Oka Satoko, Akagi Yuina, Mituyoshi Takaya, Ono Kazuo
Division of Hematology, Japanese Red Cross Society Wakayama Medical Center, Wakayama, Wakayama, Japan.
Division of Pathology, Japanese Red Cross Society Wakayama Medical Center, Wakayama, Japan.
Int J Hematol. 2025 Apr 29. doi: 10.1007/s12185-025-03994-2.
The co-occurrence of JAK2 V617F mutations and the BCR::ABL1 translocation in the same patient is rare, and the current standard treatment for aggressive myeloid blast phase chronic myeloid leukemia (CML-myeloid BP) with JAK2 V617F mutations remains inadequate, particularly in transplant-ineligible patients. Asciminib, a first-in-class allosteric inhibitor of BCR::ABL1 kinase that specifically targets the ABL1 myristoyl pocket, has emerged as a novel alternative to standard tyrosine kinase inhibitor (TKI) therapy. Ropeginterferon alfa-2b (ropegIFNα2b) is a novel site-selective, monopegylated recombinant human IFN with long-term safety and efficacy in patients with polycythemia vera (PV). Here, we report a case of successful combination therapy with asciminib and ropegIFNα2b in a patient with CML-myeloid BP who had a long history of PV with JAK2 V617F refractory to induction chemotherapy with several TKIs. The combination of asciminib and ropegIFNα2b is a promising new treatment option for these patients.
JAK2 V617F突变与BCR::ABL1易位在同一患者中同时出现的情况较为罕见，对于伴有JAK2 V617F突变的侵袭性髓系急变期慢性髓性白血病（CML-髓系BP），目前的标准治疗仍然不足，尤其是对于不适合移植的患者。阿斯科利尼布是一种一流的BCR::ABL1激酶变构抑制剂，专门靶向ABL1肉豆蔻酰口袋，已成为标准酪氨酸激酶抑制剂（TKI）治疗的一种新替代方案。聚乙二醇化干扰素α-2b（ropegIFNα2b）是一种新型的位点选择性单聚乙二醇化重组人干扰素，对真性红细胞增多症（PV）患者具有长期安全性和有效性。在此，我们报告了一例CML-髓系BP患者成功接受阿斯科利尼布和ropegIFNα2b联合治疗的病例，该患者有长期PV病史，JAK2 V617F对多种TKI诱导化疗耐药。阿斯科利尼布和ropegIFNα2b联合使用是这些患者一种有前景的新治疗选择。
