Tree-based subgroup discovery using electronic health record data: heterogeneity of treatment effects for DTG-containing therapies.

Jiabei Yang, Ann W Mwangi, Rami Kantor, Issa J Dahabreh, Monicah Nyambura, Allison Delong, Joseph W Hogan, Jon A Steingrimsson

Biostatistics (Oxford, England) 2024 Apr 15

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The rich longitudinal individual level data available from electronic health records (EHRs) can be used to examine treatment effect heterogeneity. However, estimating treatment effects using EHR data poses several challenges, including time-varying confounding, repeated and temporally non-aligned measurements of covariates, treatment assignments and outcomes, and loss-to-follow-up due to dropout. Here, we develop the subgroup discovery for longitudinal data algorithm, a tree-based algorithm for discovering subgroups with heterogeneous treatment effects using longitudinal data by combining the generalized interaction tree algorithm, a general data-driven method for subgroup discovery, with longitudinal targeted maximum likelihood estimation. We apply the algorithm to EHR data to discover subgroups of people living with human immunodeficiency virus who are at higher risk of weight gain when receiving dolutegravir (DTG)-containing antiretroviral therapies (ARTs) versus when receiving non-DTG-containing ARTs. © The Author(s) 2023. Published by Oxford University Press. All rights reserved. [br]For permissions, please e-mail: journals.permissions@oup.com.

Citation

Jiabei Yang, Ann W Mwangi, Rami Kantor, Issa J Dahabreh, Monicah Nyambura, Allison Delong, Joseph W Hogan, Jon A Steingrimsson. Tree-based subgroup discovery using electronic health record data: heterogeneity of treatment effects for DTG-containing therapies. Biostatistics (Oxford, England). 2024 Apr 15;25(2):323-335