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 - 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
PY - 2020
Y1 - 2020
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.
KW - BREMSO
KW - Bayesian analysis
KW - Beta-interferon
KW - Multiple sclerosis
KW - Prognosis natural history
KW - BREMSO
KW - Bayesian analysis
KW - Beta-interferon
KW - Multiple sclerosis
KW - Prognosis natural history
UR - http://hdl.handle.net/10807/147829
UR - http://www.elsevier.com/wps/find/journaldescription.cws_home/725776/description#description
U2 - 10.1016/j.msard.2020.101952
DO - 10.1016/j.msard.2020.101952
M3 - Article
SN - 2211-0348
VL - 40
SP - 101952-N/A
JO - Multiple Sclerosis and Related Disorders
JF - Multiple Sclerosis and Related Disorders
ER -