ReviewBMC musculoskeletal disorders2020
Gorham-Stout disease successfully treated with sirolimus (rapamycin): a case report and review of the literature.
Review in BMC musculoskeletal disorders, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 17 papers.
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Who cites it
17 citing papers in PubMed, 35 citations in OpenAlex.
- Gorham-Stout disease with thoracic involvement: pathogenic mechanisms, respiratory complications, and multimodal therapies.Orphanet journal of rare diseases · 2026Review
- Vascular malformations: from genetics to therapeutics.EMBO molecular medicine · 2026Review
- Pathological Fractures in Rare Bone Diseases: Clinical Cases.Acta medica Lituanica · 2026Article
- Phloridzin Mitigates Gorham-Stout Disease by Dual Inhibition of Osteoclastogenesis Related to the CaACS omega · 2025Article
- Chylothorax Associated with the Syndrome of Idiopathic Osteolysis Including Gorham-Stout Disease: Spontaneous Remission Without Treatment.Turkish archives of pediatrics · 2024Article
- Gorham-Stout disease: remission with sirolimus therapy.BJR case reports · 2023Article
- The role of genetic and epigenetic factors in determining the risk of spinal fragility fractures: new insights in the management of spinal osteoporosis.Quantitative imaging in medicine and surgery · 2023Review
- Case Report and Literature Review: Bisphosphonate, Sirolimus, and Atenolol Treatment in a 4-Year-Old Child Diagnosed with Gorham-Stout Disease.Pharmaceuticals (Basel, Switzerland) · 2023Article
- The spectrum of imaging manifestations of Gorham-Stout disease: a novel dynamic contrast-enhanced MR lymphangiography.Orphanet journal of rare diseases · 2023Article
- The molecular mechanism of Gorham syndrome: an update.Frontiers in immunology · 2023Review
- A new therapeutic approach in Gorham-Stout disease: a case report.Frontiers in surgery · 2023Article
- Gorham-Stout case report: a multi-omic analysis reveals recurrent fusions as new potential drivers of the disease.BMC medical genomics · 2022Article
- "A case of vanishing bone disease complicated by chylothorax- diagnosis and treatment".JRSM open · 2022Article
- Gorham-Stout disease of skull base leading to cranial settling and rhinorrhea: a case-based review.Child's nervous system : ChNS : official journal of the International Society for Pediatric Neurosurgery · 2022Review
- Current concepts from diagnosis to management in Gorham-Stout disease: a systematic narrative review of about 350 cases.EFORT open reviews · 2022Review
- Gorham-Stout disease of the mandible, manubrium and cervical spine presenting as bilateral chylothorax.BMJ case reports · 2021Article
- Stimulation of Treg Cells to Inhibit Osteoclastogenesis in Gorham-Stout Disease.Frontiers in cell and developmental biology · 2021Article
Corrections and comments
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Authors and funding
9 authors at 1 institution in 1 country.
Funding
Abstract
backgroundGorham-Stout disease (GSD) is a rare disease characterized by bone lesions and osteolysis. Therapy usually involves surgical resection. Sirolimus (Rapamycin) is used in some patients with GSD but the efficacy and safety of Sirolimus remains unclear. We propose that Sirolimus may be a novel therapeutic for GSD and present a case and review of literature that supports this. CASE PRESENTATION: We presented a 1-year-old boy with GSD involving osteolysis of the right humerus with fracture of the left femur complicated by an effusion in the right pleural cavity. X-rays showed osteolysis in the right clavicle. A large pleural effusion was observed on the right-side, and the left lung was significantly compressed. X-rays also showed a fracture of the left femur. A femoral biopsy was performed that showed necrotic tissue in the cortical bone and a large number of irregularly shaped capillaries that proliferated within the necrotic tissue. Dilated lymphatic vessels were seen adjacent to the cortex, with fibrous tissue hyperplasia. We prescribed sirolimus, which is an oral mTOR inhibitor, for two consecutive years. The boy recovered well without other progressive bone lesions and participates in normal daily activities. His growth and development are the same as that of his peers. DISCUSSION AND
conclusionGorham-Stout disease is a rare and enigmatic disease characterized by the presentation of an intraosseous lymphatic anomaly (LM), which results in progressive bone resorption. Based on this case report and a literature review, we conclude that sirolimus may be an effective alternative medication for GSD.
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