Safety, feasibility, and outcomes of deep brain stimulation in young children excluded from the FDA Humanitarian Device Exemption: analysis of the CHILD-DBS registry.
In the US, deep brain stimulation (DBS) is accessible to pediatric patients with dystonia who are 7 years of age and older through an FDA Humanitarian Device Exemption (HDE). The aim of this study was to assess the safety, feasibility, and outcomes of DBS for dystonia in children younger than 7 years of age who are excluded from the FDA HDE.
Data were collected (February 2015-December 2025) through The Child & Youth Comprehensive Longitudinal Database for DBS, which is a prospective registry of pediatric DBS from 5 tertiary pediatric hospitals in North America. Participants younger than 7 years of age who underwent DBS targeting the globus pallidus internus for the treatment of dystonia were included. Demographics, operative details, and postoperative outcomes and complications were analyzed.
Twelve children underwent 14 DBS-related surgeries, with a mean follow-up duration of 2.66 years (range 0.50-10.85 years). The mean age at the time of surgery was 4.72 years (range 2.96-6.83 years), and the mean weight was 16.5 kg (range 9.3-30.8 kg). Half of the children had prior ICU admissions for status dystonicus, and 9 DBS surgeries (64.3%) were performed urgently. Three children had transgression of stereotactic frame pins beyond the inner table of the skull, while 4 other children had wound complications requiring surgical intervention. No patients experienced neurological sequelae, and those requiring explantation of infected hardware were reimplanted successfully once the infections were treated. Overall, there was a mean reduction of 40.1% in the Burke-Fahn-Marsden movement subscale at 6 months postoperatively (p < 0.05), which persisted to 12 months. Three children died due to underlying conditions > 6 months from their respective surgeries.
DBS had demonstrable benefits in children younger than 7 years of age with severe refractory dystonia, albeit with higher surgical risks compared with older children. Further research is warranted to better inform treatment decisions and regulatory oversight.
Data were collected (February 2015-December 2025) through The Child & Youth Comprehensive Longitudinal Database for DBS, which is a prospective registry of pediatric DBS from 5 tertiary pediatric hospitals in North America. Participants younger than 7 years of age who underwent DBS targeting the globus pallidus internus for the treatment of dystonia were included. Demographics, operative details, and postoperative outcomes and complications were analyzed.
Twelve children underwent 14 DBS-related surgeries, with a mean follow-up duration of 2.66 years (range 0.50-10.85 years). The mean age at the time of surgery was 4.72 years (range 2.96-6.83 years), and the mean weight was 16.5 kg (range 9.3-30.8 kg). Half of the children had prior ICU admissions for status dystonicus, and 9 DBS surgeries (64.3%) were performed urgently. Three children had transgression of stereotactic frame pins beyond the inner table of the skull, while 4 other children had wound complications requiring surgical intervention. No patients experienced neurological sequelae, and those requiring explantation of infected hardware were reimplanted successfully once the infections were treated. Overall, there was a mean reduction of 40.1% in the Burke-Fahn-Marsden movement subscale at 6 months postoperatively (p < 0.05), which persisted to 12 months. Three children died due to underlying conditions > 6 months from their respective surgeries.
DBS had demonstrable benefits in children younger than 7 years of age with severe refractory dystonia, albeit with higher surgical risks compared with older children. Further research is warranted to better inform treatment decisions and regulatory oversight.
Authors
Jung Jung, Molot-Toker Molot-Toker, Dinger Dinger, Balachandar Balachandar, Taha Taha, Mithani Mithani, Breitbart Breitbart, Huynh Huynh, Kisteroff Kisteroff, Sriharan Sriharan, Vogt Vogt, LeBlanc-Millar LeBlanc-Millar, Ebrahimi-Fakhari Ebrahimi-Fakhari, Yang Yang, Ruppert-Gomez Ruppert-Gomez, Stone Stone, Northam Northam, Gadgil Gadgil, Raskin Raskin, Weil Weil, Hadjinicolaou Hadjinicolaou, Meijer Meijer, Fasano Fasano, Gorodetsky Gorodetsky, Ibrahim Ibrahim
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