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Pre- and post-transplant ponatinib for a patient with acute megakaryoblastic blast phase chronic myeloid leukemia with T315I mutation who underwent allogeneic hematopoietic stem cell transplantation

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Abstract

A 42-year-old female complaining of fever and night sweats was diagnosed with acute megakaryoblastic blast phase chronic myeloid leukemia (CML-BP). She had massive splenomegaly, left pleural effusion with leukemia infiltration, and moderate myelofibrosis. She received dasatinib monotherapy (140 mg/day) as for induction, after which her pleural effusion rapidly resolved and hematological remission was achieved. However, CML relapsed 4 months after starting dasatinib due to increased BCR-ABL fusion signals in the peripheral blood. The T315I mutation was also detected at the recurrence of CML. As a salvage treatment, ponatinib monotherapy (45 mg/day) was started immediately. After 5 months, BCR-ABL fusion signals decreased to 5%, and myelofibrosis improved from MF Grade 2 to 1; she then underwent allogeneic bone marrow transplantation from an unrelated donor. However, the graft failed, and cord blood transplantation (CBT) was performed. Ponatinib (15 mg/day) was continued after CBT as a maintenance treatment, with molecular complete response continuing for more than 1 year with no severe adverse events, including cardiovascular events. There is limited evidence regarding the optimal dose and schedule of ponatinib before and after allogeneic hematopoietic stem cell transplantation, especially in patients with CML-BP having T315I mutation; thus, well-designed clinical trials are warranted.

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Abbreviations

CML:

Chronic myeloid leukemia

BP:

Blast phase

TKI:

Tyrosine kinase inhibitor

allo-HSCT:

Allogeneic hematopoietic stem cell transplantation

AMKL:

Acute megakaryoblastic leukemia.

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Author notes

  1. Hirokazu Sasaki and Sachiko Mitani contributed equally to this case report.

Authors and Affiliations

  1. Department of Hematology and Oncology, Nagoya City University Graduate School of Medical Sciences, 1 Kawasumi, Mizuho-chou, Mizuho-ku, Nagoya, Aichi, 467-8601, Japan

    Hirokazu Sasaki, Shigeru Kusumoto, Yoshiaki Marumo, Arisa Asano, Takashi Yoshida, Tomoko Narita, Asahi Ito, Masaki Ri, Takashi Ishida, Hirokazu Komatsu & Shinsuke Iida

  2. Department of Hematology, Kainan Hospital, Yatomi, Japan

    Sachiko Mitani & Hiroki Yano

Authors
  1. Hirokazu Sasaki
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  2. Sachiko Mitani
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  3. Shigeru Kusumoto
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  4. Yoshiaki Marumo
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  5. Arisa Asano
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  6. Takashi Yoshida
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  7. Tomoko Narita
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  8. Asahi Ito
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  9. Hiroki Yano
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  10. Masaki Ri
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  11. Takashi Ishida
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  12. Hirokazu Komatsu
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Corresponding author

Correspondence to Shigeru Kusumoto.

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S.I. received research fundings and honoraria from Bristol-Myers Squibb.

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Sasaki, H., Mitani, S., Kusumoto, S. et al. Pre- and post-transplant ponatinib for a patient with acute megakaryoblastic blast phase chronic myeloid leukemia with T315I mutation who underwent allogeneic hematopoietic stem cell transplantation. Int J Hematol 110, 119–123 (2019). https://doi.org/10.1007/s12185-019-02628-8

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  • DOI: https://doi.org/10.1007/s12185-019-02628-8

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