Tags

Type your tag names separated by a space and hit enter

Patients with Fabry disease after enzyme replacement therapy dose reduction versus treatment switch.
J Am Soc Nephrol. 2014 Apr; 25(4):837-49.JA

Abstract

Because of the shortage of agalsidase-beta in 2009, many patients with Fabry disease were treated with lower doses or were switched to agalsidase-alfa. This observational study assessed end-organ damage and clinical symptoms during dose reduction or switch to agalsidase-alfa. A total of 105 adult patients with Fabry disease who had received agalsidase-beta (1.0 mg/kg body weight) for ≥1 year were nonrandomly assigned to continue this treatment regimen (regular-dose group, n=38), receive a reduced dose of 0.3-0.5 mg/kg (dose-reduction group, n=29), or switch to 0.2 mg/kg agalsidase-alfa (switch group) and were followed prospectively for 1 year. We assessed clinical events (death, myocardial infarction, severe arrhythmia, stroke, progression to ESRD); changes in cardiac, renal, and neurologic function; and Fabry-related symptoms (neuropathic pain, hypohidrosis, diarrhea, and disease severity scores). Organ function and Fabry-related symptoms remained stable in the regular-dose group. In contrast, estimated GFR decreased by about 3 ml/min per 1.73 m(2) (P=0.01) in the dose-reduction group, and the median albumin-to-creatinine ratio increased from 114 (0-606) mg/g to 216 (0-2062) mg/g (P=0.03) in the switch group. Furthermore, mean Mainz Severity Score Index scores and frequencies of pain attacks, chronic pain, gastrointestinal pain, and diarrhea increased significantly in the dose-reduction and switch groups. In conclusion, patients receiving regular agalsidase-beta dose had a stable disease course, but dose reduction led to worsening of renal function and symptoms. Switching to agalsidase-alfa is safe, but microalbuminuria may progress and Fabry-related symptoms may deteriorate.

Authors+Show Affiliations

Department of Medicine, Divisions of Cardiology and Nephrology, Comprehensive Heart Failure Center, Fabry Center for Interdisciplinary Therapy, and.No affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info available

Pub Type(s)

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

Language

eng

PubMed ID

24556354

Citation

Weidemann, Frank, et al. "Patients With Fabry Disease After Enzyme Replacement Therapy Dose Reduction Versus Treatment Switch." Journal of the American Society of Nephrology : JASN, vol. 25, no. 4, 2014, pp. 837-49.
Weidemann F, Krämer J, Duning T, et al. Patients with Fabry disease after enzyme replacement therapy dose reduction versus treatment switch. J Am Soc Nephrol. 2014;25(4):837-49.
Weidemann, F., Krämer, J., Duning, T., Lenders, M., Canaan-Kühl, S., Krebs, A., Guerrero González, H., Sommer, C., Üçeyler, N., Niemann, M., Störk, S., Schelleckes, M., Reiermann, S., Stypmann, J., Brand, S. M., Wanner, C., & Brand, E. (2014). Patients with Fabry disease after enzyme replacement therapy dose reduction versus treatment switch. Journal of the American Society of Nephrology : JASN, 25(4), 837-49. https://doi.org/10.1681/ASN.2013060585
Weidemann F, et al. Patients With Fabry Disease After Enzyme Replacement Therapy Dose Reduction Versus Treatment Switch. J Am Soc Nephrol. 2014;25(4):837-49. PubMed PMID: 24556354.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Patients with Fabry disease after enzyme replacement therapy dose reduction versus treatment switch. AU - Weidemann,Frank, AU - Krämer,Johannes, AU - Duning,Thomas, AU - Lenders,Malte, AU - Canaan-Kühl,Sima, AU - Krebs,Alice, AU - Guerrero González,Hans, AU - Sommer,Claudia, AU - Üçeyler,Nurcan, AU - Niemann,Markus, AU - Störk,Stefan, AU - Schelleckes,Michael, AU - Reiermann,Stefanie, AU - Stypmann,Jörg, AU - Brand,Stefan-Martin, AU - Wanner,Christoph, AU - Brand,Eva, Y1 - 2014/02/20/ PY - 2014/2/22/entrez PY - 2014/2/22/pubmed PY - 2014/5/28/medline SP - 837 EP - 49 JF - Journal of the American Society of Nephrology : JASN JO - J. Am. Soc. Nephrol. VL - 25 IS - 4 N2 - Because of the shortage of agalsidase-beta in 2009, many patients with Fabry disease were treated with lower doses or were switched to agalsidase-alfa. This observational study assessed end-organ damage and clinical symptoms during dose reduction or switch to agalsidase-alfa. A total of 105 adult patients with Fabry disease who had received agalsidase-beta (1.0 mg/kg body weight) for ≥1 year were nonrandomly assigned to continue this treatment regimen (regular-dose group, n=38), receive a reduced dose of 0.3-0.5 mg/kg (dose-reduction group, n=29), or switch to 0.2 mg/kg agalsidase-alfa (switch group) and were followed prospectively for 1 year. We assessed clinical events (death, myocardial infarction, severe arrhythmia, stroke, progression to ESRD); changes in cardiac, renal, and neurologic function; and Fabry-related symptoms (neuropathic pain, hypohidrosis, diarrhea, and disease severity scores). Organ function and Fabry-related symptoms remained stable in the regular-dose group. In contrast, estimated GFR decreased by about 3 ml/min per 1.73 m(2) (P=0.01) in the dose-reduction group, and the median albumin-to-creatinine ratio increased from 114 (0-606) mg/g to 216 (0-2062) mg/g (P=0.03) in the switch group. Furthermore, mean Mainz Severity Score Index scores and frequencies of pain attacks, chronic pain, gastrointestinal pain, and diarrhea increased significantly in the dose-reduction and switch groups. In conclusion, patients receiving regular agalsidase-beta dose had a stable disease course, but dose reduction led to worsening of renal function and symptoms. Switching to agalsidase-alfa is safe, but microalbuminuria may progress and Fabry-related symptoms may deteriorate. SN - 1533-3450 UR - https://www.unboundmedicine.com/medline/citation/24556354/Patients_with_Fabry_disease_after_enzyme_replacement_therapy_dose_reduction_versus_treatment_switch_ L2 - http://jasn.asnjournals.org/cgi/pmidlookup?view=long&pmid=24556354 DB - PRIME DP - Unbound Medicine ER -