Author Interviews, Genetic Research, NEJM, NIH / 13.12.2014

Dr. Constantine A. Stratakis, M.D., D.Sc National Institutes of Health, Clinical Research Center Bethesda, MD 20892-1862MedicalResearch.com Interview with: Dr. Constantine A. Stratakis, M.D., D.Sc National Institutes of Health, Clinical Research Center Bethesda, MD 20892-1862 Medical Research: What is the background for this study? What are the main findings? Dr. Stratakis: We have been working for years on the genetics of pituitary tumors in association with other conditions. A few years ago (attached), we studied for the first time a series of pediatric giants that we sequenced for then known genes. We found a few MEN1 and AIP mutations but all mutations were present in older kids with gigantism. This left out the youngest among the giants without any genetic defect. This was the first time I realized that I was dealing with a different disease. We started looking for additional genetic defects and when we found the Xq26 microduplications in 3 kindreds. We contacted the custodians of the largest series in the world - Dr. Beckers in Liege. He screened his cases, once we gave him the coordinates, and boom - it was there... The most significant thing here is that this is a new disease really: the early pediatric gigantism is almost exclusively due to Xq26 microduplications unless it is part of a family with another syndrome (AIP, MEN1, Carney complex). If there is no family history and you are dealing with a toddler with gigantism, based in these data, there is a more than 80% chance of having an Xq26 defect. This is pretty amazing! In addition, assuming that GPR101 is the responsible gene (which needs to be confirmed with additional studies) this identifies a new molecular pathway of increasing growth hormone secretion, most likely due to upregulation of GHRH - all of this needs to be confirmed in further human and animal studies. The Xq26 genomic micro-arrangements (which contain the GPR101, but also 3 other genes) is the big news here... (more…)
Author Interviews, Critical Care - Intensive Care - ICUs, NEJM, Nutrition / 26.11.2014

MedicalResearch.com Interview with: Sheila E. Harvey, Ph.D. CTU Manager/Senior Research Fellow ICNARC Napier House London Medical Research: What is the background for this study? Dr. Harvey: The CALORIES trial was set-up in the context of concerns about malnutrition in critically ill patients in NHS hospitals and conflicting evidence as to the optimal route for delivery of early nutritional support to critically ill patients. The enteral route is the mainstay of nutritional support in the critically ill but it is frequently associated with gastrointestinal intolerance and underfeeding. In contrast, the parenteral route, though more invasive and expensive, is more likely to secure delivery of the intended nutrition but has been associated with more risks and complications (e.g. infectious complications) compared with the enteral route. In light of the uncertainty surrounding the most effective route for delivery of early nutritional support and, given recent improvements in the delivery, formulation and monitoring of parenteral nutrition, the UK National Institute of Health Research (NIHR) Health Technology Assessment (HTA) Programme put out a “call” for a large pragmatic randomised controlled trial to be conducted in critically ill patients to determine the optimal route of delivery of early nutritional support. CALORIES was set up to test the hypothesis that early nutritional support delivered via the parenteral route is superior to early nutritional support delivered via the enteral route in adults who had an unplanned admission to an intensive care unit and who could be fed via either route. The primary outcome was all-cause mortality at 30 days. The secondary outcomes included infectious and non-infectious complications (hypoglycaemia, elevated liver enzymes, nausea requiring treatment, abdominal distension, vomiting, new or substantially worsened pressure ulcers). (more…)
Author Interviews, Dartmouth, Lung Cancer, NEJM / 07.11.2014

William C. Black, MD Professor of Radiology Department of Radiology Dartmouth-Hitchcock Medical Center Lebanon, NH 03756MedicalResearch.com Interview with: William C. Black, MD Professor of Radiology Department of Radiology Dartmouth-Hitchcock Medical Center Lebanon, NH 03756 Medical Research: What is the background for this study? What are the main findings? Dr. Black: Lung cancer is the leading cause of cancer related death in the U.S., killing more people than cancers of the colon, breast, and prostate combined. In 2011, the National Lung Screening Trial (NLST) demonstrated that screening for lung cancer with low-dose CT could reduce lung cancer mortality by 20% in adults at high risk for the disease. Since then, several medical organizations have recommended that eligible adults be offered screening. The U.S. Preventive Services Task Force (USPSTF) released a grade B recommendation for low-dose CT screening in December 2012, which means that private insurers must cover the cost of screening by January 1, 2015. The Centers for Medicare and Medicaid (CMS) is expected to issue a final decision on national coverage for CT screening in February 2015 and a preliminary decision for public comment on November 10, 2014. (more…)
Author Interviews, NEJM, Rheumatology / 06.11.2014

Professor Paul Emery Arthritis Research UK Professor of Rheumatology Director - Leeds Musculoskeletal Biomedical Research Unit, LTHT Director – Leeds Institute of Rheumatic and Musculoskeletal Medicine University of LeedsMedicalResearch.com Interview Professor Paul Emery Arthritis Research UK Professor of Rheumatology Director - Leeds Musculoskeletal Biomedical Research Unit, LTHT Director – Leeds Institute of Rheumatic and Musculoskeletal Medicine University of Leeds Medical Research: What is the background for this study? What are the main findings? Professor Emery: Joint damage and functional disability are common in people who suffer from rheumatoid arthritis (RA), even in those with early disease. We know that early aggressive treatment with biologics, such as the anti-TNF agent etanercept, results in rapid remission in many patients with moderate-to-severe rheumatoid arthritis, which can help reduce the risk of joint destruction and disability long term. However, we don’t yet know whether remission achieved with biologic therapy can be maintained after doses are reduced or therapy is withdrawn. The PRIZE trial, a “state-of-the-art” biologic treatment trial conducted in adults with early untreated rheumatoid arthritis, was designed to fill this knowledge gap. The trial included three phases:
  • 1) induction therapy with full-dose combination etanercept-methotrexate therapy;
  • 2) maintenance therapy with a reduced-dose etanercept-methotrexate regimen, methotrexate alone, or no treatment; and
  • 3) complete treatment withdrawal. After clinical remission was induced, remission was shown to be effectively maintained with the reduced-dose combination regimen but not with the biologic-free regimens.Significantly more patients who had received the reduced-dose regimen were in remission after therapy was withdrawn than patients who received no therapy after remission induction. Interestingly, however, after remission was induced with the full-dose combination regimen, no substantial progression of joint damage on x-ray was seen in patients receiving the reduced-dose regimen, methotrexate only, or no treatment.
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Author Interviews, General Medicine, Leukemia, NEJM, Transplantation / 03.11.2014

John E. Wagner, M.D. Principal Investigator Professor Director, Division of Blood and Marrow Transplantation Department of Pediatrics McKnight Presidential Endowed Chair Hageboeck Family / Children's Cancer Research Fund Endowed Chair University of Minnesota Minneapolis, MN 55455MedicalResearch.com Interview with: John E. Wagner, M.D. Principal Investigator Professor Director, Division of Blood and Marrow Transplantation Department of Pediatrics McKnight Presidential Endowed Chair Hageboeck Family / Children's Cancer Research Fund Endowed Chair University of Minnesota Minneapolis, MN 55455 Medical Research: What is the background for this study? What are the main findings? Dr. Wagner: Earlier studies of umbilical cord blood transplantation (UCB) in children with hematological malignancies demonstrated a survival rate of approximately 50%.  While single UCB transplant was very effective despite HLA mismatch, few adults had access to umbilical cord blood as a treatment option due to the cell dose requirement of 2. 5 x 10^6 nucleated cells per kilogram recipient body weight.  For this reason, at the University of Minnesota we explored the co-transplantation of two partially HLA matched umbilical cord blood units in adults as a straightforward strategy to achieving the cell dose requirement.  Early results were remarkable with survival rates higher than that observed in children.  This in turn led to the design of the BMT CTN 0501 study, a randomized trial comparing single versus double umbilical cord blood transplantation in children aged 2-21 years with hematological malignancies.  All patients received a uniform conditioning regimen of fludarabine, cyclophosphamide and total body irradiation and GVHD prophylaxis of cyclosporine A and mycophenylate mofetil.  224 patients were randomized. There were four major findings:
  • 1) survival results overall, regardless of treatment arm, have improved,
  • 2) for children, an adequately dosed single umbilical cord blood unit is sufficient, giving a survival result of 72% at one year,
  • 3) double umbilical cord blood transplant is associated with more GVHD and poorer platelet recovery but survival is comparable to an adequately dosed single unit, and
  • 4) HLA mismatch is well tolerated with potentially better disease free survival in patients transplanted with HLA mismatched umbilical cord blood , a provocative finding that requires further investigation.
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Author Interviews, Colon Cancer, NEJM / 22.10.2014

Alfredo Falcone MD Chiara Cremolini Fotios Loupakis University of Pisa and Azienda-Ospedaliero Universitaria Pisana ItalyMedicalResearch.com Interview with: Alfredo Falcone MD Chiara Cremolini Fotios Loupakis University of Pisa and Azienda-Ospedaliero Universitaria Pisana Italy Medical Research: What are the main findings of the study? Dr. Falcone: In the TRIBE study the main findings are that the use of an initial more intensive therapy with a triplet of cytotoxics (FOLFOXIRI) plus bevacizumab vs a doublet (FOLFIRI) + bevacizumab improves the outcome of metastatic colorectal cancer patients with unresectable metastases. In particular FOLFOXIRI + bevacizumab vs FOLFIRI+bevacizumab improved RECIST response-rate (65% vs 53%, p=0.006), progression-free survival which was the primary endpoint (median 12,1 vs 9,7 months, HR=0,75, p=0.003) and overall survival (median 31,0 vs 25,8 months, HR=0.79, p=0.054). These results, also compared to those reported in previous phase III studies in molecularly unselected patients, represent an important advance in the treatment of this disease. (more…)
Author Interviews, Cost of Health Care, Emergency Care, NEJM, UCLA / 16.10.2014

Daniel A. Waxman, MD, PhD Department of Emergency Medicine David Geffen School of Medicine University of California, Los Angeles RAND Corporation Santa Monica, CaliforniaMedicalResearch.com Interview with: Daniel A. Waxman, MD, PhD Department of Emergency Medicine David Geffen School of Medicine University of California, Los Angeles RAND Corporation Santa Monica, California Medical Research: What are the main findings of the study? Dr. Waxman: About 10 years ago, three states (Texas, Georgia, and South Carolina) passed laws which made it much harder for doctors to be sued for malpractice related to emergency room care.   The goal of our research was to determine whether the lower risk of being sued translated into less costly care by emergency physicians.  To figure this out, we looked at the billing records of nearly 4 million Medicare patients and compared care before and after the laws took effect, and between states that passed reform and neighboring states that didn’t change their laws.   We found that these substantial legal protections didn’t cause ER doctors to admit fewer patients to the hospital, to order fewer CT or MRI scans, or to spend less for the overall ER visit. (more…)
Author Interviews, Infections, NEJM / 08.10.2014

Anders Perner, MD, PhD Overlæge / Senior staff specialist Professor / Professor in Intensive Care Dept of Intensive Care Rigshospitalet Copenhagen DenmarkMedicalResearch.com Interview with: Anders Perner, MD, PhD Overlæge / Senior staff specialist Professor / Professor in Intensive Care Dept of Intensive Care Rigshospitalet Copenhagen Denmark Medical Research: What are the main findings of the study? Dr. Perner: In the large international randomised trial, we showed similar outcomes in patients with septic shock with anemia transfused at a lower vs. a higher hemoglobin threshold. The lower threshold group received 50 % fewer transfusions and one-third of these patients were never transfused in ICU. (more…)
Author Interviews, NEJM, OBGYNE / 03.10.2014

Dr. Jeff Peipert MD, PhD Institute for Public Health Robert J. Terry Professor, Department of Obstetrics & Gynecology, School of Medicine Washington University in St. LouisMedicalResearch.com Interview with: Dr. Jeff Peipert MD, PhD Institute for Public Health Robert J. Terry Professor, Department of Obstetrics & Gynecology, School of Medicine Washington University in St. Louis Medical Research: What are the main findings of this study? Dr. Peipert: In the Contraceptive CHOICE Project, over 70% of teenage girls and women who were provided no-cost contraception and were educated about the effectiveness and benefits of long-acting reversible contraceptive (LARC) methods selected the intrauterine device (IUD) or contraceptive implant.  This group of over 1400 young women aged 15-19 years had rates of pregnancy, birth, and abortion that were far below national rates for sexually experienced teens. (more…)
Author Interviews, Melanoma, NEJM / 30.09.2014

Georgina Long BSc PhD MBBS FRACP Associate Professor of Melanoma Biology and Translational Research Melanoma Institute Australia and the University of Sydney MedicalResearch.com Interview with: Georgina Long BSc PhD MBBS FRACP Associate Professor of Melanoma Biology and Translational Research Melanoma Institute Australia and the University of Sydney Medical Research: Could you provide some background on this project? Why did you decide to do this research project? What prior work led up to this latest paper? Dr. Long: Pre-clinically, we had data that showed that the combination of BRAF inhibitor + MEK inhibitor
  • Decreased skin proliferative toxicity seen with BRAF inhibitors alone (seen as hyperproliferative lesions in rats)
  • and delayed the emergence of resistance I.e. The tumours in the mice reduced in size more, and stayed reduced for longer.We then confirmed this concept in a randomised phase 2 study, although it was not powered for a definitive progression free survival (PFS_ difference like a phase 3 trial is, we saw a strong difference in response rate and in PFS, yet there were only 54 patients per arm.
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Author Interviews, Kidney Stones, NEJM, Radiology, UCSF / 17.09.2014

Rebecca Smith-Bindman, MD Professor in the Departments of Radiology; Epidemiology and Biostatistics; and Obstetrics, Gynecology and Reproductive Medicine UCSF San Francisco Calif.MedicalResearch.com Interview with: Rebecca Smith-Bindman, MD Professor in the Departments of Radiology; Epidemiology and Biostatistics; and Obstetrics, Gynecology and Reproductive Medicine UCSF San Francisco Calif. Medical Research: What are the main findings of the study? Dr. Smith-Bindman: New technology is rapidly developed in medicine, and its important to understand how that technology should be used to improve patient health outcomes. Sometimes the technology is far better than existing technology and it should replace the earlier technology, and sometimes it is not and therefore should not be used. In this clinical scenario – I e. patients who present to an emergency department with abdomen or back pain thought to possibly reflect kidney stones, ultrasound is a simpler, less expensive , and more readily available test in the emergency department setting and therefore if it is equal to CT with respect to patient outcomes, it should be used as the first test in these patients. Currently, CT is the test widely used for patients with suspected kidney stones. We assessed a large number of patients with suspected kidney stones seen at one of 15 large academic emergency medicine departments across the country. Patients were assigned to point of care ultrasound performed by an ED physician, radiology ultrasound or radiology CT. We assessed a broad range of patient centered outcomes and found each of the three tests we studied were equivalent in terms of these outcomes including complications related to missed diagnoses, related serious adverse events, time spent in the emergency department and repeated ED visits and hospitalizations. However, the exposure to ionizing radiation was around half as high in patients who underwent ultrasound as their first test, and thus ultrasound should be used as the first imaging test in patients with suspected nephrolithiasis. (more…)
Author Interviews, Cancer Research, NEJM / 12.09.2014

MedicalResearch.com Interview with: Dr. Charles Mullighan, M.D., MBBS(Hons), MSc Department of Pathology St. Jude Children's Research Hospital Memphis, TN 38105 MedicalResearch: What are the most important take home points from this study for practicing clinicians and their patients? Dr. Mullighan: Acute lymphoblastic leukemia (ALL) remains a leading cause of cancer death in children, and the prognosis worsens with increasing age. Current therapies are inadequate for many patients. This study has defined the genetic basis of a recently described subtype of Acute lymphoblastic leukemia called Ph-like ALL. We show that the prevalence increases with rising age, and that in both children and young adults the disease is driven by a diverse range of genetic changes that activate kinase signaling, which fuels the growth of leukemia cells. Ph-like Acute lymphoblastic leukemia currently has a poor outcome. The activated kinases may be inhibited by currently approved tyrosine kinase inhibitors (TKIs). We have shown efficacy of these inhibitors in cell lines and experimental models, and in a series of patients with Ph-like Acute lymphoblastic leukemia treated with TKIs. (more…)
Author Interviews, Infections, NEJM / 08.09.2014

MedicalResearch.com Interview with: Stephen H. Gillespie, M.D., D.Sc University of St. Andrews Medical School, St. Andrews Stephen H. Gillespie, M.D., D.Sc University of St. Andrews Medical School, St. Andrews Medical Research: What are the main findings of the study? Dr. Gillespie: REMox TB was a pioneering trial that has shown that a large-scale trial can be run efficiently in resource-poor settings with a high TB burden, adhere to the highest standards of good clinical trial practices, and deliver a clear, unequivocal result. REMoxTB was among the most rigorous Tuberculosis drug trials ever conducted in the modern era of TB treatment and among the largest ever conducted for a new TB treatment. It enrolled 1,931 patients at 50 sites in nine countries, mostly in Africa and Asia. Previously, there were thought to be regional differences in way in which patients' response to treatment across the world but we showed that a rigorous approach to trial conduct there was no evidence for that difference. The study confirmed that daily moxifloxacin was safe over four months of therapy and the moxifloxacin containing arms were more bactericidal initially. Despite its substantial anti-TB activity it did not prove possible to shorten therapy to four months.  . These findings, with the safety of moxifloxacin, and its activity against TB, support the continued clinical testing of moxifloxacin as a component of other novel regimens. (more…)
Author Interviews, HIV, Infections, NEJM / 04.09.2014

Bongani M. Mayosi, M.B., Ch.B., D.Phil. Department of Medicine, Old Groote Schuur Hospital Cape Town, South AfricaMedicalResearch.com Interview with: Bongani M. Mayosi, M.B., Ch.B., D.Phil. Department of Medicine, Old Groote Schuur Hospital Cape Town, South Africa Medical Research: What are the main findings of this study? Dr. Mayosi: In those with definite or probable tuberculous pericardial effusion: (1)       Prednisolone for 6 weeks and Mycibacterium indicus pranii  for three months had no significant effect on the combined outcome of death from all causes, cardiac tamponade requiring pericardiocentesis or constrictive pericarditis. (2)      Both therapies were associated with an increased risk of HIV-associated malignancy. (3)       However, use of prednisolone reduced the incidence of constrictive pericarditis and hospitalization. (4)       The beneficial effects of prednisolone on constriction and hospitalization were similar in HIV-positive and HIV-negative patients (more…)
Author Interviews, Heart Disease, NEJM / 01.09.2014

Gilles Montalescot M.D., Ph.D. Professor of Cardiology University of Paris VI; Director, Cardiac Care Unit Institute of Cardiology, Pitié-Salpêtrière University Hospital Paris, FranceMedicalResearch.com Interview with: Gilles Montalescot M.D., Ph.D. Professor of Cardiology University of Paris VI; Director, Cardiac Care Unit Institute of Cardiology, Pitié-Salpêtrière University Hospital Paris, France Medical Research: What are the main findings of the study? Dr. Montalescot : Among the 1862 patients with ongoing STEMI who were enrolled in the ATLANTIC study, we found no difference between those randomized to pre-hospital (in-ambulance) ticagrelor 180 mg and those randomized to in-hospital (in-catheterization laboratory) ticagrelor 180 mg in terms of either pre-PCI ST-segment elevation resolution (≥70%) or pre-PCI TIMI 3 flow in the culprit artery, which were the co-primary endpoints. There was also no difference between the groups in terms of major adverse cardiovascular events at 30 days, with the exception that rates of definite stent thrombosis were lower in the pre-hospital ticagrelor group than in the in-hospital group, both in the first 24 hours (0% versus 0.8%, p= 0.008) and at 30 days (0.2% versus 1.2%, p = 0.02). The safety of pre-hospital ticagrelor did not appear to be an issue, since the incidence of non-CABG-related major bleeding was low and similar in both treatment groups, whichever bleeding definition was used (PLATO, TIMI, STEEPLE, GUSTO, ISTH or BARC). (more…)
Author Interviews, Heart Disease, NEJM / 30.08.2014

MedicalResearch.com Interview with: John J.V. McMurray, M.D Professor of Medical Cardiology British Heart Foundation, Cardiovascular Research Centre University of Glasgow, Glasgow, United Kingdom MedicalResearch.com Interview with: John J.V. McMurray, M.D Professor of Medical Cardiology British Heart Foundation, Cardiovascular Research Centre University of Glasgow, Glasgow, United Kingdom Medical Research: What are the main findings of the study? Dr. McMurray: That compared to an evidence-based dose of an evidence-based ACE inhibitor (enalapril 10 mg bid), LCZ696 reduced the primary composite outcome of cardiovascular death or heart failure hospitalization by 20%, both the components of that composite and all-cause mortality (the latter by 16%) - all reductions are highly statistically significant and clinically important. LCZ696 treated patients also reported fewer symptoms and physical limitations due to heart failure. We think this is a remarkable finding - to beat what has been the gold-standard, cornerstone, therapy for around 25 years. The findings show conclusively that adding neprilysin inhibition to renin-angiotensin system blockade is superior to renin-angiotensin system blockade alone in patients with heart failure and reduced ejection fraction . (more…)
Author Interviews, Infections, NEJM / 27.08.2014

MedicalResearch.com Interview with: Ziad A. Memish, M.D. Alfaisal University Riyadh Saudi Arabia Medical Research: What are the main findings of the study? Dr. Memish:  This is an important study as we looked at the secondary transmission of MERS-CoV among household/family contacts.  Of the total study population of 280 contacts from 26 clusters collected over 6 months period last year, only 12 family contacts were positive for MERS-CoV. Knowing that 7 (2.5%) were positive by PCR, only additional 5 probable secondary transmission were identified by serology which is a very small fraction missed by PCR. (more…)
Author Interviews, Genetic Research, Hematology, Johns Hopkins, NEJM / 27.08.2014

Jerry Spivak, M.D Professor of Medicine and Oncology Director, Center for the Chronic Myeloproliferative Disorders John Hopkins MedicineMedicalResearch.com Interview with: Jerry Spivak, M.D Professor of Medicine and Oncology Director, Center for the Chronic Myeloproliferative Disorders John Hopkins Medicine Medical Research: What are the main findings of the study? Dr. Spivak: The main findings of this study are that polycythemia vera occurs in two clinical forms: an indolent form in which only phlebotomy may be necessary and a more aggressive form requiring myelosuppressive therapy and that these two forms of the disease can be distinguished genetically. (more…)
Author Interviews, General Medicine, Infections, NEJM / 26.08.2014

Medical Research Interview with: Brian Dannemann, MD, FACP Senior Director, JNJ Pharmaceutical Research and Development Titusville, NJ 08560 MedicalResearch: What are the main findings of the study? Dr. Dannemann : The final investigational 120-week results from the TMC207-C208 Phase 2 study demonstrated that bedaquiline (SIRTURO®) showed nearly twice an many patients in the bedaquiline group as in the placebo group were cured on the basis of the World Health Organization (WHO) outcome definitions for Multidrug-Resistant Tuberculosis which was statistically significant (38 of 66 patients  [58%] and 21 of 66 patients [32%] respectively; p = 0.003). (more…)
Author Interviews, NEJM, Respiratory / 22.08.2014

John DeVincenzo, M.D. Professor of Pediatrics Division of Infectious Diseases Professor of Microbiology, Immunology and Biochemistry University of Tennessee School of Medicine. University of Tennessee. Medical Director, Molecular Diagnostics and Virology Laboratories Le Bonheur Children's Hospital Memphis, TennesseeMedicalResearch.com Interview with: John DeVincenzo, M.D. Professor of Pediatrics Division of Infectious Diseases Professor of Microbiology, Immunology and Biochemistry University of Tennessee School of Medicine. Le Bonheur Children's Hospital Memphis, Tennessee Medical Research: What are the main findings of the study? Dr. DeVincenzo: The main findings are
  • a) This is the first time that anyone has shown that the infection caused by the RSV virus can be effectively reduced in a human after the infection has already started.
  •  b) We also show for the first time that once we reduce the amount of virus in the patient, that very quickly, they start to feel better. This clinical improvement was not expected to occur so rapidly.
  • c) The antiviral appeared safe and it was easy to give.
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Author Interviews, Flu - Influenza, NEJM, Vaccine Studies / 15.08.2014

David P. Greenberg, M.D. Vice President, Scientific & Medical Affairs, and Chief Medical Officer Sanofi Pasteur US.MedicalResearch.com Interview with: David P. Greenberg, M.D. Vice President, Scientific & Medical Affairs, and Chief Medical Officer Sanofi Pasteur US.   Medical Research: What are the main findings of the study? Dr. Greenberg: The New England Journal of Medicine published positive results from a randomized, double-blind, large-scale, multi-center efficacy trial, which found that Fluzone® High-Dose (Influenza Vaccine) was more efficacious in preventing influenza illness (“the flu”) in adults 65 years of age and older compared to standard-dose Fluzone vaccine. Fluzone High-Dose vaccine was found to be 24.2 percent (95% CI, 9.7 to 36.5) more effective in preventing influenza relative to standard-dose Fluzone vaccine for the primary endpoint (laboratory-confirmed influenza associated with typical clinical symptoms occurring at least 14 days post-vaccination caused by any viral type or subtype). In other words, investigators determined that participants in the Fluzone High-Dose vaccine group were less likely to get the flu than those in the standard-dose Fluzone vaccine group. The study safety data were consistent with previous Fluzone High-Dose vaccine studies. (more…)
Author Interviews, Infections, mBio, NEJM / 07.08.2014

Claudio Soto, PhD Professor of Neurology Director Mitchell Center for Alzheimer's disease and related Brain Disorders University of Texas Medical School at HoustonMedicalResearch.com Interview with: Claudio Soto, PhD Professor of Neurology Director Mitchell Center for Alzheimer's disease and related Brain Disorders University of Texas Medical School at Houston Medical Research: What are the main findings of the study? Dr. Soto: In this study we describe for the first time the highly sensitive detection of prions in human urine, specifically in samples from patients affected by the variant form of Creutzfeldt-Jakob disease (vCJD), which is the disease produced by infection with prions associated with bovine spongiform encephalopathy, also known as mad cow disease. For detection we used the protein misfolding cyclic amplification (PMCA) technique which amplifies the amount of abnormal prion protein in a cyclical manner conceptually analogous to the polymerize chain reaction. We detected prions in 13 of the 14 vCJD cases analyzed, and the only negative was a sample coming from a patient under treatment with a experimental drug injected directly into the brain. No false positive were observed in the more than 200 cases analyzed.  The concentration of abnormal prion protein in urine was estimated at 1x10^-16 g/ml, or 3x10^-21 moles/ml, which extrapolates to ~40-100 particles per ml of urine. (more…)
Author Interviews, Breast Cancer, Genetic Research, NEJM / 07.08.2014

Dr Marc Tischkowitz MD PhD University Lecturer (Associate Professor) and Honorary Consultant Physician in Medical Genetics Department of Medical Genetics, University of CambridgeMedicalResearch.com Interview with: Dr Marc Tischkowitz MD PhD University Lecturer (Associate Professor) and Honorary Consultant  Physician in Medical Genetics Department of Medical Genetics, University of Cambridge Medical Research: What are the main findings of the study? Dr. Tischkowitz: The PALB2 gene was first identified in 2006 and linked to breast cancer in 2007 but until now we have not had good breast cancer risk estimates for women who have inherited PALB2 mutations. This study was started in 2009 by an group of research institutions (The PALB2 Interest Group) in Canada, US, Europe (UK, Belgium, Greece, Italy, Finland) and Australia. We studied 362 individuals with PALB2 mutations from 154 families. We found that awomen with a PALB2 mutation will on average have a 35% risk of developing breast cancer by the age of 70, rising to 58% if there is a strong family history. Our study will help clinicians to better advise and manage such women. There are several new aspects.
  • It is by far the largest study to date and provides the most accurate risk estimates for PALB2 mutation carriers.
  • It shows that the breast cancer risk is modified by the family history. (more…)
Author Interviews, Hematology, NEJM, Sloan Kettering, Transplantation / 31.07.2014

MedicalResearch.com Interview with: Richard J. O'Reilly, MD Memorial Sloan Kettering Cancer Center Medical Research: What are the main findings of the study? Dr. O'Reilly: 1.       In a comparison of the results of HLA-matched sibling transplants with other established transplant approaches, including T-cell depleted half-matched parental marrow grafts, unmodified transplants from matched unrelated donors and cord blood transplants in the current era (2000-2009), transplants from donors other than HLA-matched siblings had 5 year survival outcomes similar to those of matched siblings when applied to young infants (≤ 3.5 months of age) or infants of any age that were not infected at the time of transplants. Thus any child born with SCID can now be successfully transplanted. 2.       Active infection at the time of transplant significantly reduced chances of long-term survival for all infants except those who received transplants from HLA-matched siblings. Thus, infection is a dominant determinant of transplant outcome.  Control of treatable infections prior to transplant should be a major clinical objective. 3.       Treatment with chemotherapy containing busulfan significantly enhances the likelihood of recovering a normal ability to make antibodies and fosters better recovery of T-cells that provide cell mediated immunity, and may be an acceptable risk in uninfected infants. However, use of any chemotherapy prior to transplant in an infant who is infected, greatly decreases chances of survival. In infected patients who lack a matched sibling, T-cell depleted transplants from half matched related donors had the best outcomes. (more…)
Author Interviews, NEJM, Prostate Cancer / 27.07.2014

Tomasz M. Beer, M.D. FACP OHSU Knight Cancer Institute, Oregon Health and Science University OR 97239MedicalResearch.com Interview with: Tomasz M. Beer, M.D. FACP OHSU Knight Cancer Institute Oregon Health and Science University OR 97239 Medical Research: What are the main findings of the study? Dr. Beer: In the study, we found that compared to placebo, enzalutamide improves overall survival, progression-free survival, quality of life, and delays the need for chemotherapy. Enzalutamide is superior to placebo with respect to all planned endpoints, across all subsets of the patient population in the study.  Enzalutamide treatment is associated with an excellent safety profile. (more…)
Author Interviews, Heart Disease, Lipids, NEJM / 17.07.2014

Professor Jane Armitage Professor of Clinical Trials and Epidemiology Clinical Trial Service Unit, Oxford Cardiovascular Science Oxford, United KingdomMedicalResearch.com Interview with: Professor Jane Armitage Professor of Clinical Trials and Epidemiology Clinical Trial Service Unit, Oxford Cardiovascular Science Oxford, United Kingdom Medical Research: What are the main findings of the study? Prof. Armitage: The study showed that adding extended release niacin with laropiprant (to reduce the flushing) to standard treatment including statins in people with heart disease or strokes did not improve their outcome or reduce the risk of recurrent heart attacks or strokes. (more…)
Author Interviews, Kidney Disease, NEJM, Transplantation / 15.07.2014

Paul Kimmel, M.D. Division of Kidney, Urologic and Hematologic Diseases National Institute of Diabetes and Digestive and Kidney Diseases at the National Institutes of Health Professor Division of Renal Diseases and Hypertension The George Washington UniversityMedicalResearch.com Interview with: Paul Kimmel, M.D. Division of Kidney, Urologic and Hematologic Diseases National Institute of Diabetes and Digestive and Kidney Diseases at the National Institutes of Health Professor Division of Renal Diseases and Hypertension The George Washington University Medical Research:   What are the main findings of the review? Dr. Kimmel: AKI (Acute Kidney Injury) and CKD (Chronic Kidney Disease), two syndromes approached separately in medical school  curricula  as well as in the clinical arena are inextricably intertwined.  They should be taught as a combined entity, culminating in progressive loss of renal function necessitating renal replacement therapy (dialysis or transplantation). The two syndromes increase risk for cardiovascular disease and diminished quality of life as well.  Preventive and therapeutic strategies should be directed at the combined entity. (more…)
Author Interviews, Breast Cancer, NEJM / 10.07.2014

Dr. Olivia Pagani Institute of Oncology of Southern Switzerland Ospedale San Giovanni, Switzerland MedicalResearch.com Interview with:  Dr. Olivia Pagani Clinical Director of the Breast Unit of Southern Switzerland Ospedale San Giovanni, Switzerland Medical Research: What are the main findings of the study? Dr. Pagani: The study showed that the aromatase inhibitor Exemestane is superior to Tamoxifen (both given together with ovarian function suppression) in preventing breast cancer recurrence in premenopausal women with oestrogen receptor positive early breast cancer. (more…)
Author Interviews, Gastrointestinal Disease, Genetic Research, NEJM, Pediatrics / 03.07.2014

Dr. Daniel AgardhMedicalResearch.com Interview with: Dr. Daniel Agardh M.D., Ph.D Department of Pediatrics Diabetes and Celiac Disease Unit Skåne University Hospital Malmo, Sweden, MedicalResearch: What are the main findings of the study? Dr. Agardh: In this study, we stratify the risk of celiac disease among children according to their HLA genotype and country of residence. We confirm that HLA-DQ2/2 genotype is the major risk factor for early celiac disease, but also show how the risk differs between the participating countries despite of sharing similar HLA risk. This points to the direction of an interaction between HLA and the environment that eventually lead to an autoimmune response in genetic susceptible children. (more…)
Author Interviews, Heart Disease, NEJM, Stroke / 01.07.2014

MedicalResearch.com Interview with: Dr. Tommaso Sanna MD Institute of Cardiology Catholic University of the Sacred Heart Rome, Italy MedicalResearch: What are the main findings of the study? Dr. Sanna: In patients with cryptogenic stroke, continuous ECG monitoring with an implantable device, called the Reveal XT Insertable Cardiac Monitor (ICM), discovered Atrial Fibrillation in 6.4 times more patients than conventional diagnostic strategies at six months, 7.3 times more patients at 12 months, and 8.8 times more patients at 36 months. In more detail, after 36 months of follow-up, 30% of patients with cryptogenic stroke had at least one episode of atrial fibrillation. (more…)