What is the efficacy of isotretinoin (13 cis-retinoic acid) (Accutane) in the treatment of myelodysplastic syndrome (MDS)?

Updated: Jul 24, 2018
  • Author: Emmanuel C Besa, MD; Chief Editor: Koyamangalath Krishnan, MD, FRCP, FACP  more...
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Answer

Isotretinoin or 13 cis-retinoic acid (Accutane) is the most active retinoid. In a randomized placebo-controlled trial in 70 MDS patients treated with low-dose isotretinoin (20 mg/m2/d), 1-year survival among patients with refractory anemia was 77%, compared with 36% in the placebo group. [26] This is statistically significant, although this form of therapy is not generally accepted. The author limits this treatment to patients who are not transfusion dependent.


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