ArticleJournal of patient-reported outcomes2021
Estimating health state utilities in hemophagocytic lymphohistiocytosis.
Article in Journal of patient-reported outcomes, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
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Who cites it
3 citing papers in PubMed, 7 citations in OpenAlex.
- Health state utilities associated with invasive pneumococcal disease, pneumonia, and recurrent acute otitis media in young children.Quality of life research : an international journal of quality of life aspects of treatment, care and rehabilitation · 2025Article
- A Vignette Study to Derive Health-Related Quality-of-Life Weights for Individuals with Steroid Refractory Chronic Graft-versus-Host Disease Receiving Third-Line Therapy in the United Kingdom.Journal of health economics and outcomes research · 2025Article
- A qualitative investigation into the impact of hemophagocytic lymphohistiocytosis on children and their caregivers.Orphanet journal of rare diseases · 2021Article
Corrections and comments
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Authors and funding
3 authors at 2 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundHemophagocyti.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.
methodsVignettes 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.
resultsDetailed 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).
conclusionsThis 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.
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