Prolonged complete remission od myelodysplastic syndrome treated with danazol, retinoic acid and low-dose prednisone

Myelodysplastic syndrome (MDS) is a diverse group of clonal hematologic neo plasms, Different medications have been tried in MDS; however, no effective treatment has been yet established We report a patient with MDS who achiev ed a complete remission in response to combination therapy of danazol, reti noic acid, and prednisone. A 53-year-old female presented with pancytopenia , macrocytosis, and hypercellular bone marrow with erythroid hyperplasia an d dysplasia and 10% ringed, sideroblasts, Cytogenetic studies revealed the presence of two abnormal clones. She was diagnosed as having MDS-refractory anemia and was given blood transfusions to maintain blood cell counts at a cceptable levels. At the same time, she was started on a combination of dan azol (600 mg/day), retinoic acid (100 mg/day), and prednisone (10 mg every other day). Fourteen months later, the patient was in complete hematologic remission; she had normal peripheral blood count, and the blood smear showe d normal morphology. Bone marrow studies revealed normal trilineage hematop oiesis, She was continued on the same combination treatment for 86 months, and she remained in complete clinical remission. Eighty-eight months from d iagnosis, she relapsed with acute myeloid leukemia. This is the first repor ted case of MDS-RA that sustained a complete hematologic remission for a pr olonged period in response to this combination treatment. This report indic ates that restoration of normal hematopoiesis, prolongation of disease-free survival, and delay in the transformation to acute leukemia may be achieve d by this combination of treatment in a subset of patients with MDS, especi ally refractory anemia with severe thrombocytopenia, Am. J. Hematol. 64:306 -310, 2000, (C) 2000 Wiley-Liss, Inc.