Deep Brain Stimulation in Pediatric Populations: A Scoping Review of the Clinical Trial Landscape

There has been rapid advancement in the development of deep brain stimulation (DBS) as a treatment option for adults for neurological and neuropsychiatric conditions. Here, we present a scoping review of completed and ongoing clinical trials focused on DBS in pediatric populations, highlighting key knowledge gaps.

Three databases (PubMed, OVID, and Embase) and the clinicaltrials.gov registry were queried to identify clinical trials for DBS in pediatric cohorts (age ≤18 years). Prospective and retrospective case series were excluded. No restrictions were placed on the diagnoses or measured clinical outcomes. Individual patient demographics, diagnosis, DBS target, and primary endpoints were extracted and summarized.

A total of 13 clinical trials were included in the final review, consisting of 9 completed trials (357 screened) and 4 ongoing trials (82 screened). Of the completed trials, 6 studied dystonia (both inherited and acquired; participants aged 4-18 years) and 3 studied drug-resistant epilepsy (participants aged 4-17 years). Among the 6 trials for dystonia, 5 used the Burke-Fahn-Marsden Dystonia Rating Scale (BFMDRS) as the primary endpoint. There were a total of 18 adverse events documented across 63 participants, with 5 of 9 studies reporting adverse events. Ongoing clinical trials are evaluating DBS for dystonia (N = 2), epilepsy (N = 1), and self-injurious behavior (N = 1).

This scoping review summarizes the landscape of clinical trials for DBS in children and youth. In dystonia, further research is warranted with more relevant pediatric outcome measures and for understudied patient subgroups and targets. There are also significant gaps in our understanding of evaluating the role of DBS in other neurological and neurodevelopmental disorders in pediatric populations.

There has been rapid advancement in the development of deep brain stimulation (DBS) as a treatment option for adults for neurological and neuropsychiatric conditions. Here, we present a scoping review of completed and ongoing clinical trials focused on DBS in pediatric populations, highlighting key knowledge gaps.

Three databases (PubMed, OVID, and Embase) and the clinicaltrials.gov registry were queried to identify clinical trials for DBS in pediatric cohorts (age ≤18 years). Prospective and retrospective case series were excluded. No restrictions were placed on the diagnoses or measured clinical outcomes. Individual patient demographics, diagnosis, DBS target, and primary endpoints were extracted and summarized.

A total of 13 clinical trials were included in the final review, consisting of 9 completed trials (357 screened) and 4 ongoing trials (82 screened). Of the completed trials, 6 studied dystonia (both inherited and acquired; participants aged 4-18 years) and 3 studied drug-resistant epilepsy (participants aged 4-17 years). Among the 6 trials for dystonia, 5 used the Burke-Fahn-Marsden Dystonia Rating Scale (BFMDRS) as the primary endpoint. There were a total of 18 adverse events documented across 63 participants, with 5 of 9 studies reporting adverse events. Ongoing clinical trials are evaluating DBS for dystonia (N = 2), epilepsy (N = 1), and self-injurious behavior (N = 1).

This scoping review summarizes the landscape of clinical trials for DBS in children and youth. In dystonia, further research is warranted with more relevant pediatric outcome measures and for understudied patient subgroups and targets. There are also significant gaps in our understanding of evaluating the role of DBS in other neurological and neurodevelopmental disorders in pediatric populations.