Abstract Number: OC 27.2
Meeting: ISTH 2022 Congress
Background: AHEAD (NCT02078427, started in 2011) is an ongoing, international study evaluating long-term outcomes in patients with severe/moderate hemophilia treated with octocog alfa or rurioctocog alfa pegol in routine clinical practice.
Aims: Characterize clinical outcomes in patients with moderate HA (factor VIII 1–≤5%) receiving standard or pharmacokinetic (PK)-guided prophylaxis or on-demand treatment with octocog alfa.
Methods: This is an 8-year interim read-out (data cutoff: July 01, 2021) of the prospective, noninterventional, multicenter AHEAD study (104 study centers). The primary endpoint is overall Gilbert score (primary endpoint; assessed using pain, bleeding, and physical examination). Secondary endpoints include annualized bleeding rates (ABR) and joint bleeding rates (AJBRs), EuroQol-5 dimensions (EQ-5D), and safety. Ethics committee approval and informed consent were obtained.
Results: 868 patients were enrolled; 287 had moderate HA; 116 (40%) received standard, 33 (11%) PK-guided, and 50 unspecified prophylaxis. 86 patients (30%) were treated on-demand and 2 received immune tolerance induction. Average Gilbert scores (all joints) were low across treatment groups (Mean [SD], years 1–7: standard, 0.0–3.1 [0.0–4.2; n=1–33], PK-guided: 0.0–3.0 [0.0–4.2; n=1–5], on-demand: 1.1–2.5 [1.5–2.7; n=0–15]). ABRs and AJBRs were consistently lower, and zero bleed rates were consistently higher, in patients receiving prophylaxis versus on-demand therapy (Tables 1 and 2). EQ-5D visual analog scale scores were similar between patients receiving standard (Mean, years 1–8: 60.0–99.0 [n=1–53]) and PK-guided (57.5–89.0 [n=1–10]) prophylaxis. Adverse events (AEs) were experienced by 454 (52.3%) of 868 patients, with serious AEs reported in 159 (18.3%) patients; 16 AEs (1.8%) were related to octocog alfa, of which 11 were serious AEs.
Conclusion(s): These real-world findings from the 8-year AHEAD interim data read-out suggest that patients with moderate HA may also benefit from a prophylaxis regimen with octocog alfa.
To cite this abstract in AMA style:Ozelo M, Hermans C, Khair K, Guillet B, Guerra R, Gu J, Tang L, Carcao M. Clinical Outcomes Following Prophylaxis With Octocog Alfa in Patients With Moderate Hemophilia A: 8-Year Interim Read-out of the Real-world AHEAD International Study [abstract]. https://abstracts.isth.org/abstract/clinical-outcomes-following-prophylaxis-with-octocog-alfa-in-patients-with-moderate-hemophilia-a-8-year-interim-read-out-of-the-real-world-ahead-international-study/. Accessed September 24, 2023.
« Back to ISTH 2022 Congress
ISTH Congress Abstracts - https://abstracts.isth.org/abstract/clinical-outcomes-following-prophylaxis-with-octocog-alfa-in-patients-with-moderate-hemophilia-a-8-year-interim-read-out-of-the-real-world-ahead-international-study/