Signori (2024)

Multiple sclerosis

Neurology • Registry Data • Italy, Australia, Turkey, Czech Republic, Kuwait, Switzerland, Spain, Lebanon, Canada

Study Metrics
Total Sample 4376
Treatment Group 3236
Control Group 1140
Covariates 7
PICO Comparisons 1
Quality Indicators
Transparency High
DAG Usage No
QBA Performed No

Study Information

First Author: Signori
Publication Year: 2024
DOI: https://doi.org/10.1136/jnnp-2023-332603
Preprint: No preprint

Institution & Funding

Institution Type: Academic
Institutions: University of Genova, University of Melbourne, Dokuz Eylul University, Charles University, Amiri Hospital, University of Catania, University Hospital Basel, Hospital Universitario Virgen Macarena, American University of Beirut Medical Center, Hacettepe University, Hotel-Dieu de Levis, Université de Montréal, Monash University, Alfred Hospital, Ospedale Policlinico San Martino
Funding: Declared: None
Funding Institutions: The authors have not declared a specific grant for this research from any funding agency in the public, commercial or not-­for-profit sectors.

Study Context

Disease: Multiple sclerosis
Disease Category: Neurology
Data Type: Registry
Number of Data Sources: 1
Geography: Italy, Australia, Turkey, Czech Republic, Kuwait, Switzerland, Spain, Lebanon, Canada
Eligible Sample: 4376.0
Number of Emulations: 1
Number of Treatments: 2

Analytical Methods

Matching Method: 1:1 PS matching
Analysis Method: Negative binomial regression, standard and ordinal logistic regression, linear regression

Quality Methods

Directed Acyclic Graph (DAG) Not Used
Quantitative Bias Analysis (QBA) Not Performed

Target Trial Information

Target Trial Name: TRANSFORMS
Registration Number: NCT00340834
Target Trial DOI: https://doi.org/10.1056/NEJMoa0907839

TTE vs RCT Comparisons

Detailed comparison between Target Trial Emulation results and corresponding Randomized Controlled Trial outcomes.

Annual relapse rate
TRANSFORMS
RR Efficacy
Population

Patients aged 18-55 years with relapsing-remitting multiple sclerosis (RRMS)

Intervention

Fingolimod (FTY) 0.5mg daily

Comparison

Intramuscular interferon β-1a (IFN) 30μg weekly

Outcome

Annual relapse rate

RCT Result

0.49

95% CI: [0.37, 0.64]


vs
TTE Result

0.55

95% CI: [0.45, 0.68]

Concordance Assessment
Confidence Interval Overlap: Yes
CIs overlap, suggesting concordance
Direction Agreement: Same Direction
Both point to similar conclusion

Transparency Indicators

Protocol Registration Available
Data Sharing Not Available
Code Sharing Not Available
Overall Transparency Score:
High Transparency - This study meets good transparency standards.

Conflicts & Funding

Conflicts of Interest: Declared: Financial
COI Institutions: Chiesi, BMS, Roche, Janssen, Sanofi Genzyme, Novartis, Merck, Biogen, WebMD Global, Eisai, BioCSL, Celgene, Teva, Bayer, GSK, Almirall, EMD Serono, Alexion, CSL, Pendopharm, Geneuro, Immunic, Medday
Funding Source: Declared: None
Funding Institutions: The authors have not declared a specific grant for this research from any funding agency in the public, commercial or not-­for-profit sectors.