Hematology

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.

The oral therapy is available through a REMS program.

These disorders can be fatal, making pharmacists’ role in detection crucial.

The results indicate promising rates of complete response, manageable adverse events, and dose-dependent exposure in both dose groups.

Treatment options are distinct in their mechanisms, safety profiles, and implications for patients’ quality of life.

In a study, nipocalimab helped more than 50% of patients with high-risk of hemolytic disease of the fetus and newborn achieve a safe live birth.

In November 2023, the FDA announced an investigation of CAR T-cell therapy based on several reported secondary T-cell malignancies.

The model was designed to help improve health outcomes, improve access to care, and lower costs for gene therapy.

Approximately 41% of enrolled patients responded to NX-2127, and the BTK degrader overcame resistance to nearly all BTK mutations that were identified to cause resistance.

According to the authors, the test has promise in guiding treatment but needs further refining to obtain information beyond molecular subtyping.

The findings suggest that further research on additional intervention methods and expanding access to hydroxyurea in Africa could benefit young patients with sickle cell disease.