Marrapodi Maria Maddalena, Mascolo Annamaria, Roberti Domenico, Martino Martina Di, Rafaniello Concetta, Riccardi Consiglia, Rossi Francesca
Department of Woman, Child and General and Specialist Surgery, University of Campania "Luigi Vanvitelli", Naples, Italy.
Campania Regional Centre for Pharmacovigilance and Pharmacoepidemiology, University of Campania "Luigi Vanvitelli", Naples, Italy.
Front Pediatr. 2023 Apr 24;11:1149718. doi: 10.3389/fped.2023.1149718. eCollection 2023.
Acquired aplastic anemia (AAA) in pediatric patients is a rare disorder characterized by hypocellular bone marrow and pancytopenia. Eltrombopag, an oral thrombopoietin receptor agonist, provides a hematologic improvement in adults with severe aplastic anemia (SAA) refractory to immunosuppressive therapy (IST). The association of ELT and IST was approved by the US Food and Drug Administration (FDA) for adults and children ≥2 years of age as a first-line treatment for SAA. However, the effects of ELT on pediatric patients with SAA remain controversial and limited.
We conducted a systematic review of the most recent literature from Pubmed, Web of Science, and Embase, published up to 20th December 2022, in order to evaluate the available evidence on the efficacy and safety of ELT added to IST for the treatment of SAA in the pediatric population.
Eltrombopag added to the IST has shown a good safety profile, without manifestations of excessive toxic effects, although not all the results obtained from our studies support the addition of ELT to the IST in the first-line treatment of children with SAA.
https://www.crd.york.ac.uk/prospero/, identifier: CRD42022325859.
儿童获得性再生障碍性贫血（AAA）是一种罕见疾病，其特征为骨髓细胞减少和全血细胞减少。艾曲泊帕是一种口服血小板生成素受体激动剂，可使对免疫抑制治疗（IST）难治的重度再生障碍性贫血（SAA）成人患者的血液学状况得到改善。艾曲泊帕与IST联合用药已获美国食品药品监督管理局（FDA）批准，用于≥2岁的成人和儿童SAA一线治疗。然而，艾曲泊帕对儿童SAA患者的疗效仍存在争议且有限。
我们对截至2022年12月20日发表于PubMed、科学网和Embase的最新文献进行了系统评价，以评估在IST基础上加用艾曲泊帕治疗儿童SAA的有效性和安全性的现有证据。
在IST基础上加用艾曲泊帕显示出良好的安全性，无过度毒性反应表现，尽管我们研究获得的并非所有结果都支持在儿童SAA一线治疗中在IST基础上加用艾曲泊帕。
