Treatment of myelodysplastic syndrome with isolated del(5q) including bands q31–q33 with a combination of all-trans-retinoic acid and tocopherol-α: a phase II study

All-trans-retinoic acid (ATRA) alone or in combination with cytokines and vitamins has been shown to stimulate erythropoiesis in low-risk myelodysplastic syndromes (MDS). We performed a phase II study on 29 patients with MDS and isolated del(5q) including bands q31–q33 to determine the efficacy and safety of ATRA in combination with tocopherol-α. All patients had low/intermediate-1 risk MDS according to the international prognostic scoring system. They received 45 mg/m² ATRA on days 1 to 90, and 90 mg/m² on days 91 to 180. Tocopherol dosage was 600 IU three times daily. Twenty-four patients completed dose level I, and 12 patients dose level II. Eighty-six percent of patients experienced side effects. Thirty discontinued the drug treatment due to such events as skin reactions, cheilitis, conjunctivitis, joint pain, creatinine increase, or CNS symptoms. One patient (3%) achieved a major erythroid response resulting in transfusion independence throughout the study. Four patients (14%) achieved a minor erythroid response with >50% reduction of transfusion needs. None of the participants had a cytogenetic response. There was no significant improvement in quality of life among responding patients as measured by the European Organization for the Research and Treatment of Cancer (EORTC) quality of life questionnaire. Based on these results, the combination of ATRA and tocopherol-α is not recommended for the treatment of del(5q) MDS.