Amifostine in the treatment of low-risk myelodysplastic syndromes

Background and Objectives. The phosphorylated aminothiol agent amifostine ( Ethyol) protects bone and other tissues from toxicity due to ionizing radia tion and antineoplastic drugs, and stimulates progenitors from normal and m yelodysplastic marrow. Contrasting results have been published so far on th e effectiveness of amifostine in ring cytopenia in patients with myelodys-p lastic syndromes (MDS). Design and Methods. In a pilot phase II study we treated 26 patients with l ow risk MDS (13 RA, 2 RARS, 2 CMML, 9 RAEB with blasts < 10%) with amifosti ne (200 mg/m(2) x 3/week for 4 weeks). Results. Hemoglobin concentration, reticulocyte, neutrophil and platelet co unts increased respectively in 6 (23%), 11 (42%), 13 (50%) and 9 (34%) of p atients. Red cell transfusions were reduced (> 50%) in 4/26 patients and ab olished in 1/26. Unexpectedly a significant decrease in soluble transferrin receptor level at week 4 of therapy, compared to the basal level (p<0.04), was observed in the whole population of patients. Interpretation and Conclusions. Amifostine can ameliorate cytopenia in some patients with MDS, with few and mild side effects. Neutropenia is more lik ely to be corrected than anemia or thrombocytopenia. Mechanisms underlying this biological remain to be clarified. (C)2000 Ferrata Storti Foundation.