Hematology

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.

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.

Less manufacturing time could be the difference between a patient receiving this life-saving therapy vs not.

Pharmacy Times provides a comprehensive recap of the latest advancements in hematology and breast cancer, shared at the 2023 American Society of Hematology and the San Antonio Breast Cancer Symposium conferences

The MRD-guided treatment approach has a significant advantage over chemotherapy.

It can also help identify patients who would benefit from disease-specific therapies.

Data support the quadruplet regimen as a new standard of care for transplant-eligible patients.

There is a significant efficacy-effectiveness gap in standard-of-care regimens.

Further policy changes to promote gender equity may be needed.