Hematology, Legal-Malpractice, Pharmaceutical Companies / 04.01.2025

Oxbryta, a groundbreaking treatment for sickle cell disease, brought hope to patients suffering from this debilitating condition. Approved by the FDA in 2019, the drug was designed to improve hemoglobin levels and reduce the painful complications associated with the disease. However, recent legal claims have sparked concern among patients and the pharmaceutical industry. Allegations surrounding the drug's safety and efficacy have led to a wave of lawsuits, potentially altering the landscape of sickle cell treatment. The emergence of Oxbryta injury lawsuits raises serious questions about the accountability of pharmaceutical companies and the adequacy of drug safety protocols. Patients who relied on Oxbryta to improve their quality of life now face uncertainty, with some alleging that the medication caused unexpected side effects or failed to deliver promised results. For many, these lawsuits represent a quest for justice and compensation for the harm endured, while for the broader healthcare industry, they highlight the critical need for transparency and rigorous testing. This article sheds light on the impact of Oxbryta lawsuits on patients and the pharmaceutical industry, addressing key concerns and implications for both parties. (more…)
Author Interviews, Cancer Research, Exercise - Fitness, Hematology / 16.12.2024

MedicalResearch.com Interview with: Janine Joseph, MS, MBA Senior Research Specialist Cancer Prevention & Control Roswell Park Comprehensive Cancer Center Buffalo, NY MedicalResearch.com: What is the background for this study? Response: Due to improvements in treatments, patients with multiple myeloma (MM) are experiencing longer lifespans and seeking ways to enhance their quality-of-life.  Fatigue, pain and negative psychological states such as anxiety and depression are common in cancer patients, including patients with MM.  Pain is particularly relevant, as MM can cause painful osteolytic (bone) lesions. The trials we discussed at ASH were aimed at evaluating the effect of exercise, specifically supervised resistance training and unsupervised walking exercise, on these and other states, including physical function and immune profile. (more…)
Anemia, Author Interviews, JAMA, OBGYNE / 23.08.2024

Esa M. Davis, M.D., M.P.H , F.A.A.F.P Professor of Medicine and Family and Community Medicine Associate Vice President of Community Health and Senior Associate Dean of pPopulation Health and Community Medicine University of Maryland School of Medicine Dr. Davis joined the U.S. Preventive Services Task Force in January 2021     MedicalResearch.com: What is the background for this study? What are the main findings? Response: Iron is important to overall health, and people need more iron when they are pregnant. This can make pregnant people at increased risk for iron deficiency, which can progress to anemia and cause complications for both moms and their babies. After reviewing the latest available research, the Task Force found that there is not enough evidence on whether pregnant people who do not have signs or symptoms of iron deficiency or anemia should be screened—or take iron supplements—to improve their health or the health of their baby.   (more…)
Hematology, Leukemia / 02.07.2024

bone-marrow-aspirateBone marrow aspiration is a medical procedure where a small amount of the liquid part of your bone marrow is removed for examination. A bone marrow aspirate can be essential in diagnosing and monitoring various conditions affecting your blood cells, such as anemia, leukemia, and infections. Understanding this procedure can provide insight into why your doctor may recommend it and what to expect. During a bone marrow aspiration, your healthcare provider will take a sample using a needle inserted into a larger bone, typically the hip. The process is usually performed alongside a bone marrow biopsy, which involves taking a small amount of solid bone marrow tissue. These procedures can yield valuable information about the health and functionality of your bone marrow. Preparation for bone marrow aspiration involves a few straightforward steps to ensure your comfort and safety. Knowing the potential risks and benefits can help you feel more at ease and prepared for this critical diagnostic tool. (more…)
Author Interviews, Hematology, Pain Research, Pediatrics / 10.12.2023

MedicalResearch.com Interview with: David Brousseau, MD, MS Chair of Pediatrics Nemours Children’s Health, Delaware and the Sidney Kimmel Medical College at Thomas Jefferson University  MedicalResearch.com: What is the background for this study? Response: Sickle cell disease (SCD) is an inherited red blood cell disorder – the most common genetic disorder in the United States, affecting about 100,000 Americans (1 of every 365 Black births and 1 of every 16,3000 Hispanic-American births) (source: CDC). Pain is its most common symptom. Patients may experience acute or chronic pain or both. Acute episodes of pain, or pain crises, can vary in duration and severity. Many are treated at home; however when the pain is excruciating and cannot be treated at home, they lead to Emergency Department (ED) visits and even hospitalization. Reducing pain through prompt administration of pain medication in the ED is a core principle of national guidelines for SCD care. However, little data exists on how pain scores and changes in pain scores in the ED are associated with the patient’s disposition and the odds of a return visit. (more…)
Author Interviews, Cancer Research, Hematology, Leukemia / 15.06.2023

Medical Research Interview: Dr. Daniel Thomas MD PhD FRACP FRCPA Program Leader, Blood Cancer Precision Medicine Theme at the South Australia Health Medical Research Institute Clinical Hematologist, Royal Adelaide Hospital Associate Professor, Adelaide Medical School, The University of Adelaide MedicalResearch.com: What is the background for this study? Would you briefly describe the condition of CMML? Response: Chronic myelomonocytic leukemia (CMML) is a rare, but increasingly frequent, clonal stem cell disorder that results in hyperproliferation of inflammatory monocytes, a form of white blood cells. It features both myelodysplasia and myeloproliferation. CMML is most often found in older adults and leads to anemia, decreased quality of life, and an increased risk of acute myeloid leukemia (AML). Granulocyte-macrophage colony-stimulating factor (GM-CSF) is a cytokine that stimulates production, growth, differentiation, activation, and function of myeloid cells (monocytes, neutrophils, and eosinophils). In the presence of RAS-pathway mutations, a greater sensitivity to GM-CSF contributes to the hyperproliferation of myelocytes in myelodysplastic leukemias such as CMML, juvenile myelomonocytic leukemia (JMML), and acute myeloid leukemia (AML).  In CMML, greater sensitivity to GM-CSF stimulates excessive monocytic precursor proliferation. The PREACH-M Trial, which stands for PREcision Approach to CHronic Myelomonocytic Leukemia, assesses the efficacy of lenzilumab in addition to azacitidine in treatment-naïve CMML participants with RAS-pathway mutations (KRAS, NRAS, CBL) and separately high dose ascorbate in participants with TET2 mutations who do not have RAS-pathway mutations. The study is currently underway and actively enrolling.  It is being conducted and funded by the South Australian Health and Medical Research Institute (SAHMRI).  (more…)
Anemia, Author Interviews, Diabetes, Kidney Disease, NEJM / 30.05.2023

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

MedicalResearch.com: What is the background for this study? What is dapagliflozin primarily indicated for?   Response: Dapagliflozin is a sodium glucose cotransporter 2 (SGLT2) inhibitor. The multinational, double-blinded, randomized, placebo-controlled, DAPA-CKD trial demonstrated the kidney and cardiovascular benefits of dapagliflozin in 4304 patients with chronic kidney disease (CKD) with and without type 2 diabetes (T2D). Based on the results of this and other trials, current guidelines recommend use of SGLT2 inhibitors in patients with CKD, T2D, or heart failure. Anemia is common among patients with CKD and is associated with worse clinical outcomes. Previous studies showed that SGLT2 inhibitors increase hemoglobin and hematocrit levels, but data are lacking in patients with CKD with and without T2D. In this post-hoc analysis of DAPA-CKD, we assessed the effect of dapagliflozin versus placebo on the correction and prevention of anemia in this population. (more…)
Author Interviews, Columbia, Genetic Research, Hematology, NEJM / 16.12.2021

MedicalResearch.com Interview with: Markus Y Mapara, MD Professor of Medicine Director of the Blood and Marrow Transplantation Columbia University Medical Center MedicalResearch.com: What is the background for this study? What are the main findings?  Response: Sickle cell disease is caused by a point mutation in the beta-globin gene of hemoglobin  resulting in the production of abnormal hemoglobin which leads to formation of sickle-shaped RBC under conditions of low oxygen. Sickle cell disease affects about 100,000 patients in the US which are predominantly African  American. The only curative approach is to perform an allogeneic bone marrow transplant which is however fraught with significant treatment-related risks if a matched sibling donor is not available. The current study describes the successful application of a novel gene therapy  to treat patients with sickle cell disease. The strategy is based on a gene-addition approach to introduce the genetic information for a Hemoglobin F-like molecule termed HgAT87Q into hematopoietic stem cells. The expression of this novel  hemoglobin prevents polymerization of HgbS  and has now been demonstrated to prevent the occurrence of vaso-occlusive pain crises in sickle cell disease patients. (more…)
Author Interviews, Brigham & Women's - Harvard, Clots - Coagulation, Hematology, Neurological Disorders, Pain Research / 22.05.2021

MedicalResearch.com Interview with: Daniel Chasman, PhD Pamela Rist, ScD, Yanjun Guo, MD, PhD Division of Preventative Medicine Brigham and Women’s Hospital  MedicalResearch.com: What is the background for this study? What are the main findings? Response: There has been speculation in the field about relationships between coagulation and migraine susceptibility for some time, but previous research has been largely inconclusive. In this study, we leveraged Mendelian randomization, a mode of genetic analysis that can support or refute potential causal effects on a health outcome, to examine whether hemostatic factors may contribute to risk of MA. (more…)
Author Interviews, Cost of Health Care, Genetic Research, Hematology, JAMA / 22.03.2021

MedicalResearch.com Interview with: Patrick DeMartino MD Pediatric Hematology and Oncology Fellow Doernbecher Children's Hospital Oregon Health & Science University MedicalResearch.com: What is the background for this study? Response: Dozens of gene therapies are expected to be on the market within a decade or so. Much has been written about the high prices of the therapies currently on the market (exceeding $1 million). However, only a small number of patients are eligible for these existing therapies each year. Gene therapy for sickle cell disease (SCD) appears promising and would potentially apply to a relatively large number of individuals in the U.S. We sought to explore potential affordability challenges associated with a gene therapy for SCD. (more…)
Author Interviews, Genetic Research, Hematology / 08.12.2020

MedicalResearch.com Interview with: Steven Pipe, MD Professor of Pediatrics and Pathology Laurence A. Boxer Research Professor of Pediatrics and Communicable Diseases Pediatric Medical Director, Hemophilia and Coagulation Disorders Program Director, Special Coagulation Laboratory University of Michigan MedicalResearch.com: What is the background for this study? Response: Hemophilia B is an inherited bleeding disorder where patients are missing clotting factor IX (9), a critical blood clotting protein.  Patients with a severe deficiency are at risk for traumatic and spontaneous bleeds – primarily into joints.  Repeated bleeding into joints causes more than acute pain and swelling but also leads to inflammatory and degenerative changes in joints that eventually leads to severe debilitating arthritis that can be crippling.  To try to prevent this, patients as young as infants are placed on regular infusions of clotting factor IX concentrates.  The relatively short half-life of factor IX means patients must infuse on average once to twice a week.  These can only be delivered intravenously – parents and then patients themselves have to learn this.  Prophylaxis must be continued lifelong to try to prevent bleeding events and protect joint health over the lifespan.  This is a tremendous burden on the patient and their caregivers. Even with regular prophylaxis, joint bleeds may still occur and arthropathy may still ensue.  This is because the blood levels often reach critically low levels prior to the next infusion.  Gene therapy aims to deliver a functional copy of the factor IX gene such that the patient’s own liver will make a continuous supply of factor IX that is delivered to the bloodstream.  At steady state with levels close to or within the normal range, patients would no longer be subject to bleeding events and would not require prophylaxis any longer.  We hope that such a one-time treatment would produced durable, “functionally curative” levels of factor IX. (more…)
Author Interviews, COVID -19 Coronavirus, Hematology / 04.12.2020

MedicalResearch.com Interview with: George Sakoulas, MD Sharp Memorial Hospital and Sharp Rees-Stealy Medical Group Associate Adjunct Professor UC San Diego School of Medicine San Diego MedicalResearch.com: What is the background for this study? Would you briefly explain what is meant by IVIG?   Response: IVIG stands for intravenous immunoglobulin. It is a preparation from pooled blood donors isolating antibodies in their blood and highly concentrated for use. It's uses are to either replace immunoglobulins in patients who do not produce enough of them because of some underlying immunodeficiency or, as in the case of COVID, they take advantage of the immune signaling properties of the non-variable part of the antibodies. Many immune cells have receptors that bind the non-variable (also called the Fc-gamma region), and when bound, the activity of these activated immune cells is modulated. IVIG is used in many immunologic complications of infections like Guillan-Barre, Kawasaki's disease, immune mediated encephalitis from mycoplasma, just to name a few. We decided to study IVIG because:
  1. Our anecdotal yet successful use of IVIG in treating ARDS due to influenza and other viruses.
  2. Our very definitive successful use of IVIG in a very sick patient with COVID early in the pandemic.
  3. The weak yet positive retrospective data from China of IVIG in COVID.
  4. The mechanism of IVIG inhibiting neutrophil extracellular trap (or NETS) thought to play an important role in COVID severe disease.
  5. IVIG has been around to 4 decades and many physicians have a level of prescribing comfort.
It is worth clarifying that the benefit here is NOT from neutralizing antibodies against SARS-CoV-2 virus but rather the immunomodulatory effect. It will be very interesting to see if the effect/benefit changes over time as pooled blood donors feeding the IVIG supply do develop more antibodies against SARS-CoV-2, but this is not yet known.  (more…)
Author Interviews, Hematology / 10.07.2020

MedicalResearch.com Interview with: Prof. Dr. med. Johannes Oldenburg Chairman and Director Institute of Experimental Haematology and Transfusion Medicine University Clinic Bonn AöR, Germany  MedicalResearch.com: What is the background for this study? Response: Prophylaxis for hemophilia A is the standard of care treatment for patients because it can help prevent spontaneous bleeds, as even a single bleed may cause joint damage and impact their quality of life.1,2 The Antihemophilic factor (recombinant) (rAHF) Hemophilia A (HA) outcome Database (AHEAD) study, which has been running for 6 years, evaluates long-term, real-world outcome data on effectiveness, safety and joint health in patients with hemophilia A who are receiving rAHF (ADVATE®) and ADYNOVI.  (more…)
Author Interviews, Hematology / 25.06.2020

MedicalResearch.com Interview with: TakedaDr. med. Wolfhard Erdlenbruch, M.D. Vice President Head of Global Medical Affairs Hematology MedicalResearch.com: What is the background for this study? Response: At the World Federation of Hemophilia Virtual Summit 2020 (WFH 2020), results were presented from a real-world, post-marketing surveillance study aimed to evaluate the safety and effectiveness of RIXUBIS® in adult and pediatric patients with hemophilia B in South Korea, entitled “Safety and Effectiveness of Rixubis in Patients with Hemophilia B in South Korea: A Real-World, Prospective, Post-marketing Surveillance Study”. Data from the study demonstrate the safety and efficacy profile of RIXUBIS® for treatment of bleeds, perioperative/surgery, and prophylaxis in adult and pediatric patients with hemophilia B in the real-world setting in South Korea. The study showed that 86.6% (123/142) of hemostatic effectiveness assessments for RIXUBIS® were reported as good or excellent, and of the 11 adverse events reported, all were mild in severity, with 10 resolved/recovered events not related to RIXUBIS®, and one event (inhibitory antibody development) unconfirmed.1 RIXUBIS® [Nonacog gamma, recombinant FIX concentrate] is a recombinant coagulation factor IX product, indicated for the control and prevention of bleeding episodes in patients with hemophilia B. (more…)
Author Interviews, CMAJ, Hematology / 13.01.2020

MedicalResearch.com Interview with: Stig E Bojesen Professor, chief physician, dr.med.sci. Dept of Clinical Biochemistry, Herlev Gentofte Hospital Copenhagen University Hospital Department of Clinical Medicine University of Copenhagen MedicalResearch.com: What is the background for this study? Response: Before this study, we did not know the value of an incidental finding of lymphopenia of an otherwise healthy individual from the general population. This is curious since lymphocyte count is a very simple measurement done almost every time you have a blood test done. (more…)
Author Interviews, Hematology / 19.12.2019

MedicalResearch.com Interview with: Tadeusz Robak MD, Ph.D. Professor of Hematology Medical University of Lodz, Poland MedicalResearch.com: What is the background for this study? What are the main findings?
  • Rozanolixizumab is an advanced SC anti-neonatal FcRn therapy currently in clinical development which has the potential to provide a targeted, convenient option to optimize individualized patient care.
  • The rozanolixizumab Phase II (TP0001) study was specifically designed to explore multiple dose regimens in order to inform the dosing strategy for further development in primary immune thrombocytopenia (ITP). Patients received either a single dose (1 x 15 mg/kg or 1 x 20 mg/kg) or multiple doses (5 x 4 mg/kg, 3 x 7 mg/kg, 2 x 10 mg/kg) of subcutaneous (SC) rozanolixizumab. The total dose was similar in all treatment groups, ranging from 15 to 21 mg/kg.
  • Rozanolixizumab was well tolerated by patients with primary ITP across all dose groups, consistent with previous rozanolixizumab studies. Additionally, improved platelet counts and reduced immunoglobin G (IgG) levels were seen at all doses and regimens of rozanolixizumab treatment, with higher response rate, higher percentage of responders and shorter time to response achieved by the 1 x 15 mg/kg, 1 x 20 mg/kg and the 3 x 7 mg/kg rozanolixizumab dose groups. These safety, tolerability and efficacy data support the Phase III development in patients with primary ITP.
(more…)
Author Interviews, Hematology, Lymphoma / 12.12.2019

MedicalResearch.com Interview with: Genentech Priscilla White, spokesperson Senior Manager, Corporate Relations Genentech MedicalResearch.com: What is the background for this study? What are the main findings?
  • Response: Mosunetuzumab is a T-cell engaging bispecific antibody designed to target CD20-positive B-cell blood cancers, by binding to both CD20 (on the surface of B-cells) and CD3 (on the surface of T-cells).
  • Analyses from the ongoing Phase I/Ib GO29781 study indicate that mosunetuzumab can produce durable responses in patients who have relapsed or who are refractory to prior treatment(s), including those who have relapsed or who are resistant to CAR T-cell therapy.
  • Results from this dose-escalation study showed encouraging efficacy with an objective response rate (ORR) of 62.7 percent (n=42/67) in slow-growing Non-Hodgkin Lymphoma and 37.1 percent (n=46/124) in aggressive NHL across all dose levels assessed.
  • Additionally, data demonstrated a complete response (CR) rate of 43.3 percent (n=29/67) in slow-growing NHL and 19.4 percent (n=24/124) in aggressive NHL. CRs showed durability, with 82.8 percent (n=24/29) of patients with slow-growing NHL remaining in remission up to 26 months off initial treatment and 70.8 percent (n=17/24) of patients with aggressive NHL, remaining in remission up to 16 months off initial treatment.
  • Of the participants who received prior CAR T-cell therapy, the ORR was 38.9 percent (n=7/18), and 22.2 percent (n=4/18) achieved a CR.
  • Adverse reactions included cytokine release syndrome (CRS) in 28.9 percent of patients with 20.0 percent at Grade 1 and 1.1 percent at Grade 3. Grade 3 neurological adverse events occurred in 3.7 percent of patients.
(more…)
Author Interviews, Hematology, Lymphoma / 10.12.2019

MedicalResearch.com Interview with: Steven M. Horwitz, MD Memorial Sloan Kettering Cancer Center New York, NY MedicalResearch.com: What is the background for this study? Response: Relapsed or refractory Peripheral T-Cell Lymphoma (R/R PTCL) remains a disease of significant unmet medical need. Duvelisib is an oral dual inhibitor of PI3K-δ and PI3K-γ approved for the treatment of adult patients with relapsed or refractory chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL) after at least two prior therapies, relapsed or refractory follicular lymphoma (FL) after at least two prior systemic therapies, and is being developed for the treatment of additional hematologic malignancies including R/R PTCL. In early studies, we saw a suggestion of quite good activity of duvelisib as a single agent in a range of subtypes of T-cell lymphoma. The PRIMO study is an ongoing, multi-center, open-label, registration-directed Phase 2 study evaluating duvelisib in patients with R/R PTCL that is expected to enroll approximately 120 patients. The study includes both a dose optimization phase and an expansion phase. The Primo study will be sufficiently powered to give a much more precise estimate of the activity in peripheral t cell lymphomas. However, prior to initiating the main cohort we needed to first try to identify an optimal dose. That “dose optimization cohort” is the subject of our presentation here. (more…)
Author Interviews, Hematology, Lymphoma / 10.12.2019

MedicalResearch.com Interview with: Prof. John Seymour, MBBS, Ph.D Lead investigator of the MURANO Trial Director. Department of Hematology at the Peter MacCallum Cancer Centre & Royal Melbourne Hospital in Australia MedicalResearch.com: What is the background for this study? What are the main findings?
  • MURANO is an international, multicenter, open-label, randomized Phase 3 study designed to evaluate the efficacy and safety of venetoclax in combination with rituximab compared with bendamustine in combination with rituximab in patients with relapsed/refractory (R/R) chronic lymphocytic leukemia (CLL).
  • At this year’s American Society of Hematology (ASH) annual meeting, we presented results from the four-year updated analysis from the study, which showed an 81 percent reduction in the relative risk of disease progression or death in patients randomized to the chemotherapy-free, two year fixed-duration treatment course of venetoclax plus rituximab and higher rates of minimal residual disease (MRD)-negativity compared to the standard of care regimen, bendamustine plus rituximab.
  • The long-term data further support the sustained clinical benefit of fixed-duration treatment with venetoclax in combination with rituximab for this patient population.
  • The safety profile of the combination is consistent with the known safety profile of each individual therapy alone. There were no new serious safety issues observed in the MURANO study since the last update. 
(more…)
Author Interviews, Biomarkers, Hematology, Transplantation / 09.12.2019

MedicalResearch.com Interview with: Hrishikesh Srinagesh MD The Tisch Cancer Institute at Mount Sinai MedicalResearch.com: What is the background for this study? Response: Graft-versus-host disease (GVHD) is the leading cause of non-relapse mortality (NRM) after allogeneic hematopoietic cell transplantation (HCT). Acute GVHD occurs in approximately 50% of HCT patients and targets the skin, liver, and gastrointestinal tract primarily. The change in clinical symptoms (e.g. reduction in volume of diarrhea) is used as the primary endpoint in most trials of acute GVHD treatment, but more accurate metrics are needed to predict long-term survival. Over the past decade, the Mount Sinai Acute GVHD International Consortium (MAGIC) has studied two biomarkers important in GVHD pathogenesis: suppressor of tumorigenesis 2 (ST2) and regenerating islet-derived 3 alpha (REG3). These proteins are shed into the bloodstream when the gastrointestinal tract is damaged during GVHD, the most lethal form of acute GVHD. The concentrations of ST2 and REG3 can be used generate an individual’s estimated probability of 6 month NRM known as the MAGIC Algorithm Probability (MAP). Our study evaluated whether the MAP measured at the start of GVHD treatment and four weeks later could predict long-term survival and we compared the MAP to clinical response after four weeks, the gold standard of response.  (more…)
Author Interviews, Hematology, Lymphoma / 07.12.2019

MedicalResearch.com Interview with: Constantine Tam, M.D. Hematologist and Disease Group Lead Low Grade Lymphoma and CLL at Peter MacCallum Cancer Centre Victoria, Australia, and Lead study investigator of CAPTIVATE MedicalResearch.com: What is the background for this study? Response: The Phase 2 CAPTIVATE (PCYC-1142) clinical trial evaluated 164 patients younger than 70 years (median age of 58 years) with previously untreated CLL/SLL. Patients were planned to receive ibrutinib for 3 cycles, followed by 12 cycles of ibrutinib and venetoclax in combination. Ninety percent of patients was able to complete the planned therapy. MRD status was evaluated in PB after 6, 9, and 12 cycles and in BM after 12 cycles of the combination. (more…)
Anemia, Author Interviews, Heart Disease, JAMA, Karolinski Institute / 11.07.2019

MedicalResearch.com Interview with: Dr. Niels Grote Beverborg, MD PhD Post-doctoral research fellow Department of experimental cardiology University Medical Center Groningen, Groningen, The Netherlands Integrated CardioMetabolic Center Karolinska Institutet, Stockholm Sweden  MedicalResearch.com: What is the background for this study?   Response: Iron deficiency is very prevalent worldwide and a significant cause of morbidity and mortality, especially in vulnerable populations such as patients with heart failure. It is well known that iron deficiency can be a consequence of an insufficient iron uptake or increased iron loss (termed low iron storage), or of a chronic low inflammatory state (defective iron utilization). However, so far, we had no tools to distinguish these causes from each other in patients and have not been able to assess their potential consequences. (more…)
Author Interviews, Hematology, Leukemia, Pediatrics / 05.12.2018

MedicalResearch.com Interview with: Charles G. Mullighan, MBBS (Hons), MSc, MD Member, St. Jude Faculty Co-Leader, Hematological Malignancies Program Medical Director, St. Jude Biorepository William E. Evans Endowed Chair St. Judes Children’s Research Hospital Memphis, TN MedicalResearch.com: What is the background for this study?   Response: B-lineage acute lymphoblastic leukemia (B-ALL) is the commonest form of ALL, and the commonest childhood tumor. It is a leading cause of childhood cancer death. It consists of multiple subtypes defined by genetic alterations. These are often chromosomal translocations that deregulate oncogenes or form fusion proteins. These alterations are disease initiating events and are associated with distinct patterns of leukemic cell gene expression. Most subtypes also have additional mutations that are important for cells to become fully leukemic. Identifying these initiating genetic changes is very important to identify patients that are likely to respond or do poorly with conventional therapy (multiagent chemotherapy). Also, some identify new opportunities for targeted therapy. However, using standard genetic testing approaches such as chromosomal cytogenetics, about 30% of B-ALL patients don’t have a subtype classifying alteration. (more…)
Author Interviews, Cost of Health Care, Hematology, J&J-Janssen, Thromboembolism / 04.12.2018

MedicalResearch.com Interview with: Paul Burton MD, PhD, FACC Vice President, Medical Affairs Internal Medicine Janssen Scientific Affairs, LLC. MedicalResearch.com: What is the background for this study? What are the main findings? Response: Despite being largely preventable, venous thromboembolism (VTE) is the second leading cause of death in people with cancer. The risk of VTE is five times greater in people with cancer than those without cancer, and that risk is magnified in those receiving certain types of chemotherapy, in the newly diagnosed and in those with more advanced, metastatic disease. This 6,194-patient study examined economic burden associated with VTE, and found patients newly diagnosed with cancer who are at a higher risk of a VTE had significantly higher all-cause and VTE-related health care costs compared to patients with a lower risk of VTE. (more…)
Author Interviews, Clots - Coagulation, Hematology, J&J-Janssen / 04.12.2018

MedicalResearch.com Interview with: Paul Burton MD, PhD, FACC Vice President, Medical Affairs Internal Medicine Janssen Scientific Affairs, LLC. MedicalResearch.com: What is the background for this study? What are the main findings? Response: Treatment of venous thromboembolism (VTE) is complicated among morbidly obese patients. Current guidelines do not recommend use of Factor Xa inhibitors in these patients due to limited clinical data available. That's why Janssen undertook this study to examine XARELTO® (rivaroxaban) in these patients. In this 5,780-patient retrospective study, results found patients treated with XARELTO® had a similar risk of recurrent VTE and major bleeding compared to those taking warfarin. However, treatment with XARELTO® was associated with less all-cause health care resource utilization (HCRU) (e.g., inpatient hospitalizations and outpatient visits) and reduced total medical costs compared to warfarin. Of note, patients taking XARELTO® had an average $2,829 lower total medical costs per patient per year (PPPY) than those taking warfarin, which was mainly driven by lower hospitalization costs. (more…)
Author Interviews, Cancer Research, Hematology, J&J-Janssen / 04.12.2018

MedicalResearch.com Interview with: Caroline McKay, PhD Real World Value & Evidence, Oncology Janssen Scientific Affairs MedicalResearch.com: What is the background for this study? What are the main findings? Response: Considering patient preferences in treatment decision-making in oncology is growing in importance. While recent introduction of new treatments for multiple myeloma have improved survival and the possibility of sustaining longer remission periods, regimen options still vary with respect to efficacy, safety, and dosing. Therefore, patients and providers must consider tradeoffs inherent in making treatment decisions that are growing in complexity. Despite this, there is a lack of research describing patient preferences within the context of currently available treatment regimens. To address this gap, this study examined how multiple myeloma patients evaluate, or weigh, treatment options. Key findings from the research are that treatment preferences do not appear to be static, but instead suggest that the relative importance of treatment attributes may change over time and treatment history. Further, patients place higher importance on overall survival and progression-free survival than other treatment attributes, and may be willing to accept an increase in the risk of serious side effects and reduced convenience in exchange for greater efficacy; however, when efficacy is comparable, patients appear to place greater weight on dosing frequency than on the duration of treatment administration, i.e., more frequent dosing appears to be less preferable to patients than longer administration/infusion time.  (more…)
Anemia, Author Interviews, Cancer Research, Hematology, Leukemia / 03.12.2018

MedicalResearch.com Interview with: Dr. Alan List MD President and Chief Executive Officer Moffitt Cancer Center Tampa, FL MedicalResearch.com: What is the background for this study?   Response: In patients with lower risk Myelodysplastic Syndromes (MDS), which accounts for the vast majority of patients with MDS overall, the most common symptomatic cytopenia is anemia. These patients, overtime, become dependent upon red blood cell transfusions and with that, they face a risk of iron loading as well as complications that occur with it. The standard first line therapy that we consider for these patients is erythropoietin-stimulating agents (ESAs). Patients who are transfusion dependent have a low response rate to ESAs, and responses are of short duration. There limited effective limited treatment options for those patients unresponsive or lose response to ESAs. For years, we’ve known that the transforming growth factor (TGF)-β pathway play an important pathogenetic role in suppressing red cell maturation and cell survival. Luspatercept is an agent that acts as an erythroid maturation agent by inhibiting the TGF-β signaling pathway by neutralizing a select group of TGF-β superfamily ligands.  (more…)
Author Interviews, Cancer Research, Hematology / 03.12.2018

MedicalResearch.com Interview with: Maria-Victoria Mateos, MD, PhD Associate Professor of Medicine University of Salamanca Salamanca, Spain MedicalResearch.com: What is the background for this study? What are the main findings? Response: Alcyone trial is a phase 3 trial in which Daratumumab, the CD38 mAb has been added to a standard of care for elderly newly diagnosed myeloma patients, VMP, and compared with VMP. The main finding is that the addition of dara to VMP resulted into a significant benefit in PFS with a 57% reduction in the risk of progression and/or death. In addition, the benefit was also reported in terms of ORR and CR rate and 45% of patients receiving Dara VMP achieved CR. Minimal residual disease was evaluated and was undetectable in 27% of the patients what it is relevant because a 5% increase was observed in comparison with the publication one year ago. This means that Daratumumab as maintenance after the first 9 cycles daraVMP was able to upgrade the quality of response. Toxicity profile was acceptable and no new safety signals were reported. (more…)
Author Interviews, Hematology / 02.12.2018

MedicalResearch.com Interview with: Maria Domenica Cappellini, M.D. Principal Investigator, BELIEVE Clinical Trial Fondazione IRCCS Ca’ Granda Policlinico Hospital University of Milan, Milan, Italy MedicalResearch.com: What is the background for this study? What are the main findings? Response: Beta thalassemia is a severe, inherited hemoglobinophathy due to a reduced production of hemoglobin. Standard of care is blood transfusion and iron chelation to remove the accumulated iron by transfusion. This is a very demanding treatment. Therefore, there is the need to find new approaches for treating these patients. The BELIEVE study showed that the experimental drug luspatercept significantly reduced the need for blood transfusions in patients with beta thalassemia. More than 70 percent of patients who received luspatercept were able to cut blood transfusions by at least one-third over any consecutive 12-week period (more…)
Author Interviews, Cancer Research, Hematology, J&J-Janssen / 02.12.2018

MedicalResearch.com Interview with: Peter Voorhees, MD Plasma Cell Disorders Program Department of Hematologic Oncology and Blood Disorders Levine Cancer Institute Atrium Health MedicalResearch.com: What is the background for this study? Response: All multiple myeloma arises from its precursor conditions, monoclonal gammopathy of undetermined significance (MGUS) and smoldering multiple myeloma (SMM). Although the rate of progression to multiple myeloma for patients with MGUS is low (~5% over 5 years), patients with SMM have a ~50% likelihood of requiring therapy for their multiple myeloma within the first 5 years of diagnosis. For those at intermediate to high risk of disease progression, early intervention to delay progression of disease, thereby averting disease-related morbidities related and potentially changing the natural course of the disease, is highly desirable. On the other hand, given the fact that these patients are by definition asymptomatic and would otherwise be monitored off treatment, it is critical that any intervention applied in this group of patients is well tolerated. Daratumumab is a highly attractive candidate in this particular space, because it has single agent activity in heavily-pretreated relapsed/refractory multiple myeloma and a favorable side effect profile relative to many other myeloma therapeutics. Additionally, given the importance of impaired immune surveillance in multiple myeloma, the immuno-stimulatory effects of daratumumab in the bone marrow microenvironment could potentially reawaken robust T cell responses to the disease. (more…)