Intrathecal Baclofen Therapy in Children
Since its introduction in the late 1980s, intrathecal baclofen (ITB) therapy has become the standard treatment for severe generalized spasticity and dystonia in children. Treatment with ITB decreases spasticity in the upper and lower extremities and has been associated with improved function and decreased musculoskeletal contractures. In addition, ITB decreases generalized secondary dystonia and has been associated with improved comfort and ease of care in approximately 85% and with improved function in approximately 33% of patients. Continued effectiveness of ITB in treating spasticity has been observed for up to 17 years, and its effectiveness in treating dystonia has been observed for up to 10 years. Although ITB therapy is frequently associated with complications such as infections, catheter malfunctions, and cerebrospinal fluid leaks, the benefits of therapy appear to outweigh the risks. Additional investigation is needed to determine the effects of ITB on other movement disorders such as athetosis and chorea.

Baclofen was initially synthesized in the 1920s as a GABA agonist to treat epilepsy. In the course of treating adults with epilepsy who also had spasticity, physicians observed that seizure frequency was minimally affected but that spasticity was decreased. Baclofen was given only orally until the mid-1980s, when Penn and Kroin first reported the use of ITB. Penn and colleagues subsequently confirmed its efficacy in a landmark doubleblind study. The first report of the use of ITB therapy to treat children was published in 1985 by Dralle, et al., who used it to treat a 4-year-old child who suffered from hypertonicity after a near-drowning. In 1991, we reported a double- blinded screening trial of ITB therapy in children with spastic CP, and confirmed the short-term effect of bolus injections on spasticity. In 1992, Müller reported that ITB improved spasticity in approximately 20 patients (children and young adults) younger than 20 years of age who were studied in a European multicenter trial. Since then, ITB therapy has been shown to diminish both spasticity and dystonia in children, and at times it improves function.

Although other medications such as clonidine and morphine have been given intrathecally to treat spasticity in adults, they have been used minimally in children, and evidence about their effectiveness is anecdotal.