Hematology

This interview at ATS 2025 features a discussion of a ‘Pediatric Year in Review’ presentation on new therapies for sickle cell disease.

Interim results from the ongoing trial indicated achievement of all efficacy endpoints, with no safety concerns reported.

A study reveals a significant link between iron deficiency anemia and increased ischemic stroke risk in young adults, highlighting urgent screening needs.

A mixture of thalidomide and hydroxyurea outperformed hydroxyurea alone in all primary outcomes.

Young shared with his real-world experiences with etranacogene dezaparvovec gene therapy.

Anemia can moderate the association between both conditions, increasing the risk of atherosclerotic cardiovascular disease.

Investigators believe the connection may account for a substantial number of anemia cases in children.

Anemia correction reduced perioperative rates but showed no significant impact on postoperative outcomes, emphasizing the need for prehabilitation strategies.

Despite no direct correlation being identified, this research highlights a potential association between the two conditions.

Investigators examined the safety of the recalled sickle cell disease medication through retrospective analysis of an FDA database.

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.

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.

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.