Related Experiment Video
Updated: Aug 6, 2026
Intrafemoral Injection of Human Hematopoietic Stem and Progenitor Cells into Immunocompromised Mice
Published on: December 8, 2023
Long-term experience with emicizumab in people with hemophilia A in the Canadian Bleeding Disorders Registry
Alfonso Iorio1, Federico Germini1,2, Quazi Ibrahim1
1Health Research Methods, Evidence, and Impact, McMaster University, Hamilton, ON, Canada.
Emicizumab prophylaxis has been approved in Canada for the prevention or reduction of bleeding episodes in people with congenital hemophilia A (PwHA). Using routinely collected health data from the Canadian Bleeding Disorders Registry (CBDR), this observational cohort study aimed to evaluate the long-term safety and effectiveness of emicizumab in PwHA. Deidentified data from the CBDR were collected for PwHA who had received ≥1 dose of emicizumab from 2018 up to 31 December 2023. Intrapatient comparisons of annualized bleeding rates (ABRs) for all reported bleeds before and after emicizumab were performed using a negative binomial regression model. Overall, 710 PwHA received ≥1 emicizumab dose, and 539 (75.9%) had >1 year of emicizumab exposure. During the observational period, the safety profile was in keeping with previous reports; 34 adverse events were observed, including 2 thrombotic events (1 unlikely related to emicizumab; 1 probably related to previous use of eptacog alfa and/or possibly related to previous emicizumab, and associated with a central venous access device). Over a median (first to third quartiles) follow-up time of 549 (374-690) days, 439 (61.8%) PwHA had no recorded bleeds. Mean ABR decreased from 1.00 (95% confidence interval [CI], 0.87-1.14) before emicizumab to 0.47 (95% CI, 0.40-0.55) after emicizumab (rate ratio, 0.47 [95% CI, 0.41-0.55]; P< .001). A notable decrease in bleeds and sustained bleed control were observed in this population for up to a 5-year follow-up period. Continued follow-up will allow for greater quantification of this impact over time, providing valuable information to health care practitioners and regulatory authorities.
Emicizumab prophylaxis has been approved in Canada for the prevention or reduction of bleeding episodes in people with congenital hemophilia A (PwHA). Using routinely collected health data from the Canadian Bleeding Disorders Registry (CBDR), this observational cohort study aimed to evaluate the long-term safety and effectiveness of emicizumab in PwHA. Deidentified data from the CBDR were collected for PwHA who had received ≥1 dose of emicizumab from 2018 up to 31 December 2023. Intrapatient comparisons of annualized bleeding rates (ABRs) for all reported bleeds before and after emicizumab were performed using a negative binomial regression model. Overall, 710 PwHA received ≥1 emicizumab dose, and 539 (75.9%) had >1 year of emicizumab exposure. During the observational period, the safety profile was in keeping with previous reports; 34 adverse events were observed, including 2 thrombotic events (1 unlikely related to emicizumab; 1 probably related to previous use of eptacog alfa and/or possibly related to previous emicizumab, and associated with a central venous access device). Over a median (first to third quartiles) follow-up time of 549 (374-690) days, 439 (61.8%) PwHA had no recorded bleeds. Mean ABR decreased from 1.00 (95% confidence interval [CI], 0.87-1.14) before emicizumab to 0.47 (95% CI, 0.40-0.55) after emicizumab (rate ratio, 0.47 [95% CI, 0.41-0.55]; P< .001). A notable decrease in bleeds and sustained bleed control were observed in this population for up to a 5-year follow-up period. Continued follow-up will allow for greater quantification of this impact over time, providing valuable information to health care practitioners and regulatory authorities.