ESPEYB17 3. Thyroid Follow-up paper - Graves disease (1 abstracts)
3.5. Teprotumumab for the treatment of active thyroid eye disease
Douglas RS , Kahaly GJ , Patel A , Sile S , Thompson EHZ , Perdok R , Fleming JC , Fowler BT , Marcocci C , Marinò M , Antonelli A , Dailey R , Harris GJ , Eckstein A , Schiffman J , Tang R , Nelson C , Salvi M , Wester S , Sherman JW , Vescio T , Holt RJ & Smith TJ
To read the full abstract: N Engl J Med. 2020;382:341–352.
Teprotumumab, an IGF-1R monoclonal antibody, showed promising results for the treatment of Graves’ orbitopathy in a first phase 2 study reported in 2017 [1]. Here, this randomized double-masked, placebo controlled phase 3 multicenter study confirms those initial promising results in patients with moderate-to severe Graves’ eye disease: The primary outcome of proptosis reduction by ≥2 mm after 24 weeks was reached by 83% on teprotumumab vs. 10% of controls. Also, all secondary outcomes, including objective measures of thyroid eye disease activity and subjective quality of life, were better in the teprotumumab group with clinically relevant differences vs. placebo. Adverse events were mostly mild-to-moderate, however one patient had to stop treatment due to infusion-related reaction.
In summary, this first phase 3 randomized controlled study of teprotumumab revealed a good treatment responses with a number needed to treat (NNT) of only 1.36 in the context of a low number of adverse events during the short observational period of 24 weeks. As no alternative specific treatment option exists for Graves’ orbitopathy, these data are an important next step to clinical approval and routine use in adults. Although severe thyroid eye disease is less frequent in childhood, we as pediatric endocrinologists look forward to teprotumumab studies during adolescence when Graves’ disease incidence is highest in the pediatric population.
Reference:
1. Smith TJ, Kahaly GJ, Ezra DG, Fleming JC, Dailey RA, Tang RA, Harris GJ, Antonelli A, Salvi M, Goldberg RA, Gigantelli JW, Couch SM, Shriver EM, Hayek BR, Hink EM, Woodward RM, Gabriel K, Magni G, Douglas RS. Teprotumumab for Thyroid-Associated Ophthalmopathy. N Engl J Med. 2017;376:1748–1761.
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ESPE Yearbook of Paediatric Endocrinology
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