Fairclough D L, Peterson H F, Cella D, Bonomi P
Center for Research Methodology and Biometry, AMC Cancer Research Center, Denver, CO 80214, USA.
Stat Med. 1998;17(5-7):781-96. doi: 10.1002/(sici)1097-0258(19980315/15)17:5/7<781::aid-sim821>3.0.co;2-o.
This paper considers five methods of analysis of longitudinal assessment of health related quality of life (QOL) in two clinical trials of cancer therapy. The primary difference in the two trials is the proportion of participants who experience disease progression or death during the period of QOL assessments. The sensitivity of estimation of parameters and hypothesis tests to the potential bias as a consequence of the assumptions of missing completely at random (MCAR), missing at random (MAR) and non-ignorable mechanisms are examined. The methods include complete case analysis (MCAR), mixed-effects models (MAR), a joint mixed-effects and survival model and a pattern-mixture model. Complete case analysis overestimated QOL in both trials. In the adjuvant breast cancer trial, with 15 per cent disease progression, estimates were consistent across the remaining four methods. In the advanced non-small-cell lung cancer trial, with 35 per cent mortality, estimates were sensitive to the missing data assumptions and methods of analysis.
本文在两项癌症治疗临床试验中，考量了纵向评估健康相关生活质量（QOL）的五种分析方法。两项试验的主要差异在于，在生活质量评估期间经历疾病进展或死亡的参与者比例。研究了参数估计和假设检验对因完全随机缺失（MCAR）、随机缺失（MAR）和不可忽略机制的假设而产生的潜在偏差的敏感性。这些方法包括完整病例分析（MCAR）、混合效应模型（MAR）、联合混合效应和生存模型以及模式混合模型。在两项试验中，完整病例分析均高估了生活质量。在辅助性乳腺癌试验中，疾病进展率为15%，其余四种方法的估计结果一致。在晚期非小细胞肺癌试验中，死亡率为35%，估计结果对缺失数据假设和分析方法敏感。
