Preserved High Probability of Overall Survival with Significant Reduction of Chemotherapy for Myeloid Leukemia in Down Syndrome: A Nationwide Prospective Study in Japan

摘要: Background On the basis of results previous Japanese trials for myeloid leukemia in Down syndrome (ML-DS), efficacy risk-oriented therapy was evaluated Pediatric Leukemia/Lymphoma Study Group AML-D05 study. Procedure All patients received induction chemotherapy that consisted pirarubicin, intermediate-dose cytarabine, and etoposide. Patients who achieved complete remission (CR) after initial were stratified to standard risk (SR) group four courses reduced-dose intensification therapy. did not achieve CR high (HR) intensified continuous or high-dose cytarabine. Results A total 72 eligible evaluated. One patient died sepsis during Sixty-nine SR two HR. No therapy-related deaths observed The 3-year event-free overall survival rates 83.3% ± 4.4% 87.5% 3.9%, respectively. Age at diagnosis less than 2 years a significant favorable prognostic factor relapse (P = 0.009). Conclusions attempt prospective study ML-DS unsuccessful, but despite dose reduction chemotherapeutic agents, outcome good, further might be possible specific subgroups.