PB0684 - Clinical Outcomes in Patients with Hemophilia A in Brazil: 3-years of Follow-up of the AHEAD International Study

Monday, July 11, 2022

6:30 PM – 7:30 PM

Location: Exhibition

Presentation Format: In-Person

Co-Author(s)

MC

Mônica H. Cerqueira

Researcher
Instituto de Hematologia do Estado do Rio de Janeiro (Hemorio), Rio de Janeiro, Brazil
Rio de Janeiro, Rio de Janeiro, Brazil
CF

Clarissa Ferreira

Physician
Hemocentro de Porto Alegre (HEMORGS)
Porto Alegre, Rio Grande do Sul, Brazil
CS

Claudia Santos Lorenzato

Haematologist
Centro de Hematologia e Hemoterapia do Paraná (HEMEPAR),
Curitiba, Parana, Brazil
LM

Luany Elvira Mesquita Carvalho

Director of Hematology
Centro de Hematologia e Hemoterapia do Ceará (HEMOCE)
Fortaliza, Ceara, Brazil
LC

Luciana Correa Oliveira

Physician
Hemocentro Ribeirão Preto, Universidade de São Paulo
Ribeirão Preto, Sao Paulo, Brazil
IP

Ieda S. Pinto

Researcher
Fundação Centro de Hematologia e Hemoterapia do Pará (Hemopa), Pará, Brazil
Belém, Para, Brazil

Presenting Author(s)

Alessandra Prezotti, MD, MsC

Physician
Centro de Hemoterapia e Hematologia do Espírito Santo (HEMOES)
Vitoria, Espirito Santo, Brazil

Co-Author(s)

JG

Joan Gu

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

Global Medical Lead
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
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

Background: The ongoing international AHEAD study (NCT02078427) evaluates the long-term effectiveness and safety of patients with hemophilia A (HA) receiving octocog alfa or rurioctocog alfa pegol in real-world clinical practice.

Aims: To report clinical outcomes from 3 years of follow-up from a subgroup of patients in Brazil with HA receiving octocog alfa using data from the AHEAD 6-year interim read-out (data cutoff: July 15, 2019).

Methods: AHEAD is a prospective, non-interventional, multicenter study including patients of any age with HA. The primary endpoint is joint health, assessed by Gilbert score (pain, bleeding, physical exam). Hemophilia Joint Health Score (HJHS), annualized bleeding rates (ABRs), annualized joint bleeding rates (AJBRs), quality of life, and safety were also recorded. Patients’ informed consent and ethics committee approval were obtained.

Results: A total of 203 patients were enrolled; 166 (81.8%) had severe HA (factor VIII < 1%) and 37 (18.2%) had moderate HA (factor VIII 1–5%). 190 patients received prophylaxis, 2 received on-demand treatment, and 11 received immune tolerance induction. Patient demographics stratified by HA severity and treatment regimen are shown in Table 1. Average Gilbert score, ABRs, and AJBRs were higher in patients with severe HA than those with moderate HA (Table 2). Mean ± SD total HJHS score in patients receiving prophylaxis was 16.8 ± 20.8 at year 1 (n=67), 12.0 ± 15.2 at year 2 (n=52), and 25.7 ± 30.9 at year 3 (n=6). 210 adverse events (AEs) were experienced by 81 patients (39.9%) and 18 serious AEs by 16 patients (7.9%). Of these, 6 (3%) AEs were treatment related, none of which were classified as serious.

Conclusion(s): The majority of patients were treated with prophylaxis, regardless of HA severity. Joint health was well maintained, and ABRs and AJBRs remained consistent over the 3-year observation period in patients with both moderate and severe HA treated with prophylaxis.