Author Interviews, Eli Lilly, Gastrointestinal Disease, NEJM / 02.11.2023

MedicalResearch.com Interview with: Marla C. Dubinsky, MD Professor of Pediatrics and Medicine Icahn School of Medicine at Mount Sinai Co- director, Susan and Leonard Feinstein IBD Clinical Center Mount Sinai Health System MedicalResearch.com: What is the background for this study? Would you briefly describe the condition of UC? Response: Lucent 1 and Lucent 2 were the induction and maintenance registration trials studying the efficacy and safety of mirikizumab in patients 18 years and older with moderate to severely active ulcerative colitis. Mirikizumab is a monoclonal antibody targeting the p19 subunit of IL23. Lucent-3 is the open label extension arm for those meeting inclusion criteria after completing Lucent 2. This study evaluated the long term efficacy and safety of mirikizumab in patients with ulcerative colitis who completed a total of 104 weeks of active mirikizumab treatment. Ulcerative colitis is a chronic incurable inflammatory condition of colon. Common symptoms include diarrhea, blood in the stool, abdominal cramping and bowel urgency. Bowel urgency is one of the most burdensome symptoms that a patient with you could experience. (more…)
Anemia, Author Interviews, Diabetes, Kidney Disease, NEJM / 30.05.2023

MedicalResearch.com Interview with: Prof. Heerspink Prof. Hiddo Lambers Heerspink, PhD PHARMD Department of Clinical Pharmacy and Pharmacology University Medical Center Groningen Groningen

MedicalResearch.com: What is the background for this study? What is dapagliflozin primarily indicated for?   Response: Dapagliflozin is a sodium glucose cotransporter 2 (SGLT2) inhibitor. The multinational, double-blinded, randomized, placebo-controlled, DAPA-CKD trial demonstrated the kidney and cardiovascular benefits of dapagliflozin in 4304 patients with chronic kidney disease (CKD) with and without type 2 diabetes (T2D). Based on the results of this and other trials, current guidelines recommend use of SGLT2 inhibitors in patients with CKD, T2D, or heart failure. Anemia is common among patients with CKD and is associated with worse clinical outcomes. Previous studies showed that SGLT2 inhibitors increase hemoglobin and hematocrit levels, but data are lacking in patients with CKD with and without T2D. In this post-hoc analysis of DAPA-CKD, we assessed the effect of dapagliflozin versus placebo on the correction and prevention of anemia in this population. (more…)
Author Interviews, Dermatology, Eli Lilly, NEJM / 26.03.2023

MedicalResearch.com Interview with: Jonathan Silverberg, MD, PHD, MPH Professor Director of Clinical Research Director of Patch Testing George Washington University School of Medicine and Health Sciences Washington, DC MedicalResearch.com: What is the background for this study? What are the main findings? Response: Lebrikizumab was previously shown to be safe and effective as a treatment for moderate-severe atopic dermatitis in a phase 2 study. These Phase 3 randomized placebo-controlled trials are the largest studies to date of lebrikizumab in AD. They showed that lebrikizumab was safe and highly effective for the treatment of moderate-severe atopic dermatitis. These studies will hopefully support the approval of lebrikizumab in the United States later this year. (more…)
Aging, Author Interviews, Geriatrics, NEJM, Pulmonary Disease, Respiratory, Vaccine Studies / 09.03.2023

MedicalResearch.com Interview with: Veronica Hulstrøm MD, PhD Senior Director Clinical Project Lead for RSV Older Adults GSK     MedicalResearch.com: What is the background for this study? Response: The AReSVi-006 phase III trial is designed to investigate the efficacy and safety of GSK’s respiratory syncytial virus (RSV) vaccine candidate for adults aged 60 years and above. The phase III trial is a randomized, placebo-controlled, double-blind, international trial with 24,966 participants who received either the investigational vaccine or placebo. (more…)
Author Interviews, Diabetes, NEJM / 09.02.2023

MedicalResearch.com Interview with: Brian S Kim, MD, MTR Sol and Clara Kest Professor Vice Chair of Research | Site Chair, Mount Sinai West and Morningside Director, Mark Lebwohl Center for Neuroinflammation and Sensation The Kimberly and Eric J. Waldman Department of Dermatology Precision Immunology Institute | Friedman Brain Institute Icahn School of Medicine at Mount Sinai MedicalResearch.com: What is the background for this study? Response: We generated preliminary data in mice that difelikefalin suppresses itch robustly with very little to no effect on inflammation in a model of atopic dermatitis. This led us to hypothesize that the primary mode of action of difelikefalin must be by direct modulation of sensory neurons to suppress itch and thus would be best suited for a neuropathic itch condition. These are conditions in which itch occurs relatively independently of skin inflammation as in atopic dermatitis due to hyperactive nerves. (more…)
Author Interviews, Infections, NEJM, Pediatrics, Vaccine Studies / 02.02.2023

MedicalResearch.com Interview with: Manuel García Cenoz MD,  PhDInstituto de Salud Pública de Navarra - IdiSNA, Pamplona, SpainCIBER Epidemiología y Salud Pública, Madrid MedicalResearch.com: What is the background for this study? Response: Neisseria meningitidis is a major cause of severe disease in infants and children. After the introduction of serogroup C vaccines, serogroup B meningococcus has become the main cause of invasive meningococcal disease in Europe. The multicomponent protein-based meningococcal B vaccine (4CMenB, Bexsero, GSK) was licensed in the European Union in 2013. Its authorization was based on immunogenicity studies. Although some countries have introduced the 4CMenB vaccine into the publicly-funded infant immunization programs, the low incidence of the disease has limited the conduct of post-commercialization studies of effectiveness.Meningococcal B vaccine began to be used in Spain in children in 2014, recommended by the Spanish Association of Pediatrics, and paid by their families as it was not publicly-funded. We have conducted a nationwide study including all confirmed cases of invasive meningococcal disease in Spain between October 2015 and September 2019 in children aged two months to five years. (more…)
Author Interviews, Biomarkers, Gender Differences, Kidney Disease, NEJM, Race/Ethnic Diversity / 26.01.2023

MedicalResearch.com Interview with: Prof. dr. Hans Pottel KU Leuven Kulak Department of Public Health and Primary Care Belgium MedicalResearch.com: What is the background for this study? Response:  The glomerular filtration rate (GFR) is used to diagnose patients with chronic kidney disease and is also used to adjust the dose of drugs that are eliminated by the kidneys. An accurate estimation of GFR is considered of importance in the management of kidney health in patients. In 2021 we published a new serum creatinine based equation, called the European Kidney Function Consortium (EKFC) equation (Pottel H. et al, Development and Validation of a Modified Full Age Spectrum Creatinine-Based Equation to Estimate Glomerular Filtration Rate : A Cross-sectional Analysis of Pooled Data. Ann Intern Med (2021) 174: 183-191): EKFC-eGFR = 107.3 / [Biomarker/Q]a x [0.990(Age – 40) if age > 40 years] With a = 0.322 if Biomarker/Q is less than 1, and a = 1.132 if Biomarker/Q is 1 or more. The equation can easily be interpreted: the leading coefficient equals the glomerular filtration rate (GFR) of 107.3 mL/min/1.73m², which is the average GFR in healthy children (aged > 2 years), adolescents and young adults. The average healthy GFR remains constant until the age of 40 years, and starts decreasing beyond that age. The GFR is inversely related to the ‘rescaled’ biomarker. The rescaling factor (Q) is the average biomarker value for healthy people of a specific population (e.g. children, adult men, adult women, white people, black people, …). Biomarker/Q equals ‘1’ for the average healthy person, corresponding with eGFR = 107.3 mL/min/1.73m² (up to 40 years of age). It should be noted that for serum creatinine, the Q-value depends on sex and race. Our hypothesis was that the above equation is valid for any renal biomarker, on the condition that the biomarker is appropriately scaled. We showed that the same equation was able to estimate GFR from 2 years to oldest ages. In the current study we tested and validated our hypothesis by applying the above formula for appropriately ‘rescaled’ cystatin C. (more…)
Author Interviews, Dermatology, Immunotherapy, NEJM, University of Pittsburgh / 06.10.2022

MedicalResearch.com Interview with: Rohit Aggarwal, MD, MS Rheumatology, Professor of Medicine Medical Director, Arthritis and Autoimmunity Center Sub-Specialty Education Coordinator Division of Rheumatology Department of Medicine University of Pittsburgh MedicalResearch.com: What is the background for this study? Response: Dermatomyositis is a rare autoimmune inflammatory disease that affects muscles and skin, although muscular forms without skin symptoms and vice versa are also seen. The exact etiology of the disease is not known but is thought to be immune-mediated with many patients having highly specific autoantibodies. There is no cure for dermatomyositis, but several types of treatment have been successfully used in the last years including different kinds of immunosuppressants (e.g. steroids) and intravenous immune globulins (IVIG) to improve the patient’s condition. So far, none of these treatments was approved for use in dermatomyositis based on large, randomized, placebo-controlled trials. Their effectiveness was mainly deduced from clinical experience and from small clinical trials. The ProDERM study was the first large, pivotal, randomized placebo-controlled trial to evaluate the efficacy and safety of intravenous immune globulin (IVIG) in dermatomyositis patients. (more…)
Author Interviews, Biogen, NEJM, Rheumatology / 15.09.2022

MedicalResearch.com Interview with: Nathalie Franchimont, M.D., Ph.D. Senior Vice President, Head of Multiple Sclerosis and Immunology Head of the Multiple Sclerosis and Immunology Development Unit Biogen MedicalResearch.com: What is the background for this study? What are the main findings? Response: Systemic lupus erythematosus (SLE) is a chronic autoimmune disease that affects multiple organ systems. Rash and arthritis are among the most frequent manifestations of the disease and severe organ damage can also occur especially when organs like the kidney are affected. Litifilimab (known as BIIB059) is a monoclonal antibody being studied for the potential treatment of SLE and cutaneous lupus erythematosus (CLE). The Phase 2 LILAC study evaluated litifilimab versus placebo in two parts: Part A in participants who have SLE with active joint and skin manifestations; and Part B in participants with active CLE, including chronic and subacute subtypes, with or without other organ involvement. Results from the SLE portion of the study (Part A) show litifilimab met the study’s primary endpoint by significantly reducing total active joint count compared to placebo. Total active joint count was defined as the total number of tender or swollen joints. Litifilimab was generally well tolerated, with most reported adverse events (AEs) rated as mild or moderate. Note, this Phase 2 trial was not powered to assess secondary endpoints. Based on these positive Phase 2 results, Biogen is currently enrolling participants into the Phase 3 TOPAZ-1 and TOPAZ-2 studies, which will evaluate the efficacy and safety of litifilimab in participants with active SLE worldwide. Part B results from LILAC were published separately in NEJM on July 28, 2022 and expand the body of evidence supporting litifilimab as a potential first-in-class therapy for cutaneous lupus erythematosus in addition to SLE. (more…)
Author Interviews, NEJM, Pulmonary Disease, Tobacco Research, University of Michigan / 04.09.2022

MedicalResearch.com Interview with: Meilan K Han MD, MS Henry Sewall Professor of Medicine Professor of Internal Medicine and Section Head Division of Pulmonary and Critical Care Medicine, Medical School University of Michigan MedicalResearch.com: What is the background for this study? What are the main findings? Response: In the NIH sponsored SPIROMICS study we demonstrated that symptomatic, tobacco exposed individuals have frequent exacerbations. Many of these individuals are treated with the same inhaled medications that have shown benefit in COPD, but we don’t have any evidence basis for this practice. (more…)
Author Interviews, Heart Disease, NEJM / 30.08.2022

MedicalResearch.com Interview with: Dr Holly Morgan M.B., B.Ch. Clinical Research Fellow and REVIVED investigator King's College London MedicalResearch.com: What is the background for this study? Response: Coronary artery disease is the commonest cause of heart failure.  Whilst individually tailored pharmacological and device therapy (optimal medical therapy, OMT) is the cornerstone of management of ischemic heart failure, rates of death and hospitalization for heart failure remain unacceptably high in this population.  Given the causative relationship between coronary disease and heart failure, coronary revascularization has long been considered as a treatment option for these patients.  Whilst there is randomized evidence to support surgical revascularization with coronary artery bypass grafting (1), none previously existed for percutaneous coronary intervention (PCI) in stable ischemic left ventricular dysfunction. Despite this, patients are frequently offered PCI in this setting (particularly if unsuitable for surgery); driven by the belief that hibernating myocardium will improve in function if blood flow is restored, regardless of the revascularization method.  This approach was supported in some international guidelines, though recommendations varied. The REVIVED-BCIS2 trial aimed to establish whether revascularization with PCI in addition to OMT would improve event free survival in patients with ischemic left ventricular dysfunction, when compared to OMT alone (2).  Inclusion criteria included a left ventricular ejection fraction of ≤35%, extensive coronary artery disease (British Cardiovascular Intervention Society jeopardy score ≥6, indicating significant stenoses in the left main coronary artery, proximal left anterior descending coronary artery, dominant circumflex artery, disease in multiple vessels or a combination of these) and viability in at least four dysfunctional myocardial segments which were amenable to PCI.  The main exclusion criteria were acute myocardial infarction within 4 weeks of randomisation, angina which limited the patient’s quality of life or decompensated heart failure or sustained ventricular arrhythmia within 72 hours. The primary composite outcome was all-cause death or hospitalization for heart failure; minimum follow up was 24 months.  Key secondary outcomes included the change in left ventricular ejection fraction from baseline to follow-up at six and twelve months, myocardial infarction, unplanned revascularization and quality of life assessed with the Kansas City Cardiomyopathy Questionnaire and EQ-5D-5L. (more…)
Author Interviews, Diabetes, NEJM, OBGYNE / 19.08.2022

MedicalResearch.com Interview with: Professor Caroline Crowther MB ChB, DCH, FRANZCOG, MD, DDU, FRCOG, CMFM Maternal Fetal Medicine Subspecialist Professor of Maternal & Perinatal Health Liggins Institue Waipapa Taumata Rau | University of Auckland MwdicalResearch.com: What is the background for this study? Response: Gestational diabetes is a growing and significant health problem worldwide for women affected and their babies. Treatment of gestational diabetes improves maternal and infant health but it remains unclear what degree of maternal hyperglycaemia should be used to make the diagnosis. Because of this uncertainty, recommended diagnostic criteria vary around the world. The GEMS randomised trial assessed whether use of lower glycaemic diagnostic criteria, recommended by the International Association of Diabetes and Pregnancy Study Groups would improve perinatal health, without increasing maternal risks, compared to use of higher criteria, and to assess the effects on use of the health services. (more…)
Author Interviews, Kidney Stones, NEJM, Urology / 11.08.2022

MedicalResearch.com Interview with: Michael Bailey Ph.D. Senior Principal Engineer, Applied Physics Laboratory Associate Professor. Mechanical Engineering Adjunct Associate Professor Urology MedicalResearch.com: What is the background for this study? Response: Small (< 6 mm) kidney stones are common and often are asymptomatic. Do you do surgery or wait for them to cause a problem? Or specifically here if you are getting surgery already for other stones that are causing a problem do you take the time and possibly extra risk of cleaning out the small stone in the kidney or in the other kidney? (more…)
Author Interviews, Brigham & Women's - Harvard, Endocrinology, Hip Fractures, NEJM, Osteoporosis, Vitamin D / 27.07.2022

MedicalResearch.com Interview with: Meryl S. LeBoff, MD Chief, Calcium and Bone SectionDirector of the Skeletal Health and Osteoporosis CenterDirector, Bone Density UnitDistinguished Chair in Skeletal Health and Osteoporosis Professor of Medicine, Harvard Medical School Endocrinology, Diabetes and Hypertension, Women's Health Brigham And Women's Hospital JoAnn E. Manson, MD, DrPH Professor, Epidemiology, Harvard T.H. Chan School Of Public Health Michael and Lee Bell Professor of Women's Health, Medicine, Harvard Medical School Chief, Preventive Medicine, Brigham And Women's Hospital Co-Director, Womens Health, Brigham And Women's Hospital   MedicalResearch.com:  What is the background for this study?  What are the main findings? Response: Osteoporosis is a major public health problem. Although supplemental vitamin D has been widely used to reduce the risk of fractures in the general population, studies of the effects of vitamin D on fractures, the most important bone health outcome, have been conflicting. Randomized controlled trials, the highest quality studies, from around the world have shown benefit, no effect, or even harm of supplemental vitamin D on risk of fractures. Some of the trials used bolus dosing, had small samples sizes or short study duration, and co-administered calcium. No large RCTS of this scale tested whether daily supplemental vitamin D (without co-administration with calcium) prevented fractures in the US population. To fill these knowledge gaps, we tested the hypothesis in this ancillary study to VITAL, whether daily supplemental vitamin D3 reduced the risk of incident total, non-spine and hip fractures in women and men in the US. (more…)
Author Interviews, Critical Care - Intensive Care - ICUs, NEJM, Vitamin C / 15.06.2022

MedicalResearch.com Interview with: François Lamontagne MD MSc (pharmacology) MSc (CEB) Professor of Medicine at the Université de Sherbrooke Endowed research chair on patient-centred research Dr. Neill Adhikari MDCM, M.Sc. Sunnybrook Research Institute and University of Toronto Toronto, Canada MedicalResearch.com:  What is the background for this study?  Response: The use of intravenous vitamin C for sepsis has been a hot topic for a few years. It was biologically plausible that vitamin C could reduce organ injury and death by scavenging reactive oxygen species and modulating the immune response to sepsis. It also seemed like an intervention that would be reasonably easy to administer globally should it prove beneficial. On the other hand, no intervention is benign and every aspect of health care should be rigorously studied. Regarding vitamin C, there were strongly held opinions in both camps and this motivated us to design and conduct the LOVIT trial. (more…)
Author Interviews, Brain Injury, NEJM / 09.06.2022

MedicalResearch.com Interview with: Daniel Perl MD Uniformed Services University of the Health Sciences Professor of Pathology at USUHS and Director of the CNRM's Brain Tissue Repository Uniformed Services University of the Health Sciences Bethesda, Maryland MedicalResearch.com:  What is the background for this study?  Response: Chronic traumatic encephalopathy (CTE) is a brain disorder that is predominantly seen in individuals who have suffered from repeated impact head trauma, such as occurs in former boxers or American football players.  CTE has very specific alterations in the brain and can only be diagnosed at autopsy.  Some have claimed that, in addition to former contact sport participants, individuals who served in the military and were repeatedly exposed to blast (explosions) are also at increased risk for developing CTE.  However, this claim has been based on a rather small number of anecdotal cases.  The DoD/USU Brain Tissue Repository is the only facility in the world that is exclusively dedicated to the collection and study of donated brain specimens derived from deceased active duty and retired service members.  We used the resources of this facility to examine 225 consecutively collected brain specimens for the presence of CTE.  This would to provide a view of how common CTE was in this setting and, when diagnosed, was the disease correlated with prior blast exposure, participation in contact sports and other forms of head trauma, and with certain forms of symptomatology such as development of PTSD, alcohol/substance abuse, death by suicide, etc. (more…)
Author Interviews, Boehringer Ingelheim, NEJM, Pulmonary Disease / 15.05.2022

MedicalResearch.com Interview with: Professor Luca Richeldi MD PhD Chair and Head, Division of Pulmonary Medicine Gemelli University Hospital - IRCCS Catholic University of the Sacred Heart Rome MedicalResearch.com:  What is the background for this study?  Would you briefly explain the condition of Idiopathic Pulmonary Fibrosis? Response: As you may know, Idiopathic pulmonary fibrosis (IPF) is a progressive, irreversible lung disease with high mortality. IPF is one of the more common forms of progressive fibrosing interstitial lung diseases and its symptoms of IPF include breathlessness during activity, a dry and persistent cough, chest discomfort, fatigue and weakness. IPF is considered a “rare” disease, but it affects more than 3 million people worldwide. Currently, there are two approved antifibrotic drugs that slow, but do not stop, the progression of fibrosis. Therefore, there is a need for additional treatments that can be used alone or with existing antifibrotic therapies. Pre-clinical research indicated that phosphodiesterase 4 (PDE4) inhibition is associated with anti-inflammatory and antifibrotic effects that may be beneficial in patients with idiopathic pulmonary fibrosis. In this Phase 2, double-blind, placebo-controlled trial, we investigated the efficacy and safety of BI 1015550, an oral preferential inhibitor of the PDE4B subtype, in patients with IPF. Patients were randomly assigned in a 2:1 ratio to receive BI 1015550 at a dose of 18 mg twice daily or placebo. (more…)
Author Interviews, NEJM, OBGYNE, Surgical Research / 31.03.2022

MedicalResearch.com Interview with: Professor Mohamed Abdel-Fattah, MD, FRCOG Chair in Gynaecology Consultant Gynaecologist & Sub-specialist Urogynaecologist School Medicine, Medical Sciences and Nutrition University Of Aberdeen Co-Director Aberdeen Centre For Women’s Health Research Lead – MBChB intercalated degree programme Chief Investigator – CATHETER II, FUTURE, and SIMS RCTs MedicalResearch.com:  Why was this study necessary? Response:At the time of study design, the main surgical option for treating stress urinary incontinence was the insertion of a standard mid-urethral sling, usually using a general anaesthetic. However, single incision mini-slings were introduced to clinical practice without robust assessment. They were considered promising due to several potential advantages including using less mesh more possibility to be performed under local anaesthetic. A number of small studies with short-term follow-up (i.e. low quality evidence) showed mini-slings to have similar success rates to standard mid-urethral slings, but required shorter hospital stay and was less painful immediately after surgery. Several systematic reviews at the time recommended an adequately powered robust randomised trial to compare the clinical and cost-effectiveness of mini-slings to standard mid-urethral slings with adequate term follow-up.  (more…)
Author Interviews, Biomarkers, Heart Disease, NEJM / 03.03.2022

MedicalResearch.com Interview with: PJ Devereaux MD PhD Professor of Medicine, and of Health Research Methods, Evidence and Impact McMaster University President of the Society of Perioperative Research and Care  MedicalResearch.com:  What is the background for this study?  Response: More than 1 million patients undergo cardiac surgery in the United States and Europe annually. Although cardiac surgery has the potential to improve and prolong a patient’s quality and duration of life, it is associated with complications. Prognostically important heart injury – detected by an elevated blood concentration of either cardiac troponin or creatine kinase myocardial MB isoform (CK-MB) – is one of the most common complications after cardiac surgery and is associated with increased mortality. Although elevated CK-MB was historically used to define heart injury after cardiac surgery, this assay is no longer available in many hospitals worldwide, and consensus statements have recommended high-sensitivity cardiac troponin assays as the preferred biomarker. Based on expert opinion, the Fourth Universal Definition of Myocardial Infarction suggested that a cardiac troponin concentration >10 times the upper reference limit, in patients with a normal baseline measurement, should be the threshold used in the diagnosis of heart attack along with evidence of ischemia (e.g., ischemic ST changes on an ECG) in the first 48 hours after coronary artery bypass grafting (CABG). Although the Academic Research Consortium-2 Consensus stated there was no evidence-based threshold for cardiac troponin after CABG, they endorsed a threshold for the diagnosis of heart attack of ≥35 times the upper reference limit together with new evidence of ischemia, based on expert opinion. They also defined a threshold of ≥70 times the upper reference limit as a stand-alone criterion for clinically important periprocedural myocardial injury. Globally, many hospitals now use high-sensitivity cardiac troponin assays; however, limited data are available to define a prognostically important degree of myocardial injury after cardiac surgery based on these assays. We undertook the Vascular Events in Surgery Patients Cohort Evaluation (VISION) Cardiac Surgery Study to examine clinical outcomes after cardiac surgery. A primary objective was to determine the relationship between postoperative levels of high-sensitivity cardiac troponin I and the risk of death 30 days after cardiac surgery.  (more…)
Author Interviews, Diabetes, NEJM, Pediatrics, Technology / 20.01.2022

MedicalResearch.com Interview with: Dr Julia Ware (née Fuchs) Clinical Research Associate Wellcome-MRC Institute of Metabolic Science-Metabolic Research Laboratories and Medical Research Council Metabolic Diseases Unit, University of Cambridge Addenbrooke’s Hospital, Cambridge MedicalResearch.com: What is the background for this study? Response: Management of type 1 diabetes is challenging in very young children, due to their high variability of insulin requirements and unpredictable eating and activity patterns. As a result, many young children do not meet the recommended glycemic targets, or only maintain recommended glycemic control with extensive caregiver input. This in turn leads to high management burden and reduced quality of life for the whole family. While the increasing use of continuous glucose-monitoring devices and insulin-pump therapy has led to reductions in the incidence of severe hypoglycaemia and diabetic ketoacidosis, and has been accompanied by modest improvements in glycemic control, the burden of management has remained high. Hybrid closed-loop systems (also called an artificial pancreas), in which an algorithm automatically adjusts insulin delivery on the basis of real-time sensor glucose levels, may address ongoing challenges in this age group. However, to date hybrid closed-loop studies involving very young children have been small and of short duration and the efficacy and safety of longer term use of a closed-loop system, as compared with standard therapy, was unclear.   To address this knowledge gap, we compared 16-week use of the Cambridge closed-loop algorithm with sensor-augmented pump therapy in children aged 1 to 7 years with type 1 diabetes in a multi-national randomised crossover study. (more…)
Author Interviews, NEJM, OBGYNE / 05.01.2022

MedicalResearch.com Interview with: Laura Schummers, ScD (she/her/hers) Postdoctoral Fellow, Contraception and Abortion Research Team CIHR Patient-Oriented Research Leadership Fellow Post-doctoral Trainee, ICES McMaster UBC - Department of Family Practice | Women's Health Research Institute MedicalResearch.com: What is the background for this study? Response: Canada was the first country in the world to remove all supplemental restrictions on the dispensing and administration of mifepristone, making the drug available as a normal prescription. This meant that the abortion pill could be prescribed by any doctor or nurse practitioner, dispensed by any pharmacist, and taken by patients when, where and if they choose. (more…)
Author Interviews, Columbia, Genetic Research, Hematology, NEJM / 16.12.2021

MedicalResearch.com Interview with: Markus Y Mapara, MD Professor of Medicine Director of the Blood and Marrow Transplantation Columbia University Medical Center MedicalResearch.com: What is the background for this study? What are the main findings?  Response: Sickle cell disease is caused by a point mutation in the beta-globin gene of hemoglobin  resulting in the production of abnormal hemoglobin which leads to formation of sickle-shaped RBC under conditions of low oxygen. Sickle cell disease affects about 100,000 patients in the US which are predominantly African  American. The only curative approach is to perform an allogeneic bone marrow transplant which is however fraught with significant treatment-related risks if a matched sibling donor is not available. The current study describes the successful application of a novel gene therapy  to treat patients with sickle cell disease. The strategy is based on a gene-addition approach to introduce the genetic information for a Hemoglobin F-like molecule termed HgAT87Q into hematopoietic stem cells. The expression of this novel  hemoglobin prevents polymerization of HgbS  and has now been demonstrated to prevent the occurrence of vaso-occlusive pain crises in sickle cell disease patients. (more…)
Author Interviews, COVID -19 Coronavirus, NEJM, Vaccine Studies / 13.12.2021

MedicalResearch.com Interview with: Ronen Arbel, PhD Outcomes Research, Community Medical Services Division Clalit Health Service Tel Aviv, Israel Director, Maximizing Health Outcomes Research Lab Sapir College, Sderot, Israel.  MedicalResearch.com: What is the background for this study? Response: The emergence of the SARS-CoV-2 delta variant and reduced effectiveness over time of the BNT162b2 vaccine (Pfizer-BioNTech) led to a recent Coronavirus 2019 disease (Covid-19) resurgence in early vaccinated populations. The Israeli Ministry of Health was the first in the world to approve a third (booster) dose of BNT162b2 to cope with this resurgence. (more…)
Asthma, Author Interviews, NEJM, Pediatrics, Pulmonary Disease, Vanderbilt / 08.12.2021

MedicalResearch.com Interview with: Leonard B. Bacharier, MD Janie Robinson and John Moore Lee Chair in Pediatrics Professor of Pediatrics Director - Center for Pediatric Asthma Research Scientific Director - Center for Clinical and Translational Research Section Chief - Pediatric Allergy and Immunology Division of Allergy, Immunology and Pulmonary Medicine Monroe Carell Jr. Children’s Hospital at Vanderbilt University Medical Center  MedicalResearch.com: What is the background for this study?  Is Dupilumab used for other atopic conditions, ie eczema/atopic dermatitis?   Response: Many children with moderate-severe asthma continue to experience asthma exacerbations and poor asthma control despite use of controller therapies.  Dupilumab has been shown to reduce asthma exacerbations in adolescents and adults, as well as to improve atopic dermatitis in children and adults. (more…)
Author Interviews, Brigham & Women's - Harvard, COVID -19 Coronavirus, NEJM, Vaccine Studies / 02.12.2021

MedicalResearch.com Interview with: Dr. Barbra Dickerman, PhD CAUSALab investigator and instructor Department of Epidemiology Harvard T.H. Chan School of Public Health  MedicalResearch.com: What is the background for this study? Response: Early randomized trials showed that the BNT162b2 (Pfizer-BioNTech) and mRNA-1273 (Moderna) vaccines were both remarkably effective at preventing symptomatic disease, when comparing each vaccine with no vaccine. However, head-to-head comparisons of these vaccines have been lacking, leaving open the question of which vaccine is more effective.  In this study, we analyzed the VA’s high-quality databases in a way that emulated the design of the hypothetical trial that would have answered this question. Specifically, we used the findings from the original trials to benchmark our methods and then extended them to provide novel evidence for the comparative effectiveness of these two vaccines in a real-world setting and across diverse subgroups and different time periods. (more…)
Author Interviews, Genetic Research, NEJM / 10.11.2021

MedicalResearch.com Interview with: Professor Sir Mark Caulfield Professor of Clinical Pharmacology William Harvey Research Institute Queen Mary University of London  MedicalResearch.com: What is the background for this study? Response: Rare diseases affect 6% of the population in western nations and there are approximately 10,000 different disorders and many remain without a genomic diagnosis after usual testing during their life time. In 2013 the UK Government launched the 100,000 Genomes Project and created Genomics England to investigate the role of whole genome sequencing in rare disease, cancer and infection. Whole genome sequencing gives the most comprehensive read out of the entire genome. To do this we partnered with the National Institute for Health Research (NIHR) BioResource and 9 hospitals across England1. Our New England Journal of Medicine paper published on the 11th November 2021 reports findings on the early rare disease participants who helped us pilot procedures and processes that would be used to enrol at scale across the NHS and revealed the potential benefits for rare disease1. (more…)
Asthma, Author Interviews, NEJM / 27.10.2021

MedicalResearch.com Interview with: Professor Chris Brightling, NIHR Senior Investigator Department of Respiratory Sciences University of Leicester Leicester, UK MedicalResearch.com: What is the background for this study? What are the main findings?  Response: Risankizumab is an anti-IL23 monoclonal antibody. It is a very effective licensed therapy for plaque psoriasis. IL23 has been implicated in asthma and therefore we chose to study the effect of risankizumab in people with severe asthma. The time to first asthma worsening was earlier and the frequency of asthma worsenings was higher in those treated with risankizumab versus placebo. We found that the gene expression of key molecules involved in the response to infection were decreased in airway samples in those treated with risankizumab. It is possible that the increased asthma worsening following risankizumab was related to this suppression of anti-microbial immunity.  (more…)
Author Interviews, NEJM, Stroke, University Texas / 08.09.2021

MedicalResearch.com Interview with: James Grotta, MD Director of Stroke Research Clinical Institute for Research and Innovation Memorial Hermann - Texas Medical Center Director, Mobile Stroke Unit Consortium University of Texas Health  MedicalResearch.com: What is the background for this study? Response: We have good stroke treatments (thrombolysis and thrombectomy).  Since the initial studies showing benefit of thrombolysis, it has been difficult to improve on the amount of benefit except by speeding its delivery; the earlier the treatment, the better the outcome. Biologically, treatment in the first hour is likely to have greatest benefit since the brain is less irreversibly damaged and the clot is more soluble. But treatment in the first hour is rare if it is carried out in the emergency department.  So MSUs take the emergency department to the patient. We know that Mobile Stroke Units (MSUs) can speed treatment; our study addressed if this be accomplished in the US, and how much difference does it make in outcome.  In particular, outcomes important to patients given the probable costs of implementing MSUs.  (more…)
Author Interviews, Heart Disease, NEJM, Salt-Sodium, Stroke / 03.09.2021

MedicalResearch.com Interview with: Maoyi TIAN PhD Program Head, Digital Health and Head, Injury & Trauma Senior Research Fellow The George Institute MedicalResearch.com: What is the background for this study? Response: There is clear evidence from the literature that sodium reduction or potassium supplementation can reduce blood pressure. Reduced blood pressure can also lead to a risk reduction for cardiovascular diseases. Salt substitute is a reduced sodium added potassium product combined those effects. Previous research of salt substitute focus on the blood pressure outcome. There is no evidence if salt substitute can reduce the risk of cardiovascular diseases or pre-mature death. This study provided a definitive evidence for this unaddressed question. MedicalResearch.com: What are the main findings? The main findings of the research were:
  • The salt substitute reduced the risk of stroke by 14%
  • The salt substitute reduced the risk of major adverse cardiovascular events by 13%
  • The salt substitute reduced the risk of pre-mature death by 12%
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