Likelihood inference on the relative risk in split-cluster designs
Notice bibliographique
Résumé
BACKGROUND: Split-cluster experiments are being used by investigators in health sciences when naturally occurring aggregate of individuals with nested sub-groups may be assigned to different treatments. Cited examples include the split-mouth trials, in which a subject's mouth is divided into two segments that are randomly assigned to different treatment groups. In other situation, randomization to treatment conditions may be possible at the person level within the cluster. In this case, when the treatment conditions are available within each cluster, the design is referred to as a multisite or split-cluster design (SCD). The major attractiveness of this design is that it removes a large portion of the inter-subject variation from the estimates of the treatment effect; hence, it has the potential to require lesser number of measurements than a parallel arm design with the same power. When the response variable of interest is binary, statistical methods developed to evaluate the effect of interventions depended on nonparametric methods. Though these methods are simple to apply, they are known to be less efficient. METHODS: Taking the relative risk (RR) as an effect measure, we construct a bivariate-correlated model under which a score test is applied to test H(0): RR = 1.0. Moreover, we construct Wald- and Fieller-based confidence intervals on RR. Since the efficiency of SCD increases when the interclass correlation coefficient (ρ₁₂) is high, we present a goodness-of-fit procedure for testing H(0):ρ₁₂ = 0, which may be helpful in choosing a design for a future study. RESULTS: For illustrating the proposed methodology, we consider two application data from the published literature; the first from a split-mouth trial on 23 patients evaluating the effect of chlorhexidine in the treatment of gingivitis, and second from study of mental health (depression and anxiety) as outcome measure obtained on 173 patients evaluated by two screening instruments. Moreover, we discussed the efficiency gained using our approach in these design settings. LIMITATIONS: The likelihood approach makes more assumptions as compared to previous approaches that have been described. CONCLUSIONS: We have developed a bivariate beta-binomial model, from which we can conduct a full likelihood statistical inference. Based on this model, we may construct Wald's confidence intervals and score tests, which are known to possess optimal statistical properties. For the purpose of comparison with nonparametric methods, we constructed the Fieller's confidence interval.
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Prédiction distillée sur la base complète
Imitation des enseignantsNi prévalence calibrée, ni vérité terrain. Validation humaine à venir. Apprise à partir de 10 348 étiquettes directes de Codex et de 10 348 étiquettes directes de Gemma. Le mode candidate est l'union des têtes enseignantes seuillées; le consensus est leur intersection. Ces sorties portent le statut machine_predicted_unvalidated et ne sont ni des étiquettes humaines ni des étiquettes directes de modèles de pointe.
Scores Codex et Gemma par catégorie
| Catégorie | Codex | Gemma |
|---|---|---|
| Métarecherche | 0,157 | 0,925 |
| Méta-épidémiologie (sens strict) | 0,000 | 0,000 |
| Méta-épidémiologie (sens large) | 0,002 | 0,001 |
| Bibliométrie | 0,000 | 0,000 |
| Études des sciences et des technologies | 0,000 | 0,001 |
| Communication savante | 0,000 | 0,000 |
| Science ouverte | 0,001 | 0,000 |
| Intégrité de la recherche | 0,001 | 0,002 |
| Charge utile insuffisante (le modèle a refusé de juger) | 0,003 | 0,001 |
Scores machine (provisoires)
Les deux têtes enseignantes du modèle étudiant, lues sur ce travail. Un score ordonne la base pour la relecture; il n'affirme jamais une catégorie, et le statut de validation accompagne chaque rangée tel quel.
Scores de référence d'un modèle non mature (critères de maturité non atteints, 7 itérations). Un score ordonne; il n'affirme jamais une catégorie.
score_only:v0-immature-baseline · tel quel depuis la passe de notation : score_only signifie que le nombre peut ordonner les travaux, et qu'aucune étiquette de catégorie n'en découleClassification
machine, non validéePrédiction automatique; les deux têtes enseignantes s’accordent sur ce qui est montré ici.
Le détail, modèle par modèle et score par score, se trouve en fin de page sous « Comment cette classification a été obtenue ».