Successful treatment of primary refractory anemia with a combination regimen of all-trans retinoic acid, calcitriol, and androgen.

We investigated the efficacy and safety of a combination regimen in 63 patients with primary refractory anemia (RA). The daily treatment protocol comprised all-trans retinoic acid (ATRA) (30 mg/m(2)), calcitriol (0.1 microg/m(2)), and androgen (stanozolol 3mg/m(2), or danazol 300 mg/m(2)) in three separate doses for eight consecutive weeks. Hematologic improvement was observed in 43 (68.3%) patients. The treatment administered was generally well tolerated, with no severe regimen-related toxicity. The overall survival rates at 3 and 5 years were 68.72% and 53.18%, respectively. These results indicate that this combination regimen is an effective and well-tolerated treatment for patients with RA.