Exploring the relationships between composite scores of disease severity, seizure-freedom and quality of life in Dravet syndrome.
Composite endpoint
Dravet syndrome
Quality of life
Seizures
Statistical analysis
Symptoms
Journal
Neurological research and practice
ISSN: 2524-3489
Titre abrégé: Neurol Res Pract
Pays: England
ID NLM: 101767802
Informations de publication
Date de publication:
06 Jun 2022
06 Jun 2022
Historique:
received:
10
04
2022
accepted:
02
05
2022
entrez:
6
6
2022
pubmed:
7
6
2022
medline:
7
6
2022
Statut:
epublish
Résumé
In Dravet syndrome (DS), a rare epileptic and developmental encephalopathy, the effectiveness of a new treatment is predominantly measured in terms of seizure frequency. However, this may not fully capture the impact of a treatment on the broader aspects of the syndrome and patients' health-related quality of life (HRQoL). Using a previously published survey which collected data from DS patients and their carers on the broader manifestations of their syndrome, their HRQoL, and their experience of seizures, this study created composite measures of symptom severity to offer new perspectives on the multifaceted aspects of this rare condition. Survey responses on the severity of physical and psychosocial symptoms were combined with independent assessments of disability and care need, to generate three composite symptom scores assessing the manifestations of DS (physical, psychosocial and care requirements). Variation in HRQoL was investigated in multiple regression analyses to assess the strength of association between each of these composite measures and three forms of seizure measures (seizure frequency, days with no seizures and longest interval without seizures), as experienced over a 4- and 12-week period. Composite scores were calculated for a cohort of 75 primarily paediatric patients who were enrolled in the study. Strong associations were found between each of the three composite symptom scores and each of the three seizure measures, with the regression coefficient on symptom score highly significant (p ≤ 0.001) in all nine comparisons. Separate regressions using predictors of HRQoL (Kiddy KINDL and Kid KINDL) as the dependent variable were inconclusive, identifying only behavioural/attention problems and status epilepticus as significant predictors of HRQoL. These results allow the development of a composite score that may be useful in developing a clinical understanding of the severity of DS for an individual patient and establishing their treatment goals. Where measurement of long-term sequalae of disease is not feasible, such as clinical trials, correlation of the composite score with experience of seizures and seizure-free periods may allow a better contextualisation of the results of short-term assessments. German Clinical Trials Register (DRKS), DRKS00011894. Registered 16 March 2017, http://www.drks.de/ DRKS00011894.
Sections du résumé
BACKGROUND
BACKGROUND
In Dravet syndrome (DS), a rare epileptic and developmental encephalopathy, the effectiveness of a new treatment is predominantly measured in terms of seizure frequency. However, this may not fully capture the impact of a treatment on the broader aspects of the syndrome and patients' health-related quality of life (HRQoL). Using a previously published survey which collected data from DS patients and their carers on the broader manifestations of their syndrome, their HRQoL, and their experience of seizures, this study created composite measures of symptom severity to offer new perspectives on the multifaceted aspects of this rare condition.
METHODS
METHODS
Survey responses on the severity of physical and psychosocial symptoms were combined with independent assessments of disability and care need, to generate three composite symptom scores assessing the manifestations of DS (physical, psychosocial and care requirements). Variation in HRQoL was investigated in multiple regression analyses to assess the strength of association between each of these composite measures and three forms of seizure measures (seizure frequency, days with no seizures and longest interval without seizures), as experienced over a 4- and 12-week period.
RESULTS
RESULTS
Composite scores were calculated for a cohort of 75 primarily paediatric patients who were enrolled in the study. Strong associations were found between each of the three composite symptom scores and each of the three seizure measures, with the regression coefficient on symptom score highly significant (p ≤ 0.001) in all nine comparisons. Separate regressions using predictors of HRQoL (Kiddy KINDL and Kid KINDL) as the dependent variable were inconclusive, identifying only behavioural/attention problems and status epilepticus as significant predictors of HRQoL.
CONCLUSIONS
CONCLUSIONS
These results allow the development of a composite score that may be useful in developing a clinical understanding of the severity of DS for an individual patient and establishing their treatment goals. Where measurement of long-term sequalae of disease is not feasible, such as clinical trials, correlation of the composite score with experience of seizures and seizure-free periods may allow a better contextualisation of the results of short-term assessments.
TRIAL REGISTRATION
BACKGROUND
German Clinical Trials Register (DRKS), DRKS00011894. Registered 16 March 2017, http://www.drks.de/ DRKS00011894.
Identifiants
pubmed: 35659154
doi: 10.1186/s42466-022-00186-9
pii: 10.1186/s42466-022-00186-9
pmc: PMC9169336
doi:
Types de publication
Journal Article
Langues
eng
Pagination
22Informations de copyright
© 2022. The Author(s).
Références
Neurol Res Pract. 2021 Jun 28;3(1):35
pubmed: 34176514
Adv Neurol. 2005;95:71-102
pubmed: 15508915
Epilepsy Behav. 2019 Sep;98(Pt A):88-95
pubmed: 31301455
Cureus. 2019 Jun 26;11(6):e5006
pubmed: 31497436
Epilepsia. 2019 Aug;60(8):1697-1710
pubmed: 31247127
Epilepsy Behav. 2019 Jan;90:217-227
pubmed: 30578097
Eur J Paediatr Neurol. 2021 Nov;35:111-122
pubmed: 34673401
Epilepsy Behav. 2018 Jan;78:232-242
pubmed: 29108913
Bundesgesundheitsblatt Gesundheitsforschung Gesundheitsschutz. 2007 May-Jun;50(5-6):810-8
pubmed: 17514467
Expert Rev Neurother. 2020 Oct;20(10):1065-1079
pubmed: 32799683
Dev Med Child Neurol. 2011 Apr;53 Suppl 2:1-6
pubmed: 21504424
Eur J Paediatr Neurol. 2019 May;23(3):392-403
pubmed: 30871879
Can J Neurol Sci. 2016 Jun;43 Suppl 3:S13-8
pubmed: 27264138
Epilepsia. 2011 Aug;52(8):1476-82
pubmed: 21668444
Epilepsy Behav. 2020 Jul;108:107094
pubmed: 32375095
Dev Med Child Neurol. 2019 Oct;61(10):1229-1236
pubmed: 30828793
Dev Med Child Neurol. 2018 Jan;60(1):63-72
pubmed: 28984349
J Child Neurol. 2008 Jun;23(6):690-4
pubmed: 18344453