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Propensity Score Methods in Rare Disease. A Demonstration Using Observational Data in Systemic Lupus Erythematosus.
J Rheumatol. 2020 Jul 01 [Online ahead of print]JR

Abstract

Observational studies allow researchers to understand the natural history of rheumatic conditions, risk factors for disease development, factors affecting important disease-related outcomes and estimate treatment effect from real world data. However, this design carries a risk of confounding bias. A propensity score is a balancing score that aims to minimize the difference between study groups and consequently potential confounding effects. The score can be applied in one of four methods in observational research: matching, stratification, adjustment, and inverse probability weighting. Systemic lupus erythematosus (SLE) is a rare disease characterized by relatively small sample size and/or low event rates. In this article, we review propensity score methods. We demonstrate application of propensity score methods to achieve study group balance in a rare disease using an example of risk of infection in SLE patients with hypogammaglobulinemia.

Authors+Show Affiliations

From the Division of Rheumatology, Department of Medicine, University of Toronto, Toronto, Canada; Centre for Prognosis in Rheumatic Diseases, University Health Network, Toronto, Canada; Rheumatology Unit, Department of Medicine, King Saud University, Saudi Arabia; College of Medicine Research Center, King Saud University, Saudi Arabia; Institute of Health Policy, Management and Evaluation, University of Toronto, Toronto, Ontario, Canada; The Krembil Research Institute, University Health Network, Toronto, Canada; Division of Biostatistics, Dalla Lana School of Public Health, University of Toronto, Toronto, Ontario, Canada; Program in Child Health Evaluative Sciences, SickKids Research Institute, Toronto, Ontario, Canada. Dr. Johnson is a site investigator for Scleroderma clinical trials supported by Boehringer Ingelheim, Bayer, Corbus and GlaxoSmithKline. Address correspondence to Sindhu Johnson MD PhD, Toronto Western Hospital, 399 Bathurst Street, Toronto, Ontario, Canada. Sindhu.Johnson@uhn.ca.From the Division of Rheumatology, Department of Medicine, University of Toronto, Toronto, Canada; Centre for Prognosis in Rheumatic Diseases, University Health Network, Toronto, Canada; Rheumatology Unit, Department of Medicine, King Saud University, Saudi Arabia; College of Medicine Research Center, King Saud University, Saudi Arabia; Institute of Health Policy, Management and Evaluation, University of Toronto, Toronto, Ontario, Canada; The Krembil Research Institute, University Health Network, Toronto, Canada; Division of Biostatistics, Dalla Lana School of Public Health, University of Toronto, Toronto, Ontario, Canada; Program in Child Health Evaluative Sciences, SickKids Research Institute, Toronto, Ontario, Canada. Dr. Johnson is a site investigator for Scleroderma clinical trials supported by Boehringer Ingelheim, Bayer, Corbus and GlaxoSmithKline. Address correspondence to Sindhu Johnson MD PhD, Toronto Western Hospital, 399 Bathurst Street, Toronto, Ontario, Canada. Sindhu.Johnson@uhn.ca.From the Division of Rheumatology, Department of Medicine, University of Toronto, Toronto, Canada; Centre for Prognosis in Rheumatic Diseases, University Health Network, Toronto, Canada; Rheumatology Unit, Department of Medicine, King Saud University, Saudi Arabia; College of Medicine Research Center, King Saud University, Saudi Arabia; Institute of Health Policy, Management and Evaluation, University of Toronto, Toronto, Ontario, Canada; The Krembil Research Institute, University Health Network, Toronto, Canada; Division of Biostatistics, Dalla Lana School of Public Health, University of Toronto, Toronto, Ontario, Canada; Program in Child Health Evaluative Sciences, SickKids Research Institute, Toronto, Ontario, Canada. Dr. Johnson is a site investigator for Scleroderma clinical trials supported by Boehringer Ingelheim, Bayer, Corbus and GlaxoSmithKline. Address correspondence to Sindhu Johnson MD PhD, Toronto Western Hospital, 399 Bathurst Street, Toronto, Ontario, Canada. Sindhu.Johnson@uhn.ca.From the Division of Rheumatology, Department of Medicine, University of Toronto, Toronto, Canada; Centre for Prognosis in Rheumatic Diseases, University Health Network, Toronto, Canada; Rheumatology Unit, Department of Medicine, King Saud University, Saudi Arabia; College of Medicine Research Center, King Saud University, Saudi Arabia; Institute of Health Policy, Management and Evaluation, University of Toronto, Toronto, Ontario, Canada; The Krembil Research Institute, University Health Network, Toronto, Canada; Division of Biostatistics, Dalla Lana School of Public Health, University of Toronto, Toronto, Ontario, Canada; Program in Child Health Evaluative Sciences, SickKids Research Institute, Toronto, Ontario, Canada. Dr. Johnson is a site investigator for Scleroderma clinical trials supported by Boehringer Ingelheim, Bayer, Corbus and GlaxoSmithKline. Address correspondence to Sindhu Johnson MD PhD, Toronto Western Hospital, 399 Bathurst Street, Toronto, Ontario, Canada. Sindhu.Johnson@uhn.ca.From the Division of Rheumatology, Department of Medicine, University of Toronto, Toronto, Canada; Centre for Prognosis in Rheumatic Diseases, University Health Network, Toronto, Canada; Rheumatology Unit, Department of Medicine, King Saud University, Saudi Arabia; College of Medicine Research Center, King Saud University, Saudi Arabia; Institute of Health Policy, Management and Evaluation, University of Toronto, Toronto, Ontario, Canada; The Krembil Research Institute, University Health Network, Toronto, Canada; Division of Biostatistics, Dalla Lana School of Public Health, University of Toronto, Toronto, Ontario, Canada; Program in Child Health Evaluative Sciences, SickKids Research Institute, Toronto, Ontario, Canada. Dr. Johnson is a site investigator for Scleroderma clinical trials supported by Boehringer Ingelheim, Bayer, Corbus and GlaxoSmithKline. Address correspondence to Sindhu Johnson MD PhD, Toronto Western Hospital, 399 Bathurst Street, Toronto, Ontario, Canada. Sindhu.Johnson@uhn.ca.

Pub Type(s)

Journal Article

Language

eng

PubMed ID

32611674

Citation

Almaghlouth, Ibrahim, et al. "Propensity Score Methods in Rare Disease. a Demonstration Using Observational Data in Systemic Lupus Erythematosus." The Journal of Rheumatology, 2020.
Almaghlouth I, Pullenayegum E, Gladman DD, et al. Propensity Score Methods in Rare Disease. A Demonstration Using Observational Data in Systemic Lupus Erythematosus. J Rheumatol. 2020.
Almaghlouth, I., Pullenayegum, E., Gladman, D. D., Urowitz, M. B., & Johnson, S. R. (2020). Propensity Score Methods in Rare Disease. A Demonstration Using Observational Data in Systemic Lupus Erythematosus. The Journal of Rheumatology. https://doi.org/10.3899/jrheum.200254
Almaghlouth I, et al. Propensity Score Methods in Rare Disease. a Demonstration Using Observational Data in Systemic Lupus Erythematosus. J Rheumatol. 2020 Jul 1; PubMed PMID: 32611674.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Propensity Score Methods in Rare Disease. A Demonstration Using Observational Data in Systemic Lupus Erythematosus. AU - Almaghlouth,Ibrahim, AU - Pullenayegum,Eleanor, AU - Gladman,Dafna D, AU - Urowitz,Murray B, AU - Johnson,Sindhu R, Y1 - 2020/07/01/ PY - 2020/7/3/entrez JF - The Journal of rheumatology JO - J. Rheumatol. N2 - Observational studies allow researchers to understand the natural history of rheumatic conditions, risk factors for disease development, factors affecting important disease-related outcomes and estimate treatment effect from real world data. However, this design carries a risk of confounding bias. A propensity score is a balancing score that aims to minimize the difference between study groups and consequently potential confounding effects. The score can be applied in one of four methods in observational research: matching, stratification, adjustment, and inverse probability weighting. Systemic lupus erythematosus (SLE) is a rare disease characterized by relatively small sample size and/or low event rates. In this article, we review propensity score methods. We demonstrate application of propensity score methods to achieve study group balance in a rare disease using an example of risk of infection in SLE patients with hypogammaglobulinemia. SN - 0315-162X UR - https://www.unboundmedicine.com/medline/citation/32611674/Propensity_Score_Methods_in_Rare_Disease._A_Demonstration_Using_Observational_Data_in_Systemic_Lupus_Erythematosus L2 - http://www.jrheum.org/cgi/pmidlookup?view=long&pmid=32611674 DB - PRIME DP - Unbound Medicine ER -
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