"The Child Neurology Foundation is very pleased that the FDA has approved Acthar as a treatment for infantile spasms," said Lawrence W. Brown, MD, president of the Child Neurology Foundation, associate professor of neurology and pediatrics and director of the pediatric neuropsychiatry program at Children's Hospital of Philadelphia. "This is good news for children with this rare form of childhood epilepsy, as well as for parents and families. The FDA approval will also help standardize care for IS by providing specific guidelines for physicians on prescribing Acthar, the drug that has been the most widely-accepted initial approach for IS for more than 50 years." Lawrence Brown, MD Associate Professor of Neurology and Pediatrics Children's Hospital of Philadelphia President of the Child Neurology Foundation

In infantile spasms, the goal of treatment is to eliminate the spasms and hypsarrhythmia as quickly as possible. Awareness and proper identification can result in the selection of appropriate therapy that can improve a patient’s developmental outcomes. Click here to read the AAN (American Academy of Neurology) treatment guidelines on infantile spasms

Physician's Role

Parents often come to their pediatricians with concerns about their infant's development or odd movements; sometimes it is the health professional who first suspects a serious problem.

Once a proper diagnosis is made, treatment often includes the involvement of child neurologists, neurosurgeons, and other professionals including dieticians who specialize in epilepsy care.

Medication

There is no consensus on the best initial approach for infantile spasms. Many experts recommend a short course of hormonal therapy (ACTHar) but others use oral antiepileptic drugs (vigabatrin or topiramate) or oral steroids (prednisolone). H.P. ACTHar® Gel and Sabril® vigabatrin are the only FDA approved treatments for infantile spasms. Other approaches such as the ketogenic diet or valproate can be effective if the primary treatment fails.

H.P. ACTHar® Gel has a direct effect on the brain and may work by helping the body produce natural hormones with the goal of providing rapid resolution of spasms with clinically significant improvement in the chaotic EEG pattern (hypsarrhythmia) often times within days to weeks at approved dose . Potential side effects include infection, hypertension, irritability, and changes in appetite.

Vigabatrin works by increasing the availability of the main inhibitory neurotransmitter in the brain. While relatively safe and effective, there is a risk of permanent vision loss which must be balanced against the consequences of untreated spasms. Vigabatrin is especially effective in children with tuberous sclerosis.

Off-label use of oral steroids can be effective, but fewer children respond than to hormonal treatment. Side effects are similar to Acthar.

"There are a number of different strategies that have been used over the years to try to treat these spasms. Treatment includes medications, surgical interventions, such as taking out abnormal pieces of brain that might have developed aberrantly during development, or other non-pharmacologic treatments, such as the ketogenic diet, which is a non-drug form that can be very effective for treating some patients with spasms as well. No single therapeutic intervention has proven to be universally effective, so really the clinicians have to look at all of the treatment options and try to tailor them as much as possible to the particular patient with spasms." Jong Rho, MD Head, Division of Pediatric Neurology Alberta Children's Hospital

Diet and Non-Medication Treatment Options

Ketogenic diet: This form of therapy for IS has been around since the 1920s. The ketogenic diet is a high fat, low carbohydrate diet with sufficient protein for growth. Recent reports show that a ketogenic diet may help to control spasms in children who don’t respond to other medical therapies.

High-dose IV immunoglobulin: This form of treatment has been used to treat a number of seizure disorders and there is some evidence that it can be effective in treating cryptogenic (unknown cause) infantile spasms. Because of the limited available data in treating IS, this treatment option is usually considered only after other medical therapies have failed.

Surgery: When medication treatment options fail, cortical resection (removal of the brain abnormality) can potentially control seizures and improve developmental outcomes if a localized area of brain abnormality can be identified and safely removed without causing additional deficit.