Melis D et al. (2005) Efficacy of ACE-inhibitor therapy on renal disease in glycogen storage disease type 1: a multicentre retrospective study. Clin Endocrinol 63: 19–25

Patients with glycogen storage disease type 1 (GSD1) are at risk of renal damage, associated with glomerular hyperfiltration, microalbuminuria, proteinuria and systemic arterial hypertension. New research from Italy has examined the role of angiotensin-converting enzyme (ACE) inhibitors in improving and delaying renal complications in these patients, as reported in patients with diabetic nephropathy.

Melis et al. carried out a retrospective analysis of 95 patients with GSD1, over a 10-year follow-up period. Ages ranged from 6 months to 42 years (median 14.5 years) at the time of the study. Of 89 patients for whom sufficient data were available, renal damage was recorded in 40 cases (45%), confirming the high prevalence of this complication in GSD1.

The effects of ACE inhibitors on the severity of renal disease were studied in a subset of 48 patients. Three years after glomerular hyperfiltration was first noted in these patients, the glomerular filtration rate was significantly lower in those treated with ACE inhibitors than in those who were not. No difference was found between the two groups in the extent of microalbuminuria or proteinuria. Renal disease progression appeared to be delayed by the administration of ACE inhibitors, at least in terms of the development of microalbuminuria, in patients who started therapy when hyperfiltration was first detected.

Recommending early follow-up of renal function in patients with GSD1, the authors call for a randomized, prospective study to explore the potential utility of ACE-inhibitor therapy in delaying the progression of renal disease.