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

Irene Sadek, E. Zayed, Ormille A. Hayne, L. A. Fernández
In: American Journal of Hematology · 2000 · vol. 64(4) , pp. 306–310 · doi:10.1002/1096-8652(200008)64:4<306::aid-ajh12>3.0.co;2-v · PMID:10911385 · W1979470678
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A patient with myelodysplastic syndrome-refractory anemia achieved prolonged complete hematologic remission with danazol, retinoic acid, and low-dose prednisone combination therapy, lasting 86 months.

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Abstract

Myelodysplastic syndrome (MDS) is a diverse group of clonal hematologic neoplasms. Different medications have been tried in MDS; however, no effective treatment has been yet established. We report a patient with MDS who achieved a complete remission in response to combination therapy of danazol, retinoic acid, and prednisone. A 53-year-old female presented with pancytopenia, macrocytosis, and hypercellular bone marrow with erythroid hyperplasia and 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 acceptable levels. At the same time, she was started on a combination of danazol (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 showed normal morphology. Bone marrow studies revealed normal trilineage hematopoiesis. She was continued on the same combination treatment for 86 months, and she remained in complete clinical remission. Eighty-eight months from diagnosis, she relapsed with acute myeloid leukemia. This is the first reported case of MDS-RA that sustained a complete hematologic remission for a prolonged period in response to this combination treatment. This report indicates that restoration of normal hematopoiesis, prolongation of disease-free survival, and delay in the transformation to acute leukemia may be achieved by this combination of treatment in a subset of patients with MDS, especially refractory anemia with severe thrombocytopenia.

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