Department of Leukemia, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA.
Acta Haematol. 2023;146(6):523-530. doi: 10.1159/000533875. Epub 2023 Sep 12.
Median duration of therapy with the first JAK1/2 inhibitor ruxolitinib (RUX) approved for patients with intermediate or high-risk myelofibrosis (MF) is about 3 years.
In this retrospective study, we aimed to evaluate clinical features, predictive factors, and outcome of patients presenting to our institution who were able to remain on RUX for ≥5 years (RUX ≥5y, n = 73).
Comparing baseline demographics of patients who remained on RUX ≥5y (n = 73) with patients who were on RUX for 6 months to 3 years (n = 203), we confirmed that patients on RUX ≥5y lacked advanced clinical features at the start of therapy, such as anemia, neutropenia, thrombocytopenia, higher blasts or monocytes. Predictive independent factors for staying on RUX ≥5y were hemoglobin >10 g/dL, circulating blasts <1%, platelets >150 × 109/L, neutrophils >70%, and having primary MF. Age over 65 years remained significant for outcome in patients on RUX ≥5y.
In this retrospective study, we report on the relevance of absence of advanced clinical features for long RUX therapy and confirm the role of age on outcome despite therapy.
首个被批准用于中高危骨髓纤维化（MF）患者的 JAK1/2 抑制剂芦可替尼（RUX）的治疗中位持续时间约为 3 年。
在这项回顾性研究中，我们旨在评估能够持续接受 RUX 治疗≥5 年（RUX≥5y，n=73）的患者的临床特征、预测因素和结局。
与接受 RUX 治疗 6 个月至 3 年（n=203）的患者相比，我们确认了持续接受 RUX≥5y 治疗的患者在开始治疗时没有晚期临床特征，如贫血、中性粒细胞减少症、血小板减少症、更高的原始细胞或单核细胞。能够持续接受 RUX≥5y 治疗的独立预测因素为血红蛋白>10g/dL、循环原始细胞<1%、血小板>150×109/L、中性粒细胞>70%以及原发性 MF。对于接受 RUX≥5y 治疗的患者，年龄超过 65 岁仍然是影响结局的重要因素。
在这项回顾性研究中，我们报告了无晚期临床特征与长期 RUX 治疗的相关性，并证实了年龄对尽管接受治疗但结局的影响。
