Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, National Clinical Research Center for Hematologic Disease, Research Unit of Key Technique for Diagnosis and Treatment of Hematologic Malignancies, Peking-Tsinghua Center for Life Science, Chinese Academic of Medical Sciences, Peking University Institute of Hematology, Peking University People's Hospital, Beijing, China.
Department of Pediatrics, Peking University People's Hospital, Peking University, Beijing, China.
Br J Haematol. 2024 Feb;204(2):585-594. doi: 10.1111/bjh.19086. Epub 2023 Sep 2.
Data from 200 children with high-risk acute myeloid leukaemia who underwent their first haploidentical haematopoietic stem cell transplantation (haplo-HSCT) between 2015 and 2021 at our institution were analysed. The 4-year overall survival (OS), event-free survival (EFS) and cumulative incidence of relapse (CIR) were 71.9%, 62.3% and 32.4% respectively. The 100-day cumulative incidences of grade II-IV and III-IV acute graft-versus-host disease (aGVHD) were 41.1% and 9.5% respectively. The 4-year cumulative incidence of chronic GVHD (cGVHD) was 56.1%, and that of moderate-to-severe cGVHD was 27.3%. Minimal residual disease (MRD)-positive (MRD+) status pre-HSCT was significantly associated with lower survival and a higher risk of relapse. The 4-year OS, EFS and CIR differed significantly between patients with MRD+ pre-HSCT (n = 97; 63.4%, 51.4% and 41.0% respectively) and those with MRD-negative (MRD-) pre-HSCT (n = 103; 80.5%, 73.3% and 23.8% respectively). Multivariate analysis also revealed that acute megakaryoblastic leukaemia without Down syndrome (non-DS-AMKL) was associated with extremely poor outcomes (hazard ratios and 95% CIs for OS, EFS and CIR: 3.110 (1.430-6.763), 3.145 (1.628-6.074) and 3.250 (1.529-6.910) respectively; p-values were 0.004, 0.001 and 0.002 respectively). Thus, haplo-HSCT can be a therapy option for these patients, and MRD status pre-HSCT significantly affects the outcomes. As patients with non-DS-AMKL have extremely poor outcomes, even with haplo-HSCT, a combination of novel therapies is urgently needed.
对 2015 年至 2021 年期间在我院接受首次半相合造血干细胞移植（haplo-HSCT）的 200 例高危急性髓系白血病儿童的数据进行了分析。4 年总生存率（OS）、无事件生存率（EFS）和累积复发率（CIR）分别为 71.9%、62.3%和 32.4%。100 天 II-IV 级和 III-IV 级急性移植物抗宿主病（aGVHD）的累积发生率分别为 41.1%和 9.5%。4 年慢性移植物抗宿主病（cGVHD）累积发生率为 56.1%，中重度 cGVHD 累积发生率为 27.3%。HSCT 前微小残留病（MRD）阳性（MRD+）状态与生存率降低和复发风险增加显著相关。HSCT 前 MRD+患者（n=97）与 MRD-患者（n=103）的 4 年 OS、EFS 和 CIR 差异显著，分别为 63.4%、51.4%和 41.0%，80.5%、73.3%和 23.8%。多变量分析还显示，无唐氏综合征的急性巨核细胞白血病（non-DS-AMKL）与极差的结局相关（OS、EFS 和 CIR 的风险比和 95%CI：3.110（1.430-6.763）、3.145（1.628-6.074）和 3.250（1.529-6.910）；p 值分别为 0.004、0.001 和 0.002）。因此，haplo-HSCT 可为这些患者提供一种治疗选择，HSCT 前的 MRD 状态显著影响结局。由于 non-DS-AMKL 患者的结局极差，即使接受了 haplo-HSCT，也迫切需要联合新型治疗方法。
