Guy's and St. Thomas' NHS Foundation Trust, Guy's Hospital, London, UK.
Br J Haematol. 2013 Jul;162(2):229-39. doi: 10.1111/bjh.12375. Epub 2013 May 14.
Patients with myelofibrosis (MF) have significant debilitating symptoms, physical disabilities, and poor health-related quality of life (HRQoL). Here, we report post-hoc analyses of the impact of ruxolitinib, a potent and selective JAK1 and JAK2 inhibitor, on disease-related symptoms and HRQoL in MF patients from the large phase 3 COMFORT-II study (N = 219). During the follow-up period of 48 weeks, HRQoL and MF-associated symptoms improved from baseline for ruxolitinib-treated patients but remained the same or worsened for best available therapy (BAT)-treated patients. Based on the European Organization for Research and Treatment of Cancer QoL Questionnaire core 30 items (EORTC QLQ-C30), treatment-induced differences in physical and role functioning, fatigue, and appetite loss significantly favoured ruxolitinib versus BAT from week 8 (P < 0·05) up to week 48 (P < 0·05). Ruxolitinib resulted in significantly higher response rates in global health status/QoL and Functional Assessment of Cancer Therapy-Lymphoma (FACT-Lym) summary scores versus BAT at most time points (P < 0·05). Significant improvements in the Lymphoma subscale (including symptoms of pain, fever, itching, fatigue, weight loss, loss of appetite, and other patient concerns), FACT-General, FACT-Lym trial outcome index, and FACT-Lym total were also observed with ruxolitinib versus BAT starting at week 8 and continuing thereafter. Overall, these data demonstrated that ruxolitinib improved HRQoL in MF patients and further support the use of ruxolitinib for the treatment of symptomatic MF.
骨髓纤维化(MF)患者存在明显的使人虚弱的症状、身体残疾和较差的健康相关生活质量(HRQoL)。在此,我们报告了来自大型 3 期 COMFORT-II 研究(N=219)的 MF 患者中,强效和选择性 JAK1 和 JAK2 抑制剂芦可替尼对疾病相关症状和 HRQoL 的影响的事后分析。在 48 周的随访期间,芦可替尼治疗患者的 HRQoL 和 MF 相关症状自基线开始改善,但最佳可用疗法(BAT)治疗患者的 HRQoL 和 MF 相关症状保持不变或恶化。根据欧洲癌症研究和治疗组织生活质量问卷核心 30 项(EORTC QLQ-C30),治疗引起的身体和角色功能、疲劳和食欲丧失方面的差异从第 8 周(P<0.05)开始直至第 48 周(P<0.05)时显著有利于芦可替尼而非 BAT。与 BAT 相比,在大多数时间点,芦可替尼在整体健康状况/生活质量和癌症治疗功能评估-淋巴瘤(FACT-Lym)总评分方面的反应率显著更高(P<0.05)。从第 8 周开始,芦可替尼还显著改善了淋巴瘤子量表(包括疼痛、发热、瘙痒、疲劳、体重减轻、食欲不振和其他患者关注的症状)、FACT 一般状况、FACT-Lym 试验结局指数和 FACT-Lym 总分,并且持续存在。总体而言,这些数据表明芦可替尼改善了 MF 患者的 HRQoL,并进一步支持将芦可替尼用于治疗有症状的 MF。