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Switch from enzyme replacement therapy to oral chaperone migalastat for treating fabry disease: real-life data.
- Source :
-
European journal of human genetics : EJHG [Eur J Hum Genet] 2020 Dec; Vol. 28 (12), pp. 1662-1668. Date of Electronic Publication: 2020 Jul 09. - Publication Year :
- 2020
-
Abstract
- The treatment options for Fabry disease (FD) are enzyme replacement therapy (ERT) with agalsidase alfa or beta, and the oral pharmacological chaperone migalastat. Since few data are available on the effects of switching from ERT to migalastat, we performed a single-center observational study on seven male Fabry patients (18-66 years) to assess the effects of the switch on renal, cardiac, and neurologic function, health status, pain, lyso-Gb3, α-Gal A activity and adverse effects. Data were retrospectively collected at time of diagnosis of FD (baseline, T0), and after 12 months of ERT (T1), and prospectively after 1 year of therapy with migalastat (T2). No patient died or reported renal, cardiac, or cerebrovascular events during the study period. The predefined measures for cardiac, renal and neurologic function, and FD-related symptoms and questionnaires were stable between baseline and the switch, and remained unchanged with migalastat. However, a significant improvement was observed in left ventricular mass index from baseline to T2 (p = 0.016), with a significative difference between the treatments (p = 0.028), and in median proteinuria from T2 vs T1 (p = 0.048). Moreover, scores of the BPI improved from baseline to T1, and remained stable with migalastat. Plasma lyso-Gb3 levels significantly decreased from baseline to T1 (P = 0.007) and T2 (P = 0.003), while did not significantly differ between the two treatments. α-Gal A activity increased from T0 to T2 (p < 0.0001). The frequency of adverse effects under migalastat and ERT was comparable (28% for both drugs). In conclusion, switching from ERT to migalastat is valid, safe and well tolerated.
- Subjects :
- 1-Deoxynojirimycin administration & dosage
1-Deoxynojirimycin adverse effects
1-Deoxynojirimycin therapeutic use
Administration, Oral
Adolescent
Adult
Aged
Drug Administration Schedule
Drug Tolerance
Enzyme Replacement Therapy
Humans
Isoenzymes administration & dosage
Isoenzymes adverse effects
Isoenzymes therapeutic use
Male
Middle Aged
Recombinant Proteins administration & dosage
Recombinant Proteins adverse effects
Recombinant Proteins therapeutic use
alpha-Galactosidase administration & dosage
alpha-Galactosidase adverse effects
alpha-Galactosidase therapeutic use
1-Deoxynojirimycin analogs & derivatives
Drug-Related Side Effects and Adverse Reactions epidemiology
Fabry Disease drug therapy
Subjects
Details
- Language :
- English
- ISSN :
- 1476-5438
- Volume :
- 28
- Issue :
- 12
- Database :
- MEDLINE
- Journal :
- European journal of human genetics : EJHG
- Publication Type :
- Academic Journal
- Accession number :
- 32647377
- Full Text :
- https://doi.org/10.1038/s41431-020-0677-x