Danon Disease Follow-up

Updated: Feb 09, 2016
  • Author: Keith K Vaux, MD; Chief Editor: Maria Descartes, MD  more...
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Follow-up

Further Outpatient Care

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  • Most of the cardiac care for patients with Danon disease can be provided in an outpatient setting.
    • This care should include routine echocardiography to monitor the progression of hypertrophy and to detect changes suggestive of decreased left ventricular function and evolution to dilated cardiomyopathy.
    • Holter monitoring should be followed up every 6-12 months depending on the patient's symptoms and degree of hypertrophy, as determined with echocardiography.
  • Male patients with Danon disease may require physical therapy for range-of-motion exercises and should be seen as needed by a pediatric neurologist. No medications cure or ameliorate the neuromuscular symptoms of Danon disease.
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Further Inpatient Care

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  • Inpatient care is needed for patients with Danon disease who present with life-threatening arrhythmias or clinically significant congestive heart failure (CHF).
  • When the patient develops CHF due to the dilated form of the disease, inpatient medical care may be indicated to administer intravenous inotropic agents and diuretics.
  • When arrhythmias are noted, inpatient telemetry is helpful. Implantation of an implantable cardioverter-defibrillator (ICD) or pacemaker often necessitates an overnight admission with telemetry.
  • Inpatient care is usually not required to manage the neurologic symptoms of Danon disease.
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Deterrence/Prevention

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  • Currently, no treatment prevents the onset of Danon disease. Early and repeated echocardiographic surveillance of young male relatives of anyone with Danon disease should be performed. Identification of a specific mutation in LAMP2 in a family may allow relatives to consider presymptomatic DNA testing after thorough genetic counseling.
  • Holter monitoring should also be performed when Danon disease is diagnosed.
  • An ICD should be implanted when clinically significant septal thickening of more than 30 mm is noted on echocardiography, when a poor increase in blood pressure is noted during exercise testing, when ventricular tachycardia is noted on Holter monitoring, or if the patient has a family history of sudden death, as is recommended for patients with hypertrophic cardiomyopathy (HCM). [38] The data are insufficient to make a specific recommendation about ICDs in patients with Danon disease alone.
  • From a neurologic perspective, range-of-motion exercises help to prevent joint contractures as the skeletal muscles weaken.
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Complications

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  • Potential complications for this disease include syncope or sudden death before placement of an ICD.
  • As with other forms of dilated cardiomyopathy, low-flow states can pose a risk of intracardiac thrombus formation with the potential for stroke.
  • The onset of CHF symptoms can be abrupt and should initiate a discussion about and workup for cardiac transplantation.
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Prognosis

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  • Patients with Danon disease have a poor life expectancy.
    • The prognosis for male patients is poor. Most male patients die before their third decade from either a sudden cardiac arrhythmia or CHF. [5]
    • Female patients live to their fifth decade. [5] Death can be the result of a cardiac arrhythmia or CHF.
  • Male patients have mild weakness of the skeletal muscle with slow or no deterioration over time.
    • These patients also are likely to have a learning disorder or mild mental retardation. [5, 11, 17]
    • Previously reported cases have not documented any deterioration in mental capacity over time.
  • See Mortality/Morbidity.
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Patient Education

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