Hematology

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.

Previously, glofitamab had received an accelerated approval for patients with relapsed or refractory diffuse large B-cell lymphoma who received 2 or more prior lines of systemic therapy.

This review discusses the prevalence, mechanisms of development, and evolving treatment landscape in chronic lymphocytic leukemia.

In this NCCN session, James M. Foran, MD, described the different FLT3 inhibitors that are available for FLT3-mutated AML, as well as considerations for the future.

The antibody drug conjugate was used in combination with the anti-cancer drug SG3249, and demonstrated efficacy in mouse models after 1 week.

The results demonstrated similar overall survival between patients with chronic lymphocytic leukemia who discontinued specialized follow-ups versus those who continued.

There are unique complexities when implementing bispecific antibodies, including site of care considerations, monitoring, and management of adverse events.

Unlike conventional monoclonal antibodies, BsAbs possess dual binding domains that target 2 distinct antigens simultaneously.

The mutation impacts BAF proteins and can lead to progression of follicular lymphoma, according to the investigators.

Positive phase 3 trial results showed that danicopan was more effective than placebo when treating extravascular hemolysis in paroxysmal nocturnal hemoglobinuria.

The mutations, C594Y and P735R, were found in the GNE gene and may influence sialylation, a process that is crucial for brain development.

Vadadustat (Vafseo; Akebia Therapeutics Inc) is indicated for individuals with chronic kidney disease who have been receiving dialysis for at least 3 months.

A study that analyzed data from 2000 to 2021 indicated improvements for both leukemia-free survival and overall survival for older patients with acute myeloid leukemia.

Early treatment administration with this regimen also promoted a faster decrease in viral load and shorter viral shedding in patients with hematologic diseases who were positive for COVID-19.

The test scans whole blood samples from individual human donors for the 5 parasite species that can cause malaria in humans and detects Plasmodium RNA and DNA.

In December 2023, nipocalimab (Johnson & Johnson) was granted orphan drug designation for fetal neonatal alloimmune thrombocytopenia.

The orphan drug designation (ODD) comes after positive results from a phase 2 trial that showed cevidoplenib improved platelet counts by 63.6% and 40.9% in 2 different dose groups.

Findings show that magnetic resonance imaging and lumbar puncture are not always needed when managing complications of CAR T-cell therapies, but may be beneficial in certain cases.

The approval comes after positive results from the PhALLCON study, however, further research is needed to confirm immature event-free survival findings.

This approval makes atidarsagene autotemcel the only therapy to be approved in the US for early-symptomatic early juvenile metachromatic leukodystrophy.

The authors note that understanding the DNA methylation profile in leukemia can help predict whether or not a patient will respond to treatment.

In a phase 1/2 clinical trial, lisocabtagene maraleucel helped patients with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) achieve complete response rates.

The study results also indicated that graft versus host disease status was associated with non-compliance following bone marrow transplantation.

The application was previously granted fast track designation and orphan drug designation.

If approved, epcoritamab (DuoBody CD3xCD20; AbbVie, Genmab) would be the first and only subcutaneous bispecific antibody for treatment in this patient population.

Currently, ocifisertib is being evaluated in a phase 1b and 2 trial to confirm the safety and tolerability profiles in patients with acute myeloid leukemia.

Certain patients may present asymptomatically, affecting treatment options.