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Imatinib mesylate for refractory acute myeloblastic leukemia harboring inv(16) and a C-KIT exon 8 mutation

Leukemia volume 19, pages 1673–1675 (2005)Cite this article

TO THE EDITOR

inv(16)(p13q22) generates the PEBP2β(CBFβ)-MYH11 fusion protein predicted to interfere with the function of the heterodimeric transcription factor AML1(RUNX1)/PEBP2β and contributes to impaired differentiation of hematopoietic cells.1, 2 However, studies using conditional knock-in mice with PEBP2β-MYH11 have documented that while the fusion protein is critical for leukemogenesis, additional genetic events are required for the development of acute myeloblastic leukemia (AML). Recently, mutations in the class III receptor tyrosine kinase (RTK) including C-KIT and FLT3 were frequently found in patients with inv(16) AML.2 These mutations could represent potential therapeutic targets for specific tyrosine kinase inhibitors. However, clinical efficacy of imatinib mesylate in AML patients with C-KIT mutation remains to be determined. While an approximately half of the patients with inv(16) AML generally have a favorable outcome, the treatment outcome is not universally favorable.1 In this study, we present a case of relapsed AML harboring inv(16) and a C-KIT exon 8 mutation who was successfully treated with imatinib and mild chemotherapy.

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Acknowledgements

This work was supported in part by Grants-in-Aid for Scientific Research from the Japanese Ministry of Education, Culture, Sport, Science and Technology, Grants-in-Aid for Cancer Research from Japanese Ministry of Health, Labor and Welfare, and the Public Trust Haraguchi Memorial Cancer Research Fund. We are very grateful to Kirin Brewery Co. Ltd (Tokyo, Japan) and Novartis Pharma AG (Basel, Switzerland) for providing stem cell factor and imatinib mesylate used in this study. We also thank Ms M Ichinomiya-Nakayama for technical assistances.

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  1. Department of Hematology, Kumamoto University School of Medicine, Kumamoto, Japan

    T Nanri, N Matsuno, T Kawakita, H Mitsuya & N Asou

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  1. T Nanri
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  2. N Matsuno
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Correspondence to N Asou.

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Nanri, T., Matsuno, N., Kawakita, T. et al. Imatinib mesylate for refractory acute myeloblastic leukemia harboring inv(16) and a C-KIT exon 8 mutation. Leukemia 19, 1673–1675 (2005). https://doi.org/10.1038/sj.leu.2403889

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