Medical Center of Hematology, The Second Affiliated Hospital, Army Medical University, Chongqing, China.
Front Immunol. 2022 May 27;13:910893. doi: 10.3389/fimmu.2022.910893. eCollection 2022.
Platelet graft failure (PGF) is a frequent and serious complication after Allogeneic hematopoietic stem cell transplantation (allo-HSCT) and lacks effective treatment strategies, which could affect the prognosis of patients and even cause death. The exact underlying mechanism of PGF remains unclear, and lacks standard treatment. Here, we conduct a retrospective study to evaluate the efficacy and safety of avatrombopag combined with mesenchymal stem cells (MSCs) in 16 patients with thrombocytopenia after allo-HSCT. Patients were administered the following treatment regimen: 20 mg/d avatrombopag; if the PLT count was less than 50×109/L for at least 2 weeks, the dose was increased to 40 mg/d; if the PLT count was 200-400×109/L, the dose was reduced; and if the PLT count was greater than 400×10^9/L, avatrombopag was terminated. Umbilical cord MSCs (1×106 cells/kg) infusion was performed every week for 4-6 weeks. Among the 16 patients, 13 patients (81.3%) achieved a complete response (CR), 2 patients (12.5%) got a partial response (PR), and 1 patient (6.3%) had no response (NR). The median time to obtain CR was 32 (7-426) days after treatment with avatrombopag combined with umbilical cord MSCs. The time to reach 20×109/L≤ PLT <50×109/L in the 2 patients with PR was 52 and 230 days after treatment, respectively. One patient had a severe pulmonary infection and died of cytomegalovirus pneumonia. Overall, our results indicated that combination of avatrombopag with MSCs can promote platelet recovery after transplantation, thereby improving the survival rate of patients and improving the quality of life of patients after transplantation, and providing a new method and strategy for the treatment of thrombocytopenia after allo-HSCT.
血小板植入失败（PGF）是异基因造血干细胞移植（allo-HSCT）后一种常见且严重的并发症，目前缺乏有效的治疗策略，这可能会影响患者的预后，甚至导致死亡。PGF的确切潜在机制尚不清楚，也缺乏标准的治疗方法。在这里，我们进行了一项回顾性研究，评估 avatrombopag 联合间充质干细胞（MSCs）在 16 例 allo-HSCT 后血小板减少症患者中的疗效和安全性。患者接受以下治疗方案：每天 20 mg avatrombopag；如果血小板计数（PLT）至少连续 2 周低于 50×109/L，则将剂量增加至 40 mg/d；如果 PLT 计数为 200-400×109/L，则减少剂量；如果 PLT 计数大于 400×10^9/L，则停止使用 avatrombopag。每周给予脐带 MSCs（1×106 个细胞/kg）输注 4-6 周。在 16 例患者中，13 例（81.3%）达到完全缓解（CR），2 例（12.5%）达到部分缓解（PR），1 例（6.3%）无反应（NR）。接受 avatrombopag 联合脐带 MSC 治疗后，获得 CR 的中位时间为 32（7-426）天。2 例 PR 患者达到 20×109/L≤PLT<50×109/L的时间分别为治疗后 52 天和 230 天。1 例患者发生严重肺部感染，死于巨细胞病毒肺炎。总体而言，我们的结果表明，avatrombopag 联合 MSCs 可促进移植后血小板恢复，从而提高患者的生存率，改善移植后患者的生活质量，为 allo-HSCT 后血小板减少症的治疗提供了新的方法和策略。
