Preserved High Probability of Overall Survival with Significant Reduction of Chemotherapy for Myeloid Leukemia in Down Syndrome: A Nationwide Prospective Study in Japan.
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.
['Taga|Takashi|T|', 'Watanabe|Tomoyuki|T|', 'Tomizawa|Daisuke|D|', 'Kudo|Kazuko|K|', 'Terui|Kiminori|K|', 'Moritake|Hiroshi|H|', 'Kinoshita|Akitoshi|A|', 'Iwamoto|Shotaro|S|', 'Nakayama|Hideki|H|', 'Takahashi|Hiroyuki|H|', 'Shimada|Akira|A|', 'Taki|Tomohiko|T|', 'Toki|Tsutomu|T|', 'Ito|Etsuro|E|', 'Goto|Hiroaki|H|', 'Koh|Katsuyoshi|K|', 'Saito|Akiko M|AM|', 'Horibe|Keizo|K|', 'Nakahata|Tatsutoshi|T|', 'Tawa|Akio|A|', 'Adachi|Souichi|S|']
D000293:Adolescent D000971:Antineoplastic Combined Chemotherapy Protocols D002648:Child D002675:Child, Preschool D003561:Cytarabine D018572:Disease-Free Survival D004314:Down Syndrome D004317:Doxorubicin D005047:Etoposide D005260:Female D006801:Humans D060828:Induction Chemotherapy D007223:Infant D007564:Japan D053208:Kaplan-Meier Estimate D007951:Leukemia, Myeloid D008297:Male D011446:Prospective Studies
Down syndrome acute myeloid leukemia clinical trial