Translational Health Economics Network (THEN), 39 quai de Valmy, 75010, Paris, France.
Infection Antimicrobials Modeling & Evolution (IAME), UMR 1137, Institut National de la Santé et de la Recherche Médicale (INSERM), Université Paris Diderot, Sorbonne Paris Cité, Paris, France.
Health Qual Life Outcomes. 2019 Jul 25;17(1):129. doi: 10.1186/s12955-019-1195-9.
Health state utility (HSU) is a core component of QALYs and cost-effectiveness analysis, although HSU is rarely estimated among a representative sample of patients. We explored the feasibility of assessing HSU in head and neck cancer from the French National Hospital Discharge database.
An exhaustive sample of 53,258 incident adult patients with a first diagnosis of head and neck cancer was identified in 2010-2012. We used a cross-sectional approach to define five health states over two periods: three "cancer stages at initial treatment" (early, locally advanced or metastatic stage); a "relapse state" and otherwise a "relapse-free state" in the follow-up of patients initially treated at early or locally advanced stage. In patients admitted in post-acute care, a two-parameter graded response model (Item Response Theory) was estimated from all 144,012 records of six Activities of Daily Living (ADLs) and the latent health state scale underlying ADLs was calibrated with the French EQ-5D-3 L social value set. Following linear interpolation between all assessments of the patient, daily estimates of utility in post-acute care were averaged by health state, patient and month of follow-up. Finally, HSU was estimated by health state and month of follow-up for the whole patient population after controlling for survivorship and selection in post-acute care.
Head and neck cancer was generally associated with poor HSU estimates in a real-life setting. As compared to "distant metastasis at initial treatment", mean HSU was higher in other health states, although numerical differences were small (0.45 versus around 0.54). It was primarily explained by the negative effects on HSU of an older age (38.4% aged ≥70 years in "early stage at initial treatment") and comorbidities (> 50% in other health states). HSU estimates significantly improved over time in the "relapse-free state" (from 8 to 12 months of follow-up).
HSU estimates in head and neck cancer were primarily driven by age at diagnosis, comorbidities, and time to assessment of cancer survivors. This feasibility study highlights the potential of estimating HSU within and across severe conditions in a systematic way at the national level.
健康状态效用(HSU)是 QALY 和成本效益分析的核心组成部分,尽管在具有代表性的患者样本中很少估计 HSU。我们探讨了在法国国家住院数据库中评估头颈部癌症 HSU 的可行性。
在 2010 年至 2012 年期间,我们从法国国家住院数据库中确定了一个详尽的 53258 例首次诊断为头颈部癌症的成年患者的样本。我们使用横断面方法在两个时期内定义了五个健康状态:三个“初始治疗时的癌症阶段”(早期、局部晚期或转移性阶段);一个“复发状态”和否则为“随访时无复发状态”,对于在早期或局部晚期阶段接受治疗的患者。在接受急性后护理的患者中,从所有 144012 个日常生活活动(ADL)记录中估计了一个两参数分级反应模型(项目反应理论),并使用法国 EQ-5D-3L 社会价值集对 ADL 下的潜在健康状态量表进行了校准。在对患者进行所有评估的线性插值后,按健康状态、患者和随访月平均急性后护理的效用日估计值。最后,通过在急性后护理中控制生存和选择,对头颈部癌症患者整个人群的健康状态和随访月进行 HSU 估计。
在现实环境中,头颈部癌症通常与较差的 HSU 估计相关。与“初始治疗时的远处转移”相比,其他健康状态的 HSU 较高,尽管数值差异较小(0.45 与 0.54 左右)。这主要归因于年龄(“初始治疗时的早期阶段”中有 38.4%的患者年龄≥70 岁)和合并症(其他健康状态中超过 50%)对 HSU 的负面影响。在“无复发状态”中,HSU 估计值随着时间的推移显著提高(从随访的 8 个月到 12 个月)。
头颈部癌症的 HSU 估计主要由诊断时的年龄、合并症和癌症幸存者评估时间驱动。这项可行性研究强调了在系统层面上在全国范围内对严重疾病内和跨疾病进行 HSU 估计的潜力。