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Ketogenic Diet Therapy for Drug-Resistant Epilepsy and Cognitive Impairment in Children With Tuberous Sclerosis
1Shenzhen Children's Hospital, China Medical University, Shenzhen, China.
Insights
The ketogenic diet (KD) effectively treats drug-resistant epilepsy and cognitive issues in children with Tuberous Sclerosis Complex (TSC). This safe therapy reduces seizures and may improve cognitive and behavioral outcomes in affected children.
Area of Science:
- Neurology
- Pediatrics
- Metabolic Disorders
Background:
- Tuberous Sclerosis Complex (TSC) is a genetic disorder associated with a high incidence of epilepsy and cognitive impairment in children.
- Conventional anti-seizure medications (ASMs) are often ineffective for TSC-related epilepsy, leading to the exploration of alternative therapies.
- The ketogenic diet (KD) has shown promise in managing refractory epilepsy in various pediatric populations.
Purpose of the Study:
- To evaluate the efficacy and safety of the ketogenic diet (KD) for treating drug-resistant epilepsy and cognitive impairment in children diagnosed with Tuberous Sclerosis Complex (TSC).
Main Methods:
- A multicenter retrospective study involving 53 children with TSC, drug-resistant epilepsy, or cognitive impairment.
- Intention-to-treat analysis was employed to assess seizure reduction and cognitive improvements following KD therapy over a 12-month period.
- Data were collected from January 1, 2010, to December 31, 2020.
Main Results:
- 51 out of 53 patients (96.2%) had previously failed an average of 5 ASMs.
- At 12 months, 31.4% of patients remained on KD, with 25.5% experiencing ≥50% seizure reduction and 5.9% achieving seizure freedom.
- Cognitive and behavioral improvements were observed in 70.6% of patients with psychomotor retardation.
- The most common side effects were gastrointestinal issues (37.7%) and hyperlipidemia (11.3%), which were manageable.
Conclusions:
- The ketogenic diet (KD) is a safe and effective therapeutic option for children with TSC experiencing drug-resistant epilepsy and cognitive impairment.
- KD therapy demonstrates significant potential in reducing seizure frequency and improving cognitive and behavioral outcomes in this patient population.
- The manageable side effect profile supports the consideration of KD as an adjunctive treatment for TSC.
Objective:
Tuberous sclerosis complex (TSC) is a rare disease with a high risk of epilepsy and cognitive impairment in children. Ketogenic diet (KD) therapy has been consistently reported to be beneficial to TSC patients. In this study, we aimed to investigate the efficacy and safety of KD in the treatment of drug-resistant epilepsy and cognitive impairment in children with TSC.
Methods:
In this multicenter study, 53 children (33 males and 20 females) with drug-resistant epilepsy or cognitive impairment caused by TSC were retrospectively recruited from 10 hospitals from January 1, 2010, to December 31, 2020. Intention-to-treat analysis was used to evaluate seizure reduction and cognition improvement as outcomes after KD therapy.
Results:
Of the 53 TSC patients included, 51 failed to be seizure-free with an average of 5.0 (range, 4-6) different anti-seizure medications (ASMs), before KD therapy. Although the other two patients achieved seizure freedom before KD, they still showed psychomotor development delay and electroencephalogram (EEG) abnormalities. At 1, 3, 6, and 12 months after the KD therapy, 51 (100%), 46 (90.2%), 35 (68.6%), and 16 patients (31.4%) remained on the diet therapy, respectively. At these time points, there were 26 (51.0%), 24 (47.1%), 22 (43.1%) and 13 patients (25.5%) having ≥50% reductions in seizure, including 11 (21.6%), 12 (23.5%), 9 (17.6%) and 3 patients (5.9%) achieving seizure freedom. In addition, of 51 patients with psychomotor retardation, 36 (36 of 51, 70.6%) showed cognitive and behavioral improvements. During the KD therapy, no serious side effects occurred in any patient. The most common side effects were gastrointestinal disturbance (20 of 53, 37.7%) and hyperlipidemia (6 of 53, 11.3%). The side effects were gradually relieved after adjustment of the ketogenic ratio and symptomatic treatment.
Conclusion:
KD is an effective and safe treatment for TSC-related drug-resistant epilepsy and cognitive impairment in children. KD can reduce seizure frequency and may potentially improve cognition and behavior.
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