Al-Mashdali Abdulrahman F, Salih Samah M, Vatsyayan Priyadarshini Asmita, Mudawi Deena, Elkourashy Sarah A, Cherif Honar, Mohamed Shehab F
Department of Hematology and Bone Marrow Transplant, National Center for Cancer Care and Research, Hamad Medical Corporation, Doha, Qatar.
Independent researcher, Doha, Qatar.
Blood Rev. 2025 Jul 29:101326. doi: 10.1016/j.blre.2025.101326.
While caplacizumab, a von Willebrand factor inhibitor, has dramatically improved iTTP management in adults, its role in pediatric iTTP remains understudied. This scoping review synthesizes current evidence on the safety and efficacy of caplacizumab in children with iTTP. We conducted a systematic literature search (PubMed, Scopus) up to March 2025, identifying 15 studies (36 patients) meeting inclusion criteria. The cohort (median age 14 years, range 1-17) exhibited female predominance (72 %) and diverse ethnic backgrounds. Neurological symptoms (53 %) and bleeding manifestations (petechiae, purpura, or ecchymoses in 74 %) were common presentations. Caplacizumab was administered at 5 mg (17 %), 10 mg (75 %), or 11 mg (8 %), alongside plasma exchange (22 % received >10 sessions) and immunosuppression (97 % corticosteroids, 92 % rituximab). Key outcomes included rapid platelet recovery (median 3.5 days post-caplacizumab initiation) and relatively short hospitalization (median 14 days). Adverse events were uncommon (11 % minor bleeding), with no major or life-threatening hemorrhagic complications reported. Mortality was 0 %, with all patients surviving to follow-up. During a median follow-up period of 6-12 months, 94 % of patients remained relapse-free. Notably, 22 % of patients were under 12 years of age, underscoring the off-label use of caplacizumab in younger children. These findings suggest caplacizumab is effective and safe in pediatric iTTP, mirroring outcomes observed in adults while demonstrating a favorable bleeding profile. However, variability in dosing and limited data on long-term effects highlight the need for pediatric-specific guidelines and prospective studies. This review supports caplacizumab as a promising adjunct in childhood iTTP but calls for further research to optimize its use in this vulnerable population.
虽然血管性血友病因子抑制剂卡泊珠单抗显著改善了成人免疫性血小板减少性紫癜（iTTP）的治疗，但它在儿童iTTP中的作用仍未得到充分研究。本综述综合了目前关于卡泊珠单抗治疗儿童iTTP安全性和有效性的证据。我们在截至2025年3月进行了系统的文献检索（PubMed、Scopus），确定了15项符合纳入标准的研究（36例患者）。该队列（中位年龄14岁，范围1 - 17岁）以女性为主（72%），种族背景多样。神经症状（53%）和出血表现（瘀点、紫癜或瘀斑占74%）是常见症状。卡泊珠单抗的给药剂量为5毫克（17%）、10毫克（75%）或11毫克（8%），同时进行血浆置换（22%接受超过10次治疗）和免疫抑制（97%使用皮质类固醇，92%使用利妥昔单抗）。关键结果包括血小板快速恢复（卡泊珠单抗开始使用后中位3.5天）和住院时间相对较短（中位14天）。不良事件不常见（11%为轻微出血），未报告重大或危及生命的出血并发症。死亡率为0%，所有患者均存活至随访。在中位6 - 12个月的随访期内，94%的患者未复发。值得注意的是，22%的患者年龄在12岁以下，这突出了卡泊珠单抗在年幼儿童中的超说明书使用情况。这些发现表明卡泊珠单抗在儿童iTTP中有效且安全，与成人观察到的结果相似，同时显示出良好的出血情况。然而，给药剂量的变异性和长期影响的有限数据凸显了制定儿童专用指南和前瞻性研究的必要性。本综述支持卡泊珠单抗作为儿童iTTP中有前景的辅助治疗药物，但呼吁进一步研究以优化其在这一脆弱人群中的使用。
