Short-term effect of captopril on microalbuminuria in children with glycogen storage disease type Ia

Area of Science:

• Nephrology
• Pediatric Endocrinology
• Metabolic Disorders

Background:

• Glycogen storage disease type Ia (GSD Ia) can lead to early renal dysfunction, characterized by glomerular hyperfiltration and proteinuria.
• Microalbuminuria, defined as an albumin/creatinine ratio > 2.5 mg/mmol, is a key indicator of this renal involvement.
• Previous studies suggest potential benefits of captopril in similar conditions like diabetic nephropathy.

Purpose of the Study:

• To investigate the efficacy of captopril in improving proteinuria in patients diagnosed with GSD Ia and microalbuminuria.
• To assess the impact of captopril treatment on urinary albumin excretion over a 6-month period.

Main Methods:

• A non-randomized study involving 36 GSD Ia patients, with 19 identified as having microalbuminuria.
• Eight patients with microalbuminuria received captopril at a dose of 1 mg/kg/day.
• Urinary albumin excretion was monitored periodically over 6 months; outcomes were compared between treated and untreated groups.

Main Results:

• Among 7 captopril-treated patients (one lost to follow-up), urinary albumin excretion normalized in 3 (42.9%) and decreased by at least 50% in another 3 (42.8%).
• Untreated patients showed less significant improvement, with only two experiencing a decrease > 50%.
• Patients with microalbuminuria exhibited more severe clinical and laboratory findings, including higher lactate and triglycerides, and lower bicarbonate levels.

Conclusions:

• Captopril at 1 mg/kg/day demonstrated effectiveness in at least 50% of GSD Ia patients presenting with microalbuminuria.
• The findings suggest captopril is a promising therapeutic option for managing early renal dysfunction in GSD Ia.
• Microalbuminuria in GSD Ia is associated with more severe disease manifestations, highlighting the need for early detection and intervention.