Randomized Study of 13-cis Retinoic Acid v Placebo in the Myelodysplastic Disorders

• Published in: Blood Vol. 71; no. 3; pp. 703 - 708
• Main Authors: Koeffler, H. Phillip, Heitjan, Daniel, Mertelsmann, Rol, Kolitz, Jonathan E., Schulman, Philip, Itri, Loretta, Gunter, Patricia, Besa, Emmanuel
• Format: Journal Article
• Language: English
• Published: Washington, DC Elsevier Inc 01.03.1988; The Americain Society of Hematology
• Subjects: Antineoplastic agents; Biological and medical sciences; Chemotherapy; Clinical Trials as Topic; Double-Blind Method; Hematopoiesis - drug effects; Humans; Medical sciences; Myelodysplastic Syndromes - drug therapy; Myelodysplastic Syndromes - physiopathology; Pharmacology. Drug treatments; Random Allocation; Skin Diseases - chemically induced; Sleep Stages; Tretinoin - adverse effects; Tretinoin - therapeutic use; Antineoplastic agent; Human; Myeloproliferative syndrome; Chemotherapy; Randomization; Premalignant lesion; Treatment; Leukemia; Placebo; Controlled therapeutic trial; Malignant hemopathy; Retinoids
• ISSN: 0006-4971; 1528-0020
• DOI: 10.1182/blood.V71.3.703.703

A double-blind, placebo-controlled randomized trial of 13-cis retinoic acid was performed to determine if the drug has a therapeutic effect in patients with myelodysplastic syndromes (MDS). Sixty-eight evaluable patients with MDS were randomized to receive a single, daily oral dose of either 13-cis retinoic acid (13-CRA, 100 mg/m2) or matching placebo. Treatment was continued, when possible, for a period of 6 months. Determination of response to treatment was based on clinical course, repeat bone marrow biopsies, and aspirates and blood counts (CBC) with WBC differential, platelet, and reticulocyte numbers at specified intervals. No significant difference was noted between the two treatment groups in response to test drug (P = .66). One patient (3%) in the 13-CRA group and two patients (6%) in the placebo group had a minor response. Approximately 30% of patients in both groups had progression of their disease, and progression-free survival was nearly identical. Greater than 90% of the patients receiving 13-CRA developed mild or moderate skin toxicity that was reversible with decreasing or discontinuing the drug. Our study did not find that 13-CRA exerts a beneficial therapeutic effect in patients with MDS.