Trial reportPediatrics2016
Efficacy and Safety of Sirolimus in the Treatment of Complicated Vascular Anomalies.
Trial report in Pediatrics, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 2 registered trials, which are not on this map. Cited by 343 papers, 9 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.
Phase II Clinical Trial of MEK Inhibitor Trametinib in the Treatment of Complicated Extracranial Arterial Venous Malformation (VM)
A Modular Open Label, Signal Seeking, Phase II Trial of Targeted Therapies for Patients With Slow-Flow or Fast-Flow Vascular Malformations (TARGET-VM)
Who cites it
343 citing papers in PubMed, 9 syntheses or guidelines pooled it.
- 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.Japanese journal of radiology · 2026Guideline
- Japanese clinical practice guidelines for vascular tumors, vascular malformations, lymphatic malformations, and lymphangiomatosis 2022.Surgery today · 2026Guideline
- Pooled it
- Research trends and hotspots in sclerotherapy for vascular malformations: bibliometric and visual analyses.Frontiers in physiology · 2026Pooled it
- Diagnosis and management of superficial arteriovenous malformations: French healthcare network's recommendations.Orphanet journal of rare diseases · 2025Guideline
- Global research trends and basis of venous/lymphatic malformations during 2003-2023: a bibliometric study over two decades.Frontiers in medicine · 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
- Sirolimus treatment for paediatric head and neck lymphatic malformations: a systematic review.European archives of oto-rhino-laryngology : official journal of the European Federation of Oto-Rhino-Laryngological Societies (EUFOS) : affiliated with the German Society for Oto-Rhino-Laryngology - Head and Neck Surgery · 2023Pooled it
- Sensitivity Analysis of the Efficacy of Everolimus for Neurocognitive Symptoms in PTEN Hamartoma Tumor Syndrome.Advances in therapy · 2026Trial
- Eppikajutsuto for Treatment of Lymphatic Malformations in Children: A Nonrandomized Clinical Trial.JAMA network open · 2025Trial
- Safety findings from the phase 1/2 MOSAIC study of miransertib for patients with PIK3CA-related overgrowth spectrum or Proteus syndrome.Orphanet journal of rare diseases · 2025Trial
- A phase 2 randomized, double-blind trial of ART-001, a selective PI3Kα inhibitor, for the treatment of slow-flow vascular malformations.Orphanet journal of rare diseases · 2025Trial
- Preliminary results of the European multicentric phase III trial regarding sirolimus in slow-flow vascular malformations.JCI insight · 2023Trial
- Clinical differences in sirolimus treatment with low target levels between children and adults with vascular malformations - A nationwide trial.Clinical and translational science · 2023Trial
- A randomized controlled trial of everolimus for neurocognitive symptoms in PTEN hamartoma tumor syndrome.Human molecular genetics · 2022Trial
- Neonatal Cardiac Tamponade Caused by a Pericardial Tufted Angioma: A Rare Cause of Fetal Pericardial Effusion and a Multidisciplinary Treatment Approach.Pediatric cardiology · 2026Article
- From Somatic Driver to Molecular Therapy in Retinal, Choroidal and Periocular Vascular Disease: Mosaicism, Targetable Pathways and Ocular Toxicity.International journal of molecular sciences · 2026Review
- Sirolimus for the treatment of vascular tumors and malformations in children: A 10-year single-institution retrospective study.World journal of clinical pediatrics · 2026Article
- Prenatal Use of Sirolimus in Fetal Therapy: Current Evidence, Clinical Applications, and Future Directions.Cureus · 2026Review
283 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
24 authors.
Funding
Abstract
BACKGROUND AND
objectivesComplicated vascular anomalies have limited therapeutic options and cause significant morbidity and mortality. This Phase II trial enrolled patients with complicated vascular anomalies to determine the efficacy and safety of treatment with sirolimus for 12 courses; each course was defined as 28 days.
methodsTreatment consisted of a continuous dosing schedule of oral sirolimus starting at 0.8 mg/m(2) per dose twice daily, with pharmacokinetic-guided target serum trough levels of 10 to 15 ng/mL. The primary outcomes were responsiveness to sirolimus by the end of course 6 (evaluated according to functional impairment score, quality of life, and radiologic assessment) and the incidence of toxicities and/or infection-related deaths.
resultsSixty-one patients were enrolled; 57 patients were evaluable for efficacy at the end of course 6, and 53 were evaluable at the end of course 12. No patient had a complete response at the end of course 6 or 12 as anticipated. At the end of course 6, a total of 47 patients had a partial response, 3 patients had stable disease, and 7 patients had progressive disease. Two patients were taken off of study medicine secondary to persistent adverse effects. Grade 3 and higher toxicities attributable to sirolimus included blood/bone marrow toxicity in 27% of patients, gastrointestinal toxicity in 3%, and metabolic/laboratory toxicity in 3%. No toxicity-related deaths occurred.
conclusionsSirolimus was efficacious and well tolerated in these study patients with complicated vascular anomalies. Clinical activity was reported in the majority of the disorders.
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Identifiers
What OpenQuestion holds
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.