Plitidepsin Evaluated for Refractory Multiple Myeloma

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.  Continue reading

One Blood Type Is a Risk For Bleeding Out After Trauma

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.

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Is Sickle Cell Really a Risk Factor for Stroke?

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

Dr. Hyacinth

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.

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TXA Increasingly Use in Shoulder Surgery To Reduce Transfusion Risk and Complications

MedicalResearch.com Interview with:

Shawn Anthony, MD, MBA Assistant Professor of Orthopaedics Icahn School of Medicine at Mount Sinai

Dr. Anthony

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.

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Investigational Imaging Test Can Help Determine Success or Failure of Bone Marrow Transplant

MedicalResearch.com Interview with:

Kirsten Williams, M.D. Blood and marrow transplant specialist Children’s National Health System

Dr. Williams

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.

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Hemophilia B: Rebinyn® Now Available To Treat Acute Bleeds and Surgical Bleeding

MedicalResearch.com Interview with:

Stephanie Seremetis, M.D. Corporate Vice President and Chief Medical Officer Biopharmaceuticals at Novo Nordisk

Dr. Stephanie Seremetis

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.  Continue reading

Phase 3 Darzalex Trial Demonstrated Meaningful Improvement in Patients with Newly Diagnosed Multiple Myeloma

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).

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Breakthrough Treatment With Prometic’s IV Plasminogen Treats Rare Disabling Disorder

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.

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Multiple Myeloma: Phase 3 Study of DARZALEX + VMP Reduced Risk of Disease Progression and Mortality

MedicalResearch.com Interview with:

Dr. Meletios A. Dimopoulos MD Professor and Chairman Department of Clinical Therapeutics University Athens School of Medicine Athens, Greece

Dr. Dimopoulos

DrMeletios 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.

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Daratumumab Monotherapy for Patients with Intermediate or High-Risk Smoldering Multiple Myeloma

MedicalResearch.com Interview with:

Craig C. Hofmeister, MD, MPH The Ohio State University 

Dr. Hofmeister

Craig CHofmeisterMD, 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.

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Cost and Side Effects Influence Patients’ Preferences for Leukemia Medications

MedicalResearch.com Interview with:

Carol Mansfield, PhD, Senior Research Economist Health Preference Assessment RTI Health Solutions www.rtihs.org

Dr. Mansfield

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.

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Transfusions From Previously Pregnant Donors Add Risk To Younger Male Recipients

MedicalResearch.com Interview with:

Rutger Middelburg, PhD Assistant Professor in clinical epidemiology Sanquin Research and LUMC

Dr. Middelburg

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.

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Emergency Transfusion of Patients with Unknown Blood Type with Blood Group O Rhesus D Positive Blood

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.

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Effects of Delayed Vs Early Umbilical Cord Clamping on Infant Anemia

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.

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Novel Oral Iron Formulation Can Correct Anemia in Non-Dialysis CKD

MedicalResearch.com Interview with:

Dr. Glenn M. Chertow, MD Professor Medicine, Nephrology Stanford University School of Medicine

Dr. Glenn M. Chertow

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.

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Iron Deficiency Anemia Linked to Hearing Loss

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.

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No Association Between Length of Red Blood Cell Storage and Mortality

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.

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Bleeding Risk Reduction in Relation to Predicted Factor IX Levels in Hemophilia B Patients Receiving Idelvion (rIX-FP)

MedicalResearch.com Interview with:

Jerry Powell MD Medical Director North America Commercial Operations CSL Behring

Dr. Jerry Powell

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.

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Computerized Working Memory Training in Pediatric Sickle Cell Disease

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

Dr. Steven J. Hardy

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.

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Monoclonal Antibody Crizanlizumab Reduces Sickle Cell Pain Crisis

MedicalResearch.com Interview with:

Kenneth I. Ataga, MD Division of Hematology/Oncology University of North Carolina at Chapel Hill Chapel Hill, NC

Dr. Kenneth I. Ataga

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. 

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