TY - JOUR
T1 - Preserved High Probability of Overall Survival with Significant Reduction of Chemotherapy for Myeloid Leukemia in Down Syndrome
T2 - A Nationwide Prospective Study in Japan
AU - Taga, Takashi
AU - Watanabe, Tomoyuki
AU - Tomizawa, Daisuke
AU - Kudo, Kazuko
AU - Terui, Kiminori
AU - Moritake, Hiroshi
AU - Kinoshita, Akitoshi
AU - Iwamoto, Shotaro
AU - Nakayama, Hideki
AU - Takahashi, Hiroyuki
AU - Shimada, Akira
AU - Taki, Tomohiko
AU - Toki, Tsutomu
AU - Ito, Etsuro
AU - Goto, Hiroaki
AU - Koh, Katsuyoshi
AU - Saito, Akiko M.
AU - Horibe, Keizo
AU - Nakahata, Tatsutoshi
AU - Tawa, Akio
AU - Adachi, Souichi
PY - 2016/2/1
Y1 - 2016/2/1
N2 - Background: On the basis of results of previous Japanese trials for myeloid leukemia in Down syndrome (ML-DS), the efficacy of risk-oriented therapy was evaluated in the Japanese Pediatric Leukemia/Lymphoma Study Group AML-D05 study. Procedure: All patients received induction chemotherapy that consisted of pirarubicin, intermediate-dose cytarabine, and etoposide. Patients who achieved complete remission (CR) after initial induction therapy were stratified to the standard risk (SR) group and received four courses of reduced-dose intensification therapy. Patients who did not achieve CR were stratified to the high risk (HR) group and received intensified therapy that consisted of continuous or high-dose cytarabine. Results: A total of 72 patients were eligible and evaluated. One patient died of sepsis during initial induction therapy. Sixty-nine patients were stratified to SR and two patients to HR. No therapy-related deaths were observed during intensification therapy. The 3-year event-free and overall survival rates were 83.3% ± 4.4% and 87.5% ± 3.9 %, respectively. Age at diagnosis less than 2 years was a significant favorable prognostic factor for risk of relapse (P = 0.009). Conclusions: The attempt of risk-oriented prospective study for ML-DS was unsuccessful, but despite the dose reduction of chemotherapeutic agents, the overall outcome was good, and further dose reduction might be possible for specific subgroups.
AB - Background: On the basis of results of previous Japanese trials for myeloid leukemia in Down syndrome (ML-DS), the efficacy of risk-oriented therapy was evaluated in the Japanese Pediatric Leukemia/Lymphoma Study Group AML-D05 study. Procedure: All patients received induction chemotherapy that consisted of pirarubicin, intermediate-dose cytarabine, and etoposide. Patients who achieved complete remission (CR) after initial induction therapy were stratified to the standard risk (SR) group and received four courses of reduced-dose intensification therapy. Patients who did not achieve CR were stratified to the high risk (HR) group and received intensified therapy that consisted of continuous or high-dose cytarabine. Results: A total of 72 patients were eligible and evaluated. One patient died of sepsis during initial induction therapy. Sixty-nine patients were stratified to SR and two patients to HR. No therapy-related deaths were observed during intensification therapy. The 3-year event-free and overall survival rates were 83.3% ± 4.4% and 87.5% ± 3.9 %, respectively. Age at diagnosis less than 2 years was a significant favorable prognostic factor for risk of relapse (P = 0.009). Conclusions: The attempt of risk-oriented prospective study for ML-DS was unsuccessful, but despite the dose reduction of chemotherapeutic agents, the overall outcome was good, and further dose reduction might be possible for specific subgroups.
KW - Down syndrome
KW - acute myeloid leukemia
KW - clinical trial
UR - http://www.scopus.com/inward/record.url?scp=85006320195&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=85006320195&partnerID=8YFLogxK
U2 - 10.1002/pbc.25789
DO - 10.1002/pbc.25789
M3 - Article
AN - SCOPUS:85006320195
VL - 63
SP - 248
EP - 254
JO - Pediatric Blood and Cancer
JF - Pediatric Blood and Cancer
SN - 1545-5009
IS - 2
ER -