Efficacy of ACE-inhibitor therapy on renal disease in glycogen storage disease type 1: a multicentre retrospective study

Clin Endocrinol (Oxf). 2005 Jul;63(1):19-25. doi: 10.1111/j.1365-2265.2005.02292.x.


Background: The efficacy of ACE-inhibitors in decreasing microalbuminuria and proteinuria has been reported in a few patients with glycogen storage disease type 1 (GSD1); however, no case-control study has ever been published.

Aim: The aim of the current study was to evaluate the efficacy of ACE-inhibitors in reducing glomerular hyperfiltration, microalbuminuria and proteinuria, and in delaying the progression of renal damage.

Patients and methods: Ninety-five patients (median age at the time of the study: 14.5 years) were enrolled from nine Italian referral centres for metabolic diseases. A retrospective study of a 10-year follow-up was conducted in order to compare the evolution of these parameters in treated patients with those who were not treated with ACE-inhibitors.

Results: A significant and progressive decrease of glomerular filtration rate was observed in treated patients vs. those who were not treated with ACE-inhibitors (P < 0.05). No difference was observed for microalbuminuria and proteinuria between the two groups of patients. Moreover, the ACE-inhibitors significantly delayed the progression from glomerular hyperfiltration to microalbuminuria, but not that from microalbuminuria to proteinuria.

Conclusions: The results of the present study underline the importance of a strict follow-up of renal function in GSD1 patients. The detection of glomerular hyperfiltration suggests precocious initiation of ACE-inhibitor treatment to delay the progression of renal damage. A randomized prospective study is needed to establish for certain the real effectiveness of this treatment in GSD1 patients.

Publication types

  • Multicenter Study

MeSH terms

  • Adolescent
  • Adult
  • Age of Onset
  • Albuminuria / physiopathology
  • Albuminuria / prevention & control
  • Angiotensin-Converting Enzyme Inhibitors / therapeutic use*
  • Child
  • Child, Preschool
  • Disease Progression
  • Glomerular Filtration Rate / drug effects
  • Glycogen Storage Disease Type I / complications*
  • Glycogen Storage Disease Type I / physiopathology
  • Humans
  • Infant
  • Kidney Diseases / etiology
  • Kidney Diseases / physiopathology
  • Kidney Diseases / prevention & control*
  • Proteinuria / physiopathology
  • Proteinuria / prevention & control
  • Retrospective Studies
  • Severity of Illness Index
  • Treatment Outcome


  • Angiotensin-Converting Enzyme Inhibitors