Department of Paediatrics, NHS Foundation Trust, Paediatric Haematology, Haematology and Oncology Unit, Birmingham Children's Hospital, Birmingham, UK.
Department of Paediatric Haematology, NHS Foundation Trust, Birmingham Children's Hospital, Birmingham, UK.
Haemophilia. 2021 Nov;27(6):e698-e703. doi: 10.1111/hae.14432. Epub 2021 Oct 10.
Emicizumab is a bispecific monoclonal antibody that bridges activated factor (F) IX and FX, and maintains haemostasis in patients with haemophilia A (PwHA). As a novel agent, many questions remain unanswered about the loss of emicizumab efficacy due to anti-drug antibody (ADA) development, the incidence of inhibitor recurrence in previously tolerized patients, and the risk of de novo inhibitor development.
To present real-world experience regarding tolerability, side effects, and outcomes of adverse events of emicizumab prophylaxis in paediatric PwHA.
Data on tolerability, compliance, adverse events, and laboratory results of paediatric patients receiving emicizumab prophylaxis, treated at the Haemophilia Comprehensive Care Centre, at Birmingham Children's Hospital between March 2018 and June 2021, were collected.
Our results showed that out of 52 patients, four experienced minor adverse events, two developed headaches, one developed abdominal pain and nausea, and one developed injection site reactions. Moreover, four patients experienced major adverse events, including severe headaches, major bleeding events, development of ADAs, and recurrence of inhibitors. Emicizumab prophylaxis was discontinued in three patients (5.7% of the cohort) due to adverse events. In addition, emicizumab was discontinued in one patient because of poor compliance. No adverse events were reported in previously untreated/minimally treated patients, represented by four patients in our cohort.
The real-world experience of emicizumab prophylaxis in our cohort showed that emicizumab was safe and well tolerated in paediatric PwHA with and without inhibitors. Long-term assessment is crucial to monitor major adverse events, recurrence of inhibitors, and development of ADAs.
依库珠单抗是一种双特异性单克隆抗体，可桥接激活的因子（F）IX 和 FX，并维持血友病 A 患者（PwHA）的止血功能。作为一种新型药物，由于抗药物抗体（ADA）的产生导致依库珠单抗疗效丧失、先前耐受的患者中抑制剂复发的发生率以及新产生抑制剂的风险等问题仍存在诸多未解。
介绍依库珠单抗预防治疗儿科血友病 A 患者的耐受性、副作用和不良事件结局的真实世界经验。
收集 2018 年 3 月至 2021 年 6 月期间在伯明翰儿童医院血友病综合护理中心接受依库珠单抗预防治疗的儿科患者的耐受性、依从性、不良事件和实验室结果数据。
我们的结果显示，52 名患者中有 4 名出现轻微不良事件，2 名出现头痛，1 名出现腹痛和恶心，1 名出现注射部位反应。此外，4 名患者发生了重大不良事件，包括严重头痛、大出血事件、ADA 的产生和抑制剂的复发。由于不良事件，有 3 名患者（占队列的 5.7%）停用了依库珠单抗预防治疗。此外，由于依从性差，1 名患者停用了依库珠单抗。在我们的队列中，有 4 名未曾接受过治疗/接受最小治疗的患者无不良事件报告。
我们队列中依库珠单抗预防治疗的真实世界经验表明，依库珠单抗在有或无抑制剂的儿科血友病 A 患者中安全且耐受良好。长期评估对于监测重大不良事件、抑制剂的复发和 ADA 的产生至关重要。
