Agalsidase therapy in patients with Fabry disease on renal replacement therapy: a nationwide study in Italy
- PMID: 18057066
- DOI: 10.1093/ndt/gfm813
Agalsidase therapy in patients with Fabry disease on renal replacement therapy: a nationwide study in Italy
Abstract
Background: In Fabry disease, end-stage renal disease (ESRD) and severe neurologic and cardiac complications represent the leading causes of late morbidity and mortality. A comprehensive Italian nationwide survey study was conducted to explore changes in cardiac status and renal allograft function in Fabry patients on renal replacement therapy (RRT) and enzyme replacement therapy (ERT).
Methods: This study was designed as a cross-sectional survey study with prospective follow-up. Of the 34 patients identified via searches in registries, 31 males and 2 females who received RRT and ERT (agalsidase beta in 30 patients, agalsidase alpha in 3) were included. Left ventricular mass index (LVMI), interventricular septal thickness at end diastole (IVSD), left ventricular posterior wall thickness (LVPWT) and renal allograft function were assessed at ERT baseline and subsequently at yearly intervals.
Results: The patients in the dialysis and transplant groups had been started on dialysis at age 42.0 and 37.1 years (mean), respectively, and patients in the transplant group received their renal allograft at age 39.8 years (mean). The mean age at the start of ERT was similar, 44.1 and 44.6 years, respectively. The mean RRT follow-up was 61.1 and 110.6 months for dialysis and transplant patients, respectively, whereas the ERT duration was 45.1 and 48.4 months, respectively. Cardiac parameters increased in dialysis patients. In transplant patients, mean LVMI seemed to plateau during agalsidase therapy at a lower level as compared to baseline. Decline in renal allograft function was relatively mild (-1.92 ml/min/year). Agalsidase therapy was well tolerated. Serious ERT-unrelated events occurred more often in the dialysis group.
Conclusions: Kidney transplantation should be the standard of care for Fabry patients progressing towards ESRD. Transplanted Fabry patients on ERT may do better than patients remaining on maintenance dialysis. Larger, controlled studies in Fabry patients with ESRD will have to demonstrate if ERT is able to change the trajectory of cardiac disease and can preserve graft renal function.
Similar articles
-
Effects of Baseline Left Ventricular Hypertrophy and Decreased Renal Function on Cardiovascular and Renal Outcomes in Patients with Fabry Disease Treated with Agalsidase Alfa: A Fabry Outcome Survey Study.Clin Ther. 2020 Dec;42(12):2321-2330.e0. doi: 10.1016/j.clinthera.2020.10.007. Epub 2020 Nov 17. Clin Ther. 2020. PMID: 33218740
-
Risk factors for severe clinical events in male and female patients with Fabry disease treated with agalsidase beta enzyme replacement therapy: Data from the Fabry Registry.Mol Genet Metab. 2016 Sep;119(1-2):151-9. doi: 10.1016/j.ymgme.2016.06.007. Epub 2016 Jun 13. Mol Genet Metab. 2016. PMID: 27510433 Clinical Trial.
-
Kidney transplantation in patients with Fabry disease.Transpl Int. 2009 Apr;22(4):475-81. doi: 10.1111/j.1432-2277.2008.00824.x. Epub 2009 Jan 22. Transpl Int. 2009. PMID: 19207191
-
Dialysis and transplantation in Fabry disease: indications for enzyme replacement therapy.Clin J Am Soc Nephrol. 2010 Feb;5(2):379-85. doi: 10.2215/CJN.05570809. Epub 2010 Jan 7. Clin J Am Soc Nephrol. 2010. PMID: 20056752 Review.
-
Kidney transplantation and enzyme replacement therapy in patients with Fabry disease.J Nephrol. 2013 Jul-Aug;26(4):645-51. doi: 10.5301/jn.5000214. Epub 2012 Sep 19. J Nephrol. 2013. PMID: 23023720 Review.
Cited by
-
Enzyme replacement therapy for Fabry disease: a systematic review of available evidence.Drugs. 2009 Nov 12;69(16):2179-205. doi: 10.2165/11318300-000000000-00000. Drugs. 2009. PMID: 19852524
-
Different renal phenotypes in related adult males with Fabry disease with the same classic genotype.Mol Genet Genomic Med. 2017 May 8;5(4):438-442. doi: 10.1002/mgg3.292. eCollection 2017 Jul. Mol Genet Genomic Med. 2017. PMID: 28717668 Free PMC article.
-
Fabry disease and kidney involvement: starting from childhood to understand the future.Pediatr Nephrol. 2022 Jan;37(1):95-103. doi: 10.1007/s00467-021-05076-x. Epub 2021 Apr 30. Pediatr Nephrol. 2022. PMID: 33928440 Review.
-
Safety and efficacy of enzyme replacement therapy in the nephropathy of Fabry disease.Biologics. 2008 Dec;2(4):823-43. doi: 10.2147/btt.s3770. Biologics. 2008. PMID: 19707461 Free PMC article.
-
The effect of enzyme replacement therapy on clinical outcomes in male patients with Fabry disease: A systematic literature review by a European panel of experts.Mol Genet Metab Rep. 2019 Feb 6;19:100454. doi: 10.1016/j.ymgmr.2019.100454. eCollection 2019 Jun. Mol Genet Metab Rep. 2019. PMID: 30775256 Free PMC article. Review.
Publication types
MeSH terms
Substances
LinkOut - more resources
Full Text Sources
Other Literature Sources
Medical