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 wide assortment of clinical, treatment and QoL assessments available in the MUST Trial and FUS. These analyses will provide high quality evidence to help guide patient management and to help plan the assessment of utility in future ophthalmology trials.

Public Health Relevance

The uveitides are a leading cause of blindness in the United States and often require long-term therapy. The Multicenter Uveitis Steroid Treatment (MUST) Trial and Follow-up Study, a randomized, comparative effectiveness trial of two treatment strategies, systemic therapy with oral corticosteroids and immunosuppression vs. regional therapy with the fluocinolone acetonide implant, provide the opportunity to evaluate the clinically important questions of remission, relapse, and ocular complications with long-term use of these medications. The long-term follow-up with regular assessment of a wide assortment of clinical and quality of life instruments also provides the opportunity to explore improved metrics for quantifying utility and performing cost effectiveness analyses.

National Institute of Health (NIH)
National Eye Institute (NEI)
Exploratory/Developmental Grants (R21)
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Special Emphasis Panel (ZEY1)
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Bhargava, Sangeeta
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Johns Hopkins University
Biostatistics & Other Math Sci
Schools of Public Health
United States
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