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M. K. Green, D. A. Hollsten, S. V. Owen; Treatment of Refractory Thyroid-Related Immune Orbital Disease With Cyclophosphamide. Invest. Ophthalmol. Vis. Sci. 2008;49(13):4733. doi: https://doi.org/.
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A small subset of patients with thyroid-related immune orbital disease (TRIO) develop severe signs and symptoms of the disease. Traditional treatments such as oral and intravenous steroid, orbital irradiation, and orbital decompression surgery are only partially effective. In the past 30 years, different immunosuppressant medications have been tried, with variable results. We present a retrospective case review of 18 patients who received the immunosuppressant cyclophosphamide as part of their treatment regimen. Patients on this therapy experienced improvement in symptoms, along with stabilization or improvement in visual acuity and exophthalmometry measurements.
All patients seen between 1995-2007 with a diagnosis of Graves orbital disease were reviewed. Three hundred and seventy-six patients were identified. Patients with one office visit, indicating mild orbital disease, were eliminated, leaving 262 patients. Of these, 18 patients had received cyclophosphamide and were included in the data analysis. Outcome measures included subjective improvement based on patient and physician observation, along with pre- and post-treatment visual acuity, each eye, and exophthalmometry measurements, each eye.
The prevalence of patients with TRIO severe enough to warrant referral to a hematologist-oncologist for cyclophosphamide in this study was 0.05%. Mean follow-up time after conclusion of cyclophosphamide therapy was 32.6 months (median = 17.5 months). The majority of patients experienced a subjective improvement in their symptoms. Despite a small sample size, three of the four objective measures showed clinically important effect sizes.
The incidence of severe TRIO requiring systemic therapy is known to be low (<=3%), and the incidence of very severe disease, which is refractory to systemic steroid, orbital irradiation, and orbital decompression, is even lower. The rarity of the disease has rendered the study of its treatment difficult. However, patients with very severe TRIO are at risk for vision loss due to compressive optic neuropathy, and painful exposure keratopathy, each of which can be blinding. Our work suggests that there is a treatment effect of the alkylating agent cyclophosphamide in cases of severe TRIO, specifically regarding visual acuity and proptosis.
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