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Efficacy and safety of ponatinib for patients with Philadelphia chromosome-positive acute lymphoblastic leukemia: a case series from a single institute

発表形態:
資料・解説・論説・研究報告・総合雑誌の論文
主要業績:
主要業績
単著・共著:
共著
発表年月:
2021年08月
DOI:
10.1007/s12185-021-03156-0
会議属性:
指定なし
査読:
有り
リンク情報:

日本語フィールド

著者:
*Keisuke Kidoguchi, Hiroshi Ureshino, Haruna Kizuka-Sano, Kyosuke Yamaguchi, Hiroo Katsuya, Yasushi Kubota, Toshihiko Ando, Masatomo Miura, Naoto Takahashi, Shinya Kimura
題名:
Efficacy and safety of ponatinib for patients with Philadelphia chromosome-positive acute lymphoblastic leukemia: a case series from a single institute
発表情報:
Int J Hematol 巻: 114 号: 2 ページ: 199-204
キーワード:
ABL1 tyrosine kinase inhibitor; Acute lymphoblastic leukemia; Philadelphia chromosome; Ponatinib; T315I mutation
概要:
Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph + ALL) is an aggressive leukemia that occurs in 20-40% of adult patients. Ph + ALL is caused by the Philadelphia chromosome (Ph), which consists of a t(9;22)(q34;q11) reciprocal translocation leading to the formation of a BCR-ABL1 fusion gene. The disease is treated with targeted therapy comprising ABL1 tyrosine kinase inhibitors (TKIs). Ponatinib is a third generation TKI that demonstrates higher binding affinity for ABL1 than first/second generation TKIs. Although intensive combined immunotherapy with ponatinib greatly improves the prognosis of Ph + ALL, the safety and efficacy profiles of ponatinib in Japanese patients are unclear. This retrospective study investigated five cases of Ph + ALL at a single institute to evaluate safety and efficacy profiles. Three patients achieved a deep molecular response (DMR) following combined intensive treatment with ponatinib as induction chemotherapy. Four patients received consolidative allogenic stem cell transplantation (allo-SCT) during their first complete response. Three of the four experienced early relapse within 100 days; they subsequently received ponatinib, and one of the three achieved a DMR. No patient experienced severe cardiovascular events. This case series suggests that ponatinib at a concentration of least 30 mg exhibits anti-leukemia effects in Japanese patients with Ph + ALL.
抄録:

英語フィールド

Author:
*Keisuke Kidoguchi, Hiroshi Ureshino, Haruna Kizuka-Sano, Kyosuke Yamaguchi, Hiroo Katsuya, Yasushi Kubota, Toshihiko Ando, Masatomo Miura, Naoto Takahashi, Shinya Kimura
Title:
Efficacy and safety of ponatinib for patients with Philadelphia chromosome-positive acute lymphoblastic leukemia: a case series from a single institute
Announcement information:
Int J Hematol Vol: 114 Issue: 2 Page: 199-204
Keyword:
ABL1 tyrosine kinase inhibitor; Acute lymphoblastic leukemia; Philadelphia chromosome; Ponatinib; T315I mutation
An abstract:
Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph + ALL) is an aggressive leukemia that occurs in 20-40% of adult patients. Ph + ALL is caused by the Philadelphia chromosome (Ph), which consists of a t(9;22)(q34;q11) reciprocal translocation leading to the formation of a BCR-ABL1 fusion gene. The disease is treated with targeted therapy comprising ABL1 tyrosine kinase inhibitors (TKIs). Ponatinib is a third generation TKI that demonstrates higher binding affinity for ABL1 than first/second generation TKIs. Although intensive combined immunotherapy with ponatinib greatly improves the prognosis of Ph + ALL, the safety and efficacy profiles of ponatinib in Japanese patients are unclear. This retrospective study investigated five cases of Ph + ALL at a single institute to evaluate safety and efficacy profiles. Three patients achieved a deep molecular response (DMR) following combined intensive treatment with ponatinib as induction chemotherapy. Four patients received consolidative allogenic stem cell transplantation (allo-SCT) during their first complete response. Three of the four experienced early relapse within 100 days; they subsequently received ponatinib, and one of the three achieved a DMR. No patient experienced severe cardiovascular events. This case series suggests that ponatinib at a concentration of least 30 mg exhibits anti-leukemia effects in Japanese patients with Ph + ALL.


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