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P. Y. Chang, G. P. Giuliari, P. Thakuria, D. Makhoul, M. Shaikh, C. S. Foster; Mycophenolate Mofetil as a Monotherapy in Pediatric Uveitis. Invest. Ophthalmol. Vis. Sci. 2009;50(13):2680.
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To evaluate the efficacy of mycophenolate mofetil (MMF) as a monotherapy in the treatment of pediatric uveitis.
This is a single-center, retrospective interventional case series. The charts of 52 patients with noninfectious uveitis treated with MMF at or before 18 years of age were reviewed. Primary outcome measures were degree of inflammation, adverse effects, and reduction of concomitant steroids and/or immunomodulatory therapy (IMT); visual acuity was a secondary outcome.
Twenty-five patients (48.1%) remained free of inflammation on the MMF monotherapy without the need for long-term corticosteroid therapy for more than 2 years. We referred to this group of patients as Durable Remission. Thirteen of these patients experienced no relapse over a mean follow-up of 33.3 months (range: 27-48); 7 patients had relapses treated by increasing the MMF dose; 4 patients had relapses treated with brief topical steroids; 1 patient required both topical and systemic steroids for his one flare-up. While not meeting the 2-year mark of Durable Remission, 9 patients (17.3%) achieved inflammation control on MMF alone for a mean of 13.1 months (range: 3-22). Finally, 4 other patients (7.7%) achieved inflammation control initially for a mean of 6.8 months (range: 2-11) but had to discontinue the medication due to significant adverse reactions. Adding these 13 patients to the Durable Remission group, there are a total of 38 patients (73.1% of the study population) who achieved uveitis remission on MMF alone; in these patients, inflammation control was achieved approximately 2 months after the initiation of the MMF therapy. While not a primary outcome measure, visual acuity either remained stable or improved in 94.2% of the study population. The most common adverse effects from MMF were gastrointestinal disturbances (21%), fatigue (12%), and leukopenia (10%). A total of six patients (11.5%) discontinued the medication due to significant reactions.
MMF had a high rate of inflammation control and was successful in inducing long-term disease remission in our study population. In addition, it was tolerated relatively well by the children in this study. We believe that MMF represents an effective steroid-sparing immunomodulatory therapy in noninfectious pediatric uveitis.
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