Ye Fanghua, Wang Leyuan, Qian Yujie, Deng Wenjun, Yu Yan, Yang Liangchun
Department of Pediatrics, Xiangya Hospital, Central South University, Changsha, China.
Ann Hematol. 2025 Sep 20. doi: 10.1007/s00277-025-06620-7.
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.
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。
