4.7 Article

Teprotumumab and the Evolving Therapeutic Landscape in Thyroid Eye Disease

Journal

JOURNAL OF CLINICAL ENDOCRINOLOGY & METABOLISM
Volume 107, Issue -, Pages S36-S46

Publisher

ENDOCRINE SOC
DOI: 10.1210/clinem/dgac168

Keywords

thyroid eye disease; Graves ophthalmopathy; teprotumumab; management; targeted therapy; biologic therapy

Funding

  1. Research to Prevent Blindness
  2. National Institutes of Health [P30 026877]

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Thyroid eye disease (TED) is a sight-threatening autoimmune condition with limited treatment options. However, targeted biologic therapies, such as teprotumumab, have emerged as effective alternatives to traditional steroid regimens. Collaborative efforts between endocrinologists and ophthalmologists in developing personalized treatment plans can enhance safety and outcomes for TED patients.
Context Thyroid eye disease (TED) is a sight-threatening and debilitating autoimmune condition, with limited therapies available, that often poses diagnostic and therapeutic challenges. In recent years, the treatment landscape has shifted to early intervention with targeted therapy. Methods A PubMed review of the literature was conducted for the period between 1979 and 2021. Search terms included thyroid eye disease, teprotumumab, targeted therapy, Graves disease, Graves ophthalmopathy, dysthyroid optic neuropathy, and related terms in different combinations. Novel biologic therapies for TED have emerged as alternatives to traditional steroid regimens in recent years. New insights into TED pathophysiology have uncovered the role of the insulin-like growth factor 1 receptor (IGF-1R) and led to the development of teprotumumab, an IGF-1R-inhibiting monoclonal antibody. Results Randomized clinical trials demonstrating the efficacy of teprotumumab for TED led to Food and Drug Administration approval. Teprotumumab is gradually replacing immunosuppressive agents as first-line therapy in the United States for active moderate-to-severe TED, while emerging reports also show its use in other stages of the disease. Recent data highlight risk factors for adverse events and screening protocols to maximize patient safety. Personalized therapeutic plans developed through effective partnership between endocrinologists and ophthalmologists aim to enhance the safety and outcomes of TED treatments and improve care for this complex disease. Conclusion TED management is shifting to an era of targeted therapy with multidisciplinary care. Teprotumumab has demonstrated superior efficacy to conventional treatments and has transformed our therapeutic and surgical algorithms. Clinical guidelines and additional studies are needed to further guide and refine therapy.

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