Sarah M Medland, Jamie Bainbridge, Matthew Cawson, Stuart J Mealing, Anna Yudina, Isabel Eastwood, Arne de Kreuk, Martin Besser, Eva Tsouana
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引用次数: 0
Abstract
Background/objective: Sickle cell disease (SCD) is a group of inherited health conditions affecting 7.74 million people worldwide. Regular automated red blood cell exchange (aRBCX) transfusions have been shown to improve control and management of SCD compared with manual RBCX (mRBCX). The aim of this study was to estimate the lifetime clinical and economic impact of aRBCX versus mRBCX in two United Kingdom-based populations with SCD (paediatrics initiated aged 5 years and adults initiated aged 38 years) that were clinically indicated for chronic disease-modifying transfusions (DMTs).
Methods: An individual patient-level simulation model was developed to estimate lifetime quality-adjusted life years (QALYs) and healthcare costs. DMT administration programmes aligned with recommended treatment schedules. Monte Carlo methods determined baseline characteristics and clinical event occurrence. Pragmatic review findings and expert opinion informed model parameters and assumptions. Second-order probabilistic sensitivity analysis (PSA) was performed for 1000 individuals' lifetimes over 500 iterations.
Results: Per individual, aRBCX reduced acute clinical events by 19% in both populations versus mRBCX. The time spent receiving chelation therapy reduced by 63 and 32 months for paediatric-initiated and adult-initiated individuals, respectively. Total lifetime DMT costs were reduced by £71,217 and £30,740 for paediatric-initiated and adult-initiated individuals, respectively. Overall, aRBCX increased QALYs and reduced costs by 0.29 and £112,811 in paediatric-initiated individuals and 0.24 and £61,895 in adult-initiated individuals. aRBCX was cost-effective in 100% of PSA iterations for both populations.
Conclusion: aRBCX shows potential to improve health outcomes and reduce healthcare costs for individuals with SCD initiating a chronic DMT programme.
期刊介绍：
PharmacoEconomics - Open focuses on applied research on the economic implications and health outcomes associated with drugs, devices and other healthcare interventions. The journal includes, but is not limited to, the following research areas:Economic analysis of healthcare interventionsHealth outcomes researchCost-of-illness studiesQuality-of-life studiesAdditional digital features (including animated abstracts, video abstracts, slide decks, audio slides, instructional videos, infographics, podcasts and animations) can be published with articles; these are designed to increase the visibility, readership and educational value of the journal’s content. In addition, articles published in PharmacoEconomics -Open may be accompanied by plain language summaries to assist readers who have some knowledge of, but not in-depth expertise in, the area to understand important medical advances.All manuscripts are subject to peer review by international experts. Letters to the Editor are welcomed and will be considered for publication.
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