Department of Pediatrics, The Second Affiliated Hospital of Anhui Medical University, Hefei, China.
Department of Hematology/Oncology, Children's Hospital Affiliated to Shanghai Jiaotong University, Shanghai, China.
Ann Hematol. 2023 Dec;102(12):3431-3444. doi: 10.1007/s00277-023-05389-x. Epub 2023 Aug 8.
To investigate the possible risk factors for death at post-treatment in children with acute lymphoblastic leukemia (ALL). A multivariate competing risk analysis was performed to retrospectively analyze the data of children with ALL who died after treatment with CCCG-ALL-2015 in China and to determine the possible risk factors for death at post-treatment in children with ALL. Age at the first diagnosis of ≥10 years; final risk level of high-risk; D19 minimal residual disease (MRD) (≥0.01%) and D46 MRD (≥0.01%); genetic abnormalities, such as KMT2A-rearrangement, c-Myc rearrangement, and PDGFRB rearrangement; and the presence of CNS3 (all P values, <0.05) were identified as independent risk factors, whereas the risk level at the first diagnosis of low-risk (LR) and ETV6::RUNX1 positivity was considered as independent protective factors of death in children with ALL. Among the 471 cases of death, 45 cases were treated with CCCG-ALL-2015 only, and 163 (34.61%) were treatment-related, with 62.42% due to severe infections. 55.83% of treatment-related mortality (TRM) occurred in the early phase of treatment (induction phase). TRM has a significant impact on the overall survival of pediatric patients with ALL. Moreover, the CCCG-ALL-2015 regimen has a better safety profile for treating children with ALL, with rates close to those in developed countries (registration number: ChiCTR-IPR-14005706; date of registration: June 4, 2014).
为了探讨儿童急性淋巴细胞白血病(ALL)治疗后死亡的可能危险因素。本研究采用多变量竞争风险分析方法,回顾性分析了在中国接受 CCCG-ALL-2015 方案治疗后死亡的 ALL 患儿的资料,确定 ALL 患儿治疗后死亡的可能危险因素。首次诊断时年龄≥10 岁;最终危险度为高危;D19 微小残留病(MRD)(≥0.01%)和 D46 MRD(≥0.01%);存在遗传学异常,如 KMT2A 重排、c-Myc 重排、PDGFRB 重排;和 CNS3 受累(所有 P 值均<0.05)被确定为独立危险因素,而首次诊断时为低危(LR)和 ETV6::RUNX1 阳性则被认为是 ALL 患儿死亡的独立保护因素。在 471 例死亡病例中,仅接受 CCCG-ALL-2015 方案治疗的有 45 例,其中 163 例(34.61%)与治疗相关,62.42%与严重感染有关。治疗相关死亡率(TRM)的 55.83%发生在治疗的早期(诱导期)。TRM 对 ALL 患儿的总生存有显著影响。此外,CCCG-ALL-2015 方案治疗儿童 ALL 的安全性良好,与发达国家的报告接近(注册号:ChiCTR-IPR-14005706;登记日期:2014 年 6 月 4 日)。