Expert review of hematology · 2023
A real-world evidence analysis of the impact of switching from factor VIII to emicizumab prophylaxis in patients with hemophilia A without inhibitors.
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Escobar Miguel, Bullano Michael, Mokdad Ali G, Caicedo Jorge, Schultz Bob G, Fan Qi, Verma Sumit
Аннотация
BACKGROUND: This study retrospectively compared annualized billed bleed rates (ABRb) in people with hemophilia A (PwHA) without inhibitors who switched from factor VIII (FVIII) prophylaxis to emicizumab.
RESEARCH DESIGN AND METHODS: A real-world comparison study was performed on the effect of switching from FVIII to emicizumab prophylaxis in male, non-inhibitor patients on ABRb using an all-payer claims database (APCD) dataset from 1 January 2014, to 31 March 2021. The identification period was from 1 November 2017, to 30 September 2020.
RESULTS: One hundred and thirty-one patients were included with a total of 82 and 45 bleeds in the pre- and post-switch periods, respectively. The average follow-up period pre-switch was 978.37 days (SD 555.03), whereas the average follow-up period post-switch was 522.26 days (SD 191.36). No significant differences in mean ABRb were observed pre-/post-switch (0.25 and 0.20, respectively; P = 0.4456).
CONCLUSIONS: The results of this study demonstrate no significant reduction in ABRb, suggesting that switching from FVIII to emicizumab may not deliver incremental benefits to PwHA receiving prophylactic care.
RESEARCH DESIGN AND METHODS: A real-world comparison study was performed on the effect of switching from FVIII to emicizumab prophylaxis in male, non-inhibitor patients on ABRb using an all-payer claims database (APCD) dataset from 1 January 2014, to 31 March 2021. The identification period was from 1 November 2017, to 30 September 2020.
RESULTS: One hundred and thirty-one patients were included with a total of 82 and 45 bleeds in the pre- and post-switch periods, respectively. The average follow-up period pre-switch was 978.37 days (SD 555.03), whereas the average follow-up period post-switch was 522.26 days (SD 191.36). No significant differences in mean ABRb were observed pre-/post-switch (0.25 and 0.20, respectively; P = 0.4456).
CONCLUSIONS: The results of this study demonstrate no significant reduction in ABRb, suggesting that switching from FVIII to emicizumab may not deliver incremental benefits to PwHA receiving prophylactic care.