ArticleOrphanet journal of rare diseases2026
Limb lengthening in achondroplasia: a cost-effectiveness analysis using a Markov model.
Article in Orphanet journal of rare diseases, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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Abstract
backgroundDisproportionate short stature is one of the most characteristic clinical traits of achondroplasia, a condition with a significant impact on patients' autonomy that results in a series of limitations leading to poorer health-related quality of life. Limb lengthening surgery is an effective therapeutic alternative for increasing the stature of persons with achondroplasia. The purpose of this study was to evaluate the cost-effectiveness of bone lengthening in patients with achondroplasia and to estimate the economic impact of the procedure on the Spanish health system by means of a Markov model.
resultsPatients with achondroplasia who undergo a limb lengthening procedure gain, on average, 5.954 quality-adjusted life years (QALYs) more than unoperated patients (27.197 vs. 21.243 QALYs). As the procedure entails a mean incremental cost of €49,480, the resulting incremental cost-effectiveness ratio (ICER) is €8,310/QALY. The probabilistic sensitivity analysis (PSA) performed yielded a mean quality of life gain of 6.287 QALYs and a mean cost increase of €49,131, which translates into an ICER of €7,814/QALY. The budget impact analysis was based on the hypothesis that all patients with achondroplasia born every year in the country used as a reference (Spain, n = 15) undergo the procedure. Under this assumption, a cumulative cost increase of €8,896,196 can be estimated over a 15-year period (€8,836,931 following the PSA).
conclusionsLimb lengthening surgery in patients with achondroplasia can be considered a cost-effective intervention according to the most commonly accepted willingness-to-pay thresholds in Spain and other Western countries.
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