Use of the ketogenic diet to manage refractory epilepsy in CDKL5 disorder: Experience of >100 patients

Wiley Periodicals, Inc. © 2017 International League Against Epilepsy Objective: Pathogenic variants involving the CDKL5 gene result in a severe epileptic encephalopathy, often later presenting with features similar to Rett syndrome. Cardinal features of epilepsy in the CDKL5 disorder include early o...

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Main Authors: Lim, Z., Wong, K., Olson, H., Bergin, A., Downs, Jennepher, Leonard, H.
Format: Journal Article
Published: Wiley-Blackwell Publishing, Inc. 2017
Online Access:http://hdl.handle.net/20.500.11937/58453
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author Lim, Z.
Wong, K.
Olson, H.
Bergin, A.
Downs, Jennepher
Leonard, H.
author_facet Lim, Z.
Wong, K.
Olson, H.
Bergin, A.
Downs, Jennepher
Leonard, H.
author_sort Lim, Z.
building Curtin Institutional Repository
collection Online Access
description Wiley Periodicals, Inc. © 2017 International League Against Epilepsy Objective: Pathogenic variants involving the CDKL5 gene result in a severe epileptic encephalopathy, often later presenting with features similar to Rett syndrome. Cardinal features of epilepsy in the CDKL5 disorder include early onset at a median age of 6 weeks and poor response to antiepileptic drugs. The ketogenic diet (KD) was first introduced in the 1920s as a treatment option for refractory epilepsy in children. This study investigated use of the KD in the CDKL5 disorder and its influences on seizures. Methods: The International CDKL5 Disorder Database, established in 2012, collects information on individuals with the CDKL5 disorder. Families have provided information regarding seizure characteristics, use, and side effects of the KD treatment. Descriptive statistics and time to event analyses were performed. Clinical vignettes were also provided on patients attending Boston Children's Hospital. Results: Data regarding KD use were available for 204 individuals with a pathogenic CDKL5 variant. Median age of inclusion in the database was 4.8 years (range = 0.3–33.9 years), with median age of 6 weeks (range = 1 day–65 weeks) at seizure onset. History of KD use was reported for 51% (104 of 204) of individuals, with a median duration of use of 17 months (95% confidence interval = 9–24). Changes in seizure activity after commencing KD were reported for two-thirds (69 of 104), with improvements in 88% (61 of 69). Nearly one-third (31.7%) experienced side effects during the diet. At ascertainment, only one-third (32%) remained on the diet, with lack of long-term efficacy as the main reason for diet cessation (51%, 36 of 70). Significance: Benefits of KD in the CDKL5 disorder are in keeping with previous trials on refractory epilepsies. However, poor long-term efficacy remains as a significant barrier. In view of its side effect profile, KD administration should be supervised by a pediatric neurologist and specialist dietician.
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spelling curtin-20.500.11937-584532017-11-24T05:46:57Z Use of the ketogenic diet to manage refractory epilepsy in CDKL5 disorder: Experience of >100 patients Lim, Z. Wong, K. Olson, H. Bergin, A. Downs, Jennepher Leonard, H. Wiley Periodicals, Inc. © 2017 International League Against Epilepsy Objective: Pathogenic variants involving the CDKL5 gene result in a severe epileptic encephalopathy, often later presenting with features similar to Rett syndrome. Cardinal features of epilepsy in the CDKL5 disorder include early onset at a median age of 6 weeks and poor response to antiepileptic drugs. The ketogenic diet (KD) was first introduced in the 1920s as a treatment option for refractory epilepsy in children. This study investigated use of the KD in the CDKL5 disorder and its influences on seizures. Methods: The International CDKL5 Disorder Database, established in 2012, collects information on individuals with the CDKL5 disorder. Families have provided information regarding seizure characteristics, use, and side effects of the KD treatment. Descriptive statistics and time to event analyses were performed. Clinical vignettes were also provided on patients attending Boston Children's Hospital. Results: Data regarding KD use were available for 204 individuals with a pathogenic CDKL5 variant. Median age of inclusion in the database was 4.8 years (range = 0.3–33.9 years), with median age of 6 weeks (range = 1 day–65 weeks) at seizure onset. History of KD use was reported for 51% (104 of 204) of individuals, with a median duration of use of 17 months (95% confidence interval = 9–24). Changes in seizure activity after commencing KD were reported for two-thirds (69 of 104), with improvements in 88% (61 of 69). Nearly one-third (31.7%) experienced side effects during the diet. At ascertainment, only one-third (32%) remained on the diet, with lack of long-term efficacy as the main reason for diet cessation (51%, 36 of 70). Significance: Benefits of KD in the CDKL5 disorder are in keeping with previous trials on refractory epilepsies. However, poor long-term efficacy remains as a significant barrier. In view of its side effect profile, KD administration should be supervised by a pediatric neurologist and specialist dietician. 2017 Journal Article http://hdl.handle.net/20.500.11937/58453 10.1111/epi.13813 Wiley-Blackwell Publishing, Inc. restricted
spellingShingle Lim, Z.
Wong, K.
Olson, H.
Bergin, A.
Downs, Jennepher
Leonard, H.
Use of the ketogenic diet to manage refractory epilepsy in CDKL5 disorder: Experience of >100 patients
title Use of the ketogenic diet to manage refractory epilepsy in CDKL5 disorder: Experience of >100 patients
title_full Use of the ketogenic diet to manage refractory epilepsy in CDKL5 disorder: Experience of >100 patients
title_fullStr Use of the ketogenic diet to manage refractory epilepsy in CDKL5 disorder: Experience of >100 patients
title_full_unstemmed Use of the ketogenic diet to manage refractory epilepsy in CDKL5 disorder: Experience of >100 patients
title_short Use of the ketogenic diet to manage refractory epilepsy in CDKL5 disorder: Experience of >100 patients
title_sort use of the ketogenic diet to manage refractory epilepsy in cdkl5 disorder: experience of >100 patients
url http://hdl.handle.net/20.500.11937/58453