Hematology

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.

Axatilimab in 9 mg and 22 mg vial sizes are expected to launch in early February for the treatment of chronic graft-versus-host disease.

2 phase 3 trials show iptacopan improves fatigue, quality of life, and hematologic markers in C5i-experienced or -naive patients with paroxysmal nocturnal hemoglobinuria.

An infusion of ferric carboxymaltose in the third trimester significantly reduced anemia in pregnant women, surpassing the efficacy of iron tablets.

A new study highlights notable obesity-associated disruptions of iron metabolism, with higher hemoglobin and serum ferritin levels in pre-obese and obese cohorts.

Nearly half of patients with acute coronary syndrome in a single-center analysis exhibited iron deficiency, with anemia linked to a greater impact on mortality.

Piatek discussed findings from a healthcare resource utilization study and a sentiment analysis study presented at ASH 2024.

Pipe discussed a symposium at ASH 2024 about practical implementation of gene therapy in the field.

Schultz discussed the SCtalk trial and its findings at ASH 2024.

Zaheer discussed how this research may be used as a model to help study other therapy transitions as well.

Bendapudi gave an overview of 2 studies presented at ASH 2024 from his Harvard lab.

Rani discussed an in-depth study examining quality of sleep and quality of life in children and adolescents with SCD.

A study shows that a lower non-HDL/HDL cholesterol ratio is linked to greater anemia prevalence. A threshold of 4.28 shows a 15% anemia risk drop per unit increase below this value.

Concizumab marks the first subcutaneous injection treatment for hemophilia A or B with inhibitors, demonstrating an 86% reduction in bleeds in pivotal trial data.

Iron deficiency is currently screened at a ferritin threshold of 15 ug/mL, well below an accepted 25 ug/mL threshold.

The ATEV therapy, named Symvess, provides revascularization when autologous vein graft or implantation of a synthetic graft is not feasible.

Real-world clinical data from the Adelphi PNH II Disease Specific Programme were reported at ASH 2024.

Danicopan add-on therapy demonstrated more favorable safety in the ALPHA trial than pegcetacoplan did in the PEGASUS trial in a new analysis.

Appiah-Kubi also noted that despite elevated HbF levels, very young children with SCA already had anemia and reticulocytosis.

Pipe discussed findings from the open-label extension of the ATLAS studies at ASH 2024.

Chaturvedi discussed findings from the NeST study at ASH 2024, including an increased risk of stroke in those with silent cerebral infarction progression.