Real-world treatment with elosulfase alfa in patients with MPS IVA is associated with improved endurance over time
Purpose: To assess the real-world effectiveness of enzyme replacement therapy (ERT; elosulfase alfa) on endurance in the treatment of mucopolysaccharidosis type IVA (MPS IVA) using cross-sectional data. Methods: The 6-minute walk test (6MWT) distances of ERT-treated and untreated participants from t...
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Elsevier
2025-01-01
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| Series: | Genetics in Medicine Open |
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| Online Access: | http://www.sciencedirect.com/science/article/pii/S2949774425014670 |
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| author | Barbara K. Burton Karolina M. Stepien Philippe M. Campeau Jaim Sutton Abigail Hunt Pascal Reisewitz David Hinds |
| author_facet | Barbara K. Burton Karolina M. Stepien Philippe M. Campeau Jaim Sutton Abigail Hunt Pascal Reisewitz David Hinds |
| author_sort | Barbara K. Burton |
| collection | DOAJ |
| description | Purpose: To assess the real-world effectiveness of enzyme replacement therapy (ERT; elosulfase alfa) on endurance in the treatment of mucopolysaccharidosis type IVA (MPS IVA) using cross-sectional data. Methods: The 6-minute walk test (6MWT) distances of ERT-treated and untreated participants from the Morquio A Registry Study and Morquio A Clinical Assessment Program were described for age groups of interest (5 to <7 years, 9 to <11 years, 14 to <16 years, and 20 to <30 years). Linear and quantile univariate regression were performed to explore variables associated with 6MWT (ERT, sex, age, standing height, body weight, region, and race). Multivariate regression analyses were performed using covariates identified in univariate analyses (P < .10), adjusting for confounders. Results: A total of 471 participants were included; baseline characteristics were similar within age groups. Median 6MWT distances were numerically greater in ERT-treated versus untreated participants within each age group. Quantile regression adjusting for multiple factors indicated a consistent trend of improved 6MWT with ERT treatment. Standing height was also associated with longer 6MWT in the multivariate quantile analysis, except in participants aged 5 to <7 years. Conclusion: This analysis assessed associations between ERT exposure and endurance to confirm the real-world, long-term effectiveness of elosulfase alfa in participants with MPS IVA. |
| format | Article |
| id | doaj-art-5c1b9a131cf84bbb8a27601aade3f998 |
| institution | Kabale University |
| issn | 2949-7744 |
| language | English |
| publishDate | 2025-01-01 |
| publisher | Elsevier |
| record_format | Article |
| series | Genetics in Medicine Open |
| spelling | doaj-art-5c1b9a131cf84bbb8a27601aade3f9982025-08-20T03:30:41ZengElsevierGenetics in Medicine Open2949-77442025-01-01310342810.1016/j.gimo.2025.103428Real-world treatment with elosulfase alfa in patients with MPS IVA is associated with improved endurance over timeBarbara K. Burton0Karolina M. Stepien1Philippe M. Campeau2Jaim Sutton3Abigail Hunt4Pascal Reisewitz5David Hinds6Ann and Robert H. Lurie Children’s Hospital of Chicago, Chicago, IL; Correspondence and requests for materials should be addressed to Barbara K. Burton, Ann and Robert H. Lurie Children's Hospital of Chicago, 225 E Chicago Ave, Chicago, IL 60611.Northern Care Alliance NHS Foundation Trust, Salford, United KingdomCHU Sainte-Justine Research Center, Montreal, QC, CanadaBioMarin (UK) Limited, London, United KingdomBioMarin Pharmaceutical Inc, Novato, CABioMarin Pharmaceutical Inc, Novato, CABioMarin Pharmaceutical Inc, Novato, CAPurpose: To assess the real-world effectiveness of enzyme replacement therapy (ERT; elosulfase alfa) on endurance in the treatment of mucopolysaccharidosis type IVA (MPS IVA) using cross-sectional data. Methods: The 6-minute walk test (6MWT) distances of ERT-treated and untreated participants from the Morquio A Registry Study and Morquio A Clinical Assessment Program were described for age groups of interest (5 to <7 years, 9 to <11 years, 14 to <16 years, and 20 to <30 years). Linear and quantile univariate regression were performed to explore variables associated with 6MWT (ERT, sex, age, standing height, body weight, region, and race). Multivariate regression analyses were performed using covariates identified in univariate analyses (P < .10), adjusting for confounders. Results: A total of 471 participants were included; baseline characteristics were similar within age groups. Median 6MWT distances were numerically greater in ERT-treated versus untreated participants within each age group. Quantile regression adjusting for multiple factors indicated a consistent trend of improved 6MWT with ERT treatment. Standing height was also associated with longer 6MWT in the multivariate quantile analysis, except in participants aged 5 to <7 years. Conclusion: This analysis assessed associations between ERT exposure and endurance to confirm the real-world, long-term effectiveness of elosulfase alfa in participants with MPS IVA.http://www.sciencedirect.com/science/article/pii/S29497744250146706-minute walk testCross-sectionalMucopolysaccharidosis IVAMultivariate analysisRegistry |
| spellingShingle | Barbara K. Burton Karolina M. Stepien Philippe M. Campeau Jaim Sutton Abigail Hunt Pascal Reisewitz David Hinds Real-world treatment with elosulfase alfa in patients with MPS IVA is associated with improved endurance over time Genetics in Medicine Open 6-minute walk test Cross-sectional Mucopolysaccharidosis IVA Multivariate analysis Registry |
| title | Real-world treatment with elosulfase alfa in patients with MPS IVA is associated with improved endurance over time |
| title_full | Real-world treatment with elosulfase alfa in patients with MPS IVA is associated with improved endurance over time |
| title_fullStr | Real-world treatment with elosulfase alfa in patients with MPS IVA is associated with improved endurance over time |
| title_full_unstemmed | Real-world treatment with elosulfase alfa in patients with MPS IVA is associated with improved endurance over time |
| title_short | Real-world treatment with elosulfase alfa in patients with MPS IVA is associated with improved endurance over time |
| title_sort | real world treatment with elosulfase alfa in patients with mps iva is associated with improved endurance over time |
| topic | 6-minute walk test Cross-sectional Mucopolysaccharidosis IVA Multivariate analysis Registry |
| url | http://www.sciencedirect.com/science/article/pii/S2949774425014670 |
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