Effect and Tolerability of Agalsidase Alfa in Patients with Fabry Disease Who Were Treatment Naive or Formerly Treated with Agalsidase Beta or Agalsidase Alfa.

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 patie

SUBMITTER: Goker-Alpan O 

PROVIDER: S-EPMC4484907 | biostudies-literature | 2015

REPOSITORIES: biostudies-literature