Thyroid disease

Efficacy and Safety of Teprotumumab in Thyroid Eye Disease: A Systematic Review and Meta-Analysis

Endocr PractPublished Systematic review and meta-analysis of randomised trials and observational studies

Authors' conclusion

Teprotumumab effectively reduces proptosis, improves diplopia, and lowers disease activity in TED, regardless of previous treatments, severity, or dosage, albeit with increased AEs. It has the potential to become a vital first-line treatment for TED, enhancing patient quality of life.

Study context

Population
Patients with thyroid eye disease (TED) (Graves' orbitopathy); studies from PubMed, Web of Science, EMBASE, Cochrane library and ClinicalTrials.gov up to January 1, 202410 studies: 4 RCTs, 6 observational
Participants
210 vs 193
Comparison
Teprotumumab vs placebo
  • Teprotumumabn = 210
  • Placebon = 193reference
Outcome
Efficacy in RCTs

Pooled result

As reported in the abstract

Relative risk (CI)
Teprotumumabvs Placeboproptosis response
4.18(2.72–6.43)
Teprotumumabvs Placebodiplopia regression
2.29(1.54–3.41)
Teprotumumabvs PlaceboCAS score
3.09(1.98–4.80)
SMD (CI)
Between Teprotumumab and Placeboproptosis reduction
−8.38(−9.25 - −7.52)

Further pooled results

Observational studies (teprotumumab, single group)

Patients
211
Proptosis response rate
82%
Change in proptosis
−3.31 mm
Diplopia improvement rate
0.58
Pooled effect size for Clinical Activity Score (CAS) score
0.66
  • 6 observational studies

Safety

Adverse events (AEs) and serious AEs (RCTs)

Risk of AEs and serious AEs higher with teprotumumab

AEs and serious AEs (observational studies)

AE incidence
0.78
Serious AEs
0.31

Original publication

Efficacy and Safety of Teprotumumab in Thyroid Eye Disease: A Systematic Review and Meta-Analysis. Endocr Pract. Published 12 February 2025. doi:10.1016/j.eprac.2025.01.012

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