Utility estimations of different health states of patients with type I, II, and III spinal muscular atrophy in China

A mixed approach study with patient and proxy-reported data

Bibliographic Data

ID22067044
AuthorsJiahao Hu (0000-0001-5835-1012, Shanghai Medical Information Center), Lin Zhu (0000-0001-5298-7431, Shanghai Medical Information Center), Han Bao (0009-0009-7499-4136, Sun Yat-sen University), Yuhan Liu (0000-0002-5469-5608, Shanghai Medical Information Center), Huanping Xing, Qi Kang (0000-0001-7128-6913, Shanghai Medical Information Center, corresponding author), Chunlin Jin (0000-0002-6469-7017, Shanghai Medical Information Center, corresponding author)
Year2022
Volume10
Pages1054931-1054931
Publication date2022-12-20
Peer ReviewedYes
Open AccessYes
TypeARTICLE
VenueFrontiers in Public Health (JOURNAL)
Journal identifiersISSN: 2296-2565 • E-ISSN: 2296-2565
PublisherFrontiers Media SA (PUBLISHER • CH)
DOI10.3389/fpubh.2022.1054931
PMID36605247
OpenAlexW4313447989
LanguageEN
Citations received2
References cited38

Introduction: Spinal muscular atrophy (SMA) is a rare autosomal-recessive neuromuscular disease. Health state utility values (HSUV) are used in health economic evaluation regarding the desirability of health outcomes such as a certain health state or change in health states over time. There is no utility data of Chinese patients with SMA. Materials and methods: Vignettes were developed for 10 pediatric neurologists to value the utility of Chinese patients with Type I SMA. A mixed patient/proxy derived approach using EQ-5D-Y-3L, EQ-5D-3L, and CHU9D was adopted to estimate the HSUV data of patients with Type II and III SMA, including 112 patients and 301 caregivers. Result: The utility of Type I SMA patients ranged from 0.19 to 0.72 with the health state improved from "permanent ventilation" to "walking". The utility of children patients with Type II and III SMA derived from EQ-5D-Y-3L ranged from 0.33 to 0.82 while that derived from CHU9D ranged from 0.46 to 0.75. The utility of adult patients with Type II and III SMA measured by EQ-5D-3L ranged from 0.30 to 0.83. Conclusion: The better health states the patients with SMA were in, the higher were the HSUV. The utilities derived from population with different age and disease subtypes were not statistically different when patients with SMA were in the same health states. We recommend further studies on the Chinese specific value set for EQ-5D-Y-3L and other PBMs for children to derive more robust utility data

Disease · Environmental health · Physical therapy · Population · SMA · Spinal muscular atrophy · Amyotrophic Lateral Sclerosis Research · Cerebral Palsy and Movement Disorders · Medicine · Neurogenetic and Muscular Disorders Research · Internal Medicine · Pediatrics

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