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

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 treatme...

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Autores principales: Ahn, Moon Bae, Shin, Chungwoo
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Oxford University Press 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9624916/
http://dx.doi.org/10.1210/jendso/bvac150.324
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author Ahn, Moon Bae
Shin, Chungwoo
author_facet Ahn, Moon Bae
Shin, Chungwoo
author_sort Ahn, Moon Bae
collection PubMed
description 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
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spelling pubmed-96249162022-11-14 ODP080 Bisphosphonate treatment for osteoporosis in children with Gorham-Stout syndrome: a case report Ahn, Moon Bae Shin, Chungwoo J Endocr Soc Bone & Mineral Metabolism 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 Oxford University Press 2022-11-01 /pmc/articles/PMC9624916/ http://dx.doi.org/10.1210/jendso/bvac150.324 Text en © The Author(s) 2022. Published by Oxford University Press on behalf of the Endocrine Society. https://creativecommons.org/licenses/by-nc-nd/4.0/This is an Open Access article distributed under the terms of the Creative Commons Attribution-NonCommercial-NoDerivs licence (https://creativecommons.org/licenses/by-nc-nd/4.0/), which permits non-commercial reproduction and distribution of the work, in any medium, provided the original work is not altered or transformed in any way, and that the work is properly cited. For commercial re-use, please contact journals.permissions@oup.com
spellingShingle Bone & Mineral Metabolism
Ahn, Moon Bae
Shin, Chungwoo
ODP080 Bisphosphonate treatment for osteoporosis in children with Gorham-Stout syndrome: a case report
title ODP080 Bisphosphonate treatment for osteoporosis in children with Gorham-Stout syndrome: a case report
title_full ODP080 Bisphosphonate treatment for osteoporosis in children with Gorham-Stout syndrome: a case report
title_fullStr ODP080 Bisphosphonate treatment for osteoporosis in children with Gorham-Stout syndrome: a case report
title_full_unstemmed ODP080 Bisphosphonate treatment for osteoporosis in children with Gorham-Stout syndrome: a case report
title_short ODP080 Bisphosphonate treatment for osteoporosis in children with Gorham-Stout syndrome: a case report
title_sort odp080 bisphosphonate treatment for osteoporosis in children with gorham-stout syndrome: a case report
topic Bone & Mineral Metabolism
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9624916/
http://dx.doi.org/10.1210/jendso/bvac150.324
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