Abstract
Gorham-Stout syndrome is an aggressive, non-hereditary, and rare disease affecting bone metabolism. Its etiology and pathogenesis remain elusive. The syndrome manifests with diverse clinical symptoms, often leading to frequent misdiagnoses and presenting challenges in treatment. In this study, we report a case of cranial and maxillary osteolysis in a 47-year-old female patient with somatic mutations in the VEGF-A, VEGF-B, and VEGF-C genes and the EPHB4 gene. After treatment with bisphosphonates, this patient still had persistent resorption of the mandible, but switching to a teriparatide and denosumab combination yielded substantial improvement. This study is the first report to show that teriparatide combined with denosumab can be used to treat Gorham-Stout syndrome.
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Funding
This study was financially supported by the National Natural Science Foundation of China (No. 82160321), the Medicine Leading Talent of Yunnan Province Health Care Committee (No. L-2018010), and Yunnan Provincial Clinical Medical Research Centre for Oral Diseases Research Fund (No. 2022QN002).
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Zhang, L., Wang, W., Wen, L. et al. Treatment for Gorham-Stout syndrome with a combination of teriparatide and denosumab. Osteoporos Int 35, 727–731 (2024). https://doi.org/10.1007/s00198-023-06995-1
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DOI: https://doi.org/10.1007/s00198-023-06995-1