Myelodysplastic syndrome treatment with danazol and cis‐retinoic acid

In: American Journal of Hematology · 1995 · vol. 48(4) , pp. 233–236 · doi:10.1002/ajh.2830480405 · PMID:7717370 · W2102299347
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This study evaluated danazol and cis-retinoic acid in myelodysplastic syndrome, finding minimal activity and concluding that neither compound is effective enough to justify their use for treating the condition.

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Abstract

We prospectively treated 46 patients with favorable myelodysplastic syndrome classified as refractory anemia (RA), refractory cytopenia (RC), or refractory anemia with ringed sideroblasts (RARS). These patients received one of two schedules of 13-Cis-Retinoic Acid (low dose 80 mg daily for 6 months vs. high dose 200 mg po daily for 3 months), or Danazol (800 mg po daily for 3 months), and were crossed over to the alternative drug in the absence of response or at progression. Using strict criteria of response we found little objective evidence of activity for either compound. Only two minor responses were seen among 22 patients treated with low dose 13-CRA, 1 response among 20 cases that received high dose 13-CRA, and 1 partial response and 1 minor response to Danazol among 34 cases. Neither 13-Cis-Retinoic Acid nor Danazol appear active enough in patients with favorable myelodysplastic syndrome to justify their use.

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