Abstract：
Myelodysplastic syndrome (MDS) is a rare clonal hematopoietic disorder in children. The risk stratification system and treatment strategy for adults are unfit for children. The role of hypomethylating agents (HMAs) in higher-risk childhood MDS has not been identified. This study aimed to investigate the outcomes of hematopoietic stem cell transplantation (HSCT) in children with higher-risk MDS at one single center. A retrospective study was conducted in children with higher-risk MDS undergoing HSCT between September 2019 and March 2023 at Blood Diseases Hospital CAMS. The clinical characteristics and transplantation information were reviewed and analyzed. A total of 27 patients were analyzed, including 11 with MDS with excess blasts (MDS-EB), 14 with MDS-EB in transformation (MDS-EBt) or acute myeloid leukemia with myelodysplasia-related changes (AML-MRC), and 2 with therapy-related MDS/AML (t-MDS/AML). Eight patients harbored monosomy 7. Before transplantation, induction therapy was administered to 25 patients, and 19 of them achieved bone marrow blasts <5% before HSCT. The stem cell source was unmanipulated-related bone marrow or peripheral blood stem cells for nineteen patients and unrelated cord blood for eight. All patients received decitabine-containing and Bu/Cy-based myeloablative conditioning; 26 patients achieved initial engraftment. The cumulative incidences of grade II-IV and grade III-IV acute graft-versus-host disease (GvHD) at 100 days were 65.4% and 42.3%, respectively. The incidence of cGvHD was 38.5%. The median follow-up was 26 (range 4-49) months after transplantation. By the end of follow-up, two patients died of complications and two died of disease progression. The probability of 3-year overall survival (OS) was 84.8% (95%CI, 71.1 to 98.5%). In summary, decitabine-containing myeloablative conditioning resulted in excellent outcomes for children with higher-risk MDS undergoing allogeneic HSCT.
摘要：
骨髓增生异常综合征（MDS）是儿童罕见的克隆造血疾病。成人的风险分层系统和治疗策略不适合儿童。低甲基化药物（HMA）在高危儿童MDS中的作用尚未确定。本研究旨在探讨单中心高危MDS患儿造血干细胞移植（HSCT）的疗效。在2019年9月至2023年3月期间，在血液病医院CAMS对接受HSCT的高危MDS儿童进行了回顾性研究。对患者的临床特点和移植资料进行回顾性分析。共分析27例患者,包括11个带有过量母细胞的MDS（MDS-EB），14与MDS-EB转化（MDS-EBt）或急性髓细胞性白血病与骨髓增生异常相关的变化（AML-MRC），2与治疗相关的MDS/AML（t-MDS/AML）。8名患者携带7个二分体。移植前,对25例患者进行了诱导治疗，其中19例在HSCT前达到骨髓母细胞<5%。19例患者的干细胞来源为未操作相关的骨髓或外周血干细胞，8例患者为无关的脐带血。所有患者均接受了含有地西他滨和基于Bu/Cy的清髓性预处理；26例患者实现了初始植入。100天时II-IV级和III-IV级急性移植物抗宿主病（GvHD）的累积发病率分别为65.4％和42.3％，分别。cGvHD的发生率为38.5%。中位随访时间为移植后26个月（范围4-49个月）。在后续行动结束时，2例死于并发症,2例死于疾病进展.3年总生存率（OS）的概率为84.8%（95CI，71.1～98.5%）。总之,对于接受同种异体HSCT的高危MDS患儿,含有地西他滨的清髓性预处理可带来良好的预后.
