OC 27.2 - 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

Sunday, July 10, 2022

3:00 PM – 3:15 PM

Location: ExCel Center, ICC Capital Suite Room 2-4

Presentation Format: In-Person

Co-Author(s)

Margareth C. C. Ozelo, MD, PhD

Associate Professor
Hemocentro UNICAMP, Department of Internal Medicine, School of Medical Sciences, University of Campinas, Campinas, São Paulo, Brazil
Campinas, Sao Paulo, Brazil

Presenting Author(s)

CH

Cedric Hermans

Professor
Saint-Luc University Hospital, Université Catholique de Louvain, Louvain, Belgium
Louvain, Vlaams-Brabant, Belgium

Co-Author(s)

Kate Khair, PhD

Director of Research
Haemnet
London, England, United Kingdom
BG

Benoît Guillet

Doctor
CRC-MHC, CHU de Rennes – Hôpital Pontchaillou, Rennes, France
Rennes, Bretagne, France
Randy Guerra, MD

Global Medical Lead
Takeda Development Center Americas, Inc., Cambridge, MA, USA
Cambridge, Massachusetts, United States
JG

Joan Gu

Associate Director of Biostatistics
Takeda Development Center Americas, Inc., Cambridge, MA, USA
Cambridge, Massachusetts, United States
LT

Leilei Tang

Global Medical Lead
Takeda Pharmaceuticals International AG
Zürich, Zurich, Switzerland
Manuel Carcao, MD, FRCPC

Director, Haemophilia Clinic and Haemostasis Program
Hospital for Sick Children, Toronto, Canada
Toronto, Ontario, Canada

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.