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Clinical study of treatment for childhood acute myeloid leukemia by NOPHO AML 2004 protocol combined with hematopoietic stem cell transplantation

??Objective??To analyze the effectiveness and the practicability of Nordic Society for Pediatric Hematology and Oncology??NOPHO?? AML 2004 protocol combined with hematopoietic stem cell transplantation??HSCT?? in treating childhood acute myeloid leukemia??AML?? in China. Methods??The clinical data of 53 children??aged under 14?? with AML??not including acute promyelocytic leukemia?? treated with NOPHO AML 2004 protocol??some children followed by HSCT?? between Dec. 2009 and Dec. 2015 was retrospectively analyzed. Survival was evaluated by Kaplan Meier analysis and Log-Rank test. Results??Complete remission??CR?? rate reached 86.8%. Totally 36 patients??67.9%?? finished 6 courses of chemotherapy??14 patients??26.4%??underwent HSCT after 1-3 courses of consolidation chemotherapy??3 patients gave up treatment after 1-3 courses of chemotherapy. The proleptic overall survival??pOS?? at 5 years and leukemia-free survival??pLFS?? was 72% and 56% respectively during a follow-up period of 12 to 82 months??median 29 months??. For standard-risk patients??the pLFS at 5 years reached 58%?? while high-risk patients had 46%??P??0.779??. The pLFS at 5 years in patients who only underwent chemotherapy and those with additional hematopoietic stem cell transplantation was 48% and 77% respectively??P??0.1268??. The pLFS in patients with good response??intermediate response and poor response on day 15 bone marrow examination was 75%??29% and 53% respectively??P??0.0071??. Conclusion??NOPHO AML 2004 protocol is effective and feasible for Chinese patients with childhood AML. The evaluation of blast in bone marrow on day 15 can be used as an important prognostic indicator.