Physician’s Weekly spoke recently with Roman Shinder, MD, FACS, from the Departments of Ophthalmology and Otolaryngology at SUNY Downstate Medical Center, about study results he and his colleague, Anna Artymowicz, MD, presented at ASOPRS 2021 on the use of teprotumumab as monotherapy in patients with dysthyroid optic neuropathy.


PW: Why is it important to study teprotumumab as monotherapy for dysthyroid optic neuropathy? What needs existed for your research?
RS: Dysthyroid optic neuropathy is tricky for eye care professionals to treat and the options before teprotumumab was available all carried inherent drawbacks and difficulties, not only for the clinicians, but also for patients. Once teprotumumab gained FDA approval, I wanted to know whether or not we could use it—and if so, how successful it would be—in this patient population, for many reasons, but one is that this is a critical patient population for whom if something is not done quickly, they have a real chance of losing vision that could sometimes be permanent. There was a real impetus for trying it out, and especially if it worked, trying to get it on board quickly to try to achieve a good result for these patients.


What did you and your colleagues set out to determine with this study, and how did you go about doing so (how was the study conducted)?
We found patients with clinical evidence of dysthyroid optic neuropathy, who upon evaluation were offered traditional treatment options, including ensuring they understood teprotumumab and potential associated adverse events. Four patients were recruited to start teprotumumab monotherapy. Because it is a time-sensitive diagnosis, my staff and I did all in our power to get them approved through their insurance companies and infused as quickly as possible. We were able to do that within a couple weeks from the initial assessment and diagnosis. We wanted to see whether or not teprotumumab given alone to these patients could be effective in halting their progression of disease, and if we were lucky, potentially reversing some of the signs and symptoms of their disease.


What findings from your study are important to stress to our physician readers?
In all four patients we found a very quick and robust response to the teprotumumab, in which all of the signs/symptoms/indicators that we were studying improved drastically. The important ones for ophthalmologists would be visual acuity, the visual field tests done in the office through an automated Humphrey visual field, the color vision test through Ishihara color plate testing, and pupillary assessment with light response, specifically, their relative afferent pupil defect. These were the key things that we were assessing. After only the second of eight infusions, all of these metrics improved drastically and in some cases completely reversed.


What are the implications of your findings?

It’s hard to make across-the-board recommendations with a small study of only four patients, but I do think that the results were promising, and anecdotally through other personal experience in my practice, we’ve now treated more than four patients—we just haven’t published that data—and we continue to see good results. To treating clinicians, I would say that if you are not thinking about teprotumumab in patients with dysthyroid optic neuropathy, it’s at least something you may want to consider, think about, and discuss with your patients. And if it makes sense, you can try it out, because again, the FDA approval is for overall thyroid eye disease, and dysthyroid optic neuropathy definitely falls within that umbrella. And if it’s something you’re keen on, I would really recommend working hard with your staff and being vigilant to get patients approved and infused as quickly as possible.

You can have pushback through insurance companies for a number of reasons. It’s a relatively new FDA-approved medicine, so insurance companies are trying to get familiar with approving it and using it. Also, it’s fairly expensive, so insurance companies want to be sure they know what they’re approving, and they really do a detailed review before the approval is granted. But secondarily, if a doctor and his office staff are very vigilant and don’t just leave this on the back burner for a while, things can usually run pretty quickly and smoothly, but it does require quite a bit of legwork. So, I try to do that with my office staff the very first day that the patient visits and they agreed to proceed—we take our time and make sure all the detailed records are in one place, and we submit everything as a single package. We try our best to not allow the insurance companies to come back to us saying there is a missing a document or to please send X, Y, or Z. We try to send everything in detailed fashion all upfront, so all that’s left is for them to review and typically approve.


What research needs still exist in this area?

I think larger, potentially multi-institutional, potentially prospective studies are warranted to really make a bigger recommendation by which teprotumumab should be compared to more conventional treatments for this disease that are being used, probably more widespread, such as surgical orbital decompression, systemic steroids, and orbital radius.



Artymowicz A, Shinder R, et al Teprotumumab as Monotherapy for Dysthyroid Optic Neuropathy.
Presented at: ASOPRS Fall Scientific Symposium; November 11-12, 2021; New Orleans, LA.