Jing Xiao-Yu, Li Dong-Jun, Su Sheng-Nan, Dai Qing-Kai, Sun Shu-Wen, Huang Liang, Ai Yuan, Gao Ju, Zhu Yi-Ping, Ni Jia-Qi, Lu Xiao-Xi
Department of Pediatrics, West China Second University Hospital, Sichuan University, Chengdu, China.
Key Laboratory of Obstetric and Gynecologic and Pediatric Diseases and Birth Defects, Sichuan University, Ministry of Education, Chengdu, China.
Front Immunol. 2025 Aug 1;16:1625365. doi: 10.3389/fimmu.2025.1625365. eCollection 2025.
Immune-mediated cytopenias (IMCs) following allogeneic hematopoietic stem cell transplantation (HSCT) can lead to substantial morbidity and mortality, presenting a major therapeutic obstacle. Here, we report a case of a pediatric patient with acquired aplastic anemia. Nine months after HSCT, this patient developed severe, refractory hemolytic anemia and immune-mediated thrombocytopenia (IMT). Despite treatment with corticosteroids, intravenous immunoglobulin (IVIG), rituximab, along with avatrombopag, romiplostim, acetylcysteine, and decitabine, the patient's platelet count showed no signs of improvement. Subsequently, daratumumab, a monoclonal antibody targeting CD38, was administered. This treatment induced a rapid and sustained response. Four months after initial daratumumab administration, the percentage of CD38-positive immune cells in the patient's peripheral blood increased, which was concurrent with another decline in platelet levels. After re-initiating daratumumab therapy, the patient's platelet count returned to normal levels. The only significant adverse effect noted was a delayed recovery of humoral immunity. Daratumumab, by targeting antibody-producing plasma cells, shows promise as a therapeutic alternative for refractory IMCs in post-HSCT patients.
异基因造血干细胞移植(HSCT)后的免疫介导血细胞减少症(IMCs)可导致严重的发病率和死亡率,成为主要的治疗障碍。在此,我们报告一例患有获得性再生障碍性贫血的儿科患者。HSCT九个月后,该患者出现严重的难治性溶血性贫血和免疫介导的血小板减少症(IMT)。尽管使用了皮质类固醇、静脉注射免疫球蛋白(IVIG)、利妥昔单抗,以及阿伐曲泊帕、罗米司亭、乙酰半胱氨酸和地西他滨进行治疗,但患者的血小板计数没有改善迹象。随后,给予了靶向CD38的单克隆抗体达雷妥尤单抗。该治疗引发了快速且持续的反应。首次给予达雷妥尤单抗四个月后,患者外周血中CD38阳性免疫细胞的百分比增加,同时血小板水平再次下降。重新开始达雷妥尤单抗治疗后,患者的血小板计数恢复到正常水平。唯一观察到的显著不良反应是体液免疫恢复延迟。达雷妥尤单抗通过靶向产生抗体的浆细胞,有望成为HSCT后难治性IMCs患者的一种治疗选择。