Hospital Universitario Puerta de Hierro, Madrid, Spain.
Hospital Universitario de Gran Canaria Doctor Negrín, Gran Canaria, Spain.
Ann Hematol. 2024 Nov;103(11):4537-4544. doi: 10.1007/s00277-024-05906-6. Epub 2024 Sep 13.
Patients with chronic myeloid leukemia (CML) who have failed conventional tyrosine kinase inhibitors (cTKIs) and asciminib constitute a complex group of patients with few therapeutic options. A retrospective descriptive multicenter observational study was carried out including patients with CML who had presented a therapeutic failure to ≥ 2 cTKIs and asciminib, and had received or were not candidates to ponatinib. Of the 19 patients enrolled, 8 patients failed asciminib due to intolerance and 11 due to resistance. The most common strategy for intolerant patients was to initiate a previously administered cTKI (6/8) with dose adjustments or symptomatic management of adverse events (AEs). Of the patients who failed due to resistance, only patients who underwent progenitor transplantation achieved profound long-term responses. A frequent strategy was to use ponatinib (4/11) in patients previously considered non-candidates, with poor responses in patients in whom dose reductions were used, and severe AEs in patients at standard doses. In the remaining patients, cTKIs and other agents (interferon, hydroxyurea, citarabine, busulfan) were used with poor responses. Patients who progressed to advanced stages had a dismal prognosis. With a median follow-up of 11.2 months, overall survival of the global cohort was 73%; 100% for intolerant patients, 71% for resistant patients and 25% for those who discontinue asciminib in accelerated/blastic phase.
对于那些已经对传统酪氨酸激酶抑制剂(cTKIs)和 ASCiminib 治疗失败的慢性髓性白血病(CML)患者来说,他们构成了一个治疗选择很少的复杂患者群体。进行了一项回顾性描述性多中心观察研究,纳入了那些已经对≥2 种 cTKIs 和 ASCiminib 治疗失败且已经接受或不适合ponatinib 的 CML 患者。在纳入的 19 名患者中,有 8 名因不耐受而失败,11 名因耐药而失败。对于不耐受的患者,最常见的策略是开始使用之前给予的 cTKI(6/8),并调整剂量或对症处理不良反应(AE)。对于因耐药而失败的患者,只有接受过祖细胞移植的患者才获得了长期的深度缓解。经常使用 ponatinib(4/11)的策略适用于以前被认为不适合的患者,但剂量减少的患者反应较差,标准剂量的患者出现严重的 AE。在其余患者中,使用 cTKIs 和其他药物(干扰素、羟基脲、阿糖胞苷、白消安),反应不佳。进展到晚期的患者预后不良。中位随访 11.2 个月时,全球队列的总生存率为 73%;不耐受患者为 100%,耐药患者为 71%,在加速/原始细胞危象阶段停用 ASCiminib 的患者为 25%。