Nafees Beenish, Lloyd Andrew, Dewilde Sarah
Acaster Lloyd Consulting Ltd, London, UK.
Services in Health Economics, Brussels, Belgium.
J Patient Rep Outcomes. 2021 Jan 20;5(1):12. doi: 10.1186/s41687-020-00276-9.
Hemophagocyti.c lymphohistiocytosis (HLH) is a rare and severe disorder characterized by abnormal activation of the immune system. Primary HLH causes prolonged fever, spleen and liver enlargement, and organ dysfunction, usually in infancy and early childhood and is fatal if left untreated. As effective treatment options emerge, such as emapalumab-lzsg, Health Technology Assessment bodies around the world will assess them in terms of cost-effectiveness. This study was designed to estimate quality of life weights (utilities) for such analyses.
Vignettes were developed describing HLH treatment related health states. Health states included active HLH, HLH plus neurological symptoms, receiving chemotherapy, undergoing stem cell transplant (SCT), graft versus host disease (GVHD), cure and end of life care. The vignettes were based on information from in depth interviews with clinical specialists; and qualitative research with four parents of children with primary HLH aged between 1 and 18 years old. The vignettes were then assessed in time trade off (TTO) interviews with members of the UK general public in one on one face to face interviews with trained, experienced interviewers. Preference data were analysed using the generalised estimating equations framework.
Detailed qualitative data captured the substantial burden of this disease for young children. One hundred participants completed the TTO interviews. The utility score for Active HLH was estimated as 0.32 (95% CI, 0.24 to 0.39). Values for other states were HLH plus neurological symptoms (0.27, 95%CI 0.18-0.35), receiving chemotherapy (0.26, 95%CI 0.17-0.34), undergoing SCT (0.18, 95%CI 0.07-0.28), GVHD (0.07, 95%CI -0.04-0.17), cure (0.72, 95%CI 0.67-0.77) and end of life care (- 0.17, 95%CI -0.27- -0.07).
This study has estimated utility weights for seven different HLH related states which are based on detailed input from carers and physicians and have good face validity. There are few other options for collecting these data in an ultra-rare setting.
噬血细胞性淋巴组织细胞增生症（HLH）是一种罕见且严重的疾病，其特征为免疫系统异常激活。原发性HLH通常在婴儿期和幼儿期引发长期发热、脾肿大和肝肿大以及器官功能障碍，若不治疗则会致命。随着诸如emapalumab-lzsg等有效治疗方案的出现，世界各地的卫生技术评估机构将从成本效益方面对其进行评估。本研究旨在为此类分析估算生活质量权重（效用值）。
编制了描述HLH治疗相关健康状态的情景描述。健康状态包括活动性HLH、伴有神经系统症状的HLH、接受化疗、进行干细胞移植（SCT）、移植物抗宿主病（GVHD）、治愈和临终关怀。这些情景描述基于对临床专家的深入访谈信息，以及对4名年龄在1至18岁之间的原发性HLH患儿家长的定性研究。然后，由经过培训的经验丰富的访谈员与英国普通民众进行一对一的面对面访谈，通过时间权衡法（TTO）对情景描述进行评估。使用广义估计方程框架对偏好数据进行分析。
详细的定性数据揭示了这种疾病给幼儿带来的沉重负担。100名参与者完成了TTO访谈。活动性HLH的效用得分估计为0.32（95%置信区间，0.24至0.39）。其他状态的值分别为伴有神经系统症状的HLH（0.27，95%置信区间0.18 - 0.35）、接受化疗（0.26，95%置信区间0.17 - 0.34）、进行SCT（0.18，95%置信区间0.07 - 0.28）、GVHD（0.07，95%置信区间 - 0.04 - 0.17）、治愈（0.72，95%置信区间0.67 - 0.77）和临终关怀（ - 0.17，95%置信区间 - 0.27 - - 0.07）。
本研究估算了7种不同HLH相关状态的效用权重，这些权重基于护理人员和医生的详细意见，具有良好的表面效度。在这种极为罕见的情况下，几乎没有其他收集这些数据的方法。
