Glycogen Storage Disease Type I with Persistent Proteinuria: Management After ACE Inhibitor or ARB Monotherapy Fails
Clinical Scenario
This protocol addresses Glycogen Storage Disease Type I patients with established renal involvement, specifically:
- Sustained estimated GFR >140 ml/min/1.73 m² (glomerular hyperfiltration)
- Microalbuminuria >30 µg albumin/mg creatinine
- Frank proteinuria >0.2 mg protein/mg creatinine
When First-Line Angiotensin Blockade Is Not Enough
The initial step for these patients is single-agent angiotensin blockade — either an angiotensin-converting enzyme (ACE) inhibitor or an angiotensin receptor blocker (ARB) used alone. The target of that first line is to reduce proteinuria to normal or near-normal levels and to restore normal GFR rates.
When monotherapy does not achieve those goals, this protocol defines the next step.
Next-Line Approach
The clinical objective is to further reduce proteinuria. The escalation strategy involves combined angiotensin blockade — deploying more than one type of agent together. The full regimen, specific agent selection, sequencing, and monitoring criteria are contained in the complete protocol.
References
DOI: 10.1038/gim.2014.128
- Consider initiating an ACE inhibitor or ARB with evidence of hyperfiltration (sustained estimated GFR >140 ml/min/1.73 m2).
- Initiate an ACE inhibitor or ARB for persistent microalbuminuria (>30 μg albumin/mg creatinine).
- Initiate an ACE inhibitor or ARB for frank proteinuria (>0.2 mg protein/mg creatinine).
- In cases in which there is a need for further angiotensin blockade, use of both an ACE and an ARB can prove synergistic to reduce proteinuria, with no increased rate of hyperkalemia or drug-related renal insufficiency.
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