Hematology

The FDA has approved small-molecule pyruvate kinase R activator mitapivat for the treatment of anemia in adults with alpha- or beta-thalassemia.

The approval indicates narsoplimab as the first and only treatment for hematopoietic stem cell transplant-associated thrombotic microangiopathy.

Stay updated with the latest healthcare breakthroughs, including FDA news and phase 3 trial readouts, in this week's essential news roundup.

Al-Samkari discusses a potentially novel use for bevacizumab in improving outcomes for patients with lesions from non-HHT etiologies.

Grifols’ fibrinogen concentrate is now approved for acute bleeding episodes in pediatric and adult patients with congenital fibrinogen deficiency.

FibroGen’s oral HIF-PH inhibitor showed improved transfusion independence in high–high-transfusion-burden MDS patients in a post hoc phase 3 MATTERHORN analysis.

Stay updated with the latest healthcare breakthroughs, including several new FDA approvals, in this week's essential news roundup.

A prediction model ties emergency visits and opioid doses with return visits in children with sickle cell disease pain.

The approval makes etuvetidigene autotemcel (Waskyra) the first cell-based gene therapy for the treatment of Wiskott-Aldrich syndrome.

George highlights bezuclastinib’s effect on bone marrow systemic mastocytosis, emphasizing the drug’s potential in reducing symptom burden.

Data from VAYHIT3 presented at ASH 2025 suggest efficacy and safety of ianalumab in patients with primary immune thrombocytopenia.

Morris discusses this latest trial in a long list of phase 3 studies which failed to evaluate arginine therapy’s value in vaso-occlusive pain episodes in SCD.

Al-Samkari discusses the positive results from the VAYHIT2 trial, which investigated ianalumab in combination with eltrombopag in adults with ITP.

Gwarzo discussed the shortcomings faced by many patients with SCD during pain crises, due to less frequent opioid prescription than guidelines recommend.

Announced by Cogent Biosciences, these data indicate bezuclastinib’s capacity for improving patient-reported symptoms of mast cell burden.

The approval of Ayrmid’s omidubicel-onlv makes it the first hematopoietic stem cell transplant therapy to treat patients with severe aplastic anemia.

Strunk highlights reduced length of stay and superior outpatient pain management due to palliative care in hospitalized patients.

Rusfertide shows promising results as a first-in-class therapy for polycythemia vera, demonstrating sustained hematocrit control and safety in recent studies.

GLP-1 RA use was linked to a significant survival benefit and reduced risk of iron deficiency anemia in patients with celiac disease.

Results from the phase 3 trial show consistent reductions in annualized bleeding rate after 12 months of once-weekly treatment.

A study reveals that APOE e4 carriers with atrial fibrillation on Eliquis face increased intracranial hemorrhage risk, highlighting critical genetic implications.

Explore key FDA decisions in Q4 2025, impacting treatments for atopic dermatitis, chronic rhinosinusitis, and rare diseases.

Denecimig may offer a prophylaxis bleed treatment option for a range of patients across dosing frequencies, age groups, and condition severity.

This Sickle Cell Awareness Month, HCPLive draws attention to recent advancements made in SCD treatment and how much still needs to be done to address it.

An audio recap of the top 5 stories in healthcare news from the week of 08/31-9/5.