Hematology

Increased immunoglobulin replacement therapy led to reduced infections and managed secondary immune deficiencies such as hypogammaglobulinemia.

Collaboration between community centers and academic medical centers can improve patient access to these complex therapies.

The approval marks the first and only T-cell engaging bispecific antibody administered subcutaneously to treat patients after 2 or more lines of systemic therapy.

Site-of-care considerations can pose challenges with these therapies.

These findings build off research from March 2023 that demonstrated efficacy and safety of renizgamglogene autogedtemcel in patients with sickle cell disease.

The FDA approved blinatumomab for treatment of patients with CD19-positive Philadelphia chromosome-negative B-cell precursor acute lymphoblastic leukemia.

Mim8 had a safe and well-tolerated profile, with no deaths or thrombotic events reported for patients with hemophilia A.

With this approval, Yimmugo is the first approved product from the Biotest Next Level production facility.

Trametinib may become an alternative treatment to hematopoietic stem cell transplantation in patients with relapsed or refractory juvenile myelomonocytic leukemia (JMML).

Brentuximab vedotin in combination with lenalidomide and rituximab had a favorable safety profile for patients with relapsed/refractory diffuse large B-cell lymphoma.

This article provides an overview of data supporting the utility of covalent Bruton tyrosine kinase inhibitors in chronic lymphocytic leukemia and details important considerations when choosing among agents.

A 1-time infusion of valoctocogene roxaparvovec had demonstrated sustained bleed control and factor VIII expression in patients with hemophilia A.

Individuals with very low- to intermediate-risk myelodysplastic syndromes treated with luspatercept (Reblozyl; Bristol Myers Squibb) achieved improvements in hemoglobulin levels.

After allogeneic stem cell transplantation overall survival and relapse-free survival rates in patients with relapsed or refractory acute myeloid leukemia were 52% and 47%, respectively.

Imetelstat (Rytelo; Geron Corp) is a first-in-class telomerase inhibitor.

According to investigators, the primary and key secondary end points were met during the 52-week placebo-controlled period of the trial.

Asciminib demonstrated stronger efficacy, safety, and tolerability, as well as less adverse events in patients compared with those who received investigator-selected standard-of-care tyrosine kinase inhibitors.

Patients with sickle cell disease with more than a 6-month delay transitioning from pediatric to adult care were about twice as likely to be hospitalized.

Lisocabtagene maraleucel (Breyanzi; Bristol Myers Squibb) is a CD19-directed CAR T-cell therapy that is delivered as a one-time infusion.

Researchers observed that patients aged 80 years and older with acute myeloid leukemia who were treated with venetoclax and a hypomethylating agent had prolonged overall survival.

The indication is for adult patients who have received at least 2 prior lines of systemic therapy and is based on the response rate and duration of response shown in a phase 2 trial.

Antihemophilic factor (recombinant) Fc-VWF-XTEN fusion protein-ehtl was initally approved in February 2023 for adults and children with hemophilia A for prophylaxis and on-demand treatment to control bleeding.

FDA approves groundbreaking gene therapies for sickle cell disease, revolutionizing treatment.

These developments highlight the dynamic landscape of health care innovation and the collaborative efforts driving progress in disease management and treatment.

The Rika system uses an individualized nomogram to help determine the plasma collection volume that is needed for each individual donor.

Compared with placebo, acalabrutinib plus standard of care chemoimmunotherapy demonstrated better progression-free survival in patients with mantle cell lymphoma.

With gene therapy, anti-tissue factor pathway inhibitors, and RNA interference therapy targeting antithrombin, there are many new options that will be available for the patients impacted by hemophilia.

Despite the increased survival rates in all 4 evaluated racial groups, both adult and pediatric non-Hispanic African American patients continued to have worse outcomes.

The 1-time treatment may be a better alternative for patients who don’t want to undergo frequent doses of standard of care intravenous factor IX infusions.

Advances, challenges, and promising innovations emerge.