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
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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DOI：
10.1007/s12185-019-02628-8
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发表时间：
2019-07-01
影响因子：
2.1
通讯作者：
Iida, Shinsuke
中科院分区：
文献类型：
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作者：
Sasaki, Hirokazu;Mitani, Sachiko;Iida, Shinsuke
一位42岁女性，主诉发热和盗汗，被诊断为急性巨核母细胞母细胞期慢性髓性白血病（CML-BP）。她有大量脾肿大，左胸腔积液伴白血病浸润，中度骨髓纤维化。患者接受达沙替尼单药治疗（140mg/天）诱导，术后胸腔积液迅速消除，血液学缓解。然而，由于外周血BCR-ABL融合信号增加，CML在开始达沙替尼治疗4个月后复发。在CML复发时也检测到T315I突变。作为补救性治疗，立即开始波纳替尼单药治疗（45mg/天）。5个月后，BCR-ABL融合信号下降至5%，骨髓纤维化从MF 2级改善至1级；随后，她接受了一名非亲属供体的同种异体骨髓移植。然而，移植失败，并进行脐带血移植（CBT）。在CBT后继续波纳替尼（15mg/天）作为维持治疗，分子完全缓解持续超过1年，无严重不良事件，包括心血管事件。关于同种异体造血干细胞移植前后波纳替尼的最佳剂量和时间表，特别是在有T315I突变的CML-BP患者中，证据有限；因此，精心设计的临床试验是必要的。
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 (140mg/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 (45mg/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 (15mg/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.