Recombinant human erythropoietin plus all-<i>trans</i> retinoic acid and testosterone undecanoate for the treatment of anemia in patients with lower-risk myelodysplastic syndromes: a multicenter, single-arm, prospective trial.

ABSTRACT: Erythropoiesis-stimulating agents (ESA) achieve hematological improvement-erythroid (HI-E) in only 30% of ESA-naïve lower- risk myelodysplastic syndrome (LR-MDS) patients with anemia, highlighting the need for developing novel drugs or new treatment strategies to improve the outcome of these patients. We conducted this multicenter, single-arm trial to investigate the efficacy and safety of a triple regimen consisting of recombinant human erythropoietin (rhEPO), all-trans retinoic acid (ATRA) and testosterone undecanoate in patients with anemia due to lower-risk MDS based on Revised International Prognostic Scoring System. Eligible patients received rhEPO 10,000 IU/day, oral ATRA 25 mg/m2/day and oral testosterone undecanoate 80 mg twice daily for 12 weeks. The primary endpoint was the prop

SUBMITTER: Mei C 

PROVIDER: S-EPMC12862310 | biostudies-literature | 2026 Feb

REPOSITORIES: biostudies-literature