Effect of Ruxolitinib Therapy on Myelofibrosis-Related Symptoms and Other Patient-Reported Outcomes in COMFORT-I: A Randomized, Double-Blind, Placebo-Controlled Trial
Effect of Ruxolitinib Therapy on Myelofibrosis-Related Symptoms and Other Patient-Reported Outcomes in COMFORT-I: A Randomized, Double-Blind, Placebo-Controlled Trial
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DOI：
10.1200/jco.2012.44.4489
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发表时间：
2013-04-01
影响因子：
45.3
通讯作者：
Verstovsek, Srdan
中科院分区：
文献类型：
--
作者：
Mesa, Ruben A.;Gotlib, Jason;Verstovsek, Srdan
目的评价鲁索利替尼对骨髓纤维化患者症状负担和生活质量（QoL）的影响，并评价修改后的骨髓纤维化症状评估表（MFSAF） v2.0对骨髓纤维化患者骨髓纤维化相关症状的有意义改变的能力。患者和方法comfort - i（口服JAK抑制剂治疗的对照骨髓纤维化研究）是一项双盲、安慰剂对照的III期研究，评估鲁索利替尼对中-2或高危骨髓纤维化患者的治疗效果。对以下患者报告的结果（PROs）评估进行探索性分析：改进的MFSAF v2.0（个体症状和总症状评分[TSS]）、欧洲癌症研究与治疗组织生活质量问卷-核心30 （EORTC QLQ-C30）、患者报告的结果测量信息系统（PROMIS）疲劳量表和患者总体变化印象（PGIC）。结果接受ruxolitinib治疗的患者个体骨髓纤维化相关症状得到改善，而接受安慰剂治疗的患者出现恶化（P < 0.001）。大多数（91%）ruxolitinib治疗的患者被指定为>= 50% TSS应答者（>= 50% TSS改善），他们在PGIC上自我报告他们的病情“大大改善”或“非常改善”。与接受安慰剂的患者相比，这些患者在EORTC QLQ-C30功能域和全球健康状况/生活质量方面取得了显着改善，在这些测量中经历恶化（P = 35%）的患者在这些PROs方面得到了最大的改善。结论鲁索利替尼治疗的患者在骨髓纤维化相关症状和生活质量方面取得了临床意义上的改善，但安慰剂治疗的患者出现了症状和其他不良反应的恶化。[J]中华临床杂志，31(1):1285-1292。(C)美国临床肿瘤学会2013
PurposeTo assess the effects of ruxolitinib on symptom burden and quality of life (QoL) and to evaluate the ability of the modified Myelofibrosis Symptom Assessment Form (MFSAF) v2.0 to measure meaningful changes in myelofibrosis-related symptoms in patients with myelofibrosis.Patients and MethodsCOMFORT-I (Controlled Myelofibrosis Study With Oral JAK Inhibitor Treatment-I) is a double-blind, placebo-controlled phase III study evaluating ruxolitinib in patients with intermediate-2 or high-risk myelofibrosis. Exploratory analyses were conducted on the following patient-reported outcomes (PROs) assessments: modified MFSAF v2.0 (individual symptoms and Total Symptom Score [TSS]), European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire-Core 30 (EORTC QLQ-C30), Patient Reported Outcomes Measurement Information System (PROMIS) Fatigue Scale, and Patient Global Impression of Change (PGIC).ResultsPatients receiving ruxolitinib experienced improvements in individual myelofibrosis-related symptoms, although patients receiving placebo experienced worsening (P < .001). The majority (91%) of ruxolitinib-treated patients designated as >= 50% TSS responders (>= 50% TSS improvement) self-reported their condition as either "Much improved" or "Very much improved" on the PGIC. These patients achieved significant improvements in the EORTC QLQ-C30 functional domains and Global Health Status/QoL versus patients receiving placebo, who experienced worsening on these measures (P = 35% reduction in spleen volume experienced the greatest improvements in these PROs.ConclusionRuxolitinib-treated patients achieved clinically meaningful improvements in myelofibrosis-related symptoms and QoL, but patients receiving placebo reported worsening of symptoms and other PROs. J Clin Oncol 31:1285-1292. (C) 2013 by American Society of Clinical Oncology