Classical Hematology

Hemophilia management is shifting from traditional intravenous therapies toward subcutaneous agents.

Data from a large New York State analysis found that sickle cell disease hospitalizations are becoming more severe over time, with significant regional disparities in outcomes, costs, and access to specialized care.

The safety and effectiveness of marne-cel were established in an open-label, single-arm, multicenter phase 1/2 clinical trial.

Pegvaliase becomes the only enzyme substitution therapy approved to reduce blood phenylalanine concentrations in patients with phenylketonuria.

FDA priority review of rusfertide signals a new PV option, cutting phlebotomy reliance and improving hematocrit control, with pharmacists guiding injections and safety.

FDA clears oral Wegovy pills for weight loss, while Lemme Burn Gummies tout metabolic support; plus new thalassemia anemia drug and Lucentis biosimilar.

Effective SCD pain management requires an integrated, multimodal strategy across outpatient and inpatient settings.

The FDA approved mitapivat, the first oral therapy for thalassemia due to anemia, offering hope for patients with transfusion-dependent and non–transfusion-dependent forms.

Narsoplimab gains FDA approval for treating transplant-associated thrombotic microangiopathy, showing promising survival rates in high-risk patients.

FDA approves Fesilty, a new fibrinogen concentrate, offering hope for effective treatment of congenital fibrinogen deficiency and acute bleeding episodes.

New findings highlight the need for pneumococcal booster vaccinations in children with sickle cell disease to maintain immunity and prevent serious complications.

Fitusiran and concizumab are transforming treatment options and patient outcomes in hemophilia A and B with or without inhibitors.

FDA approves Omisirge, the first cell therapy for severe aplastic anemia, offering hope for patients lacking donor matches and improving recovery rates.

Experts discuss the complexities of gene therapy for sickle cell disease, emphasizing patient support, psychosocial factors, and long-term care considerations.

With this action, Thrombate III has become the first and only antithrombin concentrate approved for adult and pediatric patients with hereditary antithrombin deficiency (hATd).

A retrospective analysis found no associations between lipid parameters and the success of blood stem cell mobilization in a cohort of healthy donors.

Experts gathered to discuss the critical need for real-time learning, multidisciplinary collaboration, and comprehensive patient and family support to address unforeseen complications in gene therapy.

Speakers at an ASTCT Gene Therapy Summit highlighted novel conditioning approaches without the use of busulfan, which can cause toxic effects in patients with hematologic malignancies.

Experts at the ASTCT Gene Therapy Summit discussed the multifaceted nature of the gene therapy process for patients with sickle cell disease.

FDA expands Vonvendi use for von Willebrand disease (VWD), enhancing treatment options for adults and children to manage bleeding effectively.

Rilzabrutinib gained FDA approval as the first BTK inhibitor for chronic immune thrombocytopenia, offering new hope for patients with persistent symptoms.

The designation is supported by promising results from the ongoing phase 1/2 BEACON clinical trial.

Mona El-Mouwfi explores how pharmacists enhance hydroxyurea therapy for sickle cell disease, improving patient outcomes and addressing treatment barriers effectively.

The approval builds upon concizumab’s previous December 2024 clearance and allows for treatment in patients with hemophilia A or B, both with or without inhibitors.

Avatrombopag gains FDA approval for pediatric ITP treatment, offering a new oral option for managing chronic thrombocytopenia in children.