As the current published evidence for the use of mycophenolate mofetil (MMF) in the treatment of Graves orbitopathy (also known as thyroid eye disease, or TED) is limited, the investigators aimed to assess the efficacy and safety of the combination of MMF and oral prednisolone for TED in a real-world setting.
This was a prospective, nonrandomized, interventional case series of 261 adults diagnosed with moderate-to-severe TED at a single eye hospital in Iran who were given MMF plus oral prednisolone. The primary outcome was overall response at the 4-, 6-, 12-, and 18-month timepoints, including improvement in visual activity, intraocular pressure (IOP), and clinical activity score (CAS).
The response rate with the combination of MMF and oral prednisolone improved at each timepoint, from 67.7% at month 3 to 94.2% at month 18. The CAS, diplopia, and proptosis also continued to improve even after discontinuation of medications. Intraocular pressure did not increase over time. There were only 6 adverse events noted during the study, and none were severe.
Due to the lack of or inconclusive evidence obtained by randomized controlled trials, the use of biologic drugs in the treatment of TED is controversial. A clinical trial study with a control group is needed to provide additional evidence for biologics’ effectiveness.
Clinicians may consider this combination of medications as a relatively safe alternative treatment option for patients with moderate-to-severe TED who do not qualify for or do not want treatment with teprotumumab.