Thirty-one patients (20 male and 11 female; median age 51 years (16-79)) wi
th high-risk acute myeloblastic leukemia (AML) (20 refractory AML and 11 se
condary AML (s-AML) (four to myelodysplastic syndrome, five to chemo/radiot
herapy, one to aplastic anemia and one blastic chronic myelogenous leukemia
(B-CML)) were treated with CBDCA (300 mg/m(2)/day x 5 days in continuous i
.v. infusion) plus intermediate-dose Ara-C (500 mg/m(2)/day x 3 days in rap
id i.v, infusion). Nine patients (29%) achieved CR (five s-AML (three myelo
dysplastic syndromes, one CML and one ALL) and four refractory AML) and 11
patients had resistant disease. There were 11 early deaths (35%). Median di
sease-free survival of the nine responders was 4 months. The main toxicity
was hematological, febrile episodes took place in nearly all the patients (
96%). The CBDCA plus Ara-C regimen showed an evident antileukemic activity
in high-risk leukemia. However, the lack of long-term disease-free survivor
s shows the need for innovative postremission strategies. The high initial
response rate seen in AML secondary to myelodysplastic syndromes (MDS) warr
ants further investigation of CBDCA in combination regimens for MDS patient
s.