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 treating 29 patients with low/intermediate-1 risk myelodysplastic syndromes (MDS) with isolated del(5q). The treatment showed limited success, with only 1 patient (3%) achieving a major erythroid response and 4 patients (14%) achieving a minor response. No cytogenetic responses were observed, and there was no significant improvement in quality of life. A high incidence of side effects was reported, including skin reactions, conjunctivitis, and headaches, leading to treatment discontinuation in some cases. The study concluded that this combination therapy was not recommended for del(5q) MDS.
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