Haemophilia Comprehensive Care Centre, Charlotte Maxeke Johannesburg Academic Hospital, Johannesburg, South Africa.
Faculty of Health Sciences, University of the Witwatersrand and NHLS, Johannesburg, South Africa.
Haemophilia. 2022 May;28 Suppl 4(Suppl 4):103-110. doi: 10.1111/hae.14524.
Emicizumab is a bispecific monoclonal antibody developed to address the unmet needs of clotting factor replacement therapy and has become the benchmark for optimal prophylaxis in managing patients with haemophilia A with inhibitors. We describe the emicizumab rollout and pharmacokinetic strategies and their use in paediatric patients.
The evolving real-world experience in using emicizumab has confirmed its safety, efficacy and pharmacokinetic profile in paediatric, adolescent and adult patients receiving emicizumab at various prophylactic dosing regimens. The emicizumab current global rollout includes over 100 countries with 29 low to middle-income countries accessing emicizumab through the World Federation of Haemophilia (WFH) Humanitarian Aid Program. The diversity of emicizumab dosing and pharmacokinetic tools such as the Calibra® and the WAPPS-Hemo platforms make it possible to achieve prophylaxis goals in line with the WFH Haemophilia treatment guidelines recommendations, with minimal drug wastage. The emerging experience from long term clinical trials and long-term real-world follow-up confirm the safety, efficacy, and pharmacokinetic profile of emicizumab in paediatric haemophilia A patients. A few questions, including inhibitor recurrence, concurrent use of emicizumab with various replacement therapies and inhibitor eradication, are being addressed through multiple ongoing clinical studies.
The current global rollout of emicizumab is remarkable, and versatile dosing regimens and evolving pharmacokinetic tools such as the Calibra® and WAPPS-Hemo platforms make it a treatment choice available also for pharmacokinetic guided personalised treatment. Data from paediatric studies are consistent with those seen in adolescent and adult Haemophilia A.
依库珠单抗是一种双特异性单克隆抗体，旨在满足凝血因子替代疗法的未满足需求，已成为管理伴抑制物的血友病 A 患者最佳预防治疗的基准。我们描述了依库珠单抗的推出情况和药代动力学策略及其在儿科患者中的应用。
在各种预防剂量方案下接受依库珠单抗治疗的儿科、青少年和成年患者的真实世界经验证实了依库珠单抗的安全性、疗效和药代动力学特征。依库珠单抗目前在全球 100 多个国家推出，其中 29 个中低收入国家通过世界血友病联盟（WFH）人道主义援助计划获得依库珠单抗。依库珠单抗的多样化剂量和药代动力学工具，如 Calibra®和 WAPPS-Hemo 平台，使得可以根据 WFH 血友病治疗指南建议实现预防目标，最大限度地减少药物浪费。来自长期临床试验和长期真实世界随访的新经验证实了依库珠单抗在儿科血友病 A 患者中的安全性、疗效和药代动力学特征。一些问题，包括抑制剂复发、依库珠单抗与各种替代疗法的同时使用和抑制剂消除，正在通过多项正在进行的临床研究来解决。
依库珠单抗的全球推出令人瞩目，多样化的剂量方案和不断发展的药代动力学工具，如 Calibra®和 WAPPS-Hemo 平台，使其成为可供选择的治疗方法，也可用于基于药代动力学的个体化治疗。儿科研究的数据与血友病 A 青少年和成年患者的数据一致。
