TREATMENT & MANAGEMENT
Unfortunately, there is no current cure or treatment that can return the heart to normal or guarantee long term survival. Although occasionally children with certain types of cardiomyopathy do improve, the vast majority do not show any recovery in heart function. If detected in the earlier stages, cardiomyopathy may be controlled with long-term drug therapy and placement of a pacemaker/ defibrillator.
If the disease is diagnosed at an advanced stage, critically ill patients may require immediate lifesaving measures such as placement of a breathing tube (mechanical ventilator) and administration of medications intravenously (i.e. dobutamine, dopamine) to improve blood pressure and heart function. Once the patient has stabilized, therapy involving oral medication, implantable devices, surgery or heart transplantation will be considered.
A child's medical management should be done in consultation with a pediatric cardiologist, and possibly a cardiothoracic surgeon, electrophysiologist, and geneticist. It is important to do a comprehensive evaluation to pinpoint the cause so that proper preventative measures can be taken. Since each type of cardiomyopathy has distinct symptoms with a varying course, patients will require an individualized treatment plan for the acute and chronic management of the disease. Several factors will determine the required therapy such as the child's age, overall health, medical history, stage of the disease, and the child's tolerance for specific medications, procedures or therapies. The physician will also try to determine the extent of risk for 1) sudden death 2) predisposition to progressive symptoms 3) atrial fibrillations and 4) predisposition to end stage heart failure. Because children grow at such rapid rates, frequent visits to the cardiologist are essential for monitoring changes in the heart and preventing complications.
Every physician will try to restore a child's health to the best possible state with a minimum of interventional procedures. In general, the aim of medical therapy for a child with dilated cardiomyopathy is to 1) control symptoms of congestive heart failure, 2) improve heart function and contracting ability and 3) prevent complications such as blood clots or arrhythmias. Treatment specifics for a child with hypertrophic cardiomyopathy are somewhat similar in that the goal is to 1) control symptoms related to heart obstruction, 2) improve filling of heart chambers and 3) prevent arrhythmias and risk of sudden death.
Various medications will be given as the first line of treatment for the disease if symptoms are present. The choice and dosage of drugs will depend on each individual's diagnosis and may be modified over time as the child grows or responds to the medication. Basically drug therapy is used to relieve heart failure symptoms, decrease the workload on the heart, decrease the oxygen requirements of the heart and regulate abnormal heartbeats. Although drug therapy is noninvasive, there are some side effects and it requires careful monitoring to prevent other complications. It is important to note that many drug therapies are based on treatment of adult cardiomyopathy. There is still some debate about whether there is increased risk of toxicity and reduced effectiveness in children because they metabolize drugs differently than adults.
Patients with dilated cardiomyopathy may be given angiotensin converting enzyme (ACE) inhibitor or anti congestive medications such as captopril, enalapril and spironolactone. These medications relax the arteries in the body and decrease the amount of work the heart needs to pump blood to the body. Lasix and aldactone are common diuretics used to reduce excess fluid in the lungs or other organs. Digoxin is used to improve the pumping function of the heart and to prevent certain types of arrhythmia. Based on the long-term benefits found on adults, other medications such as beta-blockers and aldosterone may also be used in children. Some children with severe heart dysfunction may also require anticoagulation medications such as Warfarin to prevent blood clotting or anti-arrhythmic medications such as amiodarone for ventricular arrhythmias.
For patients with hypertrophic cardiomyopathy, the most serious problems are decreased heart filling, arrhythmia and obstruction to blood flow by the thickened heart muscle. Diuretics and digoxin are not usually used with HCM patients with obstruction because these drugs can worsen the obstruction of blood flow out of the heart. Instead, beta blockers and calcium channel blockers are prescribed to patients with moderate to severe obstruction. Medications such as propranolol and verapamil may be given to decrease the outflow obstruction by slowing the heart rate and relaxing the heart. Antiarrhythmic medications such as amiodarone and disopyramide may also be required to reduce the risk of sudden cardiac death.
Patients with restrictive cardiomyopathy are at a high risk for blood clots within the heart, particularly in the enlarged upper chambers. Anticoagulation medication or blood thinners such as heparin and coumadin may be prescribed along with a mild dose of diuretics to relieve venous congestion. Beta-blockers and diuretics are also often prescribed.
Arrhythmogenic Right Ventricular Cardiomyopathy
In the rare case that a child is diagnosed with arrhythmogenic right ventricular cardiomyopathy, medications to control arrhythmias and end stage congestive heart failure will be recommended.
Left Ventricular Non-Compaction Cardiomyopathy
For the child with left ventricular non-compaction cardiomyopathy (LVCM) that has symptoms of heart failure, medical treatment with diuretics, ACE inhibitors, and possibly beta-blockers may be used to alleviate symptoms and potentially help the heart muscle structure to normalize. If the patient shows signs of significant arrhythmia, antiarrhythmic medications such as amiodarone or sotalol, have been used for prevention of sudden death. In patients felt to be at risk for clot formation, anticoagulants such as aspirin are used. Patients with associated metabolic disorders may be given a mitochondrial metabolic disorder cocktail of Q10, thiamine, ribloflavin, and carnitine. Once LVCM is diagnosed, a neurological evaluation should be considered because of the high risk of associated neuromuscular disorders.
A pacemaker or defibrillator is used when drugs are not effective in alleviating obstruction or when dangerous arrhythmias need to be regulated. The procedure involves implanting a small mechanical device under the skin of the chest or abdomen with wire leads threaded through veins into the heart. It is considered minor surgery, requiring a short hospital stay. Once a pacemaker or defibrillator is inserted, it requires careful monitoring to determine when it needs to be replaced and to ensure that the electrical settings are correct. The decision to implant a pacemaker or defibrillator depends on the specific heart problem.
A pacemaker is used to monitor and stabilize slow heartbeats. Dual chamber pacing has been used in older children and adults with obstructive hypertrophic cardiomyopathy to decrease outflow obstruction. Although this procedure is not recommended for infants or small children, recent advances have allowed for single chamber pacing in younger children.
Automatic Implantable Cardioverter