Understanding how the primary endpoint impacts the interpretation of trial results: The Japanese Adult Moyamoya bypass trial.
Extracranial-intracranial bypass
Hemorrhagic moyamoya
Moyamoya
Primary endpoint
Randomized trials
Research methodology
Journal
Neuro-Chirurgie
ISSN: 1773-0619
Titre abrégé: Neurochirurgie
Pays: France
ID NLM: 0401057
Informations de publication
Date de publication:
Mar 2023
Mar 2023
Historique:
received:
30
11
2022
accepted:
02
12
2022
medline:
28
3
2023
pubmed:
27
1
2023
entrez:
26
1
2023
Statut:
ppublish
Résumé
The meaning of a clinical trial depends to a large extent on the choice of the primary outcome measure, which can be explanatory or pragmatic. We review the Japanese Adult Moyamoya (JAM) trial, that compared surgical extracranial to intracranial (EC-IC) bypass and medical management of hemorrhagic moyamoya disease. We also review some principles which guide the selection of the primary trial endpoint. The main component of the primary outcome measure in JAM was rebleeding, a surrogate outcome that allowed investigators to demonstrate that surgical bypass had causal efficacy. However, the number of patients with a poor outcome, defined as those with a modified Rankin score (mRS)>2, would have been a more pragmatic choice. Unfortunately, the trial was too small to show that patients benefited from surgery. The JAM trial showed that EC-IC bypass can decrease rebleeding in moyamoya patients, but whether patients have better outcomes with surgery remains uncertain. Hard pragmatic clinical primary outcome measures are necessary to guide surgical care.
Sections du résumé
BACKGROUND
BACKGROUND
The meaning of a clinical trial depends to a large extent on the choice of the primary outcome measure, which can be explanatory or pragmatic.
METHODS
METHODS
We review the Japanese Adult Moyamoya (JAM) trial, that compared surgical extracranial to intracranial (EC-IC) bypass and medical management of hemorrhagic moyamoya disease. We also review some principles which guide the selection of the primary trial endpoint.
DISCUSSION
CONCLUSIONS
The main component of the primary outcome measure in JAM was rebleeding, a surrogate outcome that allowed investigators to demonstrate that surgical bypass had causal efficacy. However, the number of patients with a poor outcome, defined as those with a modified Rankin score (mRS)>2, would have been a more pragmatic choice. Unfortunately, the trial was too small to show that patients benefited from surgery.
CONCLUSION
CONCLUSIONS
The JAM trial showed that EC-IC bypass can decrease rebleeding in moyamoya patients, but whether patients have better outcomes with surgery remains uncertain. Hard pragmatic clinical primary outcome measures are necessary to guide surgical care.
Identifiants
pubmed: 36701981
pii: S0028-3770(23)00005-X
doi: 10.1016/j.neuchi.2023.101408
pii:
doi:
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Pagination
101408Commentaires et corrections
Type : CommentIn
Informations de copyright
Copyright © 2023. Published by Elsevier Masson SAS.