Pw. Wijermans et al., CONTINUOUS-INFUSION OF LOW-DOSE 5-AZA-2'-DEOXYCYTIDINE IN ELDERLY PATIENTS WITH HIGHRISK MYELODYSPLASTIC SYNDROME, Leukemia, 11(1), 1997, pp. 1-5
There is no standard therapy for elderly patients with high-risk myelo
dysplastic syndrome (MDS). The treatment options of low-dose Ara-C and
haematopoietic growth factors are disappointing in regard to response
rate or response duration. We tested the treatment with a 72-h contin
uous infusion of low-dose 5-Aza-2'-deoxycytidine (DAC) in a group of 2
9 elderly patients with high-risk MDS. In 15 patients (54%) we observe
d a response. Eight complete responses were reached, even among patien
ts with bad prognostic cytogenetic findings. The actuarial median surv
ival from the start of the therapy was 46 weeks. The only (and major)
toxicity was myelosuppression, leading to a prolonged cytopenic period
and thus leading to five toxic deaths (17%) in this high-risk patient
group. We conclude that DAC is an effective drug in the treatment of
MDS patients and that it probably works via its cytotoxic activity. My
elotoxicity is its major adverse effect.