Comparative effectiveness and safety of high-efficacy disease-modifying agents (DMAs) and moderate-efficacy DMAs in patients with multiple sclerosis

Objective: The specific aims of this study are to (1) evaluate the factors associated with the initiation of heDMAs and meDMAs, (2) examine the comparative effectiveness of relapses between heDMA users and meDMA users, and (3) assess the infection risks for heDMAs and meDMAs among patients with MS. Methods: This retrospective cohort study involved the 2015-2019 MarketScan data and included adults with MS who initiated DMAs. The main statistical analytical plan involved building three separate multivariate models to address the objective of three aims: (1) a multivariable logistic regression model to identify factors associated with the selection of heDMAs, (2) an inverse probability treatment weighted (IPTW)-negative binomial model (NB) to compare the annualized relapse rate (ARR), and (3) an IPTW- Cox proportional hazards (CPH) model to compare risk of infection between heDMA and meDMA users. Results: Among 10,003 incident DMA users, 22.92% initiated with heDMAs. The multivariable logistic regression model revealed that multiple predisposing, need, and enabling factors were associated with higher odds of initiating heDMAs. The IPTW-based NB regression found that sex moderated the relationship between the types of DMAs and ARR. Stratified analyses showed no significant difference in ARR for two cohorts among female users; however, males initiating heDMAs had 0.74 (95% CI 0.59-0.94) times significantly lower adjusted incidence rate ratio compared to their counterparts. Furthermore, heDMA use was associated with a 24% (95% CI: 1.06-1.44) increased risk of serious infection and a 21% (95% CI: 1.14-1.30) increased risk of urinary tract infection compared to meDMAs. Conclusions: Both demographic and clinical factors influenced the selection of heDMA vs. meDMA. A moderate effect of sex with a lower ARR in males with heDMAs than meDMAs. Meanwhile, MS patients initiating heDMAs are associated with a greater risk of serious infections and UTIs compared to their counterparts.

Name thesis:degree_name

Doctor of Philosophy

Discipline thesis:degree_discipline

Pharmaceutic Health Outcomes & Policy

Grantor

University of Houston

Year dc:date.issued

2024

Author dc:creator

• Li, Jieni

Advisor dc:contributor.advisor

• Aparasu, Rajender R.

Committee members dc:contributor.committeemember

• Hutton, George J.
• Essien, Ekere J.
• Lin, Ying
• Varisco, Tyler J.

• Health Sciences, Pharmacy

Language dc:language.iso

en

Handle dc:identifier.uri

https://hdl.handle.net/10657/18178

OAI identifier oai:identifier

oai:uh-ir.tdl.org:10657/18178