A1 Refereed original research article in a scientific journal
Clinical outcomes in patients switching from agalsidase beta to migalastat : A Fabry Registry analysis
Authors: Pisani, Antonio; Wilson, Kathryn M.; Batista, Julie L.; Kantola, Ilkka; Ortiz, Alberto; Politei, Juan; Al-Shaar, Laila; Maski, Manish; Crespo, Ana; Ponce, Elvira; Linhart, Aleš
Publisher: John Wiley & Sons
Publication year: 2024
Journal: Journal of Inherited Metabolic Disease
Journal name in source: Journal of inherited metabolic disease
Journal acronym: J Inherit Metab Dis
Volume: 47
Issue: 5
First page : 1080
Last page: 1095
ISSN: 0141-8955
eISSN: 1573-2665
DOI: https://doi.org/10.1002/jimd.12773(external)
Web address : https://doi.org/10.1002/jimd.12773(external)
Self-archived copy’s web address: https://research.utu.fi/converis/portal/detail/Publication/457130534(external)
Fabry Registry data were analyzed among 83 agalsidase beta-treated patients with Fabry disease who switched to migalastat. Outcomes (estimated glomerular filtration rate [eGFR], urine protein-creatinine ratio [UPCR], plasma globotriaosylceramide [GL-3], plasma globotriaosylsphingosine [lyso-GL-3], interventricular septal wall thickness [IVST], left posterior wall thickness [LPWT], left ventricular mass index [LVMI]) were assessed using linear mixed models to estimate annual change over time in the pre- and postswitch periods. eGFR decreased throughout both periods (preswitch: -0.85 mL/min/1.73 m2/year; postswitch: -1.96 mL/min/1.73 m2/year; both p < 0.0001), with steeper decline postswitch (ppre/post = 0.01) in both classic and late-onset patients. UPCR increased significantly postswitch (ppre/post = 0.003) among classic patients and was stable in both periods among late-onset patients. GL-3 trajectories worsened postswitch across phenotypes (ppre/post = 0.0005 classic, 0.02 late-onset). LPWT was stable preswitch (0.07 mm/year, p = 0.25) and decreased postswitch (-0.51 mm/year, p = 0.0005; ppre/post = 0.0009), primarily among late-onset patients. IVST and LVMI slopes varied significantly by phenotype. Among classic patients, IVST and LVMI were stable and decreasing, respectively preswitch and increasing postswitch (ppre/post = 0.02 IVST, 0.01 LVMI). Among late-onset patients, IVST significantly decreased postswitch (ppre/post = 0.0003); LVMI was stable over time (ppre/post = 0.89). Ultimately, eGFR and GL-3 trajectories worsened postswitch across phenotypes, while UPCR and cardiac measures worsened among classic and stabilized/improved among late-onset patients. These findings indicate variability in long-term outcomes after switching from ERT to migalastat, underscoring the importance of careful monitoring.
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Funding information in the publication:
This work was supported by Sanofi, the sponsor of the Fabry Registry, including the medical writing/editing support in the preparation of this manuscript from Evidera, and statistical programming support from Navitas Data Sciences. The authors are responsible for the content of this manuscript and the decision to submit the manuscript for publication. Furthermore, AP, JP, AO, IK, and AL confirm independence from the sponsor and relevance of the content.
