Treatment of myelodysplastic syndromes with daily oral idarubicin. A phase I-II study

Abstract

Background: Idarubicin, a new anthracycline analogue, is available in an oral preparation, and responses have been observed using relatively aggressive therapy in patients with myelodysplastic syndromes (MDS). The authors studied whether a chronic low-dose schedule would be effective but less myelotoxic.

Methods: Forty-two patients with MDS received daily low-dose oral idarubicin in 5-week courses that included 3 weeks of treatment, followed by a 2-week rest period. Doses were escalated when possible after the second course, and each patient was to receive six courses.

Results: Only one partial response was observed. Although no patient had fatal bone marrow toxicity, only eight patients received the full six courses, primarily because of myelosuppression.

Conclusions: This schedule of oral idarubicin is relatively safe but produces fewer responses than are reported with the high-dose pulse regimens.