Investigators Release Teprotumumab Recommendations

Macro closeup of young woman face portrait in profile side with Grave's disease hyperthyroidism symptoms of ophthalmopathy bulging eyes proptosis edema
Macro closeup of young woman face portrait in profile side with Grave’s disease hyperthyroidism symptoms of ophthalmopathy bulging eyes proptosis edema
An expert panel offers guidelines for the thyroid eye disease therapy.

Not only can teprotumumab be used as a first-line thyroid eye disease treatment, according to an expert panel, it is also appropriate for a wider range of patients than were included in the 2 clinical trials, such as those with a longer duration of disease and more broadly defined activity and severity. The group of clinical investigators issued their recommendations for best practices in the Journal of Neuro-Ophthalmology.

Patients with thyroid eye disease (TED) present with dry, red eyes, photophobia, diplopia, and pain with eye movement. TED occurs at a rate of 16 and 2.9 per 100,000 among women and men, respectively.

Historically, clinicians have treated TED with nonspecific immunosuppression via corticosteroids and surgical intervention. However, both of these options are associated with risks. Teprotumumab, an anti-insulin-like growth factor I receptor (IGF-IR) inhibitor antibody, directly targets proptosis and strabismus.

At clinical trials, patients who received TED had greater reduction in proptosis and clinical activity scores compared with placebo. In addition, teprotumumab was associated with increased quality of life. The most common side effects were muscle spasms, nausea, and alopecia.

In January of 2020, teprotumumab gained approval by the US Food and Drug Administration (FDA) for the treatment of TED.

An expert panel convened in October-November 2019 to review the current evidence of teprotumumab use for TED and to formulate best practices recommendations.

Teprotumumab should be considered a first-line therapy for significant TED, according to the panel’s recommendations. At clinical trial, the treatment was both more effective and safer than the other options, such as corticosteroids.

Teprotumumab may be given to patients with TED concomitantly with antithyroid medications. The panel stated that waiting until the patient reaches a euthyroid state was unnecessary due to the absence of evidence that thyroidal status affected efficacy or safety.

Although phase 2 and 3 clinical trials only recruited patients 18 to 80 years old, the panel stated that teprotumumab may be considered among adolescents and the elderly. For adolescents, physicians should wait until linear growth has ceased and consult with a pediatric endocrinologist.

Teprotumumab can be prescribed to patients with diabetes, however, these patients should be monitored for changes to glycemic control, as the IGF-IR pathway co-regulates glucose homeostasis. Teprotumumab should be avoided among women who are pregnant or who may become pregnant. For patients with inflammatory bowel disease (IBD), it remains unclear what outcomes will occur among these patients, as they were excluded from clinical trials.

In general, the panel advises physicians to use the dosing tested in clinical trials. The established dosing protocol was 8 infusions for 6 months. Infusion 1 was dosed at 10 mg/kg by intravenous infusions for 90 minutes. The remaining doses were increased to 20 mg/kg for 60 minutes, given no adverse reactions to dose 1.

The concomitant use of teprotumumab with biologics is strongly discouraged, but concomitant use with corticosteroids are acceptable in some cases, according to the panel.

The report concludes that teprotumumab can be safely and effectively used for the treatment of patients with TED, however, the panel did note that teprotumumab remains more costly than treatment alternatives. Clinicians and their patients should weigh the benefits against the treatment costs.

Disclosure: Multiple authors declared affiliations with the biotech, pharmaceutical, and/or device companies. Please see the original reference for a full list of authors’ disclosures.  


Douglas RS, Wang Y, Dailey RA, et al. Teprotumumab in clinical practice: recommendations and considerations from the OPTIC Trial investigators. J Neuroophthalmol. 2021;41(4):461-468. doi:10.1097/WNO.0000000000001134