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Approximately 20% of patients with transient abnormal myelopoiesis (TAM) die due to hepatic or multiorgan failure. To identify potential new treatments for TAM, we performed in vitro drug sensitivity testing (DST) using the peripheral blood samples of eight patients with TAM. DST screened 41 agents for cytotoxic properties against TAM blasts. Compared with the reference samples of healthy subjects, TAM blasts were more sensitive to glucocorticoids, the mitogen-activated protein kinase kinase (MAP2K) inhibitor trametinib, and cytarabine. Our present results support the therapeutic potential of glucocorticoids and the role of the RAS/MAP2K signalling pathway in TAM pathogenesis. © 2021 British Society for Haematology and John Wiley & Sons Ltd.

Citation

Tomoko Yokosuka, Mieko Ito, Yuki Yoshino, Ayana Hirose, Wataru Nakamura, Yukari Sakurai, Akiko Hayashi, Sachio Fujita, Naoyuki Miyagawa, Dai Keino, Fuminori Iwasaki, Satoshi Hamanoue, Masakatsu Yanagimachi, Shoko Goto, Jun-Ichi Nagai, Hiroo Ueno, Junko Takita, Yukichi Tanaka, Takashi Taga, Hiroaki Goto. Using the in vitro drug sensitivity test to identify candidate treatments for transient abnormal myelopoiesis. British journal of haematology. 2022 Feb;196(3):764-768

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PMID: 34816427

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