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Quantification of the Immunosuppressant Tacrolimus on Dried Blood Spots Using LC-MS/MS
Published on: November 8, 2015
Use of Tacrolimus for the Treatment of Pediatric Noninfectious Uveitis
Ingrid Pan1, Clara Lin2,3, Alan G Palestine3
1Children's Hospital Colorado, Aurora, CO, USA. Ingrid.pan@childrenscolorado.org.
Insights
Systemic tacrolimus shows promise for treating pediatric noninfectious uveitis refractory to standard treatments. This calcineurin inhibitor was found to be a safe and effective option in a small cohort of young patients.
Area of Science:
- Ophthalmology
- Immunology
- Pediatric Medicine
Background:
- Pediatric noninfectious uveitis often presents treatment challenges.
- Limited therapeutic options exist for refractory pediatric cases.
Purpose of the Study:
- To investigate the efficacy and safety of systemic tacrolimus in children with noninfectious uveitis.
- To evaluate tacrolimus as an adjuvant immunomodulatory treatment for pediatric refractory uveitis.
Main Methods:
- Retrospective chart review of 11 pediatric patients (≤18 years) with refractory noninfectious uveitis.
- Analysis of treatment success defined by ocular inflammation markers and topical steroid use at 6 and 12 months.
- Assessment of adverse events and need for treatment discontinuation.
Main Results:
- Treatment success was achieved in 88.9% at 6 months and 63.6% at 12 months.
- Seven patients experienced laboratory abnormalities, but tacrolimus was not discontinued.
- Therapeutic tacrolimus levels were achieved with a mean dose of 0.16 mg/kg/day.
Conclusions:
- Systemic tacrolimus appears to be a well-tolerated and effective treatment for refractory pediatric noninfectious uveitis.
- Tacrolimus offers a potentially safe therapeutic option for this challenging patient population.
Introduction:
Pediatric patients with noninfectious uveitis refractory to standard therapies have limited options. Efficacy and safety of systemic tacrolimus, a calcineurin inhibitor used successfully in adult noninfectious uveitis as an adjuvant immunomodulatory treatment, was investigated in the pediatric population at our institution.
Methods:
This was a retrospective chart review of patients ≤ 18 years old diagnosed with noninfectious uveitis who were intolerant to or failed conventional systemic immunosuppressants between January 2014 and June 2025 at a tertiary referral center. The primary outcome was treatment success, defined as two or more of the following: ≤ 0.5+ anterior chamber cell, ≤ 2 drops of topical steroids per day per eye, improvement or resolution of vitritis, macular edema, papillitis, and angiographic leakage without addition of systemic therapy at 6 and 12 months after tacrolimus initiation. Secondary outcomes included need for dose reduction or discontinuation owing to adverse effects. Descriptive statistics were used to analyze the data.
Results:
Data from 11 patients, with median age of 10 years, were analyzed. Treatment success was achieved in 88.9% and 63.6% of patients at 6 and 12 months, respectively. Although seven patients experienced laboratory abnormalities, tacrolimus was not discontinued. An average tacrolimus dose of 0.16 mg/kg/day divided every 12 h achieved therapeutic tacrolimus levels. Median duration (range) of tacrolimus therapy was 24 months (4-93 months).
Conclusions:
Tacrolimus may potentially be a well-tolerated, safe, and effective option for refractory cases of pediatric noninfectious uveitis.
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