PB0202 - Long-term effectiveness and safety of emicizumab in people with hemophilia A in the Canadian Hemophilia Bleeding Disorders Registry

Sunday, June 23, 2024

13:45 – 14:45 ICT

Room: Exhibition Hall

Presenting Author(s)

MP

Man-Chiu Poon

Professor
University of Calgary
Calgary, Alberta, Canada

Co-Author(s)

AL

Adrienne Lee

Dr
University of British Columbia
Victoria, British Columbia, Canada

Co-Author(s)

FG

Federico Germini

Assistant Professor
McMaster University
Hamilton, Ontario, Canada

Co-Author(s)

AK

Arun Keepanasseril

Assistant Clinical Professor
McMaster University
Hamilton, Ontario, Canada
QI

Quazi Ibrahim

Statistician
McMaster University
Hamilton, Ontario, Canada
LP

Letizia Polito, PhD

Principal Data Scientist
F. Hoffmann-La Roche Ltd
Basel, Basel-Stadt, Switzerland
KM

Katya Moreno

Therapeutic Area Head Hematology
F. Hoffmann-La Roche Ltd
Basel, Basel-Stadt, Switzerland
MS

Michelle Santos

Medical Affairs Partner
Hoffmann-La Roche Ltd
Mississauga, Ontario, Canada
EI

Emma Iserman

Statistical Analyst
McMaster University
Hamilton, Ontario, Canada
Davide Matino

Assistant Professor
Division of Hematology & Thromboembolism, Department of Medicine, McMaster University, Ontario, Canada
Hamilton, Ontario, Canada
AI

Alfonso Iorio

Professor
McMaster University
Hamilton, Ontario, Canada

Background: Health Canada approved emicizumab prophylaxis for the prevention or reduction of bleeding episodes in people with congenital hemophilia A (PwHA) with factor VIII inhibitors in 2018, and without inhibitors in 2019.

Aims: To present the long-term effectiveness outcomes of emicizumab in a non-interventional cohort study, using data from the Canadian Bleeding Disorders Registry (CBDR).

Methods: De-identified data were extracted from the CBDR database for PwHA who had received emicizumab at least once before December 31, 2022. Baseline demographics were collected for the overall cohort. Intra-patient comparisons of annualized bleeding rate (ABR) pre and post emicizumab initiation were calculated using a negative binomial regression model. ABR was compared across post-emicizumab periods. This study (sponsored by F. Hoffmann-La Roche Ltd) follows the principles of the Declaration of Helsinki and was ethically approved.

Results: In the CBDR, 533 PwHA received ≥1 emicizumab dose, including 151 (28%) participants with >1 year emicizumab exposure. Participant characteristics are summarized (Table 1). Mean (95% confidence interval [CI]) ABR decreased from 1.22 (1.06–1.42) pre-emicizumab (2018–prior to first dose) to 0.50 (0.41–0.60) post-emicizumab (Years 1–4; median [first quartile–third quartile] follow-up 249 [136–395] days, rate ratio [95% CI] 0.41 [0.33–0.50], p< 0.001, N=533). Within the first year of treatment, mean (95% CI) ABR decreased to 0.58 (0.48–0.71) and reduced further in the second year (0.22 [0.13–0.36]); ABR remained stable during the third and fourth years (0.20 [0.10–0.37] and 0.21 [0.07–0.61], respectively; Table 2). The safety profile was consistent with previous reports; 22 adverse events were reported, including one thromboembolic event (previously reported).

Conclusion(s): Following emicizumab, a notable decrease in bleeds and sustained bleed control were observed (Years 1–4). The CBDR allows for longitudinal follow-up of PwHA, informing healthcare practitioners and regulatory authorities of the safety and effectiveness of treatments in routine clinical practice.