Product information and disease description Place of product in therapy Approaches to treatment Current treatment options and standard of care

Heart failure medical therapy

10 evidence topics · 10 sources

Consensus statement: no disease-specific therapy; symptom-guided heart failure and antiarrhythmic medication

“Currently, there are no available therapies to specifically treat Danon disease.” (opens the source at this quote in a new tab)
“For the treatment of heart disease, clinical signs and symptoms should guide the use of medications for congestive heart failure and arrhythmias, such as diuretics and antiarrhythmics, with beta blockers as first line therapies.” (opens the source at this quote in a new tab)

Consensus statement: renin-angiotensin-aldosterone system inhibition and beta blockers without proof of efficacy

“Inasmuch as cardiomyopathy in Danon disease is characterized by progressive fibrosis and an inadvertent loss of systolic function, one should consider therapies to attenuate cardiac remodeling (ie, renin-angiotensin-aldosterone system inhibition and β-adrenergic blockers).” (opens the source at this quote in a new tab)
“Although no proof of efficacy exists in Danon disease, a similar approach to prevent the development of systolic dysfunction is a prevailing practice in another X-linked malignant cardiomyopathies, including Duchenne muscular dystrophy caused by Duchenne muscular dystrophy mutations.” (opens the source at this quote in a new tab)

Consensus statement: benefit of guideline-directed medical therapy unknown

“As reviewed, it is unclear how to evaluate the risk of sudden cardiac death, whether goal-directed medical therapy is beneficial, and the natural history of the disease in women.” (opens the source at this quote in a new tab)

Consensus statement: expected tolerance and agents to avoid

“As in any pre-existing cardiac pathologic process involving a compromise in mitochondrial function, patients with Danon disease are expected to have poor tolerance to hypertension, tachycardia, and anemia.” (opens the source at this quote in a new tab)
“Cardiotoxic medications and stimulants should be avoided.” (opens the source at this quote in a new tab)
“Stimuli activating autophagy such as strenuous physical activity and diet therapy should be decreased because of a risk of enhancing disease progression in a LAMP-2-deficient heart.” (opens the source at this quote in a new tab)

GeneReviews: standard heart failure treatment with careful volume management; benefit of neurohormonal therapy not established

“careful fluid & volume mgmt & avoidance of over-diuresis & dehydration” (opens the source at this quote in a new tab)
“The benefit of neurohormonal therapy has not been established.” (opens the source at this quote in a new tab)
“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).” (opens the source at this quote in a new tab)
“The benefit of negative inotropes or myomectomy for cardiac obstruction has not been established.” (opens the source at this quote in a new tab)

Earlier management proposal: standard heart failure approaches

“Meticulous volume/fluid management, with avoidance of dehydration or overdiuresis” (opens the source at this quote in a new tab)
“Reliance on hypertrophic cardiomyopathy guidelines for patients with hypertrophy but preserved LVEF” (opens the source at this quote in a new tab)

ESC cardiomyopathy guideline: no approved etiological therapy

“To date, there are no approved aetiological therapies for PRKAG2 syndrome and Danon disease.” (opens the source at this quote in a new tab)

Company statement on heart failure medications and disease progression

“There are no specific therapies available for the treatment of DD and medications typically utilized for the treatment of HF are not believed to modify progression to end-stage HF.” (opens the source at this quote in a new tab)

Atrioventricular block and rate-slowing medication in affected women

“although the latter arrhythmogenic condition (AV block) may have been exacerbated by the administration of beta-blocker or non-dihydropyridine calcium-channel blocker medication.” (opens the source at this quote in a new tab)

Therapies to attenuate fibrosis: registry authors' statement

“While novel therapies are being developed, contemporary therapies to attenuate fibrosis might be useful to modify the course of cardiomyopathy” (opens the source at this quote in a new tab)

Controlled trials of heart failure drugs in Danon disease

No evidence found.