Suh Jin Kyung, Lee Seong Wook, Koh Kyung-Nam, Im Ho Joon, Choi Eun Seok, Jang Seongsoo, Park Chan-Jeoung, Seo Jong Jin
Department of Pediatrics, Kyungpook National University Hospital, Daegu, Korea.
Department of Pediatrics, Korea University Ansan Hospital, Seoul, Korea.
Pediatr Transplant. 2017 Nov;21(7). doi: 10.1111/petr.13004. Epub 2017 Aug 1.
Intensified chemotherapy, HSCT, and supportive care improve the survival of pediatric patients with AML. However, no consensus has been reached regarding the role of HSCT in patients without favorable cytogenetics. We evaluated OS and EFS according to prognostic factors that affect clinical outcomes, including cytogenetics risk group, conditioning regimen, donor type, disease status at the time of HSCT, and number of chemotherapy cycles prior to HSCT in 65 pediatric patients with AML without favorable cytogenetics who underwent HSCT. Fifteen of the 65 patients died: three of TRM and 12 of disease-related mortality. The 5-year OS and EFS were 78.0% and 72.0%, respectively, and the 5-year cumulative relapse and TRM rates were 26.9% and 5.1%, respectively. Survival rates were not influenced by cytogenetic group (intermediated vs. poor), donor type (related vs. unrelated), transplant type (myeloablative vs. reduced-intensity conditioning), or number of pretransplant chemotherapy cycles (≤3 vs. >3 cycles). The low TRM rate and encouraging outcomes suggest that HSCT may be a feasible treatment for pediatric patients with AML without favorable cytogenetics.
强化化疗、造血干细胞移植（HSCT）及支持性治疗可提高急性髓系白血病（AML）患儿的生存率。然而，对于细胞遗传学特征不佳的患者，HSCT的作用尚未达成共识。我们根据影响临床结局的预后因素，对65例细胞遗传学特征不佳且接受了HSCT的AML患儿的总生存期（OS）和无事件生存期（EFS）进行了评估，这些因素包括细胞遗传学风险组、预处理方案、供体类型、HSCT时的疾病状态以及HSCT前的化疗周期数。65例患者中有15例死亡：3例死于移植相关死亡率（TRM），12例死于疾病相关死亡率。5年OS和EFS分别为78.0%和72.0%，5年累积复发率和TRM率分别为26.9%和5.1%。生存率不受细胞遗传学分组（中等风险组与不良风险组）、供体类型（亲属供体与非亲属供体）、移植类型（清髓性预处理与减低强度预处理）或移植前化疗周期数（≤3个周期与>3个周期）的影响。低TRM率及令人鼓舞的结果表明，HSCT对于细胞遗传学特征不佳的AML患儿可能是一种可行的治疗方法。
