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Symptoms, quality of life improve with teprotumumab for adults with thyroid eye disease
CHICAGO— Among adults with recent-onset thyroid eye disease, those who received the monoclonal human antibody teprotumumab experienced substantial symptom relief and reported quality of life gains compared with those assigned placebo, according to pooled data from a phase 2 and a phase 3 clinical trial of the drug presented at the American Thyroid Association annual meeting.
“In the largest placebo-controlled evaluation thus far conducted in patients with active and severe thyroid eye disease, teprotumumab reduced proptosis, inflammation and diplopia, resulting in marked improvement in quality of life, as compared with placebo over 24 weeks,” George J. Kahaly, MD, PhD, director of the Molecular Thyroid Research Laboratory and chief physician of the endocrine outpatient clinic of Johannes Gutenberg University Medical Center in Mainz, Germany, said during the presentation.
In a 24-week randomized, double-blind, placebo-controlled phase 2 study and a phase 3 clinical study, Kahaly and colleagues assigned 171 adults with active moderate to severe thyroid eye disease, diagnosed in the previous 9 months, to infusions of teprotumumab (Horizon Therapeutics; n = 84; mean age, 51.5 years; 69% women; 24% smokers) or placebo (n = 87; mean age, 51.4 years; 77% women; 30% smokers) every 3 weeks (eight infusions).
Among the teprotumumab group, proptosis was reduced by at least 2 mm for 77.4% of participants, with an average reduction from baseline of –2.63 mm vs. 14.9% of the placebo group with an average reduction from baseline of 0.31 mm at week 24. Diplopia improved by at least one grade for 69.7% of the teprotumumab group vs. 30.5% with placebo, and 61.9% of the teprotumumab group had a clinical activity score of 0 or 1, indicating minimal inflammation vs. 21.8% among the placebo group (P < .001 for all comparisons).
Participants in the teprotumumab group also reported significant improvements in quality of life, as assessed with the Graves’ orbitopathy quality of life (GO-QoL) scale, in visual functioning and appearance, according to Kahaly.
Asked during a question session after the presentation about longer-term durability of treatment effect with teprotumumab, Kahaly said most participants who responded to treatment at 24 weeks remained responders at 72 weeks.
“It is probably useful and recommended to continue the treatment and to give more than eight infusions hoping for an even higher response rate,” Kahaly said.
Asked about where teprotumumab may fit in the current treatment landscape, Kahaly said head-to-head trials are needed.
“We have never observed such convincing results for the other drugs [currently used to treat thyroid eye disease],” Kahaly said. “This is the first drug that seems to be a disease-modifying drug, and reducing proptosis by a mean 3 mm, this drug is well tolerated. There are many facts and data supporting the use of this drug and … once it will be cleared by the FDA this drug will probably become the first-line treatment.” – by Jill Rollet
Reference:
Kahaly G, et al. Oral #2. Presented at: 89th Annual Meeting of the American Thyroid Association; Oct. 30-Nov. 3, 2019; Chicago.
Disclosure: Kahaly reports Johannes Gutenberg University Medical Center has received research-associated funding from Horizon and River Vision.