Long-term outcomes of intraventricular baclofen therapy for medically refractory generalized secondary dystonia.

Abstract

Objective: Intrathecal baclofen (ITB) is commonly used to treat secondary generalized dystonia. Intraventricular baclofen (IVB) has been shown to be a safe alternative treatment with low complications. The objective of this study was to report the long-term effects of IVB.

Methods: This retrospective analysis included patients who underwent IVB therapy from April 2005 to June 2024. The decision to use IVB and the surgical technique have been previously described. Data collection included sex, race, etiology of dystonia, Gross Motor Functional Classification System scores, Barry-Albright Dystonia Scale (BADS) scores, Ashworth Scale scores, medical and surgical management of dystonia, follow-up duration, and complications. Patients whose IVB pump was removed within 1 year and those with less than 1 year of follow-up were excluded.

Results: Thirty-six patients with IVB were identified, and 27 patients (median age 12.9 years) were ultimately included. The most common cause of secondary dystonia was cerebral palsy in 21 patients (78%), followed by metabolic and neurodegenerative disorders in 4 (14%), infection in 1 (4%), and severe traumatic brain injury (TBI) in 1 (4%). The follow-up duration ranged from 1.2 to 16.7 years, with a median of 7.7 years and mean of 8.4 years. The baclofen dosage was twice as high in patients with metabolic and neurodegenerative disorders compared with those with cerebral palsy, TBI, and infection, with median dosages of 1455 μg/day and 725 μg/day, respectively. Both posttreatment BADS and Ashworth Scale scores showed statistically significant improvement. Complications included infection that necessitated pump removal in 1 patient (4%), wound dehiscence that was treated with wound revision and antibiotics in 1 patient (4%), and hydrocephalus that required CSF diversion in 4 patients (15%). In 2 patients, IVB therapy was ineffective at controlling dystonia and, thus, was discontinued after 1.3 and 2.7 years. One patient asked for the pump to be removed after 1.2 years due to "does not like the physical pump itself." Four patients (15%) developed hydrocephalus, which required CSF diversion via a ventriculoperitoneal shunt in 3 patients and a lumboperitoneal shunt in 1 patient, with no further shunt or IVB complications.

Conclusions: IVB is well tolerated in patients with generalized secondary dystonia refractory to conventional medical therapy, ITB, and deep brain stimulation. Positive long-term outcomes were reported in this cohort, with significant improvement in dystonia and overall complication rates similar to those reported with ITB.