Hematology

Investigators encourage further study of treatment and therapy methods for childhood IBD.

Patient-reported outcome measures display positive trends after hematopoietic stem cell transplantation for patients with sickle cell disease.

Ragni discussed unique advantages fitusiran offers over other approved therapies for hemophilia A or B.

Pulse oximeter bias was less negative in darkly pigmented skin, suggesting a difference in performance across skin pigment categories.

Intravenous iron supplementation was safe but showed mixed improvement in cardiovascular outcomes in the FAIR-HF2 trial at ACC.25.

Data from the phase 3 ATLAS trial supported Qfitlia's BLA, with the latest data presented at December’s ASH meeting.

The AI-powered trial was officially initiated in January 2025.

Sidonio discussed how fitusiran may improve the treatment landscape and reduce treatment burden for people with hemophilia A or B with or without inhibitors.

Results suggest that post-operation intravenous iron therapy could both prevent anemia and offset logistical issues of identifying iron deficiency.

FCM yielded a statistically significant improvement in Hb levels in patients with postpartum anemia over IS.

A new systematic review finds IV iron effective for iron non-responders but calls for further studies on the causes of oral iron failure.

Anemia was linked to lower waist circumference, lower albumin levels, and treatment regimens in females, but not males.

Pongdee discussed research presented at the AAAAI/WAO Joint Congress that focused on improving diagnosis of indolent systemic mastocytosis.

Sabato discussed how research in the field will inform how to best utilize novel therapies for indolent systemic mastocytosis now that a disease-modifying therapy is available.

Our top 5 headlines include an FDA approval, a pair of submissions, data from AAAAI 2025, and more!

This FDA News Month in Review provides a round-up of regulatory decisions from February 2025.

Fidanacogene elaparvovec was approved under the name Beqvez in April 2024.

Iron deficiency was more common in acute versus chronic heart failure, with notable differences in detection by numerous recommended definitions.

Meta-analysis suggests iron therapy lowers the risk of heart failure hospitalization and cardiovascular death in patients with chronic kidney disease.

Iron administration was linked to a decrease in platelet counts, even in the absence of preexisting thrombocytosis.

More than 90% of patients with hemophilia B eliminated factor IX prophylaxis 4 years after treatment with etranacogene dezaparvovec (HEMGENIX) gene therapy.

Interim analysis of the Phase 3 FRONTIER3 trial found Mim8 well-tolerated in children with hemophilia A with and without inhibitors.

This FDA News Month in Review provides a round-up of regulatory decisions and submissions from January 2025.

Peter Kranke, MD, MBA, MME, and colleagues call for clarifications to the August 2024 recommendations with more real-world considerations.