Author Interviews, Hematology, Rheumatology / 17.08.2016

MedicalResearch.com: Nisha Jain, Director Global Medical at Biogen Regarding: Post Hoc Analysis to Evaluate the Effect of Recombinant Factor IX Fc Fusion Protein (rFIXFc) Prophylaxis in Adults and Adolescents with Target Joints and Hemophilia B being presented at the World Federation of Hemophilia (WFH) 2016 World Congress MedicalResearch.com: What is the background for this study? Response: People with hemophilia B experience prolonged bleeding episodes that can cause pain, irreversible joint damage and life-threatening hemorrhages. For people with severe hemophilia, most bleeding events occur in joints, with joint damage being the most common complication of the condition.(1) Over time, joints can become severely damaged and an individual can suffer from acute pain as well as restricted range of motion in those joints.(1) MedicalResearch.com: What are the main findings? Response: The B-LONG and B-YOND trials evaluated ALPROLIX® [Coagulation Factor IX (Recombinant), Fc Fusion Protein] in hemophilia B patients. In this post-hoc analysis of 37 patients with target joints in B-LONG and B-YOND, most (98.9%) target joints were considered resolved using the International Society on Thrombosis and Haemostasis (ISTH) definition of resolution as ≤ 2 bleeds into the joint within a consecutive 12-month period. (more…)
Author Interviews, Exercise - Fitness, Hematology, NEJM, Stanford / 08.08.2016

MedicalResearch.com Interview with: D. Alan Nelson, MPAS, PhD Postdoctoral research fellow Stanford Medicine MedicalResearch.com: What is the background for this study? Response: The study was inspired by the uncertainty surrounding sickle cell trait (SCT) and its association with serious exertional collapse events and mortality in active populations. I conducted initial, exploratory analyses on these topics in 2014-15 while examining a range of military readiness predictors and outcomes. The early work indicated that the risk of mortality, rhabdomyolysis and other exertional events arising from SCT might be substantially lower than that suggested by prior work in the research literature. Dr. Lianne Kurina and I decided to conduct further, focused study at the Stanford University School of Medicine to confirm or refute these findings. In considering best approaches, we noted that there was an absence of prior research in which the  sickle cell trait status of an entire, large, physically-active study population was known. This limitation could introduce bias to inflate the apparent impact of a theorized predictive factor. Aside from the challenges in studying the impact of SCT on exertional outcomes, with respect to prevention, a further concern is that  sickle cell trait is a non-modifiable trait. If it were a serious risk factor for rhabdomyolysis and/or mortality, despite careful exertional injury precautions such as those employed by the Army, this might present great challenges for prevention efforts. To maximize the potential for new research to provide actionable prevention information, our interests included examining a range of modifiable risk factors for rhabdomyolysis. Dr. Kurina and I have employed large, longitudinal military datasets for about five years to examine critical military health outcomes, making this study a natural progression of our joint work. The research proceeded with the support of the Uniformed Services University of the Health Sciences, and in cooperation with a distinguished group of experts who co-authored the paper and advised the project. The study was conducted using de-identified records of all SCT-tested African American US Army soldiers on active duty during 2011 - 2014 (N = 47,944). (more…)
Anemia, Author Interviews, Hematology, Lancet, Surgical Research / 06.08.2016

MedicalResearch.com Interview with: Clinical Professor Alhossain A.Khalafallah Menzies Institute for Medical Research, University of Tasmania, Australia Consultant Haematologist Senior Staff Specialist Launceston General Hospital, Australia MedicalResearch.com: What is the background for this study? Response: 1. Iron deficiency is one of the most common nutritional deficiencies worldwide, affecting up to one third of the population worldwide. 2. Prevalence of anaemia in orthopaedic surgery ranges between 10-20% with the main cause of anaemia identified as nutritional deficiency. 3. New intravenous iron preparations have been developed at a higher purchase price than oral iron. Iron carboxymaltose, as one example, remains underutilised in the treatment of perioperative anaemia. 4. To our knowledge, this study is the first to compare the efficacy, safety and long term effect on iron stores and length of hospital stay in the postoperative anaemia setting. (more…)
Author Interviews, Endocrinology, Hematology, OBGYNE, Thyroid Disease / 24.07.2016

MedicalResearch.com Interview with: Kris Poppe, MD, PhD Co-Head Endocrine Unit CHU St-Pierre UMC Université libre de Bruxelles MedicalResearch.com: What is the background for this study?  Response: Pregnant women are often referred by gynecologists to my endocrine practice, for altered thyroid function. At that occasion, I often noticed that the women also had low iron/ferritin levels (ferritin is the iron reserve). Searching in literature did not reveal many publications on the association between iron (deficiency) and thyroid function during pregnancy and so that was the background/aim to perform this study. (more…)
Anemia, Author Interviews, Heart Disease, Hematology / 01.07.2016

MedicalResearch.com Interview with: John G. F. Cleland, MD, FRCP, FESC Department of Cardiology Hull York Medical School, University of Hull, Castle Hill Hospital, Kingston-Upon-Hull National Heart and Lung Institute Royal Brompton and Harefield Hospitals Imperial College London, United Kingdom MedicalResearch.com: What is the background for this study? What are the main findings? Response: This analysis shows that iron deficiency is very common in patients with heart failure and often leads to anaemia and that the prevalence of both iron deficiency and anaemia are highly sensitivity to the criteria used to define them. The World Health Organization defines anaemia as a haemoglobin concentration of <13g/dL in men and <12g/dL in women but doctors should realise this is the lower limit of normal and haemoglobin concentrations should ideally be about 2g/dL higher than this. A man with a haemoglobin of 12g/dL is quite severely anaemic. This study suggest that iron deficiency is common when haemoglobin drops below 14g/dL for men and 13g/dL for women. (more…)
Author Interviews, Hematology, HIV, Stem Cells, Transplantation / 15.06.2016

MedicalResearch.com Interview with: Joseph Alvarnas, MD Associate clinical professor Department of hematology and Director of value-based analytics City of Hope National Medical Center Duarte, CA MedicalResearch.com: What is the background for this study? Dr. Alvarnas: Patients with HIV infection have a significantly increased risk of non-Hodgkin lymphoma and Hodgkin lymphoma. Prior to the availability of effective anti-retroviral therapy, HIV-infected patients with lymphoma had very poor treatment outcomes. Following the availability of effective anti-HIV therapy, patient outcomes for HIV-infected patients now parallel those of non-infected patients. Historically, however, HIV infection has been used as a criterion for not offering patients autologous blood stem cell transplantation outside of centers with unique expertise. The purpose of this trial was to evaluate outcomes, complication rates, and immunological reconstitution of HIV-infected patients following autologous blood stem cell transplantation. (more…)
Anemia, Author Interviews, Hematology / 23.05.2016

MedicalResearch.com Interview with: Samir K. Ballas MD FACP Emeritus Professor of Medicine and Pediatrics Department of Medicine/Cardeza Foundation for Hematologic Research Thomas Jefferson University Philadelphia, PA Medicalresearch.com: What is the background for this study? Dr. Ballas: Previous studies have shown that the incidence of overt stroke is about 11% in children with sickle cell anemia by ages 2 - 20 years. Untreated stokes recur periodically with increasing severity and mortality. Transfusion therapy has been documented to decrease the frequency and morbidity of stroke in children by 90%. Accordingly, children who develop overt stroke are treated with chronic blood exchange transfusion to prevent the recurrence of additional strokes. When children reach the age of 18-20 years their medical care is transitioned to adult programs. This transition process is associated with several issues one of which is the discontinuation of chronic blood exchange transfusion in patients with history of overt stokes partly due to logistic considerations and partly due to lack of research in strokes in adult patients and the complications of chronic blood transfusion . Medicalresearch.com: What are the main findings? Dr. Ballas: The major finding of the study is that the discontinuation of chronic blood exchange after transition to adult programs is associated with increased mortality. All the patients who discontinued blood transfusion died within 3-5 years after transition whereas patients who continued having blood exchange transfusion survived to a mean age of 36 years at the time of writing this study. (more…)
Anemia, Author Interviews, Kidney Disease, Pharmacology / 22.02.2016

MedicalResearch.com Interview with: Dr. Navdeep Tangri Attending physician and Assistant Professor in the Division of Nephrology Department of Medicine and the Department of Community Health Sciences University of Manitoba and Dr. David Collister  Seven Oaks General Hospital Renal Program Winnipeg, Manitoba Canada.  Medical Research: What is the background for this study? What are the main findings? Response: Anemia is common in chronic kidney disease (CKD) including dialysis and its treatment with erythopoetin stimulating agents (ESAs) reduces the need for blood transfusions and has varying effects on morbidity and mortality. The optimal hemoglobin (HGB) targets for treating anemia in CKD are controversial with safety concerns around the normalization of hemoglobin levels due to an increase in cardiovascular (CV) events. The effects of ESAs on health related quality of life (HRQOL) are unclear with individualization o fhemoglobin targets being controversial as clinicians and patients attempt to balance perceived HRQOL benefits with cardiovascular risk. We performed an updated meta-analysis of randomized controlled trials (RCTs) that evaluated the treatment of anemia in CKD with ESAs that targeted higher versus lower hemoglobin targets using validated HRQOL metrics including SF-36 and KDQ. We included 17 studies and found that higher hemoglobin targets compared to lower HGB targets did result in a statistically significant difference in HRQOL and thus did not improve HRQOL beyond a clinically meaningful threshold. Any change in HRQOL was further attenuated in dialysis subgroups. (more…)
Author Interviews, Hematology, NEJM / 11.02.2016

MedicalResearch.com Interview with: Dr. Filip Callewaert PhD Senior Clinical Scientist Clinical Development, Ablynx Zwijnaarde, Belgium Medical Research: What is the background for this study? What are the main findings? Dr. Callewaert: Acquired thrombotic thrombocytopenic purpura (TTP) is a rare and life-threatening coagulation disorder, in which accumulation of ultra-large von Willebrand factor (ULvWF) multimers is implicated, leading to an increased risk of thrombus formation in small blood vessels due to excessive platelet aggregation. There are no approved pharmacological therapies for acquired TTP. Despite treatment with the current standard of care (plasma exchange and immunosuppressive therapy), mortality remains at 10-20% and there is significant neurological, cardiac, and renal morbidity. Caplacizumab is a bivalent Nanobody that binds to the A1 domain of vWF thereby preventing vWF-mediated platelet aggregation. The clinical effects of caplacizumab were demonstrated in the phase II randomised, placebo-controlled TITAN study in 75 patients with acquired TTP. Compared to placebo, there was a nearly 40% reduction in median time to platelet count normalisation in the caplacizumab group (p = 0.005). Treatment with caplacizumab reduced the use of daily plasma exchange and prevented further consumption of platelets in microthrombi and small blood vessel occlusion. In addition, there were fewer recurrences of TTP requiring re-initiation of daily plasma exchange during treatment with caplacizumab (N=3) vs. placebo (N=11). The safety profile of caplacizumab was favorable, with a slightly higher tendency of mostly mild bleeding events.  (more…)
Author Interviews, Hematology / 11.12.2015

MedicalResearch.com Interview with: Dr. Elena Santagostino, MD PhD Angelo Bianchi Bonomi Hemophilia and Thrombosis Center Ca' Granda Foundation Maggiore Hospital Policlinico, Milan, Italy Medical Research: What is the background for this study? What are the main findings? Dr. Santagostino: Two of our abstracts presented at the 57th ASH Annual Meeting are part of the PROLONG-9FP clinical program evaluating the efficacy and safety of CSL Behring’s investigational long-acting fusion protein linking recombinant coagulation factor IX with recombinant albumin (rIX-FP).  The first is an oral presentation on results from two Phase III studies and an ongoing extension study of rIX-FP for routine prophylaxis in previously-treated people with hemophilia B. The two Phase III studies included adolescents and adults (ages 12 to 61) treated with dosing intervals of up to 14 days for 12 to 18 months, and children (ages 1 to 11) who received seven-day prophylaxis treatment for approximately 12 months. Overall, the median annualized spontaneous bleeding rate (AsBR) was 0.00 for all treatment intervals. The extension study is examining longer treatment intervals, including 10- and 14-day intervals in patients younger than 12 and more prolonged treatment intervals in patients older than 18, and so far has reported favorable long-term tolerability with no serious adverse reactions such as the development of inhibitors to factor IX or antibodies to rIX-FP. A second abstract reported on a surgical sub-study in these trials found that a single pre-operative dose of rIX-FP maintained hemostasis during surgery with responses rated by investigators as “excellent” or “good.” Oven a 14-day perioperative period, patients needed six or seven infusions, and none developed inhibitors to factor IX or antibodies to rIX-FP. (more…)
Author Interviews, Cancer Research, Chemotherapy, Hematology, Immunotherapy / 09.12.2015

MedicalResearch.com Interview with: Dr. Ajai Chari MD Associate Professor Medicine, Hematology and Medical Oncology Tisch Cancer Institute Mount Sinai School of Medicine New York, NY Medical Research: What is the background for this study? Dr. Chari: This is a heavily pretreated population where the median progression free survival (PFS) of the pomalidomide dexamethasone is only 4 months and ORR is only 31%. While the anti CD38 monoclonal antibody daratumumab has single agent has activity in this setting, patients with rapidly progressive disease need combination therapy to achieve rapid and deep responses. Pomalidomide also upregulates CD38 on MM cells and like daratumumab, increases the effector cell activity against myeloma. Thus, there is a strong preclinical and clinical rationale for combining daratumumab with pomalidomide and dexamethasone. (more…)
Anemia, Author Interviews, Brigham & Women's - Harvard, JAMA, Transfusions / 07.12.2015

MedicalResearch.com Interview with: Dr. Walter H. Dzik MD Associate Pathologist, Massachusetts General Hospital Associate Professor of Pathology Harvard Medical School Medical Research: What is the background for this study? What are the main findings? Dr. Dzik: Millions of Red Blood Cell transfusions are given each year.  To maintain adequate blood inventories worldwide, Red Blood Cell units are stored under refrigerated conditions.  Previous animal and laboratory research has highlighted the fact that red cells undergo biochemical, morphologic, and biophysical changes during prolonged refrigerated blood storage.    Researchers and clnicians have questioned whether the changes that occur during storage would impair the ability of transfused Red Cells to delivery oxygen to tissues. Our study was a randomized controlled trial conducted in patients with extreme anemia and insufficient global tissue oxygenation.    We randomly assigned children with severe anemia and lactic acidosis to receive Red Blood Cells stored 1-10 days versus Red Blood Cells stored 25-35 days.   We measured the recovery from lactic acidosis in response to transfusion in the two groups.   We also measured cerebral tissue oxygenation using a non-invasive tissue oximeter.    We found that the proportion of patients who achieved reversal of lactic acidosis was the same in the two RBC storage-duration groups.   The rate of decline of lactic acidosis was also equal.   There was also no difference in cerebral oxygenation, resolution of acidosis, correction of vital signs, clinical recovery, survival and 30-day followup.    (more…)
Allergies, Anemia, Author Interviews, FDA / 18.11.2015

MedicalResearch.com Interview with: Cunlin Wang, MD, PhD Division of Epidemiology I, Office of Surveillance and Epidemiology, Center for Drug Evaluation and Research US Food and Drug Administration MedicalResearch: What is the background for this study? What are the main findings? Dr. Wang:  IV Iron has been known for its risk of anaphylactic reaction, but there has been little research on the comparative safety of individual IV Iron products from a large population-based study. This study included 688,183 new users of IV iron not on dialysis from the U.S. Medicare program over a ten-year span (January 2003 to December 2013). The main findings of the study are:  the risk for anaphylaxis at first exposure was higher for iron dextran than non-dextran IV iron products combined (iron sucrose, gluconate and ferumoxytol).  When individual IV Iron products were compared, the data suggested that iron dextran has the highest risk of anaphylaxis and Iron sucrose has the lowest risk, estimated both at the first time exposure and after cumulative exposures.  The low and high molecular weight dextran products could not be individually identified during most of study period. However,  from January 2006 through March 2008, during which the use of two dextran products could be distinguished, there was very low use of high molecular weight dextran (Dexferrum@). This suggested that the study results likely represent the risk of the low molecular weight dextran (Infed@). (more…)
Anemia, Author Interviews, Infections, Kidney Disease, UCSF / 21.10.2015

Dr. Julie H. Ishida MD San Francisco Veterans Affairs Medical Center Nephrology Section San Francisco, CAMedicalResearch.com Interview with: Dr. Julie H. Ishida MD San Francisco Veterans Affairs Medical Center Nephrology Section San Francisco, CA Medical Research: What is the background for this study? What are the main findings? Dr. Ishida: Intravenous iron is important in the treatment of anemia of end-stage renal disease, but it is biologically plausible that iron may increase infection risk. While results from epidemiologic studies evaluating the association between intravenous iron and infection in hemodialysis patients have been conflicting, guidelines for the treatment of anemia of chronic kidney disease have recommended caution in prescribing, avoidance and withholding of intravenous iron in the setting of active infection. However, no data specifically support the recommendation to withhold intravenous iron during active infection. Our study observed that among hemodialysis patients hospitalized for bacterial infection who had been receiving intravenous iron as an outpatient, continued receipt of intravenous iron was not associated with higher all-cause mortality, readmission for infection, or longer hospital stay. (more…)
Anemia, Author Interviews, Cancer Research, University Texas / 16.10.2015

MedicalResearch.com Interview with: Anil K. Sood, M.D. Professor of Gynecologic Oncology and Reproductive Medicine The University of Texas MD Anderson Cancer Center Medical Research: What is the background for this study? What are the main findings? Dr. Sood: Erythropoietin is an important drug for managing anemia, but concerns have surfaced that it might promote cancer growth. The data with the conventional epo-receptor were not convincing with regard to an explanation for why tumor growth might increase. Therefore, we considered whether there could be an alternative receptor to explain these findings. We carried out a systematic search and identified EphB4 as the alternative receptor that explained the increased tumor growth in response to epo. (more…)
Anemia, Author Interviews, Biomarkers, Kidney Disease, Mineral Metabolism / 10.08.2015

MedicalResearch.com Interview with: Lac Tran, MD Division of Nephrology and Hypertension Kaiser Permanente Los Angeles Medical Center Los Angeles, CA Medical Research: What is the background for this study? What are the main findings? Dr. Tran: Abnormal serum phosphorus levels have been associated with adverse cardiovascular outcomes and progression to renal failure.  Given phosphorus’s important biological roles in cellular replication and bone mineral metabolism, we sought to investigate the association between phosphorus levels and anemia in normal kidney function and early chronic kidney disease. Our study is a population-based study among a large racially/ethnically diverse population within the Kaiser Permanente Southern California health system. Among 155, 974 individuals, 4.1% had moderate anemia and 12.9% had mild anemia.  We found that phosphorus levels greater than 3.5 mg/dL and less than 2.0 mg/dL showed a greater likelihood for moderate anemia.  Every 0.5 mg/dL phosphorus level increase demonstrated a 16% greater likelihood for moderate anemia.  The highest phosphorus tertile of our population had a 26% greater likelihood for anemia compared to the middle tertile.  We also found that female sex, Asian race, diabetes, low albumin, and low iron saturation were risk factors for anemia. (more…)
Anemia, Author Interviews, Diabetes, Diabetologia / 18.05.2015

MedicalResearch.com Interview with: Emma English PhD Lecturer in Healthcare Science and Academic Lead for Clinical Biochemistry University of Nottingham, School of Medicine Royal Derby Hospital, UK MedicalResearch: What is the background for this study? What are the main findings? Dr. English: HbA1c is widely used for monitoring glycaemic control in people with diabetes as there is clear evidence that lowering HbA1c values leads to reductions in the rates of diabetes complications. Recently the World Health Organization and the American Diabetes Association have both advocated the use of HbA1c for the diagnosis of Type 2 diabetes at a value of ≥48 mmol/mol (6.5%). Whilst there are many advantages to the use of HbA1c as a diagnostic tool there are equally some significant limitations to its use. A widely cited confounder is anaemia, however to what extent and which types of anaemia affect HbA1c results was not clearly understood. When HbA1c was introduced as a diagnostic test in England we received many queries from healthcare professionals asking questions such as ‘at what level of anaemia should I not use HbA1c?’ and ‘should I routinely screen patients for anaemia when using HbA1c? And if so, what test should I use?’. In order to answer these questions we conducted a systematic review of the literature to determine what was known on this subject. Our findings, presented in Diabetologia, suggest that iron deficiency and iron deficiency anaemia may lead to a spuriously elevated HbA1c level, thus may lead a false positive diagnosis of diabetes. However, non-iron deficiency anaemias can lead to an artificially lower HbA1c and may lead to a false negative result where a diagnosis of diabetes would be missed. There is no clear evidence to suggest at what levels anaemia can give rise to these effects on HbA1c value and also there does not appear to be a single ideal test for identifying patients where this could be an issue. (more…)
Author Interviews, Brigham & Women's - Harvard, Hematology / 28.04.2015

MedicalResearch.com Interview with: John M. Higgins, MD MGH Center for Systems Biology Boston, MA Medical Research: What is the background for this study? What are the main findings? Dr. Higgins: Hundreds of studies over the past 8 years have shown that increased variation in the size of red blood cells (RBCs) is associated with increased risk for a very wide range of common diseases, like heart disease, many types of cancer, infection, many autoimmune diseases, and lots of other conditions.  The size of red blood cells (RBCs) in the circulation of a healthy person usually varies by about 12-14%, meaning that if you took a sample of the cells, most of the bigger cells would be about 14% larger than the smaller cells.  People whose red blood cells show more variation in size have a greater risk of developing a wide range of diseases.  Also, among patients already diagnosed with many common diseases like heart disease or cancer, those with higher RBC size variation have worse outcomes.  It is unknown how all of these different diseases could be connected to variation in the size of red blood cells.  The study explains a major cause for this connection.  We find that the human body seems to slow down the production and destruction of RBCs in just about every major disease very slightly.  Since red blood cells gradually become smaller as they age, a delay in destruction will increase the fraction of small cells, and the overall variation in size increases.  The study also describes a method to estimate a patient’s RBC clearance rate. (more…)
Anemia, Author Interviews, Kidney Disease, Vitamin C / 27.03.2015

MedicalResearch.com Interview with: Dr. Tanjim Sultana MD Department of Nephrology Lenox Hill Hospital New York Medical Research: What is the background for this study? What are the main findings? Response: Almost all dialysis patients are anemic. One quarter of patients requiring High dose Epogen have functional iron deficiency, which means they have adequate iron store but unable to utilize it. Vitamin C has been shown to mobilize iron from their storage and making it available to use in red blood cell production. Prior studies showed intravenous high dose vitamin C could increase hemoglobin levels and decrease epogen requirement. In our study we used daily low dose oral vitamin C in functional iron deficient patients to achieve the same goals. Patients taking daily low dose vitamin C for 3 months had significant decrease in their epogen dose compared to the control group. (more…)
Author Interviews, Hematology / 06.09.2014

Gabriel Popescu Associate Professor Department of Electrical and Computer Engineering & Bioengineering University of Illinois at Urbana-Champaign Beckman Institute for Advanced Science Urbana, IL 61801 MedicalResearch.com Interview with: Gabriel Popescu Associate Professor Department of Electrical and Computer Engineering & Bioengineering University of Illinois at Urbana-Champaign Beckman Institute for Advanced Science Urbana, IL Medical Research: What are the main findings of the study? Prof. Popescu: We used a new imaging method, which combines microscopy and interferometry, to measure nanoscale fluctuations in the red blood cell membrane. We found that the fluctuations, known to be due to thermal or Brownian motion, decrease with blood storage time. These results indicate that the deformability of the cells degrades with time. It means that blood functionality is lower the longer the blood is stored. (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, 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…)
Anemia, Author Interviews, Kidney Disease / 18.07.2014

MedicalResearch.com Interview with: Dennis J. Cotter President Medical Technology and Practice Patterns Institute, Inc. Bethesda, MD 20816 Medical Research: What are the main findings of the study? Answer: This is the first study to document anemia management practice patterns among predialysis CKD patients before and after publication of TREAT. Using a retrospective observational design based on a large US health plan database with over 1.2 million claims for predialysis CKD stage 3 and 4 patients, we report 4 main study findings. 1) For CKD stage 3 patients, the proportion prescribed ESA therapy declined from 17% pre-TREAT to 11% post-TREAT (a 38% decline) and for CKD stage 4 patients, from 34% to 27% (a 22% decline). 2) Prescribing of ESA therapy was declining even before TREAT, but the decline accelerated in the post-TREAT period. 3) ESA prescribing declined after TREAT regardless of anemia status; among patients with hemoglobin <10 g/dL, only 25% of stage 3 and 33% of stage 4 CKD were prescribed ESAs two years after TREAT, a notable 50% decline. 4) After adjusting for all covariates, the probability of prescribing ESAs was 35% less during a two year period after vs. before TREAT publication. (more…)
Author Interviews, Hematology, JAMA, Stem Cells, Transplantation / 03.07.2014

John F. Tisdale, MDMedicalResearch.com Interview with: Dr. John Tisdale MD Molecular and Clinical Hematology Branch National Institute of Diabetes and Digestive and Kidney Diseases, National Heart, Lung, and Blood Institute, Bethesda, Maryland MedicalResearch: What are the main findings of the study? Dr. Tisdale: Using a nonmyeloablative allogeneic HLA-match peripheral blood stem cell transplantation strategy aimed at tolerance induction, we were able to revert the phenotype in 26 of 30 adult patients with severe sickle cell disease ranging in age from 16 to 65 years. In contrast to standard transplantation strategies which rely on high doses of chemo and/or radiotherapy after which the entire bone marrow and blood system is replaced by that of the donor, our patients had a mixture of their own and that of their donor. This procedure was well tolerated, with no non-relapse mortality, and led to complete replacement of red blood cells by that of the donor in successfully engrafted patients. This replacement resulted in decreases in pain, pain medication usage, hospitalizations, and improvements in organ function. (more…)
Anemia, Author Interviews, Kidney Disease, Transfusions / 28.05.2014

MedicalResearch.com Interview with: David T. Gilbertson, Ph.D. Chronic Disease Research Group Center for Observational Research, Amgen, Inc, Thousand Oaks, CA MedicalResearch: What were the main findings of the study? Dr. Gilbertson: Since transfusion avoidance is important in patients receiving maintenance hemodialysis, development of a measure of red blood cell transfusion use to assess dialysis facility anemia management is reasonable. Because dialysis facility size varies widely, calculation of a standardized transfusion ratio (STfR) using standard methods is possible, but these methods result in significant instability in estimates for small dialysis facilities. Use of more advanced statistical methods results in standardized transfusion ratio estimates that are considerably more stable and more consistently precise across dialysis facilities of all sizes. (more…)
Anemia, Author Interviews, BMJ, OBGYNE, Pediatrics / 04.07.2013

MedicalResearch.com Interview with Batool Haider, MD, MS, DSc candidate Departments of Epidemiology and Nutrition School of Public Health Harvard University Anaemia, prenatal iron use, and risk of adverse pregnancy outcomes: systematic review and meta-analysis MedicalResearch.com: What are the main findings of the study? Dr. Haider: The main findings of the study are that iron use in the prenatal period increased maternal mean haemoglobin concentration by 4.59 (95% confidence interval 3.72 to 5.46) g/L compared with controls and significantly reduced the risk of anaemia (relative risk 0.50, 0.42 to 0.59), iron deficiency (0.59, 0.46 to 0.79), iron deficiency anaemia (0.40, 0.26 to 0.60), and low birth weight (0.81, 0.71 to 0.93). The effect of iron on preterm birth was not significant (relative risk 0.84, 0.68 to 1.03). Analysis of cohort studies showed a significantly higher risk of low birth weight (adjusted odds ratio 1.29, 1.09 to 1.53) and preterm birth (1.21, 1.13 to 1.30) with anaemia in the first or second trimester. Exposure-response analysis indicated that for every 10 mg increase in iron dose/day, up to 66 mg/day, the relative risk of maternal anaemia was 0.88 (0.84 to 0.92) (P for linear trend<0.001). Birth weight increased by 15.1 (6.0 to 24.2) g (P for linear trend=0.005) and risk of low birth weight decreased by 3% (relative risk 0.97, 0.95 to 0.98) for every 10 mg increase in dose/day (P for linear trend<0.001). Duration of use was not significantly associated with the outcomes after adjustment for dose. Furthermore, for each 1 g/L increase in mean haemoglobin, birth weight increased by 14.0 (6.8 to 21.8) g (P for linear trend=0.002); however, mean haemoglobin was not associated with the risk of low birth weight and preterm birth. No evidence of a significant effect on duration of gestation, small for gestational age births, and birth length was noted. (more…)
Author Interviews, Hematology, Surgical Research, Transfusions / 08.05.2013

MedicalResearch.com eInterview with Luca Weltert, MD Cardiac Surgery Department of the European Hospital in Rome, Presentation during the Plenary Scientific Session of the 93rd AATS Annual Meeting in Minneapolis. MedicalResearch.com: What are the main findings of the study? Dr. Luca Weltert: This study illustrates the efficacy of Human Recombinant Erythropoietin (HRE) in avoiding blood transfusions in the heart surgery setting. And aside from complicated number, tables and statistics tells that HRE can reduce Blood Transfusions as much as 65%, while at the same time not exposing patients to any substantial added risk. (more…)