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 RePORTER · NIH · R21 · $206,243 · view on reporter.nih.gov ↗

Abstract

! 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
JOHNS HOPKINS UNIVERSITY
Principal Investigator
Elizabeth Ann Sugar
Activity code
R21
Funding institute
NIH
Fiscal year
2020
Award amount
$206,243
Award type
5
Project period
2019-02-01 → 2021-01-31