非血缘脐血干细胞移植治疗高危/难治儿童急性髓系白血病160例临床分析

OBJECTIVE: To retrospectively analyze the clinical outcomes of single unrelated cord blood transplantation（UCBT）in children with high risk and refractory acute myeloid leukemia（AML）. METHODS: Between June 2008 and December 2018, a total of 160 consecutive pediatric patients with AML received single UCBT（excluding acute promyelocytic leukemia）. Myeloablative conditioning（MAC）regimen were applied. All patients received a combination of cyclosporine A（CsA）and mycophenolate mofetil（MMF）for the prophylaxis of graft-versus-host disease（GVHD）. RESULTS: The cumulative incidence of neutrophil cells engraftment at day +42 and platelet recovery at day +120 was 95.0％（95％ CI 90.0％–97.5％）at a median of 16 days after transplantation（range, 11–38 days）and 85.5％（95％ CI 83.3％–93.4％）with a median time to recovery of 35 days（range, 13–158）, respectively. Incidence of grades Ⅱ-Ⅳ and Ⅲ-Ⅳ acute GVHD and chronic GVHD were 37.3％（95％ CI 29.3％–45.2％）, 27.3％（95％ CI 20.0％–35.0％）and 22.4％（95％ CI 15.5％–28.7％）, respectively. The transplant-related mortality（TRM）at 360 day was 13.1％（95％ CI 8.4％–18.9％）. The 5-year cumulative incidence of relapse was 13.8％（95％ CI 8.5％–20.3％）. The 5-year disease-free survival（DFS）and overall survival（OS）were 71.7％（95％ CI 62.7％–77.8％）and 72.2％（95％ CI 64.1％–78.7％）, respectively. The 5-year GVHD and relapse free survival（GRFS）was 56.1％（95％ CI 46.1％–64.9％）. The 5-year cumulative recurrence rates of CR1, CR2, and NR groups were 5.3％, 19.9％, and 30.9％（P＝0.001）, and the 5-year OS rates were 79.9％（95％CI 70.3％–86.7％）, 71.1％（95％CI 50.4％–84.4％）and 52.9％（95％CI 33.0％–69.3％）（χ(2)＝7.552, P＝0.020）, respectively. CONCLUSION: For pediatric patients with high risk and refractory AML, UCBT is a safe and effective treatment option, and it is favorable to improve the survival rate in CR1 stage.