Authors :
Presenting Author: Matt Lallas, MD – Nicklaus Children’s Hospital
MARYTERY FAJARDO, MD – Nicklaus Children's Hospital
Trevor Resnick, MD – Nicklaus Children's Hospital Comprehensive Epilepsy Center
Michael Duchowny, MD – Nicklaus Children's Hospital
Prasanna Jayakar, MD, PhD – Nicklaus Children's Hospital
Nolan Altman, MD – Nicklaus Children's Hospital
MAGNO GUILLEN, PhD – NICKLAUS CHILDRENS HOSPITAL
John Ragheb, MD – Nicklaus Children's Hospital
Rationale:
Epilepsy due hypothalamic hamartoma (HH) represents a surgically treatable source of epileptic encephalopathy. As part of a study of the safety of magnetic resonance guided focused ultrasound ablation (FUS) in children with benign, central brain lesions, several patients with HH related epilepsy were treated. This report describes the subset of patients who had epilepsy and their response to FUS.Methods:
An original cohort of 10 patients were in the prospective, single arm MRI guided focused ultrasound ablation trial from 2018 to 2024 (NCT03028246) were treated using the Insightec ExAblate 4000 protocol, nine patients treated at Nicklaus Children’s Hospital, Miami, FL, and one at Children’s National Medical Center. Clinical and imaging data regarding the benign lesions and associated features were collected as part of the study. Eight patients were treated for HH. The surgical history and clinical response were reviewed for patients with HH and epilepsy associated. Results:
Six patients with both HH and Epilepsy, age 13-22 years, were treated by FUS. We have previously published three of these patients. Of the six patients, one had no prior surgery while five had prior surgery. One was seizure free from a single laser interstitial thermal therapy (LITT), though with residual encephalopathy, and the remaining four continued to have seizures as the primary concern. Two patients had only one prior surgery, including one patient who received LITT, and another who received and endoscopic transventricular resection of the HH. The other two patients received two prior surgeries, including one patient who underwent LITT twice, and another who underwent Gamma Knife radiosurgery and LITT. Of the five patients with active seizures at the time of FUS, all had a 90% or greater reduction in seizures at last follow up while three were seizure free. None had any new hypothalamic dysfunction, though pre-existing dysfunction remained. In these six patients, five were reported to have better mood, increased focus, or improved school performance.
The study is limited in that the protocol’s primary outcomes were not specific to epilepsy outcomes, but rather on safety and tolerability of the procedure in benign, centrally located brain lesions. Seizure diaries were not collected, and EEG features were not collected in a standardized manner as part of the protocol.Conclusions: This data provides an important first proof of principal that MRgFUS was well tolerated and provided beneficial seizure control and behavioral improvement in the small number of patients. MRgFUS ablation in patients with HH related epilepsy has the potential to become a preferred method of treatment if future prospective studies with larger cohorts can demonstrate similar seizure and behavioral outcomes to those described here.
Funding:
The Focused Ultrasound Foundation (grant no. FUS 530 to T.S.T.) funded this trial (clinicaltrials.gov identifier no.NCT03028246). The device manufacturer, InSightec Ltd., is our FDA regulatory sponsor