Treatment of Del(5q) Myelodysplastic Syndrome with All-Trans-Retinoic Acid and Tocopherol-Alpha

    November 2004 in “ Blood ”
    Aristoteles Giagounidis, Sabine Haase, Ulrich Germing, Claudia Schoch, Brigitte Schlegelberger, Ludwig Wilkens, Guntram Büsche, Hans Kreipe, Manuel Aivado, Michael Lübbert, Arnold Ganser, Carlo Aul
    TLDR The treatment was not recommended due to limited effectiveness and significant side effects.
    The study investigated the efficacy and safety of all-trans-retinoic acid (ATRA) combined with tocopherol-α in 29 patients with low/intermediate-1 risk myelodysplastic syndromes (MDS) and isolated del(5q). The treatment resulted in significant side effects, with 86% of patients experiencing adverse events, including serious ones like skin reactions and conjunctivitis. Only 1 patient (3%) achieved a major erythroid response, and 4 patients (14%) had a minor response, but no cytogenetic responses were observed. The study concluded that the combination of ATRA and tocopherol-α was not recommended for treating del(5q) MDS due to limited efficacy and significant side effects.
    Discuss this study in the Community →