David P. Steensma, MD, of Dana-Farber Cancer Institute, discusses early study findings on H3B-8800, which decreased the need for red blood cell or platelet transfusion in 14% of patients. This splicing modulator, used in the trial to treat patients with hematologic malignancies, also showed safety, dose-dependent target engagement, and a predictable pharmacokinetic profile (Abstract 673).
Andrew D. Zelenetz, MD, PhD, of Memorial Sloan Kettering Cancer Center, discusses the nature of biologic molecules and their importance in future therapeutic innovation.
Today, the U.S. Food and Drug Administration (FDA) approved fedratinib (Inrebic) for adults with intermediate-2 or high-risk primary or secondary (postpolycythemia vera or postessential thrombocythemia) myelofibrosis. “Prior to today, there was one FDA-approved drug to treat patients with...
New data suggest that people who have a parent, sibling, or child with blood cancer have a higher likelihood of being diagnosed with a hematologic malignancy themselves. A study by Sud et al published in Blood offers the first...
Just as newer drugs have significantly improved outcomes for patients with multiple myeloma in the past decade, newer imaging techniques are upgrading detection of the disease, leading to earlier treatment, but standards to help guide clinicians on the optimal use of advanced imaging have lagged...
The speed at which older individuals with blood cancers are able to walk 4 meters (about 13 feet) holds information about their overall health and may help to predict survival and unplanned hospital visits, according to study published by Liu et al in Blood. The association was...
In a study reported in the Journal of Clinical Oncology, Bustoros et al developed a model for predicting risk of progression from asymptomatic Waldenström’s macroglobulinemia to symptomatic disease requiring treatment. The study involved 439 patients with asymptomatic...
Earlier this week, the Centers for Medicare & Medicaid Services (CMS) proposed to improve the reimbursement currently given to hospitals that provide chimeric antigen receptor T-cell (CAR-T) therapy to patients with blood cancer as part of the Fiscal Year 2020 Inpatient Prospective Payment...
Although the pool of registered bone marrow donors has increased in recent years, a new study suggests that most patients of southern European and non-European descent are unlikely to have a suitable match if they need a bone marrow transplant. If an immediate registry search does not identify a...
Gary H. Lyman, MD, MPH, of the Fred Hutchinson Cancer Research Center/Seattle Cancer Care Alliance, discusses current strategies for the use of biosimilars to treat cancer-induced anemia and neutropenia, and the need to provide evidence for the efficacy of these agents to allay any concerns about their use.
The FDA recently issued announcements on a Fast Track designation, a Priority Review, two supplemental new drug applications, an investigational new drug application, and a marketing clearance. The agency also released a safety communication on cancer-related surgery. Fast Track Designation for...
This letter is a follow-up to a report published previously in The ASCO Post. Recent trials report comparable or improved efficacy of direct-acting oral coagulants (DOACs) over low–molecular-weight heparins (LMWH) in the treatment of cancer-related VTE (cVTE) at the expense of increased...
In a Japanese phase II trial reported in the Journal of Clinical Oncology, Hayashi et al found that standard site-specific treatment based on gene-expression profiling was not associated with better outcomes vs empirical paclitaxel and carboplatin in patients with cancer of unknown primary site....
The U.S. Food and Drug Administration (FDA) recently issued the following new designations and clearances: Fast Track Designation for Itolizumab for the Treatment of Acute Graft-vs-Host Disease The FDA granted Fast Track designation to itolizumab for the treatment of acute graft-vs-host ...
On December 21, 2018, the U.S. Food and Drug Administration (FDA) approved ravulizumab-cwvz (Ultomiris) for adult patients with paroxysmal nocturnal hemoglobinuria (PNH). PNH is a rare bone marrow failure disorder that manifests with hemolytic anemia, thrombosis, and peripheral blood cytopenias....
On December 21, the U.S. Food and Drug Administration (FDA) approved tagraxofusp-erzs (Elzonris) infusion for the treatment of blastic plasmacytoid dendritic cell neoplasm (BPDCN) in adults and in pediatric patients aged 2 years and older. “Prior to [this] approval, there had been no...
Alexander B. Pine, MD, PhD, of Yale School of Medicine, discusses a survey gathering data on health-care providers’ practices and preferences in using direct oral anticoagulant therapy to treat venous thromboembolism. Readers of The ASCO Post are invited to participate in this research by completing the survey, entitled: “Perspectives and Practices in Utilization of Direct Oral Anticoagulants in Patients With Cancer-Associated Venous Thromboembolism.” The survey takes approximately 3 to 4 minutes to complete and can be taken on a mobile device or a computer. The survey link is https://yalesurvey.ca1.qualtrics.com/jfe/form/SV_3l0HxrreWZhVtBz.
Researchers used machine learning to develop a new system to analyze genomic and clinical data to provide a personalized overall outcome that is patient-specific in myelodysplastic syndromes. In tests, the system outperformed the current standard prognostic tool, suggesting the new model may offer...
In the phase III MEDALIST clinical trial, luspatercept significantly reduced the need for frequent blood transfusions in just over half (53%) of patients with myelodysplastic syndromes (MDS) who were anemic, required regular red blood cell transfusions, and showed abnormal iron overload in red...
The largest prospective trial of hydroxyurea for sickle cell anemia (SCA) has shown that this treatment—long the standard of care for treating SCA in developed countries—is feasible, accepted, well tolerated, and safe for children living in sub-Saharan Africa. Tshilolo et al reported...
In a German phase II trial (RELAZA2) reported in The Lancet Oncology, Platzbecker et al found that minimal residual disease (MRD)–guided treatment with azacitidine was successful in preventing hematologic relapse in patients with myelodysplastic syndrome (MDS) and acute myeloid leukemia...
In a study reported in the Journal of Clinical Oncology, Frick et al found that allogeneic hematopoietic stem cell transplantation (HSCT) from donors with clonal hematopoiesis of indeterminate potential (CHIP) appears safe and not associated with poorer survival when donors are older, related...
The costs to treat blood cancer are higher than costs for other cancers, and the costs incurred by a patient diagnosed with a blood cancer do not return to precancer levels, according to a Milliman study commissioned by The Leukemia & Lymphoma Society (LLS). The study—The...
An emerging treatment known as adoptive T-cell therapy has proven effective in a phase II clinical trial for treating progressive multifocal leukoencephalopathy (PML), a rare and often fatal brain infection sometimes observed in patients with cancer and other diseases in which the immune...
In a study reported in The New England Journal of Medicine, Grinfeld et al identified distinct genomic subgroups among patients with myeloproliferative neoplasms that, when combined with clinical variables, offered the potential for individualized predictions of clinical outcomes. Study Details...
Frederick L. Locke, MD, of Moffitt Cancer Center, discusses the key studies behind the development of CAR T cells, major adverse events and management strategies, and the potential of this revolutionary treatment.
In a single-institution study reported in The New England Journal of Medicine, Duncavage et al found that mutation clearance after allogeneic hematopoietic stem cell transplantation (HSCT) was associated with better outcomes in patients with myelodysplastic syndrome. Study Details The study...
The U.S. Food and Drug Administration (FDA) has accepted for filing a biologics license application (BLA) for tagraxofusp (Elzonris, formerly SL-401) for the treatment of patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN), a rare hematologic malignancy. The FDA also granted Priority ...
The U.S. Food and Drug Administration (FDA) is warning that the antibiotic azithromycin should not be given on a long-term basis to prevent the inflammatory lung condition bronchiolitis obliterans syndrome in patients with cancers of the blood or lymph nodes who undergo a donor stem cell...
In a study reported in the Journal of Clinical Oncology, Treon et al found that ibrutinib monotherapy was highly active in patients with previously untreated Waldenström’s macroglobulinemia, with better response in patients without CXCR4 mutation. Ibrutinib has been shown to be active in ...
In a study reported in JAMA Oncology, Scragg et al found that monthly high-dose vitamin D supplementation, without calcium, was not associated with a reduced risk of developing cancer. Study Details The current analysis is a post hoc analysis of the Vitamin D Assessment (ViDA) study, which...
The U.S. Food and Drug Administration (FDA) has accepted for Priority Review a supplemental new drug application (sNDA) for ibrutinib (Imbruvica) in combination with rituximab (Rituxan) as a new treatment option for Waldenström's macroglobulinemia. If approved, the sNDA would expand the...
A new comparison study showed that among polycythemia vera patients who were resistant or intolerant to hydroxyurea, those treated with ruxolitinib (Jakavi) had a significantly reduced risk of thrombosis and death compared to those who received best available therapy. The study findings are based...
Results from a preplanned interim analysis of the phase III iNNOVATE (PCYC-1127) study evaluating the investigational use of ibrutinib (Imbruvica) in combination with rituximab (Rituxan) in relapsed/refractory and treatment-naive patients with Waldenström’s macroglobulinemia were...
Meletios A. Dimopoulos, MD, of the National and Kapodistrian University of Athens, discusses phase III findings on ibrutinib/rituximab vs placebo/rituximab in Waldenström’s macroglobulinemia (Abstract 8003).
On April 23, Epizyme, Inc, announced that U.S.-based enrollment of new patients into tazemetostat clinical trials is temporarily on hold. Following a safety report of a pediatric patient who developed a secondary lymphoma, the U.S. Food and Drug Administration (FDA) issued a partial clinical hold...
On April 17, the U.S. Food and Drug Administration (FDA) approved fostamatinib disodium hexahydrate (Tavalisse) for the treatment of thrombocytopenia in adult patients with chronic immune thrombocytopenia who have had an insufficient response to a previous treatment. Fostamatinib is an oral spleen ...
On April 11, Bellicum Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) has lifted the clinical hold on studies of BPX-501 in the U.S. The decision follows consultation with the FDA and agreement on amendments to the study protocols including guidance on monitoring...
Pediatric patients with solid tumors may have poor quality T cells compared to patients with leukemia, and certain chemotherapies were detrimental to the T cells and their potential to become chimeric antigen receptor (CAR) T cells, according to data presented during a media preview for the...
In a phase I trial reported in The Lancet Oncology, Burris et al determined the phase II dose of the next-generation PI3Kδ inhibitor umbralisib and found evidence of the agent’s activity in relapsed or refractory hematologic malignancies. Umbralisib exhibits improved PI3K isoform...
A sizable percentage of elderly patients with blood-related cancers such as leukemia and multiple myeloma are apt to show signs of diminished cognitive functioning—a decline that may impact their survival—a new study by investigators at Dana-Farber Cancer Institute and Brigham and...
On January 30, Bellicum Pharmaceuticals announced it has received notice from the U.S. Food and Drug Administration (FDA) that U.S. studies of BPX-501—an agent being studied to improve outcomes for patients undergoing stem cell transplant who lack a matched donor—have been placed...
Stephen M. Ansell, MD, PhD, of the Mayo Clinic, discusses integrating immune checkpoint inhibitors, improving efficacy, and reducing toxicity when treating blood cancers.
In a prospective longitudinal study reported in the Journal of Clinical Oncology, Sharafeldin et al found that cognitive function after hematopoietic cell transplantation (HCT) for hematologic malignancy was impaired among those receiving myeloablative allogeneic HCT, with a delayed effect being...
People with cancer face an increased risk for venous thromboembolism (VTE). Under current guidelines, cancer patients who develop VTE are prescribed low–molecular-weight heparin, an anticoagulant that must be injected under the skin daily for several months. While effective, this regimen can...
In a phase III trial, patients with acquired thrombotic thrombocytopenic purpura (TTP), a rare blood clotting disorder, who received the investigational drug caplacizumab showed significant improvements in the time it took to normalization of their platelet count compared to those receiving a...
Results from a phase II clinical trial presented by Kean et al at the 59th American Society of Hematology (ASH) Annual Meeting (Abstract 212) show that the drug abatacept (Orencia) nearly eliminated life-threatening severe acute graft-versus-host disease (GVHD) in patients receiving hematopoietic...
In the first comprehensive analysis of clinical trial enrollment among older adults with blood cancers, researchers from the U.S. Food and Drug Administration (FDA) found significant gaps in participation among those aged 75 and older when considered against the incidence of these malignancies in...
Alok A. Khorana, MD, of the Cleveland Clinic, discusses the prevalence of venous thromboembolism in cancer patients treated at U. S. emergency departments and associated costs, mortality, and hospital admissions in the United States (Abstract 219).
In a phase I trial, patients with an advanced or aggressive form of systemic mastocytosis, a rare blood disorder, had rapid and durable responses with few adverse effects following treatment with an investigational drug that targets the genetic mutation found in more than 90% of cases. Results were ...