Hematology

Phase 3 trial evaluated luspatercept-aamt and shows highly statistically significant and clinically meaningful improvement in red blood cell transfusion for those with myelodysplastic syndromes.

Phase 2 APPLY-PNH trial shows positive results superiority vs anti-C5 treatment in adult patients with PNH with residual anemia despite prior therapy.

Understanding the novel mechanisms of multiple myeloma can help to develop better next-generation therapies that overcome drug resistance.

These study findings may have implications for monitoring for the development of ICANS in patients receiving CAR T-cell therapies for cancer, according to the authors.

Panelists at the AMCP Nexus conference discuss the burden of undertreated and untreated chronic kidney disease.

A researcher suggests that understanding specific cell function in the tumor microenvironment can help create effective and individualized immunotherapy cancer treatments.

Omidubicel is a stem cell-based product that utilizes nicotinamide to inhibit differentiation and to increase the migration, bone marrow homing, and engraftment efficiency of hematopoietic progenitor cells.

Researchers identified a new immunotherapeutic target that may be effectively treated with CAR T-cell therapy in patients with pretreated multiple myeloma.

Relapses remain a challenge in treating patients with leukemia.

Biology and disease type may help determine which therapeutic options could work best for patients with Waldenstrom macroglobulinemia.

Study results show that the frequency of adverse events decreased after the patients transitioned from combination therapy to monotherapy.

Belantamab mafodotin plus lenalidomide and dexamethasone was found tolerable with no new safety signals and enhanced response rates in patients with multiple myeloma.

Ivosidenib plus azacitidine shows benefits in event-free survival, overall survival, and clinical responses compared with placebo in patients with IDH1-mutated acute myeloid leukemia.

The functional and physical well-being and other scores from the survey are more favorable for those who were treated with the therapy, investigators say.

IDH inhibitors show encouraging results in patients with myelodysplastic syndrome, but longer follow ups are needed in a larger patient population to confirm the results.

Expert discusses whether epigenetic programs regulate human T cell exhaustion and whether epigenetic-based strategies can be used to enhance T cell-based therapies.

After 4 years, the 1-time cost of valoctocogene roxaparvovec appeared to be more cost-effective than the annual cost of emicizumab prophylaxis for hemophilia A, according to a new Draft Evidence Report.

Receiving benefits from the SNAP program does not alleviate the issue, according to the analysis.

Management of marginal zone lymphoma was impacted by the COVID-19 pandemic because some treatment options can reduce B-cell-produced antibodies.

The agency’s decision data for the investigational factor VIII therapy from Sanofi and Soba is set for February 28, 2023.

The biosimilar is expected to be launched as a prefilled syringe in early 2023.

A high proportion of myeloid cells may contribute to recurrence in those with long progression-free survival by promoting cancer growth and/or suppressing antitumor immunity.

The phase 2 AXIOMATIC trials assessing milvexian met its primary endpoints, with researchers anticipating a phase 3 trial to come within the year.

BleedCEASE is intended to stop bleeding from cuts and nosebleeds.

Tranexamic acid indicated for patients with hemophilia for short-term use to reduce or prevent hemorrhage and decrease the need for replacement therapy during and following tooth extraction.

Ibrutinib (Imbruvica) is the first therapy to gain FDA approval for younger patients who had no prior treatment options for chronic graft-versus-host disease.

Zynteglo is an autologous hematopoietic stem cell-based gene therapy indicated for the treatment of adult and pediatric patients with β-thalassemia who require regular red blood cell transfusions.

The regulatory submission includes data from the MOMENTUM phase 3 clinical trial that met all primary and key second efficacy end points.

The treatment regimen is first in more than 20 years to significantly improve outcomes in individuals with the fast-growing cancer, according to the company.

Kcentra is indicated for the urgent reversal of acquired coagulation factor deficiency induced by vitamin K antagonist therapy in adult patients with acute major bleeding.