Effect and tolerability of agalsidase alfa in patients with fabry disease who were treatment naïve or formerly treated with agalsidase beta or agalsidase alfa

Ozlem Goker-Alpan, Khan Nedd, Suma Shankar, Yeong Hau H. Lien, Neal Weinreb, Anna Wijatyk, Peter Chang, Rick Martin

Research output: Chapter in Book/Report/Conference proceedingChapter

7 Scopus citations

Abstract

Objectives: In a multicenter, open-label, treatment protocol (HGT-REP-059; NCT01031173), clinical effects and tolerability of agalsidase alfa (agalα; 0.2 mg/kg every other week) were evaluated in patients with Fabry disease who were treatment naïve or switched from agalsidase beta (switch). Over 24 months, data were collected on the safety profile; renal and cardiac parameters were assessed using estimated glomerular filtration rate (eGFR), left ventricular mass index (LVMI), and midwall fractional shortening (MFS). Results: Enrolled patients included 71 switch (median [range] age, 46.6 [5–84] years; male to female [M:F], 40:31) and 29 treatment naïve (38.7 [12–74] years; M:F, 14:15). Adverse events (AEs) were consistent with the known safety profile of agalα. Two switch patients had hospitalization due to possibly/probably drug-related serious AEs (one with transient ischemic attack, one with infusion-related AEs). One switch and two treatment-naïve patients discontinued treatment because of AEs. Three patients (one each switch, treatment naïve, and previous agalα) died; no deaths were considered drug-related. There was no significant change from baseline in LVMI or MFS in either group. Similarly, eGFR remained stable; mean ± standard error annualized change in eGFR (mL/min/1.73 m2) was −2.40 ± 1.04 in switch and −1.68 ± 2.21 in treatment-naïve patients. Conclusions: This is the largest cohort of patients with Fabry disease who were started on or switched to agalα in an FDA-accepted protocol during a worldwide supply shortage of agalsidase beta. Because this protocol was primarily designed to provide access to agalα, there were limitations, including not having stringent selection criteria and the lack of a placebo group.

Original languageEnglish (US)
Title of host publicationJIMD Reports
PublisherSpringer
Pages7-15
Number of pages9
DOIs
StatePublished - Jan 1 2015
Externally publishedYes

Publication series

NameJIMD Reports
Volume23
ISSN (Print)2192-8304
ISSN (Electronic)2192-8312

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Keywords

  • Chronic kidney disease stage
  • Enzyme replacement therapy
  • Fabry disease
  • Left ventricular hypertrophy
  • Left ventricular mass index

ASJC Scopus subject areas

  • Internal Medicine
  • Endocrinology, Diabetes and Metabolism
  • Biochemistry, Genetics and Molecular Biology (miscellaneous)

Cite this

Goker-Alpan, O., Nedd, K., Shankar, S., Lien, Y. H. H., Weinreb, N., Wijatyk, A., Chang, P., & Martin, R. (2015). Effect and tolerability of agalsidase alfa in patients with fabry disease who were treatment naïve or formerly treated with agalsidase beta or agalsidase alfa. In JIMD Reports (pp. 7-15). (JIMD Reports; Vol. 23). Springer. https://doi.org/10.1007/8904_2015_422