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Propensity Score Methods in Rare Disease: A Demonstration Using Observational Data in Systemic Lupus Erythematosus.
Almaghlouth, Ibrahim; Pullenayegum, Eleanor; Gladman, Dafna D; Urowitz, Murray B; Johnson, Sindhu R.
Afiliação
  • Almaghlouth I; I. Almaghlouth, MBBS, MSc, Division of Rheumatology, Department of Medicine, University of Toronto, Ontario, Canada, and Rheumatology Unit, Department of Medicine, and College of Medicine Research Center, King Saud University, Saudi Arabia; E. Pullenayegum, PhD, Division of Biostatistics, Dalla Lana
  • Pullenayegum E; I. Almaghlouth, MBBS, MSc, Division of Rheumatology, Department of Medicine, University of Toronto, Ontario, Canada, and Rheumatology Unit, Department of Medicine, and College of Medicine Research Center, King Saud University, Saudi Arabia; E. Pullenayegum, PhD, Division of Biostatistics, Dalla Lana
  • Gladman DD; I. Almaghlouth, MBBS, MSc, Division of Rheumatology, Department of Medicine, University of Toronto, Ontario, Canada, and Rheumatology Unit, Department of Medicine, and College of Medicine Research Center, King Saud University, Saudi Arabia; E. Pullenayegum, PhD, Division of Biostatistics, Dalla Lana
  • Urowitz MB; I. Almaghlouth, MBBS, MSc, Division of Rheumatology, Department of Medicine, University of Toronto, Ontario, Canada, and Rheumatology Unit, Department of Medicine, and College of Medicine Research Center, King Saud University, Saudi Arabia; E. Pullenayegum, PhD, Division of Biostatistics, Dalla Lana
  • Johnson SR; I. Almaghlouth, MBBS, MSc, Division of Rheumatology, Department of Medicine, University of Toronto, Ontario, Canada, and Rheumatology Unit, Department of Medicine, and College of Medicine Research Center, King Saud University, Saudi Arabia; E. Pullenayegum, PhD, Division of Biostatistics, Dalla Lana
J Rheumatol ; 2020 Jul 01.
Artigo em Inglês | MEDLINE | ID: mdl-32611674
ABSTRACT
Observational studies allow researchers to understand the natural history of rheumatic conditions, risk factors for disease development, and factors affecting important disease-related outcomes, and to estimate treatment effect from real-world data. However, this design carries a risk of confounding bias. A propensity score (PS) is a balancing score that aims to minimize the difference between study groups and consequently potential confounding effects. The score can be applied in 1 of 4 methods in observational research matching, stratification, adjustment, and inverse probability weighting. Systemic lupus erythematosus (SLE) is a rare disease characterized by a relatively small sample size and/or low event rates. In this article, we review the PS methods. We demonstrate application of the PS methods to achieve study group balance in a rare disease using an example of risk of infection in SLE patients with hypogammaglobulinemia.
Texto completo: Disponível Coleções: Bases de dados internacionais Base de dados: MEDLINE Tipo de estudo: Patient_preference / Fatores de risco Aspecto: Preferência do paciente Idioma: Inglês Ano de publicação: 2020 Tipo de documento: Artigo

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Texto completo: Disponível Coleções: Bases de dados internacionais Base de dados: MEDLINE Tipo de estudo: Patient_preference / Fatores de risco Aspecto: Preferência do paciente Idioma: Inglês Ano de publicação: 2020 Tipo de documento: Artigo