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Patients with Fabry Disease after Enzyme Replacement Therapy Dose Reduction and Switch-2-Year Follow-Up.
J Am Soc Nephrol. 2016 Mar; 27(3):952-62.JA

Abstract

Because of the shortage of agalsidase-β supply between 2009 and 2012, patients with Fabry disease either were treated with reduced doses or were switched to agalsidase-α. In this observational study, we assessed end organ damage and clinical symptoms with special focus on renal outcome after 2 years of dose-reduction and/or switch to agalsidase-α. A total of 89 adult patients with Fabry disease who had received agalsidase-β (1.0 mg/kg body wt) for >1 year were nonrandomly assigned to continue this treatment regimen (regular-dose group, n=24), to receive a reduced dose of 0.3-0.5 mg/kg and a subsequent switch to 0.2 mg/kg agalsidase-α (dose-reduction-switch group, n=28), or to directly switch to 0.2 mg/kg agalsidase-α (switch group, n=37) and were followed-up for 2 years. We assessed clinical events (death, myocardial infarction, severe arrhythmia, stroke, progression to ESRD), changes in cardiac and renal function, Fabry-related symptoms (pain, hypohidrosis, diarrhea), and disease severity scores. Determination of renal function by creatinine and cystatin C-based eGFR revealed decreasing eGFRs in the dose-reduction-switch group and the switch group. The Mainz Severity Score Index increased significantly in these two groups (P=0.02 and P<0.001, respectively), and higher frequencies of gastrointestinal pain occurred during follow-up. In conclusion, after 2 years of observation, all groups showed a stable clinical disease course with respect to serious clinical events. However, patients under agalsidase-β dose-reduction and switch or a direct switch to agalsidase-α showed a decline of renal function independent of the eGFR formula used.

Authors+Show Affiliations

Internal Medicine D, Department of Nephrology, Hypertension and Rheumatology, Interdisciplinary Fabry Center Münster.Department of Medicine, Division of Nephrology and.Department of Pediatrics and Adolescent Medicine, University of Ulm, Ulm, Germany; Department of Medicine, Divisions of Cardiology and Nephrology, Comprehensive Heart Failure Center, Fabry Center for Interdisciplinary Therapy and.Department of Neurology.Internal Medicine D, Department of Nephrology, Hypertension and Rheumatology, Interdisciplinary Fabry Center Münster.Department of Medicine, Divisions of Cardiology and Nephrology, Comprehensive Heart Failure Center, Fabry Center for Interdisciplinary Therapy and Department of Neurology, University of Würzburg, Würzburg, Germany;Department of Cardiovascular Medicine, Division of Cardiology.Department of Medicine, Division of Cardiology, Charité, Campus Virchow-Klinikum, Berlin, Germany;Department of Medicine, Divisions of Cardiology and Nephrology, Comprehensive Heart Failure Center, Fabry Center for Interdisciplinary Therapy and Department of Neurology, University of Würzburg, Würzburg, Germany;Institute of Epidemiology and Social Medicine, and.Institute of Sports Medicine, Molecular Genetics of Cardiovascular Disease, University Hospital Münster, Münster, Germany;Department of Medicine, Divisions of Cardiology and Nephrology, Comprehensive Heart Failure Center, Fabry Center for Interdisciplinary Therapy and.Department of Medicine, Divisions of Cardiology and Nephrology, Comprehensive Heart Failure Center, Fabry Center for Interdisciplinary Therapy and Katharinen-Hospital Unna, Unna, Germany.Internal Medicine D, Department of Nephrology, Hypertension and Rheumatology, Interdisciplinary Fabry Center Münster, Eva.Brand@ukmuenster.de.

Pub Type(s)

Journal Article
Observational Study
Research Support, Non-U.S. Gov't

Language

eng

PubMed ID

26185201

Citation

Lenders, Malte, et al. "Patients With Fabry Disease After Enzyme Replacement Therapy Dose Reduction and Switch-2-Year Follow-Up." Journal of the American Society of Nephrology : JASN, vol. 27, no. 3, 2016, pp. 952-62.
Lenders M, Canaan-Kühl S, Krämer J, et al. Patients with Fabry Disease after Enzyme Replacement Therapy Dose Reduction and Switch-2-Year Follow-Up. J Am Soc Nephrol. 2016;27(3):952-62.
Lenders, M., Canaan-Kühl, S., Krämer, J., Duning, T., Reiermann, S., Sommer, C., Stypmann, J., Blaschke, D., Üçeyler, N., Hense, H. W., Brand, S. M., Wanner, C., Weidemann, F., & Brand, E. (2016). Patients with Fabry Disease after Enzyme Replacement Therapy Dose Reduction and Switch-2-Year Follow-Up. Journal of the American Society of Nephrology : JASN, 27(3), 952-62. https://doi.org/10.1681/ASN.2015030337
Lenders M, et al. Patients With Fabry Disease After Enzyme Replacement Therapy Dose Reduction and Switch-2-Year Follow-Up. J Am Soc Nephrol. 2016;27(3):952-62. PubMed PMID: 26185201.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Patients with Fabry Disease after Enzyme Replacement Therapy Dose Reduction and Switch-2-Year Follow-Up. AU - Lenders,Malte, AU - Canaan-Kühl,Sima, AU - Krämer,Johannes, AU - Duning,Thomas, AU - Reiermann,Stefanie, AU - Sommer,Claudia, AU - Stypmann,Jörg, AU - Blaschke,Daniela, AU - Üçeyler,Nurcan, AU - Hense,Hans-Werner, AU - Brand,Stefan-Martin, AU - Wanner,Christoph, AU - Weidemann,Frank, AU - Brand,Eva, Y1 - 2015/07/16/ PY - 2015/03/31/received PY - 2015/06/10/accepted PY - 2017/03/01/pmc-release PY - 2015/7/18/entrez PY - 2015/7/18/pubmed PY - 2016/7/9/medline KW - Fabry’s disease KW - chronic kidney disease KW - creatinine clearance KW - cystatin C clearance KW - enzyme KW - outcomes KW - replacement therapy SP - 952 EP - 62 JF - Journal of the American Society of Nephrology : JASN JO - J Am Soc Nephrol VL - 27 IS - 3 N2 - Because of the shortage of agalsidase-β supply between 2009 and 2012, patients with Fabry disease either were treated with reduced doses or were switched to agalsidase-α. In this observational study, we assessed end organ damage and clinical symptoms with special focus on renal outcome after 2 years of dose-reduction and/or switch to agalsidase-α. A total of 89 adult patients with Fabry disease who had received agalsidase-β (1.0 mg/kg body wt) for >1 year were nonrandomly assigned to continue this treatment regimen (regular-dose group, n=24), to receive a reduced dose of 0.3-0.5 mg/kg and a subsequent switch to 0.2 mg/kg agalsidase-α (dose-reduction-switch group, n=28), or to directly switch to 0.2 mg/kg agalsidase-α (switch group, n=37) and were followed-up for 2 years. We assessed clinical events (death, myocardial infarction, severe arrhythmia, stroke, progression to ESRD), changes in cardiac and renal function, Fabry-related symptoms (pain, hypohidrosis, diarrhea), and disease severity scores. Determination of renal function by creatinine and cystatin C-based eGFR revealed decreasing eGFRs in the dose-reduction-switch group and the switch group. The Mainz Severity Score Index increased significantly in these two groups (P=0.02 and P<0.001, respectively), and higher frequencies of gastrointestinal pain occurred during follow-up. In conclusion, after 2 years of observation, all groups showed a stable clinical disease course with respect to serious clinical events. However, patients under agalsidase-β dose-reduction and switch or a direct switch to agalsidase-α showed a decline of renal function independent of the eGFR formula used. SN - 1533-3450 UR - https://www.unboundmedicine.com/medline/citation/26185201/Patients_with_Fabry_Disease_after_Enzyme_Replacement_Therapy_Dose_Reduction_and_Switch_2_Year_Follow_Up_ L2 - https://jasn.asnjournals.org/cgi/pmidlookup?view=long&amp;pmid=26185201 DB - PRIME DP - Unbound Medicine ER -