UCP Research and Educational Foundation Comments: Regarding recent FDA Public Notification concerning adverse events reported in children with CP who have received treatment with Botulinum Toxin injections

Concerns about the safety of Botox injections to treat spasticity in individuals with CP have recently been raised. It has not been established whether these adverse events are causally related to Botox treatment. Serious adverse events have been reported in 4 children with cerebral palsy. The FDA is currently reviewing safety data from clinical studies submitted by the drug's manufacturers, as well as post-marketing adverse event reports and medical literature. The FDA is not advising health care professionals to discontinue prescribing these products.

After completing a review of the data, the FDA will communicate to the public its conclusions, resulting recommendations and any regulatory actions. In the interim, the FDA is not advising health care professionals to discontinue prescribing these products. UCPREF is closely monitoring this situation and will alert the CP community of any new developments.

Background

The severe adverse events resulting in death reported in the United States occurred in three children with Cerebral Palsy. All involved some degree of aspiration, respiratory infections, and other breathing complications; all 3 children had a history of respiratory compromise and dysphagia prior to treatment with BOTOX. It is unknown if treatment with Botox was causal in their deaths.

Botox use has been approved for the treatment of limb spasticity in individuals with CP in approximately 60 countries since 1995. While not approved in the US, highly trained physicians have been effectively treating spasticity (severe arm and leg muscle spasms) with Botox over the last 15 years.

The FDA is aware of the body of literature describing the use of botulinum toxins to treat limb spasticity in children and adults with CP.

Comments

Botox has been a very helpful and effective method for treating children and adults with disabling spasticity and dystonia for many years. Those being treated by a qualified and experienced physician should discuss their concerns with their physician, but there is no indication that treatment should be discontinued, and the product is not being "recalled".

It is critical that Botox only be administered by highly trained physicians who understand and fully explain the risks to patients and their care-givers. It is also important that physicians make patients and care-givers fully aware of the signs and symptoms of botulism. The symptoms include difficulty in swallowing, weakness, difficulty in breathing, difficulty in speaking. Patients and care-givers should also understand that these side effects can occur as early as one day and as late as several weeks after a Botox injection and that if they have any of these symptoms they should seek medical attention immediately.

Serious adverse events should be reported to FDA's MedWatch reporting system by completing a form on line at http://www.fda.gov/medwatch/report/hcp.htm, by faxing (1-800-FDA-0178), by mail using the postage-paid address form provided online (5600 Fishers Lane, Rockville, MD 20853-9787), or by telephone (1-800-FDA-1088).

Please contact Mindy Aisen at UCPREF with any questions - maisen@ucpresearch.org