在 COMFORT-II 研究中,接受芦可替尼治疗的骨髓纤维化患者中突变状态对结局的影响。
Impact of mutational status on outcomes in myelofibrosis patients treated with ruxolitinib in the COMFORT-II study.
机构信息
Department of Experimental and Clinical Medicine, University of Florence, Florence, Italy;
出版信息
Blood. 2014 Apr 3;123(14):2157-60. doi: 10.1182/blood-2013-11-536557. Epub 2014 Jan 23.
The JAK1/JAK2 inhibitor ruxolitinib produced significant reductions in splenomegaly and symptomatic burden and improved survival in patients with myelofibrosis (MF), irrespective of their JAK2 mutation status, in 2 phase III studies against placebo (COMFORT-I) and best available therapy (COMFORT-II). We performed a comprehensive mutation analysis to evaluate the impact of 14 MF-associated mutations on clinical outcomes in 166 patients included in COMFORT-II. We found that responses in splenomegaly and symptoms, as well as the risk of developing ruxolitinib-associated anemia and thrombocytopenia, occurred at similar frequencies across different mutation profiles. Ruxolitinib improved survival independent of mutation profile and reduced the risk of death in patients harboring a set of prognostically detrimental mutations (ASXL1, EZH2, SRSF2, IDH1/2) with an hazard ratio of 0.57 (95% confidence interval: 0.30-1.08) vs best available therapy. These data indicate that clinical efficacy and survival improvement may occur across different molecular subsets of patients with MF treated with ruxolitinib.
在两项针对安慰剂(COMFORT-I)和最佳可用疗法(COMFORT-II)的 III 期研究中,JAK1/JAK2 抑制剂芦可替尼可显著减少骨髓纤维化(MF)患者的脾肿大和症状负担,并改善生存,无论其 JAK2 突变状态如何。我们进行了全面的突变分析,以评估 166 名 COMFORT-II 患者中 14 种与 MF 相关的突变对临床结局的影响。我们发现,脾肿大和症状的反应,以及发生芦可替尼相关贫血和血小板减少的风险,在不同的突变谱中出现的频率相似。芦可替尼改善了生存,与突变谱无关,并降低了携带一组预后不良突变(ASXL1、EZH2、SRSF2、IDH1/2)的患者的死亡风险,其危险比为 0.57(95%置信区间:0.30-1.08),而不是最佳可用疗法。这些数据表明,芦可替尼治疗的 MF 患者的不同分子亚群可能出现临床疗效和生存改善。