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波纳替尼作为不适合异基因干细胞移植和/或传统化疗的急变期慢性髓性白血病患者的有效替代策略:1例报告

Ponatinib as a Valid Alternative Strategy in Patients with Blast Crisis-Chronic Myeloid Leukemia Not Eligible for Allogeneic Stem Cells Transplantation and/or Conventional Chemotherapy: Report of a Case.

作者信息

Bucelli Cristina, Cattaneo Daniele, Ferla Valeria, Zappa Manuela, de Benedittis Caterina, Soverini Simona, Iurlo Alessandra

机构信息

Hematology Division, IRCCS Ca' Granda-Maggiore Policlinico Hospital Foundation and University of Milan, Milan, Italy.

Department of Experimental, Diagnostic and Specialty Medicine, Institute of Hematology "L. and A. Seragnoli," University of Bologna, Bologna, Italy.

出版信息

Case Rep Hematol. 2017;2017:6167345. doi: 10.1155/2017/6167345. Epub 2017 Aug 14.

DOI:10.1155/2017/6167345
PMID:28890835
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC5584354/
Abstract

Currently, imatinib and dasatinib are the only tyrosine-kinase inhibitors approved in the US and Europe for the treatment of blast crisis of chronic myeloid leukemia (BC-CML) at diagnosis, while ponatinib is the only inhibitor used in patients bearing T315I mutation. Here we report the case of a 61-year-old man diagnosed with B-cell lymphoid BC-CML, initially treated with imatinib 800 mg day and then with dasatinib 140 mg day because of intolerance. A complete cytogenetic response (CCyR) was achieved at three months; however, three months later a relapse was observed, and the T315I mutation was detected. Ponatinib 45 mg once daily was then started together with a short course of chemotherapy. Bone marrow evaluation after six months of therapy showed the regaining of CCyR, together with the achievement of a deep molecular response. However, one year from ponatinib start the patient experienced a new disease relapse; he was effectively treated with ponatinib and chemotherapy once again, but in the meanwhile an ischemic stroke was detected. This case report confirms the high efficacy of ponatinib monotherapy in BC-CML patients, representing a valid option for non-allogeneic stem cells transplantation eligible cases and the only one available for those carrying the T315I mutation.

摘要

目前,伊马替尼和达沙替尼是美国和欧洲批准用于治疗初诊慢性髓性白血病急变期(BC-CML)的仅有的酪氨酸激酶抑制剂,而波纳替尼是用于携带T315I突变患者的唯一抑制剂。在此,我们报告一例61岁男性患者,诊断为B细胞淋巴性BC-CML,最初接受每日800毫克伊马替尼治疗,后因不耐受改为每日140毫克达沙替尼治疗。三个月时实现了完全细胞遗传学缓解(CCyR);然而,三个月后观察到复发,并检测到T315I突变。随后开始每日一次服用45毫克波纳替尼并联合短期化疗。治疗六个月后的骨髓评估显示CCyR恢复,同时实现了深度分子反应。然而,开始使用波纳替尼一年后,患者再次出现疾病复发;再次接受波纳替尼和化疗有效治疗,但与此同时检测到缺血性中风。本病例报告证实了波纳替尼单药治疗在BC-CML患者中的高效性,对于适合非异基因干细胞移植的病例是一种有效的选择,对于携带T315I突变的患者是唯一可用的选择。

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