Michael Recht

1. 1.1. Inheritable bleeding disorders are usually characterized by abnormal bleeding into joints, vital organs, or mucocutaneous tissues [1–4]. With the exception of von Willebrand disease, they a...

1. 1.1. In 2020, the U.S. National Bleeding Disorders Foundation (NBDF) embraced community-based participatory research (CBPR), in which the community and research team play equitable roles in ever...

1. 1.1. The National Research Blueprint (NRB) proposes a change in how inheritable bleeding disorders (BD) research in the U.S. is conducted and who it engages: a Bleeding Disorders Research Collab...

1. 1.1. The U.S. National Bleeding Disorders Foundation (NBDF) 2021 State of the Science Summit examined how research can best advance what matters most to people with inheritable bleeding disorder...

Background: The Natural History Study of the Safety, Effectiveness, and Practice of Treatment for People with Haemophilia (ATHN 7) monitors use of contemporary haemophilia therapies, including emicizumab, a bispecific antibody substituting for activated factor (F)VIII in people with haemophilia A (HA). Aim: To report participant characteristics and real‐world safety and effectiveness of emicizumab in ATHN 7. Methods: ATHN 7 is a prospective observational cohort study conducted across 26 American Thrombosis and Haemostasis Network (ATHN)‐affiliated sites. People with HA receiving emicizumab were eligible for inclusion in this analysis. Clinical information was collected quarterly through participant interview and medical record review. Safety was evaluated by documenting adverse events (AEs); effectiveness was assessed by annualized bleeding rates (ABRs). Results: At data cut‐off (31st December 2023), 257 participants were receiving emicizumab; 63 (24.5%) had FVIII inhibitors at baseline; 84.4% had severe HA. In total, 109 (42.4%) participants were <12 years old. Median (range) emicizumab exposure duration was 116.4 (0.1–206.6) weeks. Overall, 40 AEs were reported in 13 (5.1%) participants; a total of 33 injection‐site reactions were reported in 7 (2.7%) participants. No thromboses or thrombotic microangiopathy were reported. There was one fatality from haemorrhagic shock, deemed unrelated to emicizumab. Median (range) ABR for treated bleeds was 0.25 (0–8.18) for participants with FVIII inhibitors and 0.51 (0–16.25) for participants without FVIII inhibitors. Conclusion: In people with HA treated with emicizumab, no new safety signals were identified. ABRs were similar for participants with or without FVIII inhibitors, across different severities of HA. Trial Registration: ClinicalTrials.gov identifier: NCT03619863

Introduction: Eptacog beta is a novel human recombinant FVIIa approved for use in the United States, European Union, United Kingdom and Mexico for the treatment and control of bleeding in patients with haemophilia A or B with inhibitors (≥12 years). It is also indicated for perioperative care in the same patient population in Europe and the United Kingdom.Aim: To assess the incidence of rebleeding and review treatment outcomes in subjects with haemophilia with inhibitors enrolled in the phase 3 PERSEPT 1 clinical trial.Methods: To treat mild/moderate bleeding episodes (BEs), subjects administered an initial 75 or 225µg/kg dose of eptacog beta, followed (if necessary) by additional 75µg/kg doses at predefined intervals until bleed control. This analysis used subject‐reported rebleeding to determine a rebleeding incidence for the first 24 h. Rebleeding through later timepoints was an exploratory, intention‐to‐treat analysis of bleed treatment data.Results: Four hundred and sixty‐five BEs were analysed. Through 24 h, the proportion of rebleeds was 0% (initial 75µg/kg dose) and 0.5% (initial 225µg/kg dose). Through 48 h, the proportion of rebleeds was 3.2% (75µg/kg initial dose) and 5.6% (225µg/kg initial dose); the difference between initial dose strategies was not statistically significant. The majority of rebleeds were controlled with a single dose of eptacog beta and no subject who treated a rebleed required hospitalization.Conclusion: Subjects with haemophilia with inhibitors who used eptacog beta to treat mild/moderate BEs experienced a low incidence of rebleeding. Rebleeds that did occur were effectively controlled with eptacog beta (median, one dose) without the need for hospitalization.