Development and content validation of a preliminary core set of patient- and caregiver-relevant outcomes for inclusion in a potential composite endpoint for Dravet Syndrome

Nabbout, Rima; Auvin, Stéphane; Chiron, Catherine; Irwin, J. David; Mistry, Arun; Bonner, Nicola; Williamson, Nicola; Bennett, Bryan

doi:10.1016/j.yebeh.2017.08.029

Cited by 55 publications

(83 citation statements)

References 43 publications

(63 reference statements)

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“…12,14 These findings emphasize that a biopsychosocial approach to treatment should be developed for Dravet syndrome, with the implementation and evaluation of combined pharmacological and psychosocial interventions. impacts not directly attributable to the condition) to be as important as the direct impact of seizures, and that Dravet syndrome affected multiple domains of their lives.…”

Section: Discussionmentioning

confidence: 97%

“…In the previous study by Nabbout et al 14 identified previously are still relevant and should be measured in future clinical trials on Dravet syndrome. A selected endpoint model must paint a comprehensive picture of the condition, incorporating aspects such as symptoms and the impact on the child and the caregiver.…”

Section: Discussionmentioning

confidence: 98%

“…After interview analyses, conceptual mapping was performed using the French conceptual model 14 and concepts emerging from this study. The conceptual model details key concepts (i.e.…”

Section: Discussionmentioning

confidence: 99%

“…14 • Indirect seizure impacts were reported to be as important as direct impacts. 14 • Indirect seizure impacts were reported to be as important as direct impacts.…”

Section: Data Collectionmentioning

confidence: 99%

“…Research into the impact of Dravet syndrome on both patients and caregivers is increasing, 13 including recent work conducted by Nabbout et al 14 This small-scale study explored the wider impact of Dravet syndrome in both children and their caregivers, using qualitative interviews. Its aim was to create a broader picture of the condition beyond seizures and to identify potential endpoints that should be considered in devising a future composite endpoint for Dravet syndrome clinical trials.…”

mentioning

confidence: 99%

See 4 more Smart Citations

Perception of impact of Dravet syndrome on children and caregivers in multiple countries: looking beyond seizures

Nabbout

Auvin

Chiron

et al. 2019

Develop Med Child Neuro

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Section: Discussionmentioning

confidence: 97%

Section: Discussionmentioning

confidence: 98%

“…After interview analyses, conceptual mapping was performed using the French conceptual model 14 and concepts emerging from this study. The conceptual model details key concepts (i.e.…”

Section: Discussionmentioning

confidence: 99%

“…14 • Indirect seizure impacts were reported to be as important as direct impacts. 14 • Indirect seizure impacts were reported to be as important as direct impacts.…”

Section: Data Collectionmentioning

confidence: 99%

mentioning

confidence: 99%

See 3 more Smart Citations

Perception of impact of Dravet syndrome on children and caregivers in multiple countries: looking beyond seizures

Nabbout

Auvin

Chiron

et al. 2019

Develop Med Child Neuro

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Core Outcome Sets in Child Health

Lei

Shen

Yang³

et al. 2022

JAMA Pediatr

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ImportanceDeveloping core outcome sets is essential to ensure that results of clinical trials are comparable and useful. A number of core outcome sets in pediatrics have been published, but a comprehensive in-depth understanding of core outcome sets in this field is lacking.ObjectiveTo systematically identify core outcome sets in child health, collate the diseases to which core outcome sets have been applied, describe the methods used for development and stakeholder participation, and evaluate the methodological quality of existing core outcome sets.Evidence ReviewMEDLINE, SCOPUS, Cochrane Library, and CINAHL were searched using relevant search terms, such as clinical trials, core outcome, and children, along with relevant websites, such as Core Outcome Measures in Effectiveness Trials (COMET). Four researchers worked in teams of 2, performed literature screening and data extraction, and evaluated the methodological quality of core outcome sets using the Core Outcome Set–Standards for Development (COS-STAD).FindingsA total of 77 pediatric core outcome sets were identified, mainly developed by organizations or researchers in Europe, North America, and Australia and mostly from the UK (22 [29%]) and the US (22 [29%]). A total of 77 conditions were addressed; the most frequent International Classification of Diseases, 11th Revision category was diseases of the digestive system (14 [18%]). Most of the outcomes in pediatric core outcome sets were unordered (34 [44%]) or presented in custom classifications (29 [38%]). Core outcome sets used 1 or more of 8 development methods; the most frequent combination of methods was systematic review/literature review/scoping review, together with the Delphi approach and consensus for decision-making (10 [14%]). Among the 6 main types of stakeholders, clinical experts were the most frequently involved (74 [100%]), while industry representatives were rarely involved (4 [5%]). Only 6 core outcome sets (8%) met the 12 criteria of COS-STAD.Conclusions and RelevanceFuture quality of pediatric core outcome sets should be improved based on the standards proposed by the COMET initiative, while core outcome sets methodology and reporting standards should be extended to pediatric populations to help improve the quality of core outcome sets in child health. In addition, the COMET outcome taxonomy should also add items applicable to children.

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A European pilot study in Dravet Syndrome to delineate what really matters for the patients and families

et al. 2023

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We aimed to identify caregivers' opinions on the outcome measures that matter in clinical trials in individuals with Dravet syndrome (DS). We conducted a prospective European multicenter study based on an 11 closed questions survey developed by the French reference center for rare epilepsies and DS patients’ advocacy groups. Items included questions on seizures and daily life outcomes that a clinical trial on a therapy for individuals with DS should target. Statistical analyses were performed to evaluate the impact of the country of residence and of the patients’ age. The survey was answered by 153 caregivers (68%: France, 28%: Germany, and 24%: Italy) off individuals with DS. Individuals with DS included 86 males (mean age of 11.4 [interquartile: 7‐20.4] years). Families ranked as important almost all the items proposed. However, items related to daily life had the highest rank in all three countries compared to items about seizures (P = 0.02). Increase in individuals’ age was associated with a higher age at diagnosis (ρ = 0.26, P = 0.02), and a lower impact of seizure duration (ρ = −0.25, P = 0.005) and on the need of hospital referral (ρ = −0.26, P = 0.005). These data can help tailor patient‐centered outcome measures in future clinical and real‐life trials for DS.

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Development and content validation of a preliminary core set of patient- and caregiver-relevant outcomes for inclusion in a potential composite endpoint for Dravet Syndrome

Abstract: This study showed the wider potential impact of DS to extend beyond that of seizures, demonstrating that there is a need for additional patient- and caregiver-relevant concepts to be measured in clinical trials to fully identify the value of therapeutic interventions.

Cited by 55 publications

References 43 publications

Perception of impact of Dravet syndrome on children and caregivers in multiple countries: looking beyond seizures

Perception of impact of Dravet syndrome on children and caregivers in multiple countries: looking beyond seizures

Core Outcome Sets in Child Health

A European pilot study in Dravet Syndrome to delineate what really matters for the patients and families

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