HIGHLIGHTS
- •Modular sets of biomarkers can be used in clinical practice and for research.
- •Common sets of biomarkers can improve data harmonization and sharing.
- •MS populations from different centres can be heterogeneous.
- •Multi-centre real-world studies need to be designed carefully.
ABSTRACT
Background
Worldwide multiple sclerosis (MS) centers have coordinated their efforts to use data
acquired in clinical practice for real-world observational studies. In this retrospective
study, we aim to harmonize outcome measures, and to evaluate their heterogeneity within
the Rising Italian Researchers in MS (RIReMS) study group.
Methods
RIReMS members filled in a structured questionnaire evaluating the use of different
outcome measures in clinical practice. Thereafter, thirty-four already-published papers
from RIReMS centers were used for heterogeneity analyses, using the DerSimonian and
Laird random-effects method to compute the between-study variance (τ2).
Results
Based on questionnaire results, we defined basic modules for diagnosis and follow-up,
consisting of outcome measures recorded by all participating centers at the time of
diagnosis, and, then, at least annually; we also defined more detailed/optional modules,
with outcome measures recorded less frequently and/or in the presence of specific
clinical indications. Looking at heterogeneity, we found 5-year variance in age at
onset (ES=27.34; 95%CI=26.18, 28.49; p<0.01; τ2=4.76), and 7% in female percent (ES=66.42; 95%CI=63.08, 69.76; p<0.01; τ2=7.15). EDSS variance was 0.2 in studies including patients with average age <36.1
years (ES=1.96; 95%CI=1.69, 2.24; p<0.01; τ2=0.19), or from 36.8 to 41.1 years (ES=2.70; 95%CI=2.39, 3.01; p<0.01; τ2=0.18), but increased to 3 in studies including patients aged >41.4 years (ES=4.37;
95%CI=3.40, 5.35; p<0.01; τ2=2.96). The lowest variance of relapse rate was found in studies with follow-up duration
≤2 years (ES=9.07; 95%CI=5.21, 12.93; p = 0.02; τ2=5.53), whilst the lowest variance in EDSS progression was found in studies with follow-up
duration >2 years (ES=5.41; 95%CI=3.22, 7.60; p = 0.02; τ2=1.00).
Discussion
We suggest common sets of biomarkers to be acquired in clinical practice, that can
be used for research purposes. Also, we provide researchers with specific indications
for improving inclusion criteria and data analysis, ultimately allowing data harmonization
and high-quality collaborative studies.
Keywords
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Article info
Publication history
Published online: July 11, 2020
Accepted:
July 11,
2020
Received in revised form:
June 25,
2020
Received:
May 5,
2020
Identification
Copyright
© 2020 Elsevier B.V. All rights reserved.
