[Alglucosidase alfa (Myozyme ®) in late-onset Pompe disease]
Agencia de Evaluación de Tecnologías Sanitarias de Andalucía
Record ID 32014001015
Authors' objectives: The main objective of the study was to evaluate the efficacy, safety and efficiency of alglucosidase alfa in patients with late-onset Pompe disease.
Authors' recomendations: There is no evidence to suggest that alglucosidase alfa treatment increases survival of patients with late-onset Pompe's disease. Treatment with alglucosidase alfa does not increase quality of life (assessed by the SF-36 questionnaire) of patients with late-onset Pompe's disease. However, improved functional capacity, measured by the distance walked in the 6MWT (from 25m to 148m, according to published studies). Overall, this is a very modest improvement. Although it is statistically significant, its clinical relevance is unclear. However, results are very heterogeneous among patients, showing different degrees of treatment response. Respiratory function did not improve in a clinical relevant way but was stabilised. Regarding muscle strength, no significant improvement was achieved. Patients with better outcomes were those with mild to moderate disease. In patients where clinically relevant improvements occur, the greater progress happened in the first 26 weeks. Then the benefit was usually maintained during the study. However, there was no literature to suggest that the benefits remain longer term. Alglucosidase alfa treatment was generally well tolerated, although there was no evidence of long-term safety. The budget impact for the Andalusian Public Health System to treat with alglucosidase alfa 4 patients with late-onset Pompe's disease was estimated at 0.27 % of the budget in hospital pharmacy.
Project Status: Completed
Year Published: 2014
URL for published report: https://www.aetsa.org/publicacion/alglucosidasa-alfa-myozyme-en-enfermedad-de-pompe-de-comienzo-tardio/
English language abstract: An English language summary is available
Publication Type: Not Assigned
- Glycogen Storage Disease Type II
- Enzyme Replacement Therapy
Organisation Name: Andalusian Health Technology Assessment Area
Contact Address: Area de Evaluacion de Tecnologias Sanitarias Sanitarias de Andalucia (AETSA) Avda. Innovación, s/n Edificio Arena 1. Sevilla (Spain) Tel. +34 955 006 309
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Copyright: Andalusian Agency for Health Technology Assessment (AETSA)
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