Centre for Health Economics and Policy Analysis, and Department of Clinical Epidemiology and Biostatistics, McMaster University, Hamilton, Ontario, Canada.
Pediatr Blood Cancer. 2012 Oct;59(4):717-24. doi: 10.1002/pbc.24096. Epub 2012 Jan 31.
The objective was to quantify the health-related quality of life (HRQL) of children treated for acute lymphoblastic leukemia (ALL) and identify specific disabilities for remediation.
Two types of subjects were included: ALL patients 5 plus years old in a multi-center clinical trial and general population control groups. Patients were assessed during all four major phases of active treatment and approximately 2 years after treatment. Health status and HRQL were measured using HEALTH UTILITIES INDEX® (HUI®) Mark 2 (HUI2) and Mark 3 (HUI3). HRQL scores were used to calculate quality-adjusted life years (QALYs). Excess disability rates identified attributes for remediation.
HUI assessments (n = 749) were collected during the five phases. Mean HRQL increased from induction through the post-treatment phase (P < 0.001). There were no significant demographic or treatment effects on HRQL, except for type of asparaginase during continuation therapy (P = 0.005 for HUI2 and P = 0.007 for HUI3). Differences in mean HRQL scores between patients and controls were important (P < 0.001) during the active treatment phases but not during the post-treatment phase. Relative to controls, patients lost approximately 0.2 QALYs during active treatment. Disability was evident in mobility/ambulation, emotion, self-care and pain, and declined over time.
Patients with ALL experienced important but declining deficits in HRQL during active treatment phases: Equivalent to losing approximately 2 months of life in perfect health. HRQL within the 2-years post-treatment phase was similar to controls. The policy challenge is to develop new treatment protocols producing fewer disabilities in mobility/ambulation, emotion, self-care, and pain without compromising survival.
本研究旨在量化接受急性淋巴细胞白血病（ALL）治疗的儿童的健康相关生活质量（HRQL），并确定具体的残疾需要修复。
本研究纳入了两种类型的研究对象：多中心临床试验中 5 岁以上的 ALL 患者和一般人群对照组。患者在整个积极治疗的四个主要阶段以及治疗后约 2 年进行评估。健康状况和 HRQL 使用健康效用指数®（HUI®）标记 2 （HUI2）和标记 3 （HUI3）进行评估。HRQL 评分用于计算质量调整生命年（QALYs）。识别出多余残疾率的属性以进行修复。
共收集了五个阶段的 HUI 评估（n=749）。HRQL 从诱导期到治疗后阶段逐渐增加（P<0.001）。除了延续治疗中使用的门冬酰胺酶类型（HUI2 为 P=0.005，HUI3 为 P=0.007）外，HRQL 不受人口统计学或治疗因素的影响。在积极治疗阶段，患者与对照组之间的 HRQL 评分差异具有统计学意义（P<0.001），但在治疗后阶段无差异。与对照组相比，患者在积极治疗期间丧失了约 0.2 个 QALYs。在活动性治疗阶段，患者在移动/活动能力、情绪、自我照顾和疼痛方面存在明显残疾，且随时间推移而下降。
ALL 患者在积极治疗阶段经历了重要但逐渐减轻的 HRQL 缺陷：相当于在完美健康状态下丧失约 2 个月的生命。治疗后 2 年内的 HRQL 与对照组相似。政策挑战是制定新的治疗方案，在不影响生存的情况下减少移动/活动能力、情绪、自我照顾和疼痛方面的残疾。
