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伊马替尼和博纳吐单抗成功挽救了一名对达沙替尼和化疗耐药的患有TERF2::PDGFRB融合基因的小儿B淋巴细胞白血病患者。

Imatinib and blinatumomab successfully rescued a pediatric B-ALL patient with TERF2::PDGFRB fusion resistant to dasatinib and chemotherapy.

作者信息

Ye Fanghua, Wang Leyuan, Qian Yujie, Deng Wenjun, Yu Yan, Yang Liangchun

机构信息

Department of Pediatrics, Xiangya Hospital, Central South University, Changsha, China.

出版信息

Ann Hematol. 2025 Sep 20. doi: 10.1007/s00277-025-06620-7.

Abstract

PDGFRB-rearranged acute lymphoblastic leukemia (ALL), an ABL-class Ph-like ALL subtype, typically exhibits chemotherapy resistance and poor prognosis. Precise diagnosis and therapies are crucial for improving outcomes. Here, we report a case of a 9-year-old male harboring a TERF2::PDGFRB fusion, exhibiting primary resistance to dasatinib, but achieving a remarkable response to imatinib. Subsequent combination therapy with blinatumomab induced sustained bone marrow remission. These findings highlight the variability in tyrosine kinase inhibitors (TKIs) sensitivity among PDGFRB-rearranged ALL cases, supporting early treatment switching upon poor response. Notably, the combination of blinatumomab and imatinib may be an effective treatment strategy for PDGFRB-rearranged ALL.

摘要

血小板衍生生长因子受体β(PDGFRB)重排的急性淋巴细胞白血病(ALL)是ABL类费城样ALL亚型,通常表现出化疗耐药性且预后不良。精确的诊断和治疗对于改善预后至关重要。在此,我们报告一例9岁男性病例,其携带TERF2::PDGFRB融合基因,对达沙替尼原发性耐药,但对伊马替尼有显著反应。随后使用博纳吐单抗的联合治疗诱导了持续的骨髓缓解。这些发现突出了PDGFRB重排的ALL病例中酪氨酸激酶抑制剂(TKIs)敏感性的变异性,支持在反应不佳时尽早更换治疗方案。值得注意的是,博纳吐单抗和伊马替尼联合使用可能是治疗PDGFRB重排的ALL的有效治疗策略。

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