Bleeding Disorders

A collection of features and news articles published in ASH Clinical News related to bleeding disorders.

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Small Retrospective Study Suggests Thrombopoietin Receptor Agonists Are Safe, Likely Effective for ITP During...

Preliminary findings from an observational multicenter study suggest that temporary off-label use of thrombopoietin receptor agonists (TPO-RAs) for the treatment of severe and/or refractory...
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Extended Half-Life Factor VIII Fusion Protein Effective in Previously Untreated Hemophilia A

The use of extended half-life recombinant factor VIII Fc fusion protein (rFVIIIFc) appeared to be safe and effective in young boys with previously untreated...
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FLT180a: A New Gene Therapy Candidate for Severe Hemophilia B

In a small study of patients with severe hemophilia B, the novel adeno-associated virus (AAV) gene therapy FLT180a was associated with increased factor IX...
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Caplacizumab Produces Faster, Sustained Platelet Count Responses Than Placebo in aTTP

Patients with acquired thrombotic thrombocytopenic purpura (aTTP) who were treated with caplacizumab – anti–von Willebrand factor humanized immunoglobulin – experienced a faster and more...

FDA’s Rejection of Hemophilia Gene Therapy Raises Questions for Gene Therapy’s Future

Following a priority review, the FDA chose not to approve valoctocogene roxaparvovec, a one-time gene therapy for hemophilia A, seeking more evidence to support...
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Systemic Bevacizumab Improves Anemia, Reduces Bleeding in Hereditary Hemorrhagic Telangiectasia

Treatment with systemic bevacizumab reduced bleeding and significantly increased hemoglobin levels in patients with the rare bleeding disorder hereditary hemorrhagic telangiectasia (HHT), according to...

Early FVIII Prophylaxis Improves Long-Term Joint Outcomes in Severe Hemophilia A

Joint bleeding results in acute and chronic pain for patients living with severe hemophilia A, but joint outcomes improve when prophylactic factor VIII (FVIII)...
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CRISPR-Based Therapy Targeting BCL11 Shows Clinical Benefit in SCD and Beta-Thalassemia

Treatment with the CRISPR/Cas9 gene-editing therapy CTX001 was associated with successful neutrophil and platelet engraftment in 2 patients with transfusion-dependent β-thalassemia (TDT) and 1...

FDA Approves Coagulation Factor VIIa (Recombinant)-jncw for Hemophilia

The FDA has approved coagulation factor VIIa (recombinant)-jncw to control bleeding in patients aged 12 years and older with hemophilia A or B with...

High Bleeding Events in SCD Warrant Consideration of In-Hospital Ulcer Prophylaxis

By the time they reach age 40, 21% of patients with sickle cell disease (SCD) will have experienced a bleeding event, with most events...