Berrueco Rubén, González-Forster Elisa, Deya-Martinez Angela, Solsona María, García-García Ana, Calzada-Hernández Joan, Yiyi Luo, Vlagea Alexandru, Ruiz-Llobet Anna, Alsina Laia
Pediatric Hematology Department, Hospital Sant Joan de Déu, Barcelona, Spain.
Institut de Recerca Sant Joan de Déu de Barcelona (IRSJD), Barcelona, Spain.
Front Pediatr. 2023 Oct 17;11:1174671. doi: 10.3389/fped.2023.1174671. eCollection 2023.
Second-line treatments of autoimmune cytopenias (AC) are not well-defined in children. Mycophenolate mofetil (MMF) is an immunosuppressant agent that has been demonstrated to be safe and effective in this setting. A retrospective observational study was conducted in 18 children with prolonged AC who received MMF, in order to describe clinical and biological markers of response. The overall response rate of MMF at 20-30 mg/kg per day was 73.3%. All patients with Evans syndrome ( = 9) achieved complete response. Among the patients with monolineage AC ( = 9), those with an underlying inborn errors of immunity (IEI), tended to respond better to MMF. No biological markers related to treatment response were found. Rather, lymphocyte subpopulations proved useful for patient selection as a marker suggestive of IEI along with immunoglobulin-level determination.
儿童自身免疫性血细胞减少症（AC）的二线治疗方法尚未明确界定。霉酚酸酯（MMF）是一种免疫抑制剂，已证明在这种情况下是安全有效的。对18例接受MMF治疗的持续性AC患儿进行了一项回顾性观察研究，以描述反应的临床和生物学指标。每天20 - 30mg/kg的MMF总体反应率为73.3%。所有埃文斯综合征患者（n = 9）均实现完全缓解。在单系AC患者（n = 9）中，那些存在潜在先天性免疫缺陷（IEI）的患者对MMF的反应往往更好。未发现与治疗反应相关的生物学指标。相反，淋巴细胞亚群与免疫球蛋白水平测定一起，被证明作为提示IEI的标志物对患者选择有用。
