Descriptive and prognostic value of patient-reported outcomes: The bortezomib experience in relapsed and refractory multiple myeloma
Descriptive and prognostic value of patient-reported outcomes: The bortezomib experience in relapsed and refractory multiple myeloma
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DOI：
10.1200/jco.2005.04.0824
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发表时间：
2006-02-20
影响因子：
45.3
通讯作者：
de la Loge, C
中科院分区：
文献类型：
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作者：
Dubois, D;Dhawan, R;de la Loge, C
硼替佐米是一种硼酸二肽，最近被作为治疗多发性骨髓瘤（MM）的新方法引入。这项工作的目的是评估患者报告结果（PRO）在解释硼替佐米临床试验结果中的附加价值。作为SUMMIT（蛋白酶体抑制治疗的未控制多发性骨髓瘤研究）研究的一部分，研究人员对222名复发、难治性多发性骨髓瘤患者进行硼替佐米治疗。患者在几个时间点接受以下四项PRO测量：欧洲癌症研究与治疗组织（EORTC）核心生活质量问卷（QLQ-C30）和骨髓瘤特异性模块（QLQ-MY24），慢性疾病治疗功能评估（FACIT）疲劳量表，癌症治疗功能评估(FACT)/妇科肿瘤组（GOG）神经毒性（Ntx）量表。最小重要差异（MID）阈值用于定义患者为改善、稳定或恶化。通过生存分析来评估PRO数据的预测能力。结果在总体人群中，基线和最佳终点之间存在正变化。与临床反应一致，反应组之间PRO评分的变化具有统计学意义，完全缓解（CB）或部分缓解（PR）患者PRO评分改善，轻微缓解或无变化的患者PRO评分基本稳定，进展性疾病患者PRO评分大部分恶化。神经病变相关症状的评分变化相当稳定。相比之下，CR或PR患者的疲劳评分显著改善。当应用不同的MID阈值时，在所有变化组定义的几个领域中，改善患者的比例超过35%。此外，生存分析结果表明，PRO数据可以提供额外的预后信息，以补充临床数据。结论在临床试验中，PRO评估在进一步解释临床反应、不良反应影响及患者预后方面具有补充价值。
Bortezomib, a boronic acid dipeptide, has been recently introduced as a new approach to treating multiple myeloma (MM). The goal of this work was to evaluate the added value of patient-reported outcomes (PRO) in the interpretation of bortezomib clinical trial outcomes.Patients and Methods Two hundred two patients with relapsed, refractory MM were treated with bortezomib as part of the SUMMIT (Study of Uncontrolled Multiple Myeloma Managed with Proteasome Inhibition Therapy) study. Patients were administered the following four PRO measures at several time points: the European Organisation for Research and Treatment of Cancer (EORTC) core Quality of Life Questionnaire (QLQ-C30) and the myeloma-specific module (QLQ-MY24), the Functional Assessment of Chronic Illness Therapy (FACIT) Fatigue scale, and the Functional Assessment of Cancer Therapy (FACT)/Gynecologic Oncology Group (GOG) Neurotoxicity (Ntx) scale. Minimal important difference (MID) thresholds were used to define patients as improved, stable, or worsened. A survival analysis was conducted to assess the predictive power of PRO data.Results For the total population, there was a positive change between baseline and best end point. Consistent with the clinical responses, change in PRO scores showed statistically significant differences between response groups with PRO improvement in patients with complete response (CB) or partial response (PR), mostly stable scores in patients with minor response or no change, and deterioration in most scores for patients with progressive disease. Change in scores for neuropathy-related symptoms was reasonably stable. In contrast, fatigue scores significantly improved for patients with CR or PR. When various MID thresholds were applied, the proportion of improved patients exceeded 35% for several domains within all change group definitions. Moreover, survival analysis results demonstrated the additional prognostic information PRO data can provide to supplement clinical data.Conclusion This study demonstrated the complementary value for PRO assessments in further interpreting clinical response, the impact of adverse effects, and patient prognosis in clinical trials.