Activated prothrombin complex concentrate to treat bleeding events in acquired hemophilia A: BAHAS study

[Objective] Activated prothrombin complex concentrate (aPCC) is a bypassing agent indicated to treat bleeds in patients with acquired hemophilia A (AHA). Nevertheless, its efficacy and safety in the real-world setting have not often been addressed.[Methods] We report the experience of Spanish reference centers for coagulation disorders and from acquired hemophilia Spanish Registry (AHASR) from August 2012 to February 2021. Follow-up period of 30 days after aPCC withdrawal.[Results] Thirty patients with a median age of 70 years old, suffering from 51 bleeds treated with aPCC were finally evaluated. As first-line treatment, aPCC stopped bleeding in 13 of 14 (92.9%) cases. aPCC as the second line after recombinant factor VIIa failure, stopped bleeding in all cases. In 17 patients, aPCC was used far from initial bleed control as prophylaxis of rebleeding with 94% effectiveness. No thromboembolic episodes were communicated. One patient developed hypofibrinogenemia, which did not prevent aPCC from halting bleeding. No other serious adverse events possibly or probably associated with aPCC were reported.[Conclusions] This data support aPCC as hemostatic treatment in AHA with high effectiveness and excellent safety profile in acute bleeds and as extended use to prevent rebleedings, even in aging people with high cardiovascular risk.Shire IIR-ES-002899.Peer reviewe

Expert opinion paper on the treatment of hemophilia A with emicizumab

ABSTRACTIntroduction Prophylaxis with emicizumab, a bispecific monoclonal antibody that mimics FVIII function, has shown encouraging results in clinical trials in terms of efficacy and safety. However, current experience is limited, and many areas of concern to clinicians have yet to be reviewed.Areas covered This paper reviews the experience of hemophilia A patients treated with emicizumab based on the results of clinical trials and real-life studies. The authors place special emphasis on issues such as the management of these patients in situations of hemorrhage and/or surgical interventions, joint health or laboratory monitoring.Expert opinion Treatment with emicizumab has been shown to improve joint health and reduce bleeding, of particular interest to patients with inhibitors and high bleeding rates. However, there are still concerns about its administration in neonates and previously untreated patients due to limited reported experience. Laboratory monitoring is not strictly necessary due to the stable pharmacokinetics emicizumab has been shown to exhibit, however, tests that globally assess hemostasis may be useful especially in cases of bleeding or surgery. The authors are also of the opinion that prophylaxis before minor surgery is not necessary and that major surgeries can be safely performed with additional prophylactic coagulation factor