麻豆传媒

NEW ORLEANS -- Patients taking the new, ultra-expensive thyroid eye disease drug teprotumumab (Tepezza) are typically tolerating ear-related side effects, with some saying they're more interested in protecting their vision than their hearing, reported an ophthalmologist.

One patient, for example, insisted on continuing the treatment despite hearing loss, said Suzanne Freitag, MD, of Massachusetts General Hospital in Boston, during a presentation here at the American Academy of Ophthalmology annual meeting. "She was arguing with me: 'I want my eyes, I don't care as much about my ears.'"

Hearing loss is the most significant side effect linked to teprotumumab, which received FDA approval in 2020 and can cost up to $400,000 for a full 6-month treatment course, said Michael Kazim, MD, of Columbia University in New York City. The drug treats thyroid eye disease, also known as Graves' eye disease, an autoimmune disorder that can cause bulging eyes and a long list of other ocular problems.

In addition to teprotumumab, much cheaper drugs for the disease, such as steroids, are available, noted Kazim.

Freitag said her clinic has treated more than 100 patients with teprotumumab. An analysis showed that 36% reported ear/hearing changes, the most common side effect other than muscle spasms (55%), and more common than several others, including fatigue (30%), nausea (25%), dysgeusia (18%), and diarrhea (17%). The ear effects aren't just loss of hearing, she explained; patients also mentioned tinnitus and a feeling of fullness in the ear.

Despite these effects, however, 96% of patients surveyed said their side effects were mild or tolerable. "I find this a powerful addition to my armamentarium," Freitag noted. "We used to tell patients with moderate and severe thyroid eye disease to expect that they would not look the same again when their disease was running out. For many patients, this is no longer true."

Another study presented by Connie Sears, MD, of Stanford University in California, offered a somewhat similar perspective on the tolerability and side effect profile of teprotumumab. Researchers tracked 51 patients at three centers who were taking teprotumumab to treat thyroid eye disease. At a mean follow-up of 35 weeks, 78% reported adverse effects related to treatment, including muscle cramps (58%), diarrhea (40%), hair loss (33%), fatigue (23%), and hearing loss (23%). Symptoms persisted in 30% of patients at last follow-up. Treatment was paused in 4% of patients, and halted in 12%.

Finally, a study of 1,101 patients (71% women, mean age 58), presented at the AAO meeting by Raymond Douglas, MD, PhD, of Cedar-Sinai Medical Center in Los Angeles, reported a similar percentage of patients who stopped treatment with teprotumumab: 8% due to adverse events, 1% for noncompliance, fewer than 1% for "site-of-care challenges" or payer restrictions, and fewer than 1% for "patient or physician concern."

Among those who stopped treatment for any reason, the most common adverse effects -- not necessarily the causes of the discontinuation decisions -- were muscle spasms (49 patients), fatigue (28), nausea (27), diarrhea (26), hearing loss (21), and tinnitus (7).