Acquired hemophilia A: emerging treatment options

Maissaa Janbain,1 Cindy A Leissinger,1 Rebecca Kruse-Jarres2 1Louisiana Center for Bleeding and Clotting Disorders, Tulane University Medical Center, New Orleans, LA, USA; 2Washington Center for Bleeding Disorders, Blood Works NW, Seattle, WA, USA Abstract: Acquired hemophilia A is a rare autoimmune disorder caused by an autoantibody (inhibitor) to factor VIII (FVIII) that interferes with its coagulant function and predisposes to severe, potentially life-threatening hemorrhage. Disease management focuses on controlling bleeding, primarily with the use of bypassing therapy and recombinant porcine FVIII, and permanently eradicating the autoantibody using various immunosuppressants. Treatment challenges include delayed diagnosis, difficulty achieving hemostasis and durable remissions, and complications associated with the use of hemostatic and immunosuppressive therapy in a primarily older patient population. Keywords: autoantibodies, factor VIII, hemostasis, inhibitors, inhibitor eradicatio

VWF-FVIII concentrates in the treatment of inherited von Willebrand disease: A single-centre retrospective study.

Dear Editor, Von Willebrand disease (VWD) is the most common inherited bleeding disorder affecting both genders. Its prevalence ranges between 0.6% and 1.3% of the general population, although the number of symptomatic VWD patients appears much lower, comprised between 0.005% and 0.01%.1 VWD is a heterogeneous bleeding disorder caused by quantitative or qualitative defects of von Willebrand factor (VWF), with an impact on both primary and secondary haemostasis and variable clinical presentation. A partial quantitative defect of VWF is present in Type 1 and absolute in Type 3 VWD. Concerning Type 2 VWD, this condition is characterized by various qualitative defects of VWF, further subdivided into 2A, 2B, 2M and 2N Subtypes.2 Accordingly, the clinical presentation of VWD can range from asymptomatic individuals to patients with severe spontaneous haemophilia‐like joint bleeding, while the most common symptoms comprise mucocutaneous bleeding and bleeding from minor wounds, menorrhagia and haemorrhages following surgery or dental extractions. [...