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

Daniela Melis, G. Parenti, R. Gatti, R. Della Casa, R. Parini, E. Riva, A. B. Burlina, C. Dionisi Vici, M. Di Rocco, F. Furlan, M. Torcoletti, F. Papadia, A. Donati, V. Benigno, G. Andria

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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.

Original languageEnglish
Pages (from-to)19-25
Number of pages7
JournalClinical Endocrinology
Issue number1
Publication statusPublished - Jul 2005

ASJC Scopus subject areas

  • Endocrinology


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