Thota Usha Rani, Martha Sreelatha, Ravula Chaitanya Jyothi, Cherukuri Nirmala
Department of Pediatrics, Institute of Child Health - Niloufer Hospital for Women and Children, Osmania Medical College, Hyderabad, Telangana, India.
Indian J Pediatr. 2024 Sep 25. doi: 10.1007/s12098-024-05263-2.
To assess the effectiveness and tolerability of emicizumab prophylaxis in hemophilia A (HA). Emicizumab is a novel therapeutic drug which is the first and only non-factor replacement agent licensed for use in people with HA.
Pediatric patients aged 1 mo to 12 y with severe HA and frequent / life threatening bleeding events, with or without coagulation protein factor VIII inhibitors were enrolled (n = 18) in this observational pre-post study. Patients were switched from therapy involving on-demand or prophylactic factor VIII/bypassing agents/immune tolerance induction to emicizumab prophylaxis and followed up for 52 wk.
One year before initiating emicizumab, a total of 229 bleeding events occurred among the enrolled children. After emicizumab prophylaxis, 5 patients had one episode of bleeding event each with a mean bleeding duration of 1.2 d in one year. The mean annualized bleeding rate significantly reduced from 12.7 ± 8.61 events pre-emicizumab prophylaxis to 0.28 ± 0.46 events post-emicizumab prophylaxis (p < 0.001). Out of the total cohort (n = 18), 72.2% of patients (n = 13) had no bleeding events (95% Confidence interval: 46.4-89.3) while on emicizumab. The mean annualized joint bleeding rate reduced from 9.72 ± 7.44 to 0.17 ± 0.38 (p < 0.001). The target joint resolution was 100% and no adverse events were noted.
Emicizumab was found to be effective and safe as a prophylactic agent for the treatment of severe HA with and without factor VIII inhibitors. Emicizumab prophylaxis can optimize treatment outcomes and promote a better quality of life in children with severe HA.
评估艾美赛珠单抗预防治疗甲型血友病（HA）的有效性和耐受性。艾美赛珠单抗是一种新型治疗药物，是首个也是唯一获许可用于HA患者的非凝血因子替代药物。
本观察性前后对照研究纳入了18例年龄在1个月至12岁之间、患有严重HA且有频繁/危及生命的出血事件、有或无凝血蛋白因子VIII抑制剂的儿科患者。患者从按需或预防性使用因子VIII/旁路制剂/免疫耐受诱导治疗转换为艾美赛珠单抗预防治疗，并随访52周。
在开始使用艾美赛珠单抗前一年，纳入的儿童共发生229次出血事件。在使用艾美赛珠单抗预防治疗后，5例患者每人发生1次出血事件，一年中的平均出血持续时间为1.2天。年化出血率均值从艾美赛珠单抗预防治疗前的12.7±8.61次显著降至预防治疗后的0.28±0.46次（p<0.001）。在整个队列（n = 18）中，72.2%的患者（n = 13）在使用艾美赛珠单抗时无出血事件（95%置信区间：46.4 - 89.3）。年化关节出血率均值从9.72±7.44降至0.17±0.38（p<0.001）。目标关节症状缓解率为100%，未观察到不良事件。
发现艾美赛珠单抗作为预防治疗严重HA（无论有无因子VIII抑制剂）的药物是有效且安全的。艾美赛珠单抗预防治疗可优化治疗效果，提高严重HA患儿的生活质量。
