Pharmerit International, Bethesda, MD, USA.
Pharmerit International, Rotterdam, The Netherlands.
Pharmacoeconomics. 2019 Jan;37(1):85-92. doi: 10.1007/s40273-018-0704-8.
Acute myeloid leukaemia (AML) is an aggressive haematological cancer associated with significant humanistic impact. The current study assessed how the general public in the United Kingdom (UK) values AML health states.
The composite time trade-off (cTTO) methodology was employed to elicit health state utilities in AML. Pertinent AML literature related to symptom and quality-of-life impact including physical, functional and emotional well-being, as well as the safety profile of AML treatments, were taken into consideration for drafting health state descriptions. Ten health states included in the study were newly diagnosed AML, induction, consolidation, maintenance, long-term follow-up, relapsed/refractory, stem-cell transplant (SCT) procedure, SCT recovery, SCT long-term follow-up with complications and SCT long-term follow-up without complications. The descriptions were validated by haematologists and nurse specialists for clinical accuracy and completeness. A total of 210 individuals from the general UK population participated in the cTTO interviews. Descriptive statistics were computed for health state utility values.
The mean age of the participants was 44.0 years (standard deviation [SD] 14.9, range 18-81) and comprised 129 (61.4%) female participants. The utility values ranged from 0.94 (SD 0.13) for SCT long-term follow-up without complications to - 0.21 (SD 0.62) for the SCT procedure.
The study provides health utilities for a range of AML health states, with the SCT procedure health state being valued worse than death. The utilities obtained in this study can be employed as inputs in cost-effectiveness analyses of AML therapies.
急性髓系白血病（AML）是一种侵袭性血液系统癌症，对人类健康具有重大影响。本研究评估了英国公众对 AML 健康状况的重视程度。
采用复合时间权衡（cTTO）方法来评估 AML 的健康状况效用。相关 AML 文献涉及症状和生活质量影响，包括身体、功能和情绪健康，以及 AML 治疗的安全性概况，用于起草健康状况描述。本研究纳入了 10 种健康状况，包括新诊断的 AML、诱导、巩固、维持、长期随访、复发/难治性、干细胞移植（SCT）程序、SCT 恢复、SCT 长期随访伴并发症和 SCT 长期随访无并发症。这些描述经过血液学家和护士专家的验证，以确保其临床准确性和完整性。共有 210 名来自英国普通人群的参与者参与了 cTTO 访谈。计算了健康状况效用值的描述性统计数据。
参与者的平均年龄为 44.0 岁（标准差 [SD] 14.9，范围 18-81），其中 129 名（61.4%）为女性。效用值范围从 SCT 长期随访无并发症的 0.94（SD 0.13）到 SCT 程序的-0.21（SD 0.62）。
本研究提供了一系列 AML 健康状况的健康效用值，其中 SCT 程序健康状况的价值低于死亡。本研究中获得的效用值可作为 AML 治疗成本效益分析的输入。
