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Health State Utilities for Sickle Cell Disease: A Catalog Prepared From a Systematic Review

Published:September 04, 2021DOI:https://doi.org/10.1016/j.jval.2021.08.002

      Highlights

      • Although the emerging curative therapies bring hope to patients with sickle cell disease (SCD), concerns about their high costs have been raised. Cost-effectiveness analysis can be used to align the costs of the curative therapies with their expected health benefits.
      • Our study is the first to comprehensively summarize and catalog health state utilities (HSUs) for SCD and its specific complications and HSUs tied to its treatments, which can inform future cost-effectiveness analyses in SCD.
      • We identify the research gaps, such as the need for more empirical studies to elicit HSUs associated with the SCD and its treatment complications from patients and HSUs from their caregivers.

      Abstract

      Objectives

      Sickle cell disease (SCD) is a complex, chronic condition that impairs health-related quality of life of affected individuals and their caregivers. As curative therapies emerge, comprehensive cost-effectiveness models will inform their value. These models will require descriptions of health states and their corresponding utility values that accurately reflect health-related quality of life over the disease trajectory. The objectives of this systematic review were to develop a catalog of health state utility (HSU) values for SCD, identify research gaps, and provide future directions for preference elicitation.

      Methods

      Records were identified through searches of PubMed and Embase, Tufts Medical Center Cost-Effectiveness Analysis Registry, reference lists of relevant articles, and consultation with SCD experts (2008-2020). We removed duplicate records and excluded ineligible studies. For included studies, we summarized the study characteristics, methods used for eliciting HSUs, and HSU values.

      Results

      Five studies empirically elicited utilities using indirect methods (EQ-5D) (n = 3) and Short Form-6 Dimension (n = 2); these represent health states associated with general SCD (n = 1), SCD complications (n = 2), and SCD treatments (n = 3). Additionally, we extracted HSUs from 7 quality-adjusted life-years-based outcome research studies. The HSU among patients with general SCD without specifying complications ranged from 0.64 to 0.887. Only 36% of the HSUs used in the quality-adjusted life-year-based outcomes research studies were derived from individuals with SCD. No study estimated HSUs in caregivers.

      Conclusions

      There is a dearth of literature of HSUs for use in SCD models. Future empirical studies should elicit a comprehensive set of HSUs from individuals with SCD and their caregivers.

      Keywords

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      References

      1. Sickle cell disease. National Heart, Lung, and Blood Institute.
        • Tanabe P.
        • Spratling R.
        • Smith D.
        • Grissom P.
        • Hulihan M.C.E.
        understanding the complications of sickle cell disease.
        Am J Nurs. 2019; 119: 26-35
        • Chaturvedi S.
        • DeBaun M.R.
        Evolution of sickle cell disease from a life-threatening disease of children to a chronic disease of adults: the last 40 years.
        Am J Hematol. 2016; 91: 5-14
        • Panepinto J.A.
        • Bonner M.
        Health-related quality of life in sickle cell disease: past, present, and future.
        Pediatr Blood Cancer. 2012; 59: 377-385
        • Esham K.S.
        • Rodday A.M.
        • Smith H.P.
        • et al.
        Assessment of health-related quality of life among adults hospitalized with sickle cell disease vaso-occlusive crisis.
        Blood Adv. 2020; 4: 19-27
        • Fiuza-Luces C.
        • Simpson R.J.
        • Ramírez M.
        • Lucia A.
        • Berger N.A.
        Physical function and quality of life in patients with chronic graft-versus-host-disease: a summary of preclinical and clinical studies and a call for exercise intervention trials in patients.
        Bone Marrow Transplant. 2016; 51: 13-26
        • Webb E.J.D.
        • O’Dwyer J.
        • Meads D.
        • Kind P.
        • Wright P.
        Transforming discrete choice experiment latent scale values for EQ-5D-3L using the visual analogue scale.
        Eur J Health Econ HEPAC Health Econ Prev Care. 2020; 21: 787-800
        • Marra C.A.
        • Woolcott J.C.
        • Kopec J.A.
        • et al.
        A comparison of generic, indirect utility measures (the HUI2, HUI3, SF-6D, and the EQ-5D) and disease-specific instruments (the RAQoL and the HAQ) in rheumatoid arthritis.
        Soc Sci Med. 2005; 60: 1571-1582
      2. Cure sickle cell initiative. National Heart, Lung, and Blood Institute.
        • Ikawa Y.
        • Miccio A.
        • Magrin E.
        • Kwiatkowski J.L.
        • Rivella S.
        • Cavazzana M.
        Gene therapy of hemoglobinopathies: progress and future challenges.
        Hum Mol Genet. 2019; 28: R24-R30
        • Harrison C.
        First gene therapy for β-thalassemia approved.
        Nat Biotechnol. 2019; 37: 1102-1103
        • Towse A.
        • Fenwick E.
        Uncertainty and cures: discontinuation, irreversibility, and outcomes-based payments: what is different about a one-off treatment?.
        Value Health. 2019; 22: 677-683
        • Higgins J.P.T.
        • Thomas J.
        • Chandler J.
        • et al.
        Cochrane handbook for systematic reviews of interventions version 6.0 (updated July 2019). Cochrane.
        www.training.cochrane.org/handbook
        Date accessed: July 1, 2019
        • Trikalinos T.A.
        • Dahabreh I.J.
        • Wallace B.C.
        • Schmid C.H.
        • Lau J.
        Towards a framework for communicating confidence in methodological recommendations for systematic reviews and meta-analyses. Agency for Healthcare Research and Quality.
        • Moher D.
        • Liberati A.
        • Tetzlaff J.
        • Altman D.G.
        • PRISMA Group
        Preferred Reporting Items for Systematic Reviews and Meta-Analyses: the PRISMA statement.
        PLoS Med. 2009; 6e1000097
        • Ara R.
        • Brazier J.
        • Peasgood T.
        • Paisley S.
        The identification, review and synthesis of health state utility values from the literature.
        Pharmacoeconomics. 2017; 35: 43-55
        • Brazier J.
        • Ara R.
        • Azzabi I.
        • et al.
        Identification review, and use of health state utilities in cost-effectiveness models: An ISPOR Good Practices for Outcomes Research Task Force Report.
        Value Health. 2019; 22: 267-275
        • Anie K.A.
        • Grocott H.
        • White L.
        • Dzingina M.
        • Rogers G.
        • Cho G.
        Patient self-assessment of hospital pain, mood and health-related quality of life in adults with sickle cell disease.
        BMJ Open. 2012; 2e001274
        • Arnold S.D.
        • Jin Z.
        • Sands S.
        • Bhatia M.
        • Kung A.L.
        • Satwani P.
        Allogeneic hematopoietic cell transplantation for children with sickle cell disease is beneficial and cost-effective: a single-center analysis.
        Biol Blood Marrow Transplant. 2015; 21: 1258-1265
        • Ojelabi A.O.
        • Bamgboye A.E.
        • Ling J.
        Preference-based measure of health-related quality of life and its determinants in sickle cell disease in Nigeria.
        PLoS One. 2019; 14e0223043
        • Payne K.A.
        • Rofail D.
        • Baladi J.-F.
        • et al.
        Iron chelation therapy: clinical effectiveness, economic burden and quality of life in patients with iron overload.
        Adv Ther. 2008; 25: 725-742
        • Spackman E.
        • Sculpher M.
        • Howard J.
        • et al.
        Cost-effectiveness analysis of preoperative transfusion in patients with sickle cell disease using evidence from the TAPS trial.
        Eur J Haematol. 2014; 92: 249-255
        • Bradt P.
        • Spackman E.
        • Synnott P.
        • et al.
        Crizanlizumab, Voxelotor, and L-glutamine for sickle cell disease: effectiveness and value. Institute for Clinical and Economic review.
        • Cherry M.G.
        • Greenhalgh J.
        • Osipenko L.
        • et al.
        The clinical effectiveness and cost-effectiveness of primary stroke prevention in children with sickle cell disease: a systematic review and economic evaluation.
        Health Technol Assess. 2012; 16: 1-129
        • McLeod C.
        • Fleeman N.
        • Kirkham J.
        • et al.
        Deferasirox for the treatment of iron overload associated with regular blood transfusions (transfusional haemosiderosis) in patients suffering with chronic anaemia: a systematic review and economic evaluation.
        Health Technol Assess. 2009; 13 (iii-121)
        • O’Brien S.H.
        • Hankins J.S.
        Decision analysis of treatment strategies in children with severe sickle cell disease.
        J Pediatr Hematol Oncol. 2009; 31: 873-878
        • Lubeck D.
        • Agodoa I.
        • Bhakta N.
        • et al.
        Estimated life expectancy and income of patients with sickle cell disease compared with those without sickle cell disease.
        JAMA Netw Open. 2019; 2e1915374
        • Serjeant G.R.
        The natural history of sickle cell disease.
        Cold Spring Harb Perspect Med. 2013; 3a011783
      3. Complications and treatments of sickle cell disease. Centers for Disease Control and Prevention.
        • Sanders G.D.
        • Neumann P.J.
        • Basu A.
        • et al.
        Recommendations for conduct, methodological practices, and reporting of cost-effectiveness analyses: second panel on cost-effectiveness in health and medicine [published correction appears in JAMA. 2016;316(18):1924].
        JAMA. 2016; 316: 1093-1103
        • Madani B.M.
        • Al Raddadi R.
        • Al Jaouni S.
        • Omer M.
        • Al Awa M.-I.
        Quality of life among caregivers of sickle cell disease patients: a cross sectional study.
        Health Qual Life Outcomes. 2018; 16: 176
        • van den Tweel X.W.
        • Hatzmann J.
        • Ensink E.
        • et al.
        Quality of life of female caregivers of children with sickle cell disease: a survey.
        Haematologica. 2008; 93: 588-593
        • Farrell A.T.
        • Panepinto J.
        • Carroll C.P.
        • et al.
        End points for sickle cell disease clinical trials: patient-reported outcomes, pain, and the brain.
        Blood Adv. 2019; 3: 3982-4001
        • McClish D.K.
        • Smith W.R.
        • Levenson J.L.
        • et al.
        Comorbidity, Pain, Utilization, and Psychosocial Outcomes in Older versus Younger sickle cell adults: the PiSCES Project.
        BioMed Res Int. 2017; 20174070547
        • Dampier C.
        • LeBeau P.
        • Rhee S.
        • et al.
        Health-related quality of life in adults with sickle cell disease (SCD): a report from the comprehensive sickle cell centers clinical trial consortium.
        Am J Hematol. 2011; 86: 203-205
        • Treadwell M.J.
        • Hassell K.
        • Levine R.
        • Keller S.
        Adult sickle cell quality-of-life measurement information system (ASCQ-me).
        Clin J Pain. 2014; 30: 902-914