Prospective study of a pirarubicin, intermediate-dose cytarabine, and etoposide regimen in children with Down syndrome and acute myeloid leukemia: the Japanese Childhood AML Cooperative Study Group.
OBJECTIVE
To evaluate a less intensive chemotherapeutic regimen specifically designed for patients with Down syndrome (DS) and acute myeloid leukemia (AML), and to determine the prognostic factors for event-free survival.


METHODS
Seventy-two patients with AML-DS were treated with remission induction chemotherapy consisting of pirarubicin (25 mg/m2/d for 2 days), cytarabine (100 mg/m2/d for 7 days), and etoposide (150 mg/m2/d for 3 days). Patients received four courses of intensification therapy of the same regimen. Prophylaxis for CNS leukemia was not included.


RESULTS
All but two patients were younger than 4 years, and 67 of the 72 patients (93%) were diagnosed as acute megakaryoblastic leukemia (AMKL). Seventy of the 72 patients (97.2%) achieved a complete remission (CR), and the estimated 4-year event-free survival (EFS) rate was 83% +/- 9%. Nine patients relapsed, and one died as a result of pneumonia during CR. Multivariate analysis revealed that the presence of monosomy 7 was a greater risk factor of adverse outcome (odds ratio = 5.67; P = .027).


CONCLUSIONS
A less intensive chemotherapeutic regimen produces excellent outcomes in standard-risk AML-DS patient. Risk-oriented therapy should be considered for future trials in AML-DS.
Department of Pediatrics, Institute of Clinical Medicine, University of Tsukuba, Tsukuba, Japan. kazukok@md.tsukuba.ac.jp
Kazuko
K
Kudo
Seiji
S
Kojima
Ken
K
Tabuchi
Hiromasa
H
Yabe
Akio
A
Tawa
Masue
M
Imaizumi
Ryoji
R
Hanada
Kazuko
K
Hamamoto
Ryoji
R
Kobayashi
Akira
A
Morimoto
Hideki
H
Nakayama
Masahiro
M
Tsuchida
Keizo
K
Horibe
Hisato
H
Kigasawa
Ichiro
I
Tsukimoto
D000971:Antineoplastic Combined Chemotherapy Protocols / Q000008:administration & dosage*; D002648:Child; D002675:Child, Preschool; D003561:Cytarabine / Q000008:administration & dosage; D018572:Disease-Free Survival; D004314:Down Syndrome / Q000150:complications*; D004317:Doxorubicin / Q000008:administration & dosage / Q000031:analogs & derivatives; D005047:Etoposide / Q000008:administration & dosage; D005260:Female; D006801:Humans; D007223:Infant; D007947:Leukemia, Megakaryoblastic, Acute / Q000150:complications / Q000188:drug therapy*; D008297:Male; D011446:Prospective Studies; D016896:Treatment Outcome
D003561:Cytarabine; D005047:Etoposide; D004317:Doxorubicin; C027260:pirarubicin