Trial reportPediatric blood & cancer2019
Efficacy of systemic sirolimus in the treatment of generalized lymphatic anomaly and Gorham-Stout disease.
Trial report in Pediatric blood & cancer, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT00975819 (A Phase 2 Study - Clinical Trial Assessing Efficacy and Safety of the mTOR Inhibitor Sirolimus in the Treatment of Complicated Vascular Anomalies), which is not on this map. Cited by 61 papers, 6 of them syntheses that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
A Phase 2 Study - Clinical Trial Assessing Efficacy and Safety of the mTOR Inhibitor Sirolimus in the Treatment of Complicated Vascular Anomalies
Who cites it
61 citing papers in PubMed, 6 syntheses or guidelines pooled it, 121 citations in OpenAlex.
- Japanese Clinical Practice Guidelines for Vascular Tumors, Vascular Malformations, Lymphatic Malformations, and Lymphangiomatosis 2022.The Journal of dermatology · 2026Guideline
- Japanese clinical practice guidelines for vascular tumors, vascular malformations, lymphatic malformations, and lymphangiomatosis 2022.Surgery today · 2026Guideline
- Japanese clinical practice guidelines for vascular tumors, vascular malformations, lymphatic malformations, and lymphangiomatosis 2022.Japanese journal of radiology · 2026Guideline
- Clinical features and current management experience in Gorham-Stout disease: a systematic review.Orphanet journal of rare diseases · 2025Pooled it
- Sirolimus for vascular anomalies in the first year of life: a systematic review.Journal of perinatology : official journal of the California Perinatal Association · 2024Pooled it
- Japanese clinical practice guidelines for vascular tumors, vascular malformations, lymphatic malformations, and Lymphangiomatosis 2022.Pediatrics international : official journal of the Japan Pediatric SocietyGuideline
- Therapeutic Efficacy of Sirolimus in Skeletal Manifestations of Gorham-Stout Disease in Adults: A Systematic Review.Cureus · 2026Review
- Imaging of complex lymphatic anomalies in children.Pediatric radiology · 2026Review
- Japanese Clinical Practice Guidelines for Vascular Tumors, Vascular Malformations, Lymphatic Malformations, and Lymphangiomatosis 2022.Journal of plastic and reconstructive surgery · 2026Review
- The role of the lymphatic system in musculoskeletal system health and disease: research progress and future directions.Bone research · 2026Review
- Lymphatic vascular aging and age-related bone loss: current status and future perspectives.Frontiers in immunology · 2026Review
- Vascular malformations: from genetics to therapeutics.EMBO molecular medicine · 2026Review
- Multimodal treatment strategies in generalized lymphatic anomaly with pleural effusion: a case study and literature review.Kardiochirurgia i torakochirurgia polska = Polish journal of cardio-thoracic surgery · 2025Article
- Lymphatic malformations involving the thorax in children: a retrospective cohort study.BMC pulmonary medicine · 2025Article
- Gorham-Stout disease of the craniovertebral junction causing basilar impression and Chiari malformation type I: illustrative case.Journal of neurosurgery. Case lessons · 2025Article
- Sclerothrapy of giant lymphatic malformation in neonates.Journal of perinatology : official journal of the California Perinatal Association · 2025Article
- Generalized lymphatic anomaly in a pediatric patient manifesting as a rare presentation of hemorrhagic pleural effusion: a case report.BMC pediatrics · 2025Article
- Exploring the genetic alterations of Gorham-Stout disease.Frontiers in endocrinology · 2025Review
- Surgical treatment of Gorham-Stout disease combined with scoliosis: a case report and literature review.BMC musculoskeletal disorders · 2024Review
- Gorham-Stout disease and multiple cervical lymphangiomas: case report.Multidisciplinary respiratory medicine · 2024Article
1 more citing papers are in PubMed but not listed here.
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
10 authors at 8 institutions in 3 countries.
Funding
Abstract
backgroundGeneralized lymphatic anomaly (GLA) and Gorham-Stout disease (GSD) are rare complicated lymphatic malformations that occur in multiple body sites and are associated with significant morbidity and mortality. Treatment options have been limited, and conventional medical therapies have been generally ineffective. Emerging data suggest a role for sirolimus as a treatment option for complex lymphatic anomalies. PROCEDURE: Disease response was evaluated by radiologic imaging, quality of life (QOL), and clinical status assessments in children and young adults with GLA and GSD from a multicenter systematic retrospective review of patients treated with oral sirolimus and the prospective phase 2 clinical trial assessing the efficacy and safety of sirolimus in complicated vascular anomalies (NCT00975819). Sirolimus dosing regimens and toxicities were also assessed.
resultsEighteen children and young adults with GLA (n = 13) or GSD (n = 5) received oral sirolimus. Fifteen patients (83%) had improvement in one or more aspects of their disease (QOL 78%, clinical status 72%, imaging 28%). No patients with bone involvement had progression of bone disease, and the majority had symptom or functional improvement on sirolimus. Improvement of pleural and pericardial effusion(s) occurred in 72% and 50% of affected patients; no effusions worsened on treatment.
conclusionsSirolimus appears effective at stabilizing or reducing signs/symptoms of disease in patients with GLA and GSD. Functional impairment and/or QOL improved in the majority of individuals with GLA and GSD with sirolimus treatment.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.