Author Interviews, Hematology, NEJM / 07.09.2018

MedicalResearch.com Interview with: Dr. Johnny Mahlangu  MBBCh Faculty of Health Sciences University of the Witwatersrand and National Health Laboratory Service Johannesburg, South Africa MedicalResearch.com: What is the background for this study? What are the main findings? Response: Current unmet needs in patients with haemophilia without inhibitors are the high disease burden imposed by the frequent injections which have to be given intravensously . Emicizumab which is given subcutaneously weekly or fortnightly aims to address these unmet needs. (more…)
ASCO, Author Interviews, Cancer Research, Hematology / 05.06.2018

MedicalResearch.com Interview with: PharmaMarDr. Javier Gómez García Senior Manager. Biostatistics and Data Management PharmaMar Madrid Area, Spain MedicalResearch.com: What is the background for this study? What are the main findings? Response: Multiple myeloma accounts for approximately 1 percent of all cancers and slightly more than 10 percent of all hematologic malignancies. At present, more than 80,000 new cases are reported worldwide each year, and the disease prevalence is increasing. Plitidepsin is a synthetic cyclic depsipeptide isolated from the marine tunicate Aplidium albicans targeting the proto-oncogene eEF1A2, which is over-expressed in multiple myeloma cells. In ADMYRE trial 255 relapsed and refractory multiple myeloma patients with at least three but not more than six prior regimens, including at least bortezomib and lenalidomide/thalidomide, were randomized at 2:1 ratio to receive plitidepsin 5 mg/m2 D1 and 15 plus DXM 40 mg D1, 8, 15 and 22 (P+DXM), or DXM 40 mg D1,8,15 and 22 (DXM) every four weeks. P+DXM met the primary endpoint, progression-free survival (PFS) assessed by an Independent Review Committee, showing a 35% risk reduction in the probability of progression or death. Indeed, a 20% risk reduction in the probability of death was also observed in spite of the fact that 44% of patients from control arm (DXM) switched to P+DXM arm after progression. Therefore, survival in the control arm might have been extended by the effect of plitidepsin, increasing the post progression survival and overestimating the survival observed in the control arm and consequently underestimating the actual difference between treatment arms. Several methods were used to assess the impact of crossover and the robustness of the results even when penalizations were applied; PharmaMar believes that strong evidence in terms of survival benefit in favour of P+DXM has been established.  (more…)
Accidents & Violence, Author Interviews, Critical Care - Intensive Care - ICUs, Emergency Care, Hematology / 05.05.2018

MedicalResearch.com Interview with: Dr. Wataru Takayama Tokyo Medical and Dental University Department of Emergency and Disaster Medicine Tokyo, Japan MedicalResearch.com: What is the background for this study? Response: ABO blood type is a potential risk of various diseases and various conditions. Furthermore, ABO blood type has a profound influence on hemostasis. Hemorrhage is the leading cause of death in patients with trauma, we assessed the association between the difference in blood types and the outcomes of death. MedicalResearch.com: What are the main findings? Response: Blood type O was the independent risk factor for all-cause in-hospital mortality and death due to exsanguination, TBI, and other causes after adjusting for potential confounders. This is the first study to report the association between ABO blood types and mortality in patients with severe trauma. (more…)
Author Interviews, Emory, Hematology, JAMA, Neurological Disorders, Stroke / 23.04.2018

MedicalResearch.com Interview with : Dr. Hyacinth I Hyacinth MD Aflac Cancer and Blood Disorder Center, Emory Children’s Center, Department of Pediatrics, Emory University School of Medicine Atlanta, GA 30322 MedicalResearch.com: What is the background for this study? This study was conducted against the backdrop of a significantly higher risk for stroke among African Americans compared to non-Hispanic Whites, despite adjusting for traditional risk factors. Also, sickle cell disease is a well-known genetic risk factor for stroke and recent studies show that sickle cell trait is a risk factor for chronic kidney disease, venous thromboembolism and pulmonary embolism, all of which are potential risk factors for stroke. (more…)
Author Interviews, Hematology, Surgical Research, Transfusions / 19.03.2018

MedicalResearch.com Interview with: Shawn Anthony, MD, MBA Assistant Professor of Orthopaedics Icahn School of Medicine at Mount Sinai MedicalResearch.com: What is the background for this study? What are the main findings?  Response: Rates of total shoulder arthroplasty are increasing, especially with an aging population.  Blood loss requiring transfusion is less common than in total hip or knee replacements but still required in some patients.  Tranexamic acid (TXA) is increasingly used to reduce blood loss in lower extremity arthroplasty but limited data exists for its effectiveness and safety in patients undergoing shoulder arthroplasty. We aimed to utilize national data to assess frequency of use and effectiveness of TXA in shoulder arthroplasty patients. While utilization of TXA has become very common in total hip and knee arthroplasty, TXA is still used in less than 50% of patients undergoing shoulder arthroplasty as of 2016.  TXA use was associated with a 36% decrease in transfusion risk and a 35% decreased risk for combined complications. Moreover, TXA use was associated with 6.2% shorter hospital stay. (more…)
Abuse and Neglect, Hematology, Lancet, Medical Imaging, Transplantation / 07.03.2018

MedicalResearch.com Interview with: Kirsten Williams, M.D. Blood and marrow transplant specialist Children’s National Health System  MedicalResearch.com: What is the background for this study? What are the main findings?  Response: This study addressed a life-threatening complication of bone marrow transplantation called bone marrow failure. Bone marrow transplantation has provided a cure for patients with aggressive leukemias or acquired or genetic marrow dysfunction. The process of bone marrow transplantation involves giving chemotherapy and/or radiation, which removes the diseased blood cells from the bone marrow. After this, new bone marrow stem cells are infused from a healthy individual. They travel to the bone marrow and start the slow process of remaking the blood system. Because these new cells start from infancy, it takes upwards of four to five weeks for new mature healthy cells to emerge into the blood, where they can be identified. Historically, there has been no timely way to determine if the new cells have successfully repopulated unless they can be seen in the blood compartment. This condition of bone marrow failure is life-threatening, because patients don't have white blood cells to protect them from infection. Once bone marrow failure is diagnosed, a second new set of stem cells are infused, often after more chemotherapy is given. However, for many individuals this re-transplantation is too late, because severe infections can be fatal while waiting cells to recover. We were the first group to use a new imaging test to understand how the newly infused bone marrow cells develop inside the patient. We have recently published a way to detect the new bone marrow cell growth as early as five days after the cells are given. We used an investigational nuclear medicine test to reveal this early cell growth, which could be detected weeks before the cells appear in the blood. This radiology test is safe, does not cause any problems and is not invasive. It is called FLT (18F-fluorothymidine) and the contrast is taken up by dividing hematopoietic stem cells. The patients could even see the growth of their new cells inside the bone marrow (which they very much enjoyed while waiting to see recovery of the cells in their blood). We could use the brightness of the image (called SUV) to determine approximately how many weeks remained before the cells were visible in the blood. Finally, we actually could see where the new cells went after they were infused, tracking their settling in various organs and bones. Through this, we could see that cells did not travel directly to all of the bones right away as was previously thought, but rather first went to the liver and spleen, then to the mid-spine (thorax), then to the remainder of the spine and breastplate, and finally to the arms and legs. This pattern of bone marrow development is seen in healthy developing fetuses. In this case, it occurs in a similar pattern in adults undergoing bone marrow transplant. (more…)
Author Interviews, Hematology, NEJM, Orthopedics, Thromboembolism / 22.02.2018

MedicalResearch.com Interview with: Dr. David R. Anderson, MD, FRCPC, FACP Faculty of Medicine Dean, Professor Dean, Faculty of Medicine Division of Hematology, Department of Medicine & Nova Scotia Health Authority MedicalResearch.com: What is the background for this study? What are the main findings? Response: Blood clots in the lungs (pulmonary embolism) and veins of the legs (deep vein thrombosis) are well recognized complications following total hip and knee arthroplasty surgeries.  Prior to the routine use of antithrombotic prophylaxis, pulmonary embolism was the most common cause of death following these procedures.  Oral anticoagulants such as rivaroxaban are commonly prescribed for the indication of preventing blood clots following total hip or knee arthroplasty.  For maximal benefit these agents are continued following surgery for up to five weeks following total hip arthroplasty and for two weeks following total knee arthroplasty. There is evidence that aspirin has some benefit for the prevention of deep vein thrombosis and pulmonary embolism following total hip or knee arthroplasty.  However there is less evidence for its benefit than for oral anticoagulants.  We reasoned that aspirin would potentially be an attractive alternative for extended out of hospital prophylaxis following total hip or knee arthroplasty for patients who received a short course (5 days )of rivaroxaban following surgery.  Aspirin would be attractive for this indication because of its low cost, ease of use, and low rates of side effects. Our study demonstrated that in a randomized controlled trial involving a large group (over 3400) of patients undergoing total hip or knee arthroplasty that extended therapy with aspirin was comparable to rivaroxaban for the prevention of deep vein thrombosis and pulmonary embolism following surgery.  Low rates of complications (< 1%) were observed with both treatment arms.  We also found that rates of clinically important bleeding complications (the most common side effect with antithrombotic drugs) were uncommon and similar with the two agents. (more…)
Author Interviews, Hematology, Novo Nordisk, Surgical Research / 15.02.2018

MedicalResearch.com Interview with: Stephanie Seremetis, M.D. Corporate Vice President and Chief Medical Officer Biopharmaceuticals at Novo Nordisk MedicalResearch.com: What is the background for this announcement? Response: We’re proud and excited to make Rebinyn® (Coagulation Factor IX (Recombinant), GlycoPEGylated) available as a new extended half-life treatment for hemophilia B management. Rebinyn® is an injectable medicine used to treat and control bleeding in adults and children with hemophilia B. It can be used to treat bleeds when they occur and to manage bleeding during surgery. Rebinyn® is not used for routine prophylaxis or for immune tolerance induction in patients with hemophilia B. Hemophilia B is a serious, chronic, inherited bleeding disease that affects about 5,000 people in the U.S. People living with hemophilia B have low levels of clotting Factor IX protein in the blood, often resulting in prolonged or spontaneous bleeding, especially into the muscles, joints or internal organs.  (more…)
Author Interviews, Cancer Research, Hematology / 21.12.2017

MedicalResearch.com Interview with: janseen-oncologyMaria-Victoria Mateos, MD, PhD University Hospital of Salamanca/IBSAL Salamanca, Spain MedicalResearch.com: What is the background for this study? What are the main findings? Response: The Phase 3 ALCYONE study data showed DARZALEX (daratumumab) in combination with bortezomib, melphalan, and prednisone (VMP) significantly improved clinical outcomes, including reducing the risk of disease progression or death by 50 percent, in newly diagnosed patients with multiple myeloma who are ineligible for autologous stem cell transplantation (ASCT) at a median follow-up of 16.5 months (Hazard Ratio [HR] = 0.50; 95 percent CI [0.38-0.65], p<0.0001). The median progression-free survival (PFS) for DARZALEX-VMP had not yet been reached, compared to an estimated median PFS of 18.1 months for patients who received VMP alone. In addition to reducing the risk of disease progression or death, DARZALEX significantly improved the overall response rate (ORR) as compared to VMP alone, including more than doubling rates of stringent complete response, significantly improved rates of very good partial response or better and complete response or better (CR). The most common (≥10 percent) Grade 3/4 treatment-emergent adverse events (TEAEs) for DARZALEX-VMP vs. VMP were neutropenia (40 percent vs. 39 percent), thrombocytopenia (34 percent vs. 38 percent), anemia (16 percent vs. 20 percent) and pneumonia (11 percent vs. 4 percent). One patient in each arm discontinued treatment due to pneumonia, and 0.9 percent of patients discontinued DARZALEX due to an infection. Twenty-eight percent of patients experienced infusion reactions (IRs) due to DARZALEX.. In the DARZALEX-VMP arm, 42 percent of patients experienced a serious adverse event (SAE), compared to 33 percent in the VMP arm. The study findings were as a late-breaking abstract (Abstract #LBA-4) at the 59th American Society of Hematology (ASH) Annual Meeting in Atlanta, and simultaneously published in the New England Journal of Medicine (NEJM). (more…)
Author Interviews, Hematology / 16.12.2017

MedicalResearch.com Interview with: Dr. Charles T. Nakar, MD Indiana Hemophilia and Thrombosis Center Pediatrics Indianapolis, IN   MedicalResearch.com: What is the background for this study? Response: Congenital plasminogen deficiency is a rare genetic disorder that is caused by mutations in the PLG gene. Mutations in this gene lead to clinical manifestations such as fibrinous deposits on mucous membranes leading to disruption of tissue or organ function. These symptoms, when untreated, lead to significant morbidity and decreased quality of life. Life-threatening episodes may occur especially when the respiratory system is affected. There is currently no established approach to treatment of type 1 plasminogen deficiency and the available topical and systemic therapies (e.g. FFP, corticosteroids, immunomodulatory drugs, anticoagulants, amongst others) lack consistent efficacy. Patients may undergo multiple surgeries to remove lesions, but this approach typically leads to regrowth of lesions. Prometic’s intravenous plasminogen replacement therapy represents the first direct treatment for this serious disorder. (more…)
Author Interviews, Cancer Research, Hematology, NEJM / 15.12.2017

MedicalResearch.com Interview with: Dr. Meletios A. Dimopoulos MD Professor and Chairman Department of Clinical Therapeutics University Athens School of Medicine Athens, Greece MedicalResearch.com: What is the background for this study? What are the main findings? Response: Updated data from the Phase 3 POLLUX trials showed DARZALEX, in combination with lenalidomide and dexamethasone, reduced the risk of disease progression or death by 56 percent, compared to lenalidomide and dexamethasone alone (Hazard Ratio [HR]=0.44; 95 percent CI [0.34-0.55], p<0.0001). After a median follow-up of 32.9 months, the median progression-free survival (PFS) in the DARZALEX arm has not been reached, compared with a median PFS of 17.5 months for patients who received lenalidomide and dexamethasone alone. DARZALEX in combination with lenalidomide and dexamethasone also significantly increased the overall response rate (ORR) compared to lenalidomide and dexamethasone alone (93 percent vs. 76 percent, p<0.0001), including rates of complete response (CR) or better (55 percent vs. 23 percent, p<0.0001). DARZALEX also showed significantly higher (>3-fold) MRD-negative rates compared to lenalidomide and dexamethasone alone. These data were featured as an oral presentation (Abstract #739) at the 59th American Society of Hematology (ASH) Annual Meeting in early December. (more…)
Author Interviews, Hematology / 13.12.2017

MedicalResearch.com Interview with: Craig C. Hofmeister, MD, MPH The Ohio State University  MedicalResearch.com: What is the background for this study? What are the main findings? Response: Preliminary data presented from the randomized, open-label, Phase 2 CENTAURUS (SMM2001) study evaluated three dosing schedules for DARZALEX monotherapy in patients with intermediate or high-risk smoldering multiple myeloma. A total of 123 patients were enrolled, with a median time since initial smoldering multiple myeloma diagnosis of 6.83 months (0.4-56). Patients were randomized to one of three treatment arms receiving DARZALEX 16 mg/kg intravenously in 8-week cycles: 1.) a long-intense dosing schedule (LONG) where DARZALEX was administered weekly in Cycle 1, every other week in Cycle 2-3, every 4 weeks in Cycle 4-7, and every 8 weeks up to Cycle 20; 2.) an intermediate dosing schedule (INT), where DARZALEX was given weekly for 1 cycle, and every 8 weeks up to Cycle 20 and; 3.) a short intense dosing schedule (SHORT), where DARZALEX was given weekly for 1 cycle. Results from the study showed DARZALEX monotherapy had a tolerable safety profile in patients with intermediate or high-risk smoldering multiple myeloma, with the most common treatment-emergent adverse events (TEAEs) being fatigue, cough, headache and insomnia. The efficacy endpoints included overall response rate, progression free survival, time to next treatment, and overall survival rate at 4 years. These study results serve as the basis for a Phase 3 study for DARZALEX in smoldering multiple myeloma, which is actively enrolling. These findings demonstrated DARZALEX had a manageable safety profile in patients with intermediate or high-risk smoldering multiple myeloma. (more…)
Author Interviews, Cost of Health Care, Hematology / 28.11.2017

MedicalResearch.com Interview with: Carol Mansfield, PhD, Senior Research Economist Health Preference Assessment RTI Health Solutions www.rtihs.org  MedicalResearch.com: What is the background for this study? Response: As the most prevalent form of leukemia, chronic lymphocytic leukemia (CLL) affects approximately 130,000 people in the United States. More than 20,000 new cases are diagnosed each year. In recent years, more treatment options–each with its own associated benefits, side effects, and price tag–have been approved. This leaves patients and physicians with a variety of factors they must consider when choosing a treatment plan. While every patient wants the most effective drug with the fewest side effects, most people don’t have that option available. By asking patients to make tradeoffs and rank their preferences, we can form an understanding of how patients approach their treatment. This study showed that patients with CLL value medicines that provide the longest progression-free survival, but are willing to trade some benefits for a lower risk of serious adverse events. Additionally, we found that cost clearly has an impact on which treatment a patient would choose. When patients get prescribed something they can’t afford, they are forced to make very difficult choices. (more…)
Author Interviews, Gender Differences, Hematology, JAMA / 17.10.2017

MedicalResearch.com Interview with: Rutger Middelburg, PhD Assistant Professor in clinical epidemiology Sanquin Research and LUMC  MedicalResearch.com: What is the background for this study? Response: Six years ago we found transfusions from female donor to be associated with increased mortality among male recipients, especially under 50 years of age. This was an unexpected observation and we considered the probability of a false positive finding (i.e. a chance association) to be relatively high. We therefore immediately started a follow-up study with two main objectives. First, we wanted to confirm our findings in an independent and much larger cohort. Second, since some complications of blood transfusion are known to be related to pregnancy history of the donor, we wanted to study a possible relationship with previous pregnancy of the blood donors. (more…)
Author Interviews, Hematology, Lancet / 09.04.2017

MedicalResearch.com Interview with: Dr. med. Kathleen Selleng, OÄ, QB Hämotherapie Universitätsmedizin Greifswald Institut für Immunologie und Transfusionsmedizin, Abt. Transfusionsmedizin Sauerbruchstraße Greifswald Deutschland MedicalResearch.com: What is the background for this study? What are the main findings? Response: Red blood cell concentrates (RBCs) of blood group O RhD negative are frequently used as universal blood for emergency transfusions in patients with unknown blood type. This leads to an over-proportional use of these red blood cell concentrates and regular shortages of O RhD negative RBCs. Due to these shortages, patients with known RhD negative blood type sometimes have to be transfused with RhD positive RBCs. The present study shows that the overall risk to induce an anti-D by transfusing all emergency patients with unknown blood type with O RhD positive RBCs is in the range of 3 to 6%, while this risk is much higher (20-30%) in RhD negative patients which have to be transfused with RhD positive RBCs due to RhD negative RBC shortages. (more…)
Anemia, Author Interviews, Global Health, JAMA, OBGYNE / 17.01.2017

Ola Andersson, MD, PhD Department of Women’s and Children’s Health Uppsala University, Uppsala, Sweden MedicalResearch.com Interview with: Dr. Ola Andersson MD, PhD Uppsala University, Uppsala, Sweden MedicalResearch.com: What is the background for this study? Response: Anemia affects over 40% of all children under 5 years of age in the world. Anemia can impinge mental and physical performance, and is associated with long-term deterioration in growth and development. Iron deficiency is the reason for anemia in approximately 50% of the children.is. When clamping of the umbilical cord is delayed, ie after 3 minutes, iron deficiency up to 6 months of age can be prevented, but it has not been shown to prevent iron deficiency or anemia in older infants. At birth, approximately 1/3 of the child's blood is in the placenta. If clamping of the umbilical cord is done immediately (early cord clamping), the blood will remain in the placenta and go to waste (or can be stored in stem cell banks). If instead clamping is postponed for 3 minutes, most of the blood can flow back to the child as an extra blood transfusion, consisting of about one deciliter (1/2 cup) of blood, equivalent to about 2 liters (half a US gallon) of an adult. A blood donor leaves 0.4-0.5 liters of blood. Blood contains red blood cells that contain hemoglobin. Hemoglobin carries oxygen to the tissues of the body. Hemoglobin contains a lot of iron, and the extra deciliter of blood may contain iron that corresponds to 3-4 months of the need for an infant. The World Health Organization (WHO) recommends umbilical cord clamping at 1 minute or later, American College of Obstetricians and Gynecologists (ACOG) recommends umbilical cord clamping at 30-60 seconds or later. (more…)
Anemia, Author Interviews, Kidney Disease, Pharmacology, Stanford / 16.01.2017

MedicalResearch.com Interview with: Dr. Glenn M. Chertow, MD Professor Medicine, Nephrology Stanford University School of Medicine MedicalResearch.com: What is the background for this study? What are the main findings? Response: Iron deficiency is common in persons with moderate to advanced (non-dialysis-dependent) chronic kidney disease (CKD), for a variety of reasons. Conventional iron supplements tend to be poorly tolerated and of limited effectiveness. In earlier studies of patients treated with ferric citrate for its effect as a phosphate binder, we saw increases in transferrin saturation and ferritin (markers of iron stores) and hemoglobin and hematocrit (the “blood count”). Therefore, we thought we should test the safety and efficacy of ferric citrate specifically for the treatment of iron deficiency anemia (IDA). With respect to the key findings, more than half (52%) of patients treated with ferric citrate experienced a sizeable (>=1 g/dL) increase in hemoglobin over the 16-week study period compared to fewer than one in five (19%) patients treated with placebo. Rates of adverse events (“side effects”) were similar to placebo; diarrhea in some patients and constipation in others were the most common. There were also favorable effects of ferric citrate on laboratory metrics of bone and mineral metabolism. (more…)
Anemia, Author Interviews, ENT, Hearing Loss, JAMA / 31.12.2016

MedicalResearch.com Interview with: Kathleen Schieffer, BS, PhD Candidate Biomedical Sciences and Clinical and Translational Science Clinical and Translational Science Fellow Hershey, PA 17033 MedicalResearch.com: What is the background for this study? Response: Hearing loss is common in the United States, with its prevalence increasing with each decade of life. Iron deficiency anemia is a common, reversible condition, associated with negative health outcomes. The inner ear is highly sensitive to ischemic damage and previous animal studies have shown that iron deficiency anemia alters the inner ear physiology. Understanding the association between iron deficiency anemia and hearing loss may open new possibilities for treatment. (more…)
Annals Internal Medicine, Author Interviews, Hematology, Karolinski Institute / 21.12.2016

MedicalResearch.com Interview with: Märit Halmin, MD, PhD student Department of Medical Epidemiology and Biostatistics, Karolinska Institutet,  Stockholm, Sweden MedicalResearch.com: What is the background for this study? Response: During recent years the possible negative effects among recipients of stored red blood cells have been investigated.  Despite a large number of studies, including four randomized trials, no consensus exists. We therefore performed the hitherto largest register based cohort study of transfused patients, assessing the association between length of storage of red blood cells and mortality. Our design allowed for detection of small but still clinically significant effect, if such exists. (more…)
Author Interviews, Hematology / 16.12.2016

MedicalResearch.com Interview with: Jerry Powell MD Medical Director North America Commercial Operations CSL Behring MedicalResearch.com: What is the background for this study? Response: The new IDELVION results presented at the American Society of Hematology (ASH) are from a pooled analysis of clinical studies from the global PROLONG-9FP clinical development program. The analysis assessed the relationship between estimated factor IX activity levels and clinical bleeding risk in adult hemophilia B patients treated with IDELVION using prophylaxis or on-demand (episodic) treatment. The PROLONG-9FP clinical development program included five Phase I through Phase III open-label, multicenter studies evaluating the pharmacokinetics, safety and efficacy of IDELVION in children and adults with hemophilia B. (more…)
Author Interviews, Hematology, Memory, Pediatrics, Technology / 09.12.2016

MedicalResearch.com Interview with: Steven J. Hardy, Phd Licensed Clinical Psychologist Divisions of Hematology and Oncology Children’s National Health System Assistant Professor of Pediatrics and Psychiatry & Behavioral Sciences George Washington School of Medicine and Health Sciences Washington, DC MedicalResearch.com: What is the background for this study? What are the main findings? Response: Children with sickle cell disease exhibit neurocognitive deficits as a consequence of either silent or overt cerebral infarction or disease-related non-infarct central nervous system effects (likely resulting from chronic anemia and hypoxic events). These complications often lead to impairment in executive functioning (e.g., working memory, attention, inhibition, cognitive flexibility), which can make it difficult to focus in class, plan for long-term school projects, remember and carry out multi-step tasks or assignments, and stay organized. The literature on interventions to reduce neurocognitive sequelae of sickle cell disease is extremely limited. Our research team investigated a promising home-based, computerized cognitive training program (Cogmed) involving repeated practice on performance-adapted exercises targeting working memory with a sample of youth (ages 7 – 16) with sickle cell disease. Of the participants who have enrolled in the study (n = 70), 49% exhibited working memory deficits (<25% in the general population have a working memory deficit) and were randomized to an eight-week waitlist or to begin Cogmed immediately. Participants who used Cogmed demonstrated significant improvements on multiple measures of working memory, while those randomized to the waitlist group only exhibited such improvements after receiving Cogmed. Approximately 25% of participants completed the recommended number of Cogmed sessions (20 – 25 sessions). However, analyses revealed that participants who completed at least 10 sessions (about 50% of the participants) showed comparable levels of working memory improvement. (more…)
Anemia, Author Interviews, Hematology, Pain Research / 06.12.2016

MedicalResearch.com Interview with: Kenneth I. Ataga, MD Division of Hematology/Oncology University of North Carolina at Chapel Hill Chapel Hill, NC MedicalResearch.com: What is the background for this study? What are the main findings? Response: The available treatments for acute painful episodes (also referred to as vaso-occlusive crises), the most common complication of sickle cell disease, are limited. Findings from the Phase II SUSTAIN study showed that crizanlizumab (formerly SelG1) at 5 mg/kg reduced the median rate of sickle cell disease-related pain crises per year by 45.3% vs. placebo in patients with or without concomitant hydroxyurea therapy. In addition, clinically meaningful reductions in the frequency of painful crises were observed regardless of sickle cell disease genotype.  (more…)
Accidents & Violence, Anemia, Author Interviews, Hematology, Surgical Research, Technology / 05.12.2016

MedicalResearch.com Interview with: Allan Doctor, MD Pediatric Critical Care Medicine Professor of Pediatrics and (Associate) Biochemistry Washington University School of Medicine & Saint Louis Children’s Hospital St. Louis, Missouri MedicalResearch.com: What is the background for this study? What are the main findings? Response: Our research team has developed the first nanoscale artificial cells designed to emulate vital functions of natural red blood cells. If ultimately confirmed safe for use in humans, this nanotechnology-based product, called ‘ErythroMer’, could represent a new and innovative alternative to blood transfusions that would be especially valuable in situations where stored blood is needed, but difficult to obtain or use, such as in pre-hospital or battlefield settings. The artificial cells are designed to be freeze-dried, stored for extended periods at ambient temperatures, and simply reconstituted with water for immediate use. This year, the National Academy of Sciences estimated that 30,000 civilian trauma deaths/year are preventable and of these, two-thirds arise from hemorrhage in the pre-hospital phase of care. One key goal for our team is to advance treatment for trauma victims or soldiers in austere environments by initiating resuscitation in the field, particularly when transport is prolonged. ErythroMer could be a blood substitute that medics carry in their pack and literally take it out, add water, and inject. There are currently no simple, practical means to bring transfusion to most trauma victims outside of hospitals. Delays in resuscitation significantly impact outcomes; it is our goal to push timely, effective care to field settings. (more…)
Anemia, Author Interviews, Kidney Disease / 30.11.2016

MedicalResearch.com Interview with: Dorota Drozdz M.D., Ph.D Jagiellonian University Kraków Response: In Poland and Portugal we use EPO beta for anemia treatment. Our interest was to find differences in clinical patterns taking in consideration that both countries are adherent to KDIGO recommendations an guidelines. We found that in both countries the mean hemoglobin (Hb) level and percentage of patients in target Hb level (10-12 g/dl on ESA treatment) are the same, but the approaches were different – in Poland the ESA dose was statistically lower than in Portugal and iron dose was statistically higher than in Portugal. Most other lab tests results were similar. Future secondary outcomes analysis should answer the question, which method is safer. (more…)
Author Interviews, Lancet, Outcomes & Safety, Thromboembolism, Transfusions / 28.11.2016

MedicalResearch.com Interview with: Mary A.M. Rogers, PhD, MS Research Associate Professor Research Director, Patient Safety Enhancement Program Department of Internal Medicine University of Michigan Ann Arbor, MI MedicalResearch.com: What is the background for this study? Response: Peripherally inserted central catheters (PICCs) are commonly used for vascular access in hospitalized patients. Previous studies have shown that PICCs of larger gauge (diameter) increase the risk of developing venous thromboembolism (blood clots in the deep veins that sometimes travel to the lung). Red blood cell transfusion is also known to increase the risk of venous thromboembolism. Because PICCs are often used to transfuse blood, we designed a study to investigate whether the method of transfusion delivery influences the risk of developing venous thromboembolism. (more…)
Anemia, Author Interviews, OBGYNE / 07.11.2016

MedicalResearch.com Interview with: Daniel C Benyshek, PhD Professor, Department of Anthropology Adjunct Professor, UNLV School of Medicine Co-Director, Metabolism, Anthropometry and Nutrition Lab University of Nevada, Las Vegas MedicalResearch.com: What is the background for this study? What are the main findings? Response: Maternal placentophagy is ubiquitous among nearly all terrestrial mammals, but is rare to non-existent among humans in the historic and cross-cultural records. Recently, however, human maternal placentophagy has emerged as a popular trend among a small but growing number of women in many industrialized countries. Most women engaging in the practice today consume their processed placenta in capsule form, taken daily, over several weeks postpartum. While human maternal placentophagy advocates claim many maternal health benefits from the practice, including improved postpartum mood, increased breast-milk production, and improved energy, among others, no carefully designed, placebo-controlled studies have evaluated these claims. Our randomized, double-blind, placebo-controlled pilot study (N=23) investigated some of these claims. Our study found that the postpartum iron status of participants who consumed their own encapsulated placenta (based on the three week daily intake recommendation of one prominent placenta encapsulation service), was no different from those women who consumed the same amount of beef placebo. (more…)
Anemia, Author Interviews, Genetic Research, Hematology / 27.10.2016

MedicalResearch.com Interview with: Peter M. Glazer, MD, PhD Robert E. Hunter Professor of Therapeutic Radiology and Professor of Genetics; Chair, Department of Therapeutic Radiology Yale University MedicalResearch.com: What is the background for this study? What are the main findings? Response: It is generally recognized that gene editing in blood stem cells could provide a strategy for treatment of inherited disorders such as sickle cell disease and thalassaemia. Recent excitement has focused on CRISPR/Cas9 technology because of it is so easy to use. However, the CRISPR approach introduces an active DNA cutting enzyme into cells, which can lead to off-target cuts in the genome. As an alternative, we have pursued triplex-forming peptide nucleic acids (PNAs) designed to bind site-specifically to genomic DNA via strand invasion and formation of PNA/DNA/PNA triplexes. PNAs consist of a charge-neutral peptide-like backbone and nucleobases enabling hybridization with DNA with high affinity. PNA/DNA/PNA triplexes recruit the cell’s own DNA repair machinery to initiate site-specific editing of the genome when single-stranded ‘donor DNAs’ are co-delivered as templates containing the desired sequence modification. We found that triplex-forming PNAs substituted at the gamma position yielded high levels of gene editing in blood stem cells in a mouse model of human β-thalassaemia. Injection of thalassemic mice with nanoparticles containing gamma PNAs and donor DNAs ameliorated the disease phenotype, with sustained elevation of blood hemoglobin levels into the normal range and up to 7% β-globin gene correction in stem cells, with extremely low off-target effects. We conclude that the combination of nanoparticle delivery and next generation PNAs may offer a minimally invasive treatment for genetic disorders of the blood that can be achieved safely and simply by intravenous administration. (more…)
Author Interviews, Hematology, PAD / 21.09.2016

MedicalResearch.com Interview with: MedicalResearch.com: What is the background for this study? What are the main findings? Response: Critical limb ischemia (CLI) is the most severe form of peripheral arterial disease whereby a severe obstruction of the arteries markedly reduces blood flow to the extremities (hands, feet and legs) causing severe pain, skin ulcers, sores, or gangrene.  Up to 30% of patients with CLI do not qualify for conventional interventions, such as a bypass or angioplasty, putting them at risk for amputation. The MOBILE trial (MarrOwStim™ PAD Kit for the Treatment of Critical LimB IschemIa in Subjects with Severe Peripheral ArteriaL DiseasE) was designed to assess the safety and efficacy of using autologous concentrated bone marrow aspirate (cBMA), cells derived from the patient's own bone marrow, to restore blood flow and prevent amputations in patients with CLI. MOBILE is a Phase 3, double-blind, randomized, placebo-controlled trial that evaluated 152 patients with CLI at 24 centers in the U.S. Patients were randomized to receive cBMA or placebo via injection at 40 sites on the symptomatic leg.  The cBMA was obtained from each patient using the MarrowStim PAD kit.  The placebo group underwent a sham bone marrow aspiration and received needle punctures in the index leg. The primary efficacy endpoint was amputation-free survival, defined as freedom from all causes of death and/or major amputation, at 52 weeks after treatment. Other endpoints included changes in blood flow in the leg, wound healing, measures of pain and quality of life, and distance walked in 6 minutes. The trial completed in June 2016, and a preliminary analysis found that cBMA demonstrated a meaningful improvement in amputation-free survival and a comparable safety profile to placebo. (more…)
Anemia, Author Interviews, Hematology, Lancet, Surgical Research / 02.09.2016

MedicalResearch.com Interview with: Alhossain A. Khalafallah, Clinical Professor Menzies Institute for Medical Research, University of Tasmania, Australia Consultant Haematologist Launceston General Hospital Australia MedicalResearch.com: What is the background for this study? Response: There are limited data regarding the effect of postoperative anemia on patient’s outcomes. The issue of postoperative anemia was noticeably to affect a large cohort of patients world-wide. This study was aiming at comparing the new approach with a single ferric carboxymaltose infusion versus standard or routine usual care for management of postoperative anemia. (more…)
AHA Journals, Anemia, Author Interviews, Hematology, Stroke / 30.08.2016

MedicalResearch.com Interview with: Raphae Barlas M.A 3rd year MBChB student The Institute of Applied Health Sciences Aberdeen MedicalResearch.com: What is the background for this study? What are the main findings? Response: Anemia and stroke are both common conditions. While previous studies have found an association between anemia on admission and increased mortality in stroke patients, this was not consistent throughout the literature. We aimed to comprehensively assess this association by conducting our own observational study, consisting of 8000 patients from UK regional stroke registry data. We then aggregated our findings into a systematic review and meta-analysis of the existing literature for a total study population of approximately 30,000 patients. (more…)