Abstract Number: PB1163
Meeting: ISTH 2022 Congress
Theme: Hemophilia and Rare Bleeding Disorders » Hemophilia - Clinical
Background: Many patients with haemophilia A (HA) and B (HB) continue to have unmet needs in disease management and treatment burden. Real-world evidence may help to further describe these unmet needs, ultimately improving haemophilia management.
Aims: To describe real-world unmet medical needs of patients with HA/HB with or without inhibitors using historical data.
Methods: Historical data were collected from patients entering the non-interventional study, explorer6 (NCT03741881). Male patients ≥12 years old with severe HA (FVIII activity < 1%), severe/moderate HB (FIX activity ≤2%) or HA/HB with inhibitors of any severity were included. Patients with previous/current concizumab or emicizumab treatment were excluded. Each patient was treated according their country’s standard-of-care (SoC). Historical data including demographics and treatment regimen were collected, analysed and described.
Results: In total, data from 231 patients (Figure 1) from 109 centres were collected. Median age was 28 years (range, 12–78 years). For the prophylaxis (PPX) group, the mean (median) annualised bleeding rates (ABR) for patients without inhibitors were HA 6.6 (2.0), HB 5.4 (2.0); and for patients with inhibitors HA 22.0 (9.0), HB 18.0 (10.0). For the episodic treatment group, the mean (median) ABRs for patients without inhibitors were HA 30.3 (24.0), HB 19.3 (12.0); and for patients with inhibitors HA 25.2 (14.0), HB 17.9 (18.0) (Table 1). At baseline, 65.6% of patients on episodic and 34.1% of patients on PPX had ≥1 target joint.
Conclusion(s): Here we present data from >30 countries, describing the historic haemophilia characteristics of patients with HA/HB, with or without inhibitors. Many patients had a high ABR and target joints despite receiving their country’s SoC treatment (episodic or PPX). This reflects a high unmet medical need, particularly for those receiving episodic treatment or those with inhibitors. Emerging therapy options could help improve health outcomes in these patients.
To cite this abstract in AMA style:
Windyga J, Tran H, Fujii T, Lyu C, Villarreal Martinez L, Sathar J, Stasyshyn O, Zozulya N, Brown Frandsen R, Eskelund C, Apte S, Mahlangu J. Real-World Unmet Needs of Patients with Haemophilia A/B with or without Inhibitors: Historical Haemophilia Characteristics from Patients Entering a Non-Interventional Study [abstract]. https://abstracts.isth.org/abstract/real-world-unmet-needs-of-patients-with-haemophilia-a-b-with-or-without-inhibitors-historical-haemophilia-characteristics-from-patients-entering-a-non-interventional-study/. Accessed May 18, 2024.« Back to ISTH 2022 Congress
ISTH Congress Abstracts - https://abstracts.isth.org/abstract/real-world-unmet-needs-of-patients-with-haemophilia-a-b-with-or-without-inhibitors-historical-haemophilia-characteristics-from-patients-entering-a-non-interventional-study/