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KMID : 1022920230110020071
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Journal of Korean Academy of Social & Managed Care Pharmacy
2023 Volume.11 No. 2 p.71 ~ p.81
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Generation of Real-World Evidence on Effectiveness and Safety of Emicizumab: A Systematic Literature Review
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Jang Ye-Won
Lee Han-Kil
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Abstract
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BACKGROUNDS: Emicizumab, a bispecific monoclonal antibody approved by the Food and Drug Administration in 2017 for the treatment of hemophilia, has seen growing usage in South Korea. However, a systematic review of its effectiveness and safety in real-world settings is lacking.
METHODS : We performed a systematic review following the Preferred Reporting Items for Systematic reviews and Meta-Analyses guideline, searching the PubMed and Cochrane Library databases. Two researchers independently searched and reviewed the literature in two stages, applying predefined inclusion/exclusion criteria and extracting key information from the selected studies. We assessed the quality of studies with the Risk of Bias Assessment tool for Non-randomized Studies (RoBANS). We evaluated emicizumab’s effectiveness and safety using the annual bleeding rate (ABR) and serious adverse events (SAEs) like thrombotic events.
RESULTS: Out of 491 relevant studies, 28 studies (1,359 patients) conducted in 14 countries from 2015 to 2022 were included. Among the 17 studies reporting pre- and post-emicizumab ABR changes, 16 demonstrated a decline in ABR, with five indicating a reduction of over 90%. The post-emicizumab ABR varied from 0.09 to 1.4, with 87.1% of cases showing fewer than two bleeds. Among 17 studies (782 patients) examining SAEs, thrombotic events occurred in two studies, affecting four patients (0.51%). The quality assessment by the RoBANS identified a high risk in outcome blinding but low in other domains.
CONCLUSION: The real-world effectiveness and safety of emicizumab are consistent with its clinical trial outcomes, suggesting the need for larger and long-term observational studies in the future.
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KEYWORD
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annual bleeding rate, emicizumab, hemophilia, systematic review
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