# Leveraging long-term follow-up in the MUST Trial and Follow-up Study: Remission, Ocular outcomes and Quantifying utility in patients with severe uveitis (MUST-ROQ)

> **NIH NIH R21** · JOHNS HOPKINS UNIVERSITY · 2020 · $206,243

## Abstract

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Project Summary/Abstract
The uveitides are a collection of diseases characterized by intraocular inflammation and are the 5th leading
cause of blindness in the US with an estimated cost of treating similar to that of treating diabetic retinopathy.
Non-infectious, intermediate, posterior, and panuveitides are potentially blinding diseases and typically are
treated with oral corticosteroids and immunosuppression (systemic therapy). The Multicenter Uveitis Steroid
Treatment (MUST) Trial was a randomized, comparative effectiveness trial comparing 2 treatment paradigms
for these diseases: systemic therapy with corticosteroids and immunosuppression vs. a strictly regional
therapy, the fluocinolone acetonide implant (Retisert®, Bausch and Lomb, Tampa, FL). Patients were recruited
over the course of 3 years and followed for a minimum of 2 years after randomization,the time-point for the
primary outcome assessment for the trial. The MUST Follow-Up Study (FUS) extended follow-up for an
additional 5 years with most patients remaining on their assigned therapy. While the visual acuity outcomes
were comparable at 2-years, visual acuity results at 7 years favored systemic therapy and the ocular risks
associated with implant therapy that were observed at 2 years (cataract, elevated intraocular pressure, and
glaucoma) did not decline. There were no substantial differences in systemic side effects between the two
therapies. At 7 years, 18% of eyes assigned to systemic therapy had been switched to implants, largely due to
failure to control the inflammation with systemic therapy. The long-term results suggest that systemic therapy
may be preferred as the initial therapy, and that implant therapy may be a viable option for individuals who fail
to achieve control of their inflammation with systemic therapy or cannot tolerate it. Nonetheless, additional
information is needed to help guide individual patient care. Control of inflammation is critical for preserving
vision with the induction of a sustained, drug-free remission (inactive disease off all treatments) the ultimate
goal if possible. Yet, there is a paucity of data on this important outcome. Understanding the incidence,
duration, and risk-factors related to remission in both treatment groups as well as additional information on the
risks of ocular complications for systemic therapy would have a direct impact on patient management for
individuals with uveitis. Utility assessments often focus on general metrics, which may not be sensitive enough
at identifying disease- and treatment-specific effects to inform care decisions. Alternate metrics (e.g. using
vision-related quality of life (QoL) or clinical assessments) are needed to address this issue. This analysis
grant application, Leveraging long-term follow-up in the MUST Trial and Follow-up Study: Remission, Ocular
Complications, and Quantifying utility (MUST-ROQ), is designed to answer these important questions by
making use of the extensive follow-up and ...

## Key facts

- **NIH application ID:** 9851391
- **Project number:** 5R21EY029757-02
- **Recipient organization:** JOHNS HOPKINS UNIVERSITY
- **Principal Investigator:** Elizabeth Ann Sugar
- **Activity code:** R21 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $206,243
- **Award type:** 5
- **Project period:** 2019-02-01 → 2021-01-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/9851391

## Citation

> US National Institutes of Health, RePORTER application 9851391, Leveraging long-term follow-up in the MUST Trial and Follow-up Study: Remission, Ocular outcomes and Quantifying utility in patients with severe uveitis (MUST-ROQ) (5R21EY029757-02). Retrieved via AI Analytics 2026-05-22 from https://api.ai-analytics.org/grant/nih/9851391. Licensed CC0.

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