Quantifying the effect of Fabrazyme (agalsidase beta) on the preservation of renal function in Fabry disease

A meta‑analysis of data from ten studies suggests that Fabrazyme preserves renal function in patients with classic Fabry disease compared to untreated patients¹

Background

• Glomerular filtration rate (GFR) decline indicates the progression of nephropathy in Fabry disease
• The effect of Fabrazyme treatment on GFR decline is not well established as the strength of the data from individual studies is limited by their small populations
• This publication reports results from a meta‑analysis of ten studies on classic Fabry disease using patient‑level data, which allows inclusion of patients on an individual basis and the adjustment for patient characteristics across settings

Objective

• To determine the long‑term effect of Fabrazyme on eGFR

Methods

• Four clinical trials and six studies from a systematic literature review (SLR). The SLR were restricted to patients with classic Fabry disease who met the eligibility criteria from phase III and phase IV Fabrazyme trials
• 315 patients were included in total, including 161 patients treated with Fabrazyme
• Analysis was undertaken with summary measures approach (SMA)
  • Step 1: an estimated rate of change in eGFR per year was obtained using linear regression on each patient individually
  • Step 2: the estimated slope coefficients from Step 1 were modelled using quantile regression with covariates of interest
• ERT was given for a median of 65 months (range, 13–69 months)

Relevance to clinical practice

• This meta‑analysis of 10 studies on patients with classic Fabry disease suggests that treatment with Fabrazyme slows down renal decline
• Patients conserved their renal function better than untreated patients as assessed by eGFR and uPCR
• Starting treatment earlier may delay the time to end stage renal disease compared with patients who are left untreated