Emicizumab 105mg/0.7ml solution for injection vials

Haemophilia A in paediatric patients presents a lifelong risk of spontaneous and trauma‐induced haemorrhage, leading to progressive joint damage, disability and impaired quality of life. Emicizumab, a bispecific monoclonal antibody administered subcutaneously, offers sustained haemostatic protection and has shown promising outcomes in children.

Abstract licence: CC BY-NC

Background Emicizumab, a bispecific factor IXa- and factor X-directed antibody indicated for routine prophylaxis of bleeding episodes in people with hemophilia A, can impose a significant financial burden. We conducted a systematic review to evaluate the reporting quality of existing pharmacoeconomic studies on emicizumab, and to synthesize its cost-effectiveness for hemophilia A treatment. Methods Databases including PubMed, Embase, Cochrane Library, National Health Service Economic Evaluation Database, Health Technology Assessment, China National Knowledge Infrastructure, VIP China Science and Technology Journal database, and WanFang were searched for pharmacoeconomic studies on emicizumab. The general information, methods, and results of the retrieved studies were analyzed. The reporting quality of the studies was evaluated with the Consolidated Health Economic Evaluation Reporting Standards (CHEERS) 2022 checklist. Results A total of 163 studies were retrieved, and 17 studies were further analyzed. Emicizumab was compared to bypassing agents (BPAs), recombinant factor VIII (rFVIII), recombinant factor VIII Fc fusion protein (rFVIIIFc), and gene therapy. The reporting quality of the studies is generally good with an average score of 79.64% (22.3/28) based on the CHEERS 2022 checklist. Current studies revealed that emicizumab prophylaxis was more cost-effective compared to BPAs in people with hemophilia A with inhibitors. However, its cost-effectiveness compared to rFVIII was unclear and varied across different countries. In addition, rFVIIIFc and valoctocogene roxaparvovec were more cost-effective than emicizumab for people with HA without inhibitors. Conclusion Emicizumab prophylaxis was more cost-effective compared to BPAs in people with hemophilia A with inhibitors. Cost-effectiveness analyses with more accurate cost estimations of different countries should provide more convincing evidence for clinical decision-making. Systematic review registration Identifier CRD 42023429349, https://www.crd.york.ac.uk/PROSPERO/view/CRD42023429349.

Abstract licence: CC BY

Background Hemophilia A is a genetic disorder characterized by deficiency or dysfunction of the factor VIII clotting protein, leading to serious bleeding disorders. Conventional treatment involves the exogenous administration of factor VIII. However, this therapy faces significant challenges, including the development of inhibitors and the need for frequent intravenous administration. Emicizumab, a recombinant bispecific monoclonal antibody that can be administered subcutaneously, offers a novel therapeutic alternative by mimicking the action of factor VIII. Methods This systematic review evaluates the efficacy, safety, and patient satisfaction with emicizumab in patients with hemophilia A without inhibitors. A comprehensive literature search was conducted using the MEDLINE, SciELO, and LILACS databases. The included studies were original articles on the use of emicizumab in hemophilia A patients without inhibitors and reviews, short communications, expert comments, and case reports were excluded. Data extraction and analysis were performed using predefined criteria. Results A total of 471 articles were identified, with 28 meeting the inclusion criteria. Studies demonstrated robust evidence of the efficacy of emicizumab in reducing bleeding episodes, with significant reductions in the Annualized Bleeding Rate and Annualized Joint Bleeding Rate. Safety profiles were favorable, with mainly minor adverse events reported. High patient satisfaction scores highlighted improvements in quality of life and treatment adherence. Conclusion Emicizumab represents a significant advancement in hemophilia A treatment, offering superior efficacy, safety, and patient satisfaction compared to traditional therapies. Future research should focus on long-term outcomes and specific subpopulations to further validate these findings.

Abstract licence: CC BY