Propensity Score Methods in Rare Disease: A Demonstration Using Observational Data in Systemic Lupus Erythematosus

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...

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Bibliographic Details
Published in:Journal of rheumatology 2021-03, Vol.48 (3), p.321-325
Main Authors: Almaghlouth, Ibrahim, Pullenayegum, Eleanor, Gladman, Dafna D, Urowitz, Murray B, Johnson, Sindhu R
Format: Article
Language:English
Subjects:
Bias
Humans
Lupus Erythematosus, Systemic - complications
Lupus Erythematosus, Systemic - drug therapy
Observational Studies as Topic
Propensity Score
Rare Diseases
Risk Factors
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Summary: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.
ISSN:0315-162X
1499-2752
DOI:10.3899/jrheum.200254