Rae Charlene, Furlong William, Feeny David, Couchman Rana, Silverman Lewis, Sallan Stephen, Laverdiere Caroline, Clavell Luis, Michon Bruno, Kelly Kara, Larsen Eric, Pullenayegum Eleanor, Athale Uma, Barr Ronald
Centre for Health Economics and Policy Analysis.
Departments of Health Research Methods, Evidence, and Impact.
J Pediatr Hematol Oncol. 2018 Nov;40(8):580-587. doi: 10.1097/MPH.0000000000001235.
Health-related quality of life (HRQL) improved progressively during therapy and beyond in children treated for acute lymphoblastic leukemia on the Dana-Farber Cancer Institute (DFCI) 95-01 protocol. This study aimed to validate that trajectory in a successor study (DFCI 00-01) and to compare the HRQL of patients in the 2 studies. Children aged above 5 years were assessed during each phase of treatment (N=4) and 2 years after completion of therapy. Health status and HRQL were measured using Health Utilities Index (HUI) instruments, HUI2 and HUI3. Quality-adjusted life years (QALYs) were calculated and compared with the general population, and patients treated on DFCI 95-01. Over 5 intervals and 758 HUI assessments, mean HRQL increased progressively from remission induction to the time after treatment (P<0.001). During intensification, high-risk patients had lower HRQL than standard-risk patients (P<0.001). During remission induction, patients on DFCI 95-01 had lower HRQL than patients on DFCI 00-01. Patients on DFCI 00-01 had ~0.2 and 0.3 fewer QALYs than controls, measured by HUI2 and HUI3, respectively. QALYs for DFCI 00-01 patients during treatment were similar to those for DFCI 95-01 patients. The trajectory of improvement in HRQL during the treatment of acute lymphoblastic leukemia in children was confirmed.
采用达纳-法伯癌症研究所（DFCI）95-01方案治疗的急性淋巴细胞白血病患儿，其与健康相关的生活质量（HRQL）在治疗期间及之后逐步改善。本研究旨在在后续研究（DFCI 00-01）中验证这一变化轨迹，并比较两项研究中患者的HRQL。对5岁以上儿童在治疗的每个阶段（N = 4）以及治疗完成后2年进行评估。使用健康效用指数（HUI）工具HUI2和HUI3测量健康状况和HRQL。计算质量调整生命年（QALYs），并与一般人群以及采用DFCI 95-01方案治疗的患者进行比较。在5个时间间隔和758次HUI评估中，平均HRQL从缓解诱导期到治疗后呈逐步上升趋势（P<0.001）。在强化治疗期间，高危患者的HRQL低于标准风险患者（P<0.001）。在缓解诱导期，采用DFCI 95-01方案治疗的患者HRQL低于采用DFCI 00-01方案治疗的患者。采用DFCI 00-01方案治疗的患者，通过HUI2和HUI3测量，其QALYs分别比对照组少约0.2和0.3。DFCI 00-01方案治疗患者在治疗期间的QALYs与DFCI 95-01方案治疗患者相似。儿童急性淋巴细胞白血病治疗期间HRQL改善的轨迹得到了证实。
