Treatment of unfavorable myelodysplastic syndrome with all-trans-retinoic acid and subcutaneous Ara C

Thirty patients with high-risk myelodysplastic syndrome, defined as>5% bone marrow blasts, were treated with a combination of oral all-trans-retinoic acid (45 mg/m² daily) and subcutaneous AraC (10 mg/m²) on days 1-14 of each 28-35 day cycle repeated for 2-6 cycles. Complete remission lasting 9, 12, and 15 months was achieved in three patients. Partial and minor response did not translate into meaningful clinical improvement, like complete responders. Overall incidence of leukemia transformation and survival of this cohort of patients was no different from the expected outcome for a group of patients with similar characteristics. Copyright (C) 1999 Elsevier Science Ltd.