A method to compare prospective and historical cohorts to evaluate drug effects. Application to the analysis of early treatment effectiveness of intramuscular interferon-β1a in multiple sclerosis patients

BREMSO-group

Research output: Contribution to journalArticle

Abstract

BACKGROUND: Disease modifying therapy have changed the natural evolution of multiple sclerosis (MS), with efficacy demonstrated in randomized clinical trials. Standard-of-care effectiveness is needed to complement clinical trial data and highlight outcomes in real-world practice, but comparing prospective patients with historical cohorts likely introduces biases. To address these potential biases, assigning a patient with a score that expresses his/her disease prognosis before starting a therapy may make it possible to evaluate the unbiased ability of the therapy to modify disease natural history. Thus, we aimed at analyzing the effectiveness of intramuscular interferon-β1a (im IFN-β1a) matching by BREMSO score (Bayesian Risk Estimate for Multiple Sclerosis at Onset) a prospective real-world cohort of treated patients with a historical cohort of untreated patients.

MATERIAL AND METHODS: We observed 108 newly diagnosed, treatment naïve MS patients over 12 months of treatment with im IFN-β1a. BREMSO score was used to assign a value to each patient, giving the real-world treated patients comparable with the Historical untreated patients, on the basis of the same risk to have unfavorable evolution.

RESULTS: A significantly higher percentage of relapse-free patients is observed in IFN-β1a treated cohort vs. Historical untreated cohort (79.6% vs. 59.3%, p < 0.01). Clinical relapses risk is reduced by 2.2 times in treated patients (p = 0.01).

CONCLUSIONS: We propose a promising method to manage observational data in a relatively unbiased way, in order to analyze real-world treatment effectiveness.

Original languageEnglish
Pages (from-to)101952
JournalMultiple Sclerosis and Related Disorders
Volume40
DOIs
Publication statusE-pub ahead of print - Jan 21 2020

Fingerprint

Interferons
Multiple Sclerosis
Pharmaceutical Preparations
Therapeutics
Recurrence
Standard of Care
Natural History
Randomized Controlled Trials
Clinical Trials

Cite this

@article{e2e6fae782c64fac925fddcf268cad65,
title = "A method to compare prospective and historical cohorts to evaluate drug effects. Application to the analysis of early treatment effectiveness of intramuscular interferon-β1a in multiple sclerosis patients",
abstract = "BACKGROUND: Disease modifying therapy have changed the natural evolution of multiple sclerosis (MS), with efficacy demonstrated in randomized clinical trials. Standard-of-care effectiveness is needed to complement clinical trial data and highlight outcomes in real-world practice, but comparing prospective patients with historical cohorts likely introduces biases. To address these potential biases, assigning a patient with a score that expresses his/her disease prognosis before starting a therapy may make it possible to evaluate the unbiased ability of the therapy to modify disease natural history. Thus, we aimed at analyzing the effectiveness of intramuscular interferon-β1a (im IFN-β1a) matching by BREMSO score (Bayesian Risk Estimate for Multiple Sclerosis at Onset) a prospective real-world cohort of treated patients with a historical cohort of untreated patients.MATERIAL AND METHODS: We observed 108 newly diagnosed, treatment na{\"i}ve MS patients over 12 months of treatment with im IFN-β1a. BREMSO score was used to assign a value to each patient, giving the real-world treated patients comparable with the Historical untreated patients, on the basis of the same risk to have unfavorable evolution.RESULTS: A significantly higher percentage of relapse-free patients is observed in IFN-β1a treated cohort vs. Historical untreated cohort (79.6{\%} vs. 59.3{\%}, p < 0.01). Clinical relapses risk is reduced by 2.2 times in treated patients (p = 0.01).CONCLUSIONS: We propose a promising method to manage observational data in a relatively unbiased way, in order to analyze real-world treatment effectiveness.",
author = "BREMSO-group and Giulia Mallucci and Francesco Patti and {Brescia Morra}, Vincenzo and Maria Buccafusca and Lucia Moiola and Amato, {Maria Pia} and Elisabetta Ferraro and Maria Trojano and Mauro Zaffaroni and Massimiliano Mirabella and Gianluca Moscato and Katrin Plewnia and Valentina Zipoli and Elisa Puma and Roberto Bergamaschi",
note = "Copyright {\circledC} 2020. Published by Elsevier B.V.",
year = "2020",
month = "1",
day = "21",
doi = "10.1016/j.msard.2020.101952",
language = "English",
volume = "40",
pages = "101952",
journal = "Multiple Sclerosis and Related Disorders",
issn = "2211-0348",
publisher = "Elsevier",

}

TY - JOUR

T1 - A method to compare prospective and historical cohorts to evaluate drug effects. Application to the analysis of early treatment effectiveness of intramuscular interferon-β1a in multiple sclerosis patients

AU - BREMSO-group

AU - Mallucci, Giulia

AU - Patti, Francesco

AU - Brescia Morra, Vincenzo

AU - Buccafusca, Maria

AU - Moiola, Lucia

AU - Amato, Maria Pia

AU - Ferraro, Elisabetta

AU - Trojano, Maria

AU - Zaffaroni, Mauro

AU - Mirabella, Massimiliano

AU - Moscato, Gianluca

AU - Plewnia, Katrin

AU - Zipoli, Valentina

AU - Puma, Elisa

AU - Bergamaschi, Roberto

N1 - Copyright © 2020. Published by Elsevier B.V.

PY - 2020/1/21

Y1 - 2020/1/21

N2 - BACKGROUND: Disease modifying therapy have changed the natural evolution of multiple sclerosis (MS), with efficacy demonstrated in randomized clinical trials. Standard-of-care effectiveness is needed to complement clinical trial data and highlight outcomes in real-world practice, but comparing prospective patients with historical cohorts likely introduces biases. To address these potential biases, assigning a patient with a score that expresses his/her disease prognosis before starting a therapy may make it possible to evaluate the unbiased ability of the therapy to modify disease natural history. Thus, we aimed at analyzing the effectiveness of intramuscular interferon-β1a (im IFN-β1a) matching by BREMSO score (Bayesian Risk Estimate for Multiple Sclerosis at Onset) a prospective real-world cohort of treated patients with a historical cohort of untreated patients.MATERIAL AND METHODS: We observed 108 newly diagnosed, treatment naïve MS patients over 12 months of treatment with im IFN-β1a. BREMSO score was used to assign a value to each patient, giving the real-world treated patients comparable with the Historical untreated patients, on the basis of the same risk to have unfavorable evolution.RESULTS: A significantly higher percentage of relapse-free patients is observed in IFN-β1a treated cohort vs. Historical untreated cohort (79.6% vs. 59.3%, p < 0.01). Clinical relapses risk is reduced by 2.2 times in treated patients (p = 0.01).CONCLUSIONS: We propose a promising method to manage observational data in a relatively unbiased way, in order to analyze real-world treatment effectiveness.

AB - BACKGROUND: Disease modifying therapy have changed the natural evolution of multiple sclerosis (MS), with efficacy demonstrated in randomized clinical trials. Standard-of-care effectiveness is needed to complement clinical trial data and highlight outcomes in real-world practice, but comparing prospective patients with historical cohorts likely introduces biases. To address these potential biases, assigning a patient with a score that expresses his/her disease prognosis before starting a therapy may make it possible to evaluate the unbiased ability of the therapy to modify disease natural history. Thus, we aimed at analyzing the effectiveness of intramuscular interferon-β1a (im IFN-β1a) matching by BREMSO score (Bayesian Risk Estimate for Multiple Sclerosis at Onset) a prospective real-world cohort of treated patients with a historical cohort of untreated patients.MATERIAL AND METHODS: We observed 108 newly diagnosed, treatment naïve MS patients over 12 months of treatment with im IFN-β1a. BREMSO score was used to assign a value to each patient, giving the real-world treated patients comparable with the Historical untreated patients, on the basis of the same risk to have unfavorable evolution.RESULTS: A significantly higher percentage of relapse-free patients is observed in IFN-β1a treated cohort vs. Historical untreated cohort (79.6% vs. 59.3%, p < 0.01). Clinical relapses risk is reduced by 2.2 times in treated patients (p = 0.01).CONCLUSIONS: We propose a promising method to manage observational data in a relatively unbiased way, in order to analyze real-world treatment effectiveness.

U2 - 10.1016/j.msard.2020.101952

DO - 10.1016/j.msard.2020.101952

M3 - Article

C2 - 32007656

VL - 40

SP - 101952

JO - Multiple Sclerosis and Related Disorders

JF - Multiple Sclerosis and Related Disorders

SN - 2211-0348

ER -