Table 5.

Danon Disease: Treatment of Manifestations

Manifestation/
Concern		TreatmentConsiderations/Other
Hypertrophic
cardiomyopathy
  • Institute hypertrophic cardiomyopathy guidelines 1 for those w/cardiac hypertrophy & preserved left-ventricular ejection fraction.
  • Timely consideration of implantable cardiac defibrillators
The benefit of negative inotropes or myomectomy for cardiac obstruction has not been established.
Heart failure Standard treatment, 2 incl careful fluid & volume mgmt & avoidance of over-diuresis & dehydrationThe benefit of neurohormonal 3 therapy has not been established.
Timely consideration of cardiac transplantation in those w/progressive symptoms or significant ↓ in left-ventricular ejection fractionDurable mechanical circulatory support should be used w/caution given biventricular involvement of disease.
Cardiac pre-
excitation &
arrhythmia
  • Consideration of electrophysiologic studies & ablation therapy
  • Timely consideration of implantable cardiac defibrillators
Efficacy of ablations for accessory pathways & arrhythmia foci may be ↓ in those w/Danon disease.
Skeletal muscle
weakness 		Physical therapy
DD/ID See Developmental Delay / Intellectual Disability Management Issues.
Retinopathy Use of low vision aids 4Consultation w/agencies for the visually impaired 5
Family/
Community
  • Ensure appropriate social work involvement to connect families w/local resources, respite, & support.
  • Coordinate care to manage multiple subspecialty appointments, equipment, medications, & supplies.
Consider involvement in adaptive sports or Special Olympics.

DD = developmental delay; ID = intellectual disability

1.
2.
3.

Neurohormonal therapy includes drugs that target the sympathetic nervous system (beta-blockers, alpha-blockers) and the renin-angiotensin system (ACE inhibitors, angiotensin receptor blockers, mineralocorticoid receptor antagonists).

4.

Low vision aids such as magnifiers and closed circuit television may provide useful reading vision for individuals with reduced central acuity and constricted visual fields.

5.

In the US, publicly funded agencies at the state level provide services for the blind or those with progressive eye disorders; services include vocational training, mobility training, and skills for independent living.

From: Danon Disease

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