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document
Randomized study of 13-cis retinoic acid v placebo in the myelodysplastic disorders . 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 .
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Intervention_Pharmacological
13-cis retinoic acid
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Intervention_Control
placebo
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Intervention_Pharmacological
13-cis retinoic acid
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Intervention_Pharmacological
13-cis retinoic acid ( 13-CRA
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Intervention_Control
matching placebo .
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Outcome_Physical
therapeutic effect
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Outcome_Other
clinical course , repeat bone marrow biopsies
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Outcome_Other
aspirates and blood counts ( CBC ) with WBC differential , platelet , and reticulocyte numbers at specified intervals
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Outcome_Physical
response
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Outcome_Physical
progression of their disease
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Outcome_Mortality
progression-free survival
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Outcome_Physical
mild or moderate skin toxicity
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Outcome_Physical
therapeutic effect
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Participant_Condition
myelodysplastic disorders .