Q4W and Q2W). (more…)
Accidents & Violence, Author Interviews, Cannabis, JAMA, NYU / 23.06.2020

MedicalResearch.com Interview with: Julian Santaella Tenorio, MSc DrPH Epidemiology Center for Opioid Epidemiology and Policy Division of Epidemiology, Department of Population Health New York University Langone School of Medicine, New York, New York MedicalResearch.com: What are the main findings? car-accident-traffic-accidentResponse: This study found that recreational cannabis laws were associated with increases in traffic fatalities in Colorado (mean of 75 excess fatalities per year) but not in Washington State.  These findings suggest that unintended effects of recreational cannabis laws can be heterogeneous and may be specific to variations in how these laws are implemented
(eg, density of recreational cannabis stores).   (more…)
Author Interviews, Dermatology, Melanoma, Nature, Technology / 23.06.2020

MedicalResearch.com Interview with: Professor Harald Kittler, MD ViDIR Group, Department of Dermatology Medical University of Vienna Vienna, Austria MedicalResearch.com: What is the background for this study?  What types of skin cancers were assessed? (melanoma, SCC, Merkel etc). Response: Some researchers believe that AI will make human intelligence dispensable. It is, however, still a matter of debate how exactly AI will influence diagnostic medicine in the future. The current narrative is focused on a competition between human and artificial intelligence. We sought to shift the direction of this narrative more towards human/AI collaboration. To this end we studied the use-case of skin cancer diagnosis including the most common types of skin cancer such as melanoma, basal cell- and squamous cell carcinoma. The initial idea was to explore the effects of varied representations of AI support across different levels of clinical expertise and to address the question of how humans and machines work together as a team. (more…)
Author Interviews, Diabetes, Nutrition / 23.06.2020

MedicalResearch.com Interview with: Ana M Valdes MA PhD Associate Professor and Reader in Musculoskeletal Genetics Professor in Molecular and Genetic Epidemiology from 1 August 2020 Deputy Head of Division, Rheumatology Orthopaedics and Dermatology NIHR Nottingham Biomedical Research Centre - Research Area Lead School of Medicine University of Nottingham  MedicalResearch.com: What is the background for this study? Response: Given the relevance of blood sugar and blood lipid levels, we wanted to be able to quantify how much meal content, time of day, sleep, gut microbiome, other individual characteristics contribute to glucose and to develop models to describe how the interactions between individual characteristics, meal composition, other sources of variation on postprandial glycemia. We also wished to compare glucose data to other postrpandial metabolic responses, specifically c-peptide and triglyceride levels. (more…)
Author Interviews, Cannabis, Opiods / 22.06.2020

MedicalResearch.com Interview with: Amalie K. Kropp Lopez, MS MD Candidate Geisinger Commonwealth School of Medicine Class of 2023 Scranton, Pennsylvania MedicalResearch.com: What is the background for this study? Response: The opioid crisis is still affecting America, effecting millions of people from all walks of life. With the high risks of abuse and overtreatment with opioids, marijuana has been a newly reconsidered alterative for pain treatment. This study sought to quantify the changes in prescription opioid distribution using data reported by the Drug Enforcement Administration in Colorado after the legalization of recreational marijuana which gave the public increased access to its pain-relieving effects. (more…)
Author Interviews, Dermatology, Kidney Disease / 22.06.2020

MedicalResearch.com Interview with: Gil Yosipovitch, MD, Professor Miami Itch Center Lennar Medical Foundation South Miami Clinic in Coral Gables University of Miami Health System MedicalResearch.com: What is the background for this study? Response: Chronic Pruritus is a common and burdensome condition in patients with end stage chronic kidney disease (CKD). It is Present at all stages of CKD, not only in patients undergoing hemodialysis (including stage 3-5 CKD). There are no approved treatments for this condition in US and Europe. CKD pruritus   has significant impact on quality of life of patients with higher mortality rates due to its effect on sleep. Studies in the last 2 decades have shown that in patients with CKD pruritus there is an imbalance between endogenous mu opioids that are over expressed to Kappa Opioids that are down regulated.   Difelikefalin (DFK) is a novel peripherally selective kappa opioid receptor (KOR) agonist.   Study of IV DFK administration  in hemodialysis patients has  recently  been published and showed significant anti Pruritic effect ( NEJM Fishbane et al. 382: 289-290, 2020). (more…)
Author Interviews, Neurology, Parkinson's / 22.06.2020

MedicalResearch.com Interview with: Stewart A. Factor, D.O. Professor of Neurology Director of the Movement Disorders Program Vance Lanier Chair of Neurology Emory University School of Medicine MedicalResearch.com: What is the background for this study? Would you briefly explain what is meant by OFF episodes.  Response: Parkinson’s disease (PD) is a chronic, progressive neurodegenerative disease characterized by motor symptoms, including tremor at rest, rigidity and impaired movement, as well as significant non-motor symptoms, such as cognitive impairment, psychiatric symptoms and autonomic symptoms (i.e. urinary issues, constipation, low blood pressure). It is the second-most common neurodegenerative disease after Alzheimer’s disease and it is predicted that the prevalence of Parkinson’s disease will double by the year 2040. The symptoms of PD are in substantial part, due to loss of dopamine nerve cells in the brain. The current standard of care for PD includes replacing the dopamine loss by the use of oral carbidopa/levodopa. Levodopa is a precursor of dopamine, converted in the brain. OFF episodes have been a significant unmet need in Parkinson’s disease since the emergence of levodopa. Initially, levodopa controls PD symptoms in a continuous fashion throughout the day. With time the response becomes less predictable and patients experience a re-emergence or worsening of PD symptoms. These episodes are what we mean by OFF episodes. OFF episodes can be characterized, in part, by re-emergence of motor symptoms including tremor, stiffness or slowed movement that can happen at any point during the day. OFF episodes typically begin within the first five years of treatment and occur at the end of a dose. This is referred to as end of dose failure or wearing off. Within the first four to six years after diagnosis, regardless of disease severity, up to 60 percent of people with PD experience OFF episodes. With time these episodes become longer, more severe and disabling, more frequent and less predictable as PD progresses. They can take up more than half the day OFF episodes may alter a persons’ ability to perform everyday activities by slowing or even precluding their completion. The result is significant burden and distress for people living with Parkinson’s disease (PD) and their care partners. CTH-300 was a Phase 3, 12-week, randomized, double-blind, placebo-controlled, parallel group, study examining the efficacy, safety and tolerability of apomorphine hydrochloride sublingual film (KYNMOBI) in people with levodopa-responsive PD complicated by OFF episodes. The primary endpoint was a mean change in the score from pre-dose in the Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Part III Motor Examination at 30 minutes after dosing at the 12-week visit of the maintenance treatment phase. The key secondary endpoint was the percentage of people with PD with a patient-rated full ON (or best) response within 30 minutes at the 12-week visit of the maintenance treatment phase. (more…)
Author Interviews, Prostate Cancer / 22.06.2020

MedicalResearch.com Interview with: Dr Hayley Schultz | Research Associate BPharmSci(Hons), PhD UniSA Clinical and Health Sciences University of South Australia Adelaide SA   MedicalResearch.com: What is the background for this study? Response: Zytiga is a blockbuster medication for treating prostate cancer containing the active ingredient abiraterone acetate, however the formulation is incredibly inefficient. Patients must take 1000 mg per day and fast for 2 hours prior to and 1 hour following its administration. This is because the drug is very water insoluble and has an oral bioavailability of less than 10%. This means only up to 10% of the dose is absorbed from the intestine and enters systemic circulation where it can have a therapeutic effect. The rest of the drug, at least 90% of the dose, moves through the gastrointestinal tract undissolved and is wasted down the toilet. Patients are required to fast when taking the medication as the drugs absorption is heavily and unpredictable increased in the presence of fatty and oily food. We developed a more efficient oral formulation for abiraterone acetate with a greater oral bioavailability, to reduce the dose of the drug and its side effects. (more…)
Author Interviews, Heart Disease, JAMA, Opiods, Surgical Research, University of Pennsylvania / 20.06.2020

MedicalResearch.com Interview with: Chase Brown, MD Associate Fellow, Leonard Davis Institute of Health Economics Integrated Cardiac Surgery Resident Hospital of the University of Pennsylvania Nimesh D. Desai, MD, PhD Director, Thoracic Aortic Surgery Research Program Associate Professor of Surgery Hospital of the University of Pennsylvania MedicalResearch.com: What is the background for this study? Dr. Chase Brown:  Opioid use in the United States is a public health emergency. We know that opioids prescribed after general surgery operations to patients who never received them within the year prior to their surgery are at increased risk for continuing to take opioids months later. However, this has not been studied in patients undergoing cardiac surgery, who often times have more severe post-operative pain. Our goal in this study was to determine how many patients after cardiac surgery and are opioid naive are continuing to take opioids within 90-180 days after their surgery.   (more…)
Author Interviews, Cannabis, Emergency Care / 20.06.2020

MedicalResearch.com Interview with: Ryan Vandrey, Ph.D. Associate Professor Behavioral Pharmacology Research Unit Johns Hopkins University School of Medicine Baltimore, MD 21224  MedicalResearch.com: What is the background for this study? Response: The background for this study is that 33 states in the U.S. have legalized medicinal cannabis use and millions of people are using cannabis for therapeutic purposes, but we have very little data on the broad health impacts of medicinal cannabis use. We surveyed medicinal cannabis users and non-using controls who had a variety of health problems and found that the cannabis users reported better health, quality of life, and less healthcare utilization compared with controls.  Because we worried about group characteristics accounting for the differences observed, we then did an analysis of people who switched groups over time (e.g. non-users who later initiated cannabis use or cannabis users who later quit) and found the same differences emerged in the same individuals over time.  Important to note here is that not all individuals who used cannabis benefited from it and that most participants were using high CBD varieties of cannabis in conjunction with more traditional treatments. (more…)
Author Interviews, Neurological Disorders, Personalized Medicine / 19.06.2020

MedicalResearch.com Interview with: https://polyneuron.com/Pascal Hänggi, PhD Chief Scientific Officer Polyneuron Pharmaceuticals   MedicalResearch.com: What is the background for this study? Response: Anti-MAG neuropathy is a rare form of acquired demyelinating neuropathy. The disease onset normally presents after the age of 50 years and is 2.7 times more frequent in men than in women, with a prevalence of about 1 in 100,000. It is caused by the production of monoclonal anti-MAG IgM antibodies that recognize the HNK-1 epitope. The myelin-associated glycoprotein MAG is a mediator for the formation and maintenance of the myelin sheaths. There is strong evidence that the binding and deposition of anti-MAG IgM autoantibodies on myelin sheath is responsible for the demyelination, which clinically manifests itself as a peripheral neuropathy affecting primarily sensory nerves. However, the causes and the exact mechanisms behind the expansion of anti-MAG IgM producing B-cell and plasma cell clones are not fully understood. Most off-label treatments aim to reduce pathogenic autoantibody titers by depleting  autoantibody-producing B cell clones which interfere with antibody-effector mechanisms, or physically remove autoantibodies from the circulation. Most frequently, the anti-CD20 monoclonal antibody rituximab is used to treat anti-MAG neuropathy patients. However, all of these treatment options often lack of selectivity, efficiency, or can induce severe adverse effects in some patients. Polyneuron has designed PN-1007 to highly selectively target the IgM autoantibodies that cause anti-MAG neuropathy. PN-1007 is a glycopolymer that mimics the natural HNK-1 carbohydrate epitope found on myelin of peripheral nerves and binds to the circulating disease-causing antibodies. By eliminating these pathogenic antibodies, PN-1007 may protect the integrity of the neuronal myelin sheaths of anti-MAG neuropathy patients. (more…)
Author Interviews, Dermatology, Melanoma, Technology / 17.06.2020

MedicalResearch.com Interview with: Chi Hwan Lee PhD Assistant Professor of Biomedical Engineering and Mechanical Engineering, and by Courtesy, of Materials Engineering, and Speech, Language, & Hearing Sciences Purdue University  MedicalResearch.com: What is the background for this study? Response: Conventional melanoma therapies, including chemotherapy and radiotherapy, suffer from the toxicity and side effects of repeated treatments due to the aggressive and recurrent nature of melanoma cells. Less-invasive topical chemotherapies by utilizing miniaturized polymeric microneedles are emerged as an alternative, but the sustained, long-lasting release of drug cargos remains challenged due to the rapidly dissolving behavior of polymers (typically, within 15 min-2 hrs). In addition, the size of the microneedles is still large for small, curvilinear and sensitive areas of tissues such as cornea (for ocular melanoma). (more…)
Author Interviews, Diabetes, JAMA / 17.06.2020

MedicalResearch.com Interview with: Richard E. Pratley, MD AdventHealth Samuel E. Crockett Chair in Diabetes Research Medical Director | AdventHealth Diabetes Institute Senior Investigator and Diabetes Program Lead AdventHealth Translational Research Institute  MedicalResearch.com: What is the background for this study? Response: Historically, older individuals with T1D have been underrepresented or excluded from clinical trials. Older individuals with T1D are at particularly high risk for hypoglycemia. Because of their long duration of diabetes, they often have impaired counterregulatory responses and hypoglycemia unawareness. Hypoglycemia in older individuals is particularly dangerous because it can lead to falls and fractures, cognitive impairment and cardiovascular events, including death. There has been the misperception that older individuals are less able to use technology to manage their diabetes.  This study dispelled that notion.   (more…)
Abbvie, Author Interviews, OBGYNE / 16.06.2020

MedicalResearch.com Interview with: https://www.abbvie.com/   Ayman Al-Hendy, M.D., Ph.D. Investigator for the ELARIS UF-2 clinical trials Professor of Gynecology Director of Translational Research University of Illinois at Chicago   Dr. Al-Hendy discusses the recent announcement that the FDA has approved  ORIAHNN™ for the management of heavy menstrual bleeding due to uterine fibroids in pre-menopausal women. MedicalResearch.com: What is the background for this approval? Uterine fibroids, commonly referred to as uterine leiomyomas, are the most common type of non-cancerous tumor known to impact women of reproductive age (30-50 years old). In fact, studies show that uterine fibroids can occur in up to 70 percent of European American women and over 80 percent of African American women by age 50. As a result of uterine fibroids, women can experience a range of symptoms, the most common being heavy menstrual bleeding (i.e. prolonged and/or frequent bleeding), which can lead to other health effects such as anemia, fatigue, pelvic pain, urinary frequency etc. Uterine fibroid treatment recommendations have historically been based on the size and location of the fibroid(s). When treating larger and more complicated fibroids, healthcare providers have typically believed that surgery is their best course of action, which has made uterine fibroids the leading reason for the hysterectomies performed in the U.S. The FDA approval of ORIAHNN was based on improving care for uterine fibroid sufferers who have had a negative impact on their quality of life due to disruptive symptoms. What makes the approval of ORIAHNN so exciting, is that women now have an oral therapy to directly address heavy menstrual bleeding due to uterine fibroids.  (more…)
Author Interviews, Cancer Research, Leukemia / 16.06.2020

MedicalResearch.com Interview with: Courtney D. DiNardo, M.D., MSCE Department of Leukemia, Division of Cancer Medicine The University of Texas MD Anderson Cancer Center MedicalResearch.com: What is the background for this study? Response: At this year’s virtual European Hematology Association (EHA) meeting, we are presenting late-breaking data from the Phase 3 VIALE-A trial. The randomized double-blind, placebo-controlled trial evaluated venetoclax in combination with azacitidine in previously-untreated patients with acute myeloid leukemia (AML) who are ineligible for standard induction therapy compared to azacitidine plus placebo.  (more…)
Author Interviews, JAMA, Multiple Sclerosis, Neurology / 15.06.2020

MedicalResearch.com Interview with: Cris S Constantinescu,  MD, PhD, FRCP Professor, Division of Clinical Neuroscience Research Group in Clinical Neurology University of Nottingham Queen's Medical Centre Nottingham UK MedicalResearch.com: What is the background for this study? Response: The study is in some way a test of the hygiene or old friends hypothesis, whereby eradication, through improved hygiene, of some parasites that have existed in the human gut for thousands of years and have suppressed inflammatory reactions, leads to an increase in inflammatory conditions. This has been used to explain the increased autoimmune and inflammatory diseases in the developed world. Healthy volunteer studies at the University of Nottingham showed therapeutic hookworm infection to be safe and well tolerated up to about 50 larvae, and then safety studies in people with airway hyperreactivity and inflammatory bowel disease raised no concern. Following a study in Argentina showing that people with MS have milder disease when they have a natural co-existing asymptomatic infection with intestinal parasites, we (Professor Pritchard, immunoparasitologist and myself) decided to test hookworm in MS, and for the first time used 25 larvae in a patient study. (more…)
Author Interviews, Flu - Influenza, Pediatrics, Pediatrics, Vaccine Studies / 15.06.2020

MedicalResearch.com Interview with: Allison Kempe, MD, MPH Ergen Family Endowed Chair in Pediatric Outcomes Research Professor of Pediatrics, University of Colorado School of Medicine Director of ACCORDS (Adult and Child Consortium for Health Outcomes Research and Delivery Science) University of Colorado School of Medicine | Children’s Hospital Colorado  MedicalResearch.com: What is the background for this study? Response: In 2019 the WHO designated vaccine hesitancy as one of the ten leading threats to global health. Although studies have assessed parental vaccine hesitancy in different localities and estimated vaccine refusals nationally, there is little recent US national data on the prevalence of hesitancy about routine childhood vaccines and national hesitancy rates for influenza vaccine have never been assessed. We used a hesitancy scale developed by the WHO to estimate levels of parental hesitancy for both routine childhood and childhood influenza vaccination  (more…)
Author Interviews, Diabetes, Duke, Telemedicine / 15.06.2020

MedicalResearch.com Interview with: Matthew J. Crowley, MD Core Investigator, Durham Center of Innovation to Accelerate Discovery and Practice Transformation (ADAPT) Affiliated Investigator, VA Office of Rural Health Staff Physician, Endocrinology Section, Durham VA Health Care System Elizabeth Kobe, BS Medical Student Durham VA Health Care System Duke University School of Medicine MedicalResearch.com: What is the background for this study? With whom were the telehealth sessions performed? (MDs, PAs, Dieticians etc). Response: Diabetes management in rural populations is especially challenging due to limited access to specialty care and self-management programs. Telehealth is a potential strategy for extending high-quality diabetes care to rural areas. The Veterans Health Administration (VHA) has a robust Home Telehealth (HT) system that is currently used for telemonitoring patient blood glucose values. In order to address the challenges of managing diabetes in rural areas in a clinically feasible manner, we strategically designed an intensive diabetes management intervention – Advanced Comprehensive Diabetes Care (ACDC) – for delivery using existing VHA HT infrastructure and clinical staffing. ACDC is a 6-month telehealth intervention that combines telemonitoring with module-based self-management support and medication management. ACDC is delivered entirely by existing clinical staff (a clinical HT nurse and a medication manager (typically a PharmD)) through bimonthly, 30-minute calls. Our initial randomized controlled trial found that ACDC improved HbA1c by a clinically and statistically significant  -1.0% relative to usual care at 6 months, while also improving blood pressure and diabetes self-care. Our goal with the present work was to improve diabetes care in clinical practice for rural Veterans whose type 2 diabetes remained uncontrolled despite receiving available services. To this end, we partnered with the VA Office of Rural Health to implement ACDC into VA sites across the country with large rural populations.  (more…)
Author Interviews / 15.06.2020

MedicalResearch.com Interview with: Nicholas J. Vogelzang, MD, FASCO, FACP Medical Oncologist at Comprehensive Cancer Centers of Nevada Associate Chair, US Oncology Research Genitourinary Committee  MedicalResearch.com: What is the background for this study? Response: According to the American Cancer Society, prostate cancer is the second leading cause of cancer death in men in the U.S. New approaches are needed to target the androgen receptor (AR), a critical driver of metastatic castration resistant prostate cancer (mCRPC). Current agents work by decreasing androgen levels (abiraterone) or blocking androgen binding to AR (enzalutamide). Despite rapid and dramatic responses to standards of care, all patients with metastatic disease progress to the castration resistant state and their tumors continue to be dependent on the AR signaling axis.1 Study design:
  • “3 + 3” dose escalation; starting dose = 35 mg, orally, once daily with food
  • Dose increases dependent on toxicities
    • Range 25% to 100% based on severity of AEs
Inclusion criteria:
  • Men with mCRPC, regardless of AR status
  • At least two prior systemic therapies, at least one of which was abiraterone or enzalutamide
  • Disease progression on most recent therapy
    • Rising PSA or 2+ new lesions upon bone scan
Endpoints:
  • Primary:
    • Define the maximum tolerated dose and recommended phase 2 dose
  • Secondary:
    • Pharmacokinetics
    • Anti‐tumor activity (PSA50, RECIST criteria)
  • Exploratory:
    • Biomarkers
      • ctDNA mutational profiling
      • AR levels in optional paired biopsies
      • AR and AR‐ V7 levels in circulating tumor cells (CTCs) 
(more…)
Author Interviews, Dermatology, Pediatrics, Pharmaceutical Companies, Regeneron / 15.06.2020

MedicalResearch.com Interview with: Amy S Paller, MD Chair, Department of Dermatology Director, Skin Biology and Diseases Resource-Based Center Walter J. Hamlin Professor of Dermatology Professor of Dermatology and Pediatrics (Dermatology) Feinberg School of Medicine Northwestern University  Dr. Paller discusses the FDA approval of Dupixent® (dupilumab) for children aged 6 to 11 years with moderate-to-severe atopic dermatitis (eczema), whose disease is not adequately controlled with topical prescription therapies or when those therapies are not advisable.  MedicalResearch.com: What is the background for this announcement? Would you briefly discuss what is meant by atopic dermatitis and how it affects children? Response: “Atopic dermatitis, the most common form of eczema, is a chronic inflammatory disease that often appears as a rash on the skin. Moderate-to-severe atopic dermatitis is characterized by rashes that can potentially cover much of the body and can include intense, persistent itching, skin lesions and skin dryness, cracking, redness or darkness, crusting and oozing. Itch is one of the most burdensome symptoms for patients and can be debilitating. This recent FDA approval expands the use of Dupilumab in the U.S. to include children aged 6 to 11 years with uncontrolled moderate-to-severe atopic dermatitis, making it the only biologic medicine approved for this use in this population. Dupilumab is also approved in the U.S. to treat patients aged 12 years and older with moderate-to-severe atopic dermatitis. Moderate-to-severe atopic dermatitis can place a particularly substantial burden on young children aged 6 to 11 years and their families. Limited treatment options leave many of these children to cope with intense, unrelenting itch and skin lesions. Families of these children can spend countless hours helping them to manage their disease.” (more…)
Author Interviews, Circadian Rhythm, COVID -19 Coronavirus, Sleep Disorders / 15.06.2020

MedicalResearch.com Interview with: Dr. Christine Blume PhD Centre for Chronobiology Psychiatric Hospital of the University of Basel Transfaculty Research Platform Molecular and Cognitive Neurosciences Basel MedicalResearch.com: What is the background for this study? Response: In modern societies, human rest-activity rhythms and sleep are between the often-conflicting poles of external social time (e.g., work hours and leisure activities) and an individual’s internal biological time. This can lead to so-called “social jetlag”, which has repeatedly been associated with detrimental health effects. With the restrictions to control the pandemic, social timing relaxed as people many started working from home and public life came to a standstill. In an online survey with 435 respondents, we investigated the effects of the phase with the strictest COVID-19 restrictions on the relationship between social and biological rhythms as well as sleep during a six-week period (mid-March until end of April 2020) in three European societies (Austria, Germany, Switzerland). (more…)
Author Interviews, Gout, Rheumatology / 15.06.2020

MedicalResearch.com Interview with: Brian LaMoreaux, M.D., M.S. Medical Director, Medical Affairs Horizon Therapeutics  MedicalResearch.com: What is the background for this study? Response: Pegloticase is a PEGylated biologic therapy for patients with uncontrolled gout who have not improved on or could not tolerate conventional urate-lowering therapies. All biologics have the ability to engender anti-drug antibodies (ADAs) and it is known that some patients given pegloticase develop ADAs that cause them to stop treatment prior to receiving a complete course of therapy. In other rheumatic autoimmune diseases, DMARDs such as methotrexate or azathioprine are used as standard of care to prevent the development of ADAs to biologics. These DMARDs often allow patients to remain on biologic therapies longer and receive the full therapeutic benefits while minimizing adverse events. While pegloticase has been used traditionally as monotherapy, recent case series have demonstrated the therapeutic benefit of immunomodulator co-administration, allowing more patients to receive a full course of pegloticase therapy. Little has been published on how widespread this practice is and whether it has changed over time. (more…)
Author Interviews, Gout, Heart Disease / 14.06.2020

MedicalResearch.com Interview with: Brian LaMoreaux, M.D., M.S. Medical Director, Medical Affairs Horizon Therapeutics MedicalResearch.com: What is the background for this study? Response: This is an independent study by Dr. Gurkipal Singh for which Horizon provided support and funding. Heart failure is the eighth leading cause of death in the US, with a 38% increase in the number of deaths due to HF from 2011 to 2017. Dr. Gurkirpal Singh conducted this analysis on heart failure hospitalization rates in patients with gout in the US to estimate their clinical and economic impact. Gout and hyperuricemia have previously been recognized as significant risk factors for heart failure, but there is little nationwide data on the clinical and economic consequences of these comorbidities. (more…)
Author Interviews, Gout, Rheumatology / 14.06.2020

MedicalResearch.com Interview with: https://www.horizontherapeutics.com/Brian LaMoreaux, M.D., M.S. Medical Director, Medical Affairs Horizon Therapeutics    MedicalResearch.com: What is the background for this study? Response: Pegloticase is an infused biologic approved to treat uncontrolled gout. The drug is highly effective, but patients can develop anti-drug antibodies that may accelerate clearance of pegloticase from the circulation. Randomized clinical trials have shown that 42% of patients treated with bi-weekly pegloticase had a serum uric acid (sUA) below 6.0 mg/dl at 3 and 6 months. Mild-to-moderate immunomodulation has been shown to lower the prevalence of anti-drug antibody formation in patients with other autoimmune diseases (rheumatoid arthritis, Crohn’s disease, juvenile idiopathic arthritis). Case reports and case series in the literature suggest that low to-moderate doses of methotrexate or azathioprine may also decrease anti-drug antibody formation in uncontrolled gout patients treated with pegloticase.   (more…)