Treatment of Glycogen Storage Disease Type IV in Dilated Cardiomyopathy

This protocol covers the management of Glycogen storage disease type IV (GSD IV) in patients who have developed dilated cardiomyopathy (DCM) without hypertrophic cardiomyopathy. The cardiac phenotype directly determines the treatment pathway.

Clinical Scenario

The patient has confirmed GSD IV with dilated cardiomyopathy. Hypertrophic cardiomyopathy is not present. This distinction is clinically important: the absence of hypertrophic cardiomyopathy and the presence of a dilated phenotype shape which interventions are appropriate.

Treatment Approach

When the cardiomyopathy associated with GSD IV does not respond to medical management, a surgical intervention approach becomes the relevant next step.

Full protocol details — including specific options, selection criteria, and sequencing — are available via the link below.

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References

For individuals with DCM, use of either β-blockers (either β1-selective, or combination non-selective β1/α1 blocker, such as carvedilol), or either angiotensin-converting enzyme (ACE) inhibitors or angiotensin receptor blockers (ARBs), is reasonable.
For cases of GSD IV with cardiomyopathy refractory to medical management, surgical intervention, including mechanical support and cardiac transplantation, can be considered.

DOI: 10.1016/j.ymgme.2023.107525

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