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Bleeding Disorders

ASH 2025 Highlight: Beyond Immunosuppression: Two New Ways to Treat ITP

David Kuter gave this presentation at ASH 2025: Immune thrombocytopenic purpura is now recognized as a disorder characterized by both reduced production and increased destruction—this understanding drives treatment selection toward achieving complete remission, rather than just disease suppression. Increase Platelet Production The latest TPO receptor agonist, hetrombopag, now in phase 3 trials, offers a direct […]
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November 2025 QQ Summary: Historic Coag Factor Nomenclature

Our November Quick Question summary about Stuart-Prower Factor, linked to a 2023 article by Dave McGlasson about coagulation factor nomenclature. The summary and McGlasson’s article attracted a comment from Dr. Emmanuel Favaloro, which described the nomenclature of traditional and current VWF activity assays.  Dr. Favaloro cited three open-access articles on this topic (click to obtain the […]
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An Illustrated Review of Hemophilia B.

Click here for the illustrated open-access review, Hermans C, Astermark J, Trakymienė SŠ, Jiménez-Yuste, V. Haemophilia B: an illustrative review of current challenges and opportunities.  Res Prac Thromb Haemostasis, 2025;9, 103229. Abstract Background Hemophilia B is a genetic bleeding disorder caused by a deficiency of clotting factor IX, which presents unique challenges in clinical management. […]
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VWF Factor Assays in Caplacizumab Therapy

Check with your medical library or join ISTH to obtain Colpani P, Baronciani L, Mancini I, et al. Evaluation of different platelet-dependent von Willebrand factor activity assays to assess the in vivo inhibitory effect of caplacizumab on the von Willebrand factor-platelet interaction. J Thromb Haemost. 2025:S1538-7836(25)00545-8. doi: 10.1016/j.jtha.2025.08.020. PMID: 40915571. Abstract Background: Caplacizumab, a humanized […]
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A Case Study: Congenital Hypofibrinogenemia

Check your medical library for Wu Y, Qin K, Xiang L, et al. Congenital hypofibrinogenemia with bleeding risk: mutations in the FGA, FGB, and FGG genes. Lab Med. 2025:lmaf042. doi: 10.1093/labmed/lmaf042. PMID: 41233956. Abstract Introduction: Congenital hypofibrinogenemia is a genetic disorder caused by defects in the fibrinogen gene. We identified a case of congenital hypofibrinogenemia […]
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Gene Transfer Therapy Updates

Yesterday, 10-28-25, we reported BioMarin’s press release announcing divestiture of their hemophilia A gene transfer therapy, valoctocogene roxaparvovec (Roctavian®). Previously, on February 25, 2025, Pfizer announced their intention to discontinue hemophilia B gene transfer therapy, fidanacogene elaparvovec-dzkt (Becvez®). Press releases from both indicate underperformance in sales. The remaining hemophilia B therapy, etranacogene dezaparvovec-drlb (HEMGENIX®), CSL […]
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