Novel observational study protocol to develop a prediction model that identifies patients with Graves' ophthalmopathy insensitive to intravenous glucocorticoids pulse therapy

Abstract

Introduction: Intravenous glucocorticoids pulse therapy is the first-line treatment for moderate-to-severe and active Graves' ophthalmopathy, with a large proportion of patients having poor efficacy and exposed to the risk of glucocorticoids adverse effects. We introduce a novel protocol to develop a prediction model designed to identify patients with Graves' ophthalmopathy who are not likely to benefit from intravenous glucocorticoids pulse therapy before administration, so that these patients can advance the time to receive appropriate treatment. Existing prediction models for prognosis of Graves' ophthalmopathy have usually focused on traditional clinical indicators without adequate consideration of orbital soft tissue changes. Our protocol for model development will address this limitation by using artificial intelligence models to quantify facial morphological changes.

Methods and analysis: This study is a single-centre, prospective and observational study. A sample size of 278 patients with moderate-to-severe and active Graves' ophthalmopathy will be prospectively recruited at ophthalmology clinic of Shanghai Ninth People's Hospital to collect clinical and artificial intelligence model's baseline data as potential variables to develop the prediction model. They will receive 12-week intravenous glucocorticoids pulse therapy according to the 2021 European Group on Graves' Orbitopathy treatment guideline. After standard medication course and following 12-week observation, patients will be evaluated for the effectiveness of treatment in our ophthalmology clinic and divided into glucocorticoids-sensitive and glucocorticoids-insensitive groups. The model will be developed by means of multivariate logistic regression to select the best variables for the prediction of glucocorticoids treatment efficacy before administration. The result of the study will provide evidence for the use of a prediction model to personalise treatment options for patients with moderate-to-severe and active Graves' ophthalmopathy.

Ethics and dissemination: The study received approval from the Ethics Committee of Shanghai Ninth People's Hospital (ethical approval number: SH9H-2020-T211-1. Findings will be disseminated via peer-reviewed publications and conference presentations.

Trial registration number: ChiCTR2000036584 (Pre-results).

Keywords: clinical trials; ophthalmology; thyroid disease.