ODP080 Bisphosphonate treatment for osteoporosis in children with Gorham-Stout syndrome: a case report

Abstract

Abstract Background Gorham-Stout disease (GSD) is a rare disease defined by progressive bone disappearance due to massive unicentric or multicentric osteolysis with proliferation of vascular structures. Symptoms and signs vary depending on the site of invasion. Including some self-limiting GSD, most treatments are palliative and limited to symptom management. Various treatments have been tried for GSD, but there are no standardized treatment protocols. Treatment includes radiological treatment, surgery, and medications such as bone absorption inhibitors (calcitonin, bisphosphonate (BP)), vitamin D and immune modulators (interferon alpha-2b, Sirolimus) Case presentation: A 4-year-old female patient presented with prolonged back pain after a fall. Pathologic compression fracture in T9, L1 vertebrae identified in simple spine image, and GSD was confirmed by MRI and biopsy finding as hemangioma in vertebrae. This patient has been treated with a combination therapy of Sirolimus and BP for 1 year. BP treatment consist with oral BP (Alendronate) between 3 intravenous BP (Pamidronate) every 4 months. After a year of treatment, BMD improved from initial -1.3 TBLH (z-score) to -0.5/0.4/0.6 Spine/Both femur (z-score), but MRI findings suggest mild aggravation of multiple compression fractures of spines of T9-L5. After 1-year of treatment, at present, the girl is alive and staying with tolerable condition. Conclusions Combination treatment with bisphosphonate and Sirolimus for childhood GSD can be helpful in slowing the demineralization of GSD without any significant adverse effect. Presentation: No date and time listed