A method to compare prospective and historical cohorts to evaluate drug effects. Application to the analysis of early treatment effectiveness of intramuscular interferon-ß1a in multiple sclerosis patients.
Mult Scler Relat Disord
; 40: 101952, 2020 May.
Article
em En
| MEDLINE
| ID: mdl-32007656
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-ß1a (im IFN-ß1a) 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 ANDMETHODS:
We observed 108 newly diagnosed, treatment naïve MS patients over 12 months of treatment with im IFN-ß1a. 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-ß1a 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.Palavras-chave
Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Avaliação de Resultados em Cuidados de Saúde
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Interferon beta-1a
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Fatores Imunológicos
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Esclerose Múltipla
Tipo de estudo:
Clinical_trials
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Etiology_studies
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Incidence_studies
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Observational_studies
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Prognostic_studies
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Risk_factors_studies
Limite:
Adult
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Female
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Humans
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Male
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Middle aged
Idioma:
En
Revista:
Mult Scler Relat Disord
Ano de publicação:
2020
Tipo de documento:
Article