The 4
Historical controls
MAS
Refractory SJIA
Refractory SJIA trial design
Journal
Pediatric rheumatology online journal
ISSN: 1546-0096
Titre abrégé: Pediatr Rheumatol Online J
Pays: England
ID NLM: 101248897
Informations de publication
Date de publication:
03 Jan 2024
03 Jan 2024
Historique:
medline:
4
1
2024
pubmed:
4
1
2024
entrez:
3
1
2024
Statut:
epublish
Résumé
The substantial morbidity and mortality associated with refractory systemic JIA underlies the need for new treatment approaches. However, progress in this area has been limited by the difficulty of enrolling these patients in clinical trials with traditional designs, particularly in patients presenting with the life-threatening macrophage activation syndrome. At the NextGen 2022 conference, there was group consensus that using historical cohorts as a control group to avoid the need for a placebo-arm or drug withdrawal was highly desirable and might be acceptable for clinical trials in MAS to support medication efficacy and safety. However, if historic controls were used in a trial, it would be important to ensure that the historic cohort matches the study group in terms of clinical characteristics (such as disease severity and exposure to other medications), and that disease outcome in both groups is assessed using the same outcome measures. The discussions at the NextGen 2022 conference focused on the potential strategies to achieve these goals.
Identifiants
pubmed: 38172909
doi: 10.1186/s12969-023-00866-z
pii: 10.1186/s12969-023-00866-z
doi:
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Pagination
150Informations de copyright
© 2023. The Author(s).
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