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Use of pragmatic randomized trials in multiple sclerosis: A systematic overview.
Hirt, Julian; Janiaud, Perrine; Düblin, Pascal; Nicoletti, Giovanni Jacopo; Dembowska, Kinga; Nguyen, Thao Vy Thi; Woelfle, Tim; Axfors, Cathrine; Yaldizli, Özgür; Granziera, Cristina; Kuhle, Jens; Kappos, Ludwig; Hemkens, Lars G.
Afiliación
  • Hirt J; Research Center for Clinical Neuroimmunology and Neuroscience Basel (RC2NB), University Hospital Basel and University of Basel, Basel, Switzerland/Department of Clinical Research, University Hospital Basel and University of Basel, Basel, Switzerland/Department of Health, Eastern Switzerland Universi
  • Janiaud P; Research Center for Clinical Neuroimmunology and Neuroscience Basel (RC2NB), University Hospital Basel and University of Basel, Basel, Switzerland/Department of Clinical Research, University Hospital Basel and University of Basel, Basel, Switzerland.
  • Düblin P; Department of Clinical Research, University Hospital Basel and University of Basel, Basel, Switzerland.
  • Nicoletti GJ; Department of Emergency Medicine, University Hospital Basel, Basel, Switzerland.
  • Dembowska K; Research Center for Clinical Neuroimmunology and Neuroscience Basel (RC2NB), University Hospital Basel and University of Basel, Basel, Switzerland/MSc program in epidemiology, Swiss TPH, University of Basel, Basel, Switzerland.
  • Nguyen TVT; Research Center for Clinical Neuroimmunology and Neuroscience Basel (RC2NB), University Hospital Basel and University of Basel, Basel, Switzerland/MSc program in epidemiology, Swiss TPH, University of Basel, Basel, Switzerland.
  • Woelfle T; Research Center for Clinical Neuroimmunology and Neuroscience Basel (RC2NB), University Hospital Basel and University of Basel, Basel, Switzerland.
  • Axfors C; Research Center for Clinical Neuroimmunology and Neuroscience Basel (RC2NB), University Hospital Basel and University of Basel, Basel, Switzerland/Department of Clinical Research, University Hospital Basel and University of Basel, Basel, Switzerland.
  • Yaldizli Ö; Research Center for Clinical Neuroimmunology and Neuroscience Basel (RC2NB), University Hospital Basel and University of Basel, Basel, Switzerland/Department of Clinical Research, University Hospital Basel and University of Basel, Basel, Switzerland.
  • Granziera C; Research Center for Clinical Neuroimmunology and Neuroscience Basel (RC2NB), University Hospital Basel and University of Basel, Basel, Switzerland/Department of Clinical Research, University Hospital Basel and University of Basel, Basel, Switzerland.
  • Kuhle J; Research Center for Clinical Neuroimmunology and Neuroscience Basel (RC2NB), University Hospital Basel and University of Basel, Basel, Switzerland/Department of Clinical Research, University Hospital Basel and University of Basel, Basel, Switzerland.
  • Kappos L; Research Center for Clinical Neuroimmunology and Neuroscience Basel (RC2NB), University Hospital Basel and University of Basel, Basel, Switzerland/Department of Clinical Research, University Hospital Basel and University of Basel, Basel, Switzerland.
  • Hemkens LG; Research Center for Clinical Neuroimmunology and Neuroscience Basel (RC2NB), University Hospital Basel and University of Basel, Basel, Switzerland/Department of Clinical Research, University Hospital Basel and University of Basel, Basel, Switzerland.
Mult Scler ; 30(4-5): 463-478, 2024 Apr.
Article en En | MEDLINE | ID: mdl-38253528
ABSTRACT

BACKGROUND:

Pragmatic trials are increasingly recognized for providing real-world evidence on treatment choices.

OBJECTIVE:

The objective of this study is to investigate the use and characteristics of pragmatic trials in multiple sclerosis (MS).

METHODS:

Systematic literature search and analysis of pragmatic trials on any intervention published up to 2022. The assessment of pragmatism with PRECIS-2 (PRagmatic Explanatory Continuum Indicator Summary-2) is performed.

RESULTS:

We identified 48 pragmatic trials published 1967-2022 that included a median of 82 participants (interquartile range (IQR) = 42-160) to assess typically supportive care interventions (n = 41; 85%). Only seven trials assessed drugs (15%). Only three trials (6%) included >500 participants. Trials were mostly from the United Kingdom (n = 18; 38%), Italy (n = 6; 13%), the United States and Denmark (each n = 5; 10%). Primary outcomes were diverse, for example, quality-of-life, physical functioning, or disease activity. Only 1 trial (2%) used routinely collected data for outcome ascertainment. No trial was very pragmatic in all design aspects, but 14 trials (29%) were widely pragmatic (i.e. PRECIS-2 score ⩾ 4/5 in all domains).

CONCLUSION:

Only few and mostly small pragmatic trials exist in MS which rarely assess drugs. Despite the widely available routine data infrastructures, very few trials utilize them. There is an urgent need to leverage the potential of this pioneering study design to provide useful randomized real-world evidence.
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Ensayos Clínicos Pragmáticos como Asunto / Esclerosis Múltiple Tipo de estudio: Clinical_trials / Prognostic_studies Aspecto: Patient_preference Límite: Humans Idioma: En Revista: Mult Scler Asunto de la revista: NEUROLOGIA Año: 2024 Tipo del documento: Article Pais de publicación: Reino Unido
Texto completo
Añadir a Mi BVS
Imprimir
XML
PubMed Links
Buscar en Google

Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Ensayos Clínicos Pragmáticos como Asunto / Esclerosis Múltiple Tipo de estudio: Clinical_trials / Prognostic_studies Aspecto: Patient_preference Límite: Humans Idioma: En Revista: Mult Scler Asunto de la revista: NEUROLOGIA Año: 2024 Tipo del documento: Article Pais de publicación: Reino Unido