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Sirolimus on Gorham-Stout disease. Case report
Vanessa García1, Gloria Alonso-Claudio1, M-Teresa Gómez-Hernández2
1Departamento de Medicina Interna. Hospital Clínico Universitario de Salamanca. Salamanca. España.
Insights
Gorham-Stout disease (GSD), a rare bone disorder, can manifest with pleural effusion. Sirolimus treatment showed promising remission in a patient with this GSD presentation.
Area of Science:
- Oncology
- Vascular Biology
- Rare Diseases
Background:
- Gorham-Stout disease (GSD) is a rare, idiopathic condition characterized by abnormal vascular proliferation leading to bone matrix destruction.
- GSD commonly affects the bone but can present with extraskeletal manifestations.
Observation:
- A 43-year-old woman presented with sternal pain, dyspnea, abdominal mass, and serous-hematic pleural effusion.
- Imaging revealed lesions on the left ribs, confirmed by biopsy showing lymphatic vascular proliferation in chest wall bone tissue.
Findings:
- Sirolimus treatment initially induced disease remission within one month.
- Treatment discontinuation due to metrorrhagia led to symptom recurrence; re-initiation resulted in symptom disappearance within four weeks.
Implications:
- This case highlights sirolimus as a potential therapeutic alternative for Gorham-Stout disease with pleural effusion.
- Further research is warranted to establish sirolimus efficacy and safety in managing GSD complications.
Background:
Gorham-Stout disease (GSD) is a rare disease of unknown etiology characterized by vascular proliferation that produces destruction of bone matrix.
Case Description:
This case is about 43 year old woman who begins with pain in sternum, dyspnea, abdominal mass and, serous-hematic pleural effusion. Imaging tests were performed showing lesions on 6th and 10th left ribs archs. Later, a thoracotomy was performed observed absence of the end of the 6th and lung, pleural and costal biopsy was token. The histologic features described lymphatic vascular proliferation in bone tissue of chest wall. Other pathologies were excluded and in view of the findings, GSD diagnosis was made.
Treatment And Outcome:
treatment was initiated with sirolimus achieving remission of the disease after the first month; however, because the presence of metrorrhagia the treatment was discontinued, reappearing symptoms afterwards. For that reason the treatment was restarted getting disappearance of the symptoms again, 4 weeks later.
Clinical Relevance:
we present the first clinical cases of EGS with pleural effusion with response to sirolimus treatment that could be an alternative to the current therapy.
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