Teprotumumab for Thyroid Eye Disease: A Systematic Review and Meta-Analysis of Randomized Controlled Trials and Real-World Studies.
Aimin Sun, Xing Wang, Junyu Wang et al.
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In 30 seconds
This systematic review and meta-analysis assessed intravenous teprotumumab efficacy and safety in adults with thyroid eye disease, combining randomized controlled trials (RCTs) and real-world studies (RWS). Across 36 studies, proptosis response favored teprotumumab in RCTs (RR, 4.55; 95% confidence interval [CI], 2.52-8.23); in RWS, 82% of patients achieved a proptosis response (95% CI, 0.78-0.86; 11 studies, 482 patients). Diplopia also improved, while hyperglycemia and hearing impairment were more frequent with treatment in RCTs and were reported in routine practice.
Key findings
- Proptosis response favored teprotumumab in RCTs (RR, 4.55; 95% CI, 2.52-8.23); 82% responded in RWS (95% CI, 0.78-0.86; 11 studies, 482 patients).
- Diplopia response favored teprotumumab in RCTs (RR, 2.13; 95% CI, 1.51-2.98); 60% improved in RWS (95% CI, 0.51-0.68; 6 studies, 204 patients).
- Hyperglycemia was more frequent with teprotumumab in RCTs (RR, 2.67; 95% CI, 1.04-6.85) and occurred in 17% of patients in RWS.
- Hearing impairment was increased in RCTs (RR, 4.05; 95% CI, 1.50-10.96) and occurred in 18% of patients in RWS.
Why it matters
The synthesis brings together controlled-trial and routine-practice evidence, showing that improvements in proptosis and diplopia are observed across both settings. It also highlights clinically relevant metabolic and auditory adverse events, which should be considered alongside expected benefits when assessing treatment suitability and follow-up needs.
What to keep in mind
The evidence combines randomized trials with real-world studies, which may differ in design and patient populations; real-world response estimates are based on subsets of the included studies.