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


OBJECTIVE
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.


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.
Melis
D
D
Dipartimento di Pediatria, Università Federico II, Napoli, Italy. danlis@tin.it
Parenti
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Gatti
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Casa
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Riva
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Dionisi Vici
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Furlan
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Torcoletti
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Papadia
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Benigno
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Andria
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D000293:Adolescent; D000328:Adult; D017668:Age of Onset; D000419:Albuminuria / Q000503:physiopathology / Q000517:prevention & control; D000806:Angiotensin-Converting Enzyme Inhibitors / Q000627:therapeutic use*; D002648:Child; D002675:Child, Preschool; D018450:Disease Progression; D005919:Glomerular Filtration Rate / Q000187:drug effects; D005953:Glycogen Storage Disease Type I / Q000150:complications* / Q000503:physiopathology; D006801:Humans; D007223:Infant; D007674:Kidney Diseases / Q000209:etiology / Q000503:physiopathology / Q000517:prevention & control*; D011507:Proteinuria / Q000503:physiopathology / Q000517:prevention & control; D012189:Retrospective Studies; D012720:Severity of Illness Index; D016896:Treatment Outcome
D000806:Angiotensin-Converting Enzyme Inhibitors