Autophagy Stimulation Improves Erythroid Proliferative Capacity in Models of SF3B1 Mutant MDS

    April 2017 in “ Leukemia research ”
    Valeria Visconte, Vera Ademà, Yasmine Hirbawi, Bartlomiej Przychodzen, Katherine A. Kelly, Cassandra M. Hirsch, Michael J. Clemente, Suresh Kumar Balasubramanian, Hetty E. Carraway, Daniel J. Lindner, Mikkael A. Sekeres, Heesun J. Rogers, James G. Phillips, T. Radivoyevitch, Steffan T. Nawrocki, Jennifer S. Carew, J. Maciejewski
    TLDR Tofacitinib helped most teenagers in the study regrow hair with mild side effects.
    In a retrospective study of 13 adolescent patients with alopecia areata (AA) aged 12 to 17 years, treatment with the Janus kinase 1/3 inhibitor, tofacitinib, resulted in clinically significant hair regrowth in 9 patients. The median percent change in the Severity of Alopecia Tool (SALT) score was 93% (mean 61%, range 1%-100%) after an average treatment duration of 6.5 months. The adverse events reported were mild. However, the study had limitations such as its retrospective design, small sample size, and absence of a control group. Despite these limitations, the findings suggest that tofacitinib is a promising therapy for AA in adolescents and warrants further investigation in prospective clinical trials.
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