Glycogen storage disease type IV
ICD-10 E74.0 · ICD-11 5C51.3.4

Treatment of Glycogen Storage Disease Type IV with Dilated Cardiomyopathy

This protocol addresses the management of Glycogen storage disease type IV (GSD IV) in individuals who present with dilated cardiomyopathy (DCM) and do not have hypertrophic cardiomyopathy. The cardiac phenotype directly informs agent selection and the overall therapeutic approach.

Clinical Scenario

The patient has confirmed dilated cardiomyopathy in the absence of hypertrophic cardiomyopathy. This distinction is clinically significant: the presence of DCM without the hypertrophic variant defines a specific pharmacological management pathway within GSD IV care.

Treatment Approach (partial overview)

Management in this setting centers on combination cardiac pharmacotherapy — a beta-blocker paired with a renin-angiotensin system agent forms the therapeutic foundation. Additional agents may be considered depending on symptom status. The complete structured regimen, including full agent selection, sequencing, and criteria for additional therapy, is available in the protocol.

Instant Access to Structured Evidence-Based Regimens

References

  1. 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.
  2. For advanced DCM, β-blockers can be combined with ACE inhibitors or ARBs at the discretion of the provider.
  3. Further, for symptomatic individuals with DCM, the use of diuretics is reasonable.
DOI: 10.1016/j.ymgme.2023.107525
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