Observational studyOrphanet journal of rare diseases2018
Functional improvements in patients with lymphangioleiomyomatosis after sirolimus: an observational study.
Observational study in Orphanet journal of rare diseases, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 11 papers, 2 of them syntheses that pooled it.
What it found
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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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
11 citing papers in PubMed, 2 syntheses or guidelines pooled it.
- The efficacy and safety of pharmacological treatments for lymphangioleiomyomatosis.Respiratory research · 2020Pooled it
- The efficacy and adverse events of mTOR inhibitors in lymphangioleiomyomatosis: systematic review and meta-analysis.Orphanet journal of rare diseases · 2018Pooled it
- Six-minute walk distance predicting the risk of mortality in lymphangioleiomyomatosis patients.Orphanet journal of rare diseases · 2026Article
- Synergistic effects of mTOR inhibitors with VEGFR3 inhibitors on the interaction between TSC2-mutated cells and lymphatic endothelial cells.Science China. Life sciences · 2025Article
- Associations of VEGF-D levels with clinical manifestations in lymphangioleiomyomatosis: a cross-sectional analysis of 631 cases.Orphanet journal of rare diseases · 2025Article
- Lymphangioleiomyomatosis and mTOR inhibitors in real world-a narrative review.Journal of thoracic disease · 2023Review
- Long-term clinical course and outcomes in patients with lymphangioleiomyomatosis.Respiratory research · 2022Article
- Efficacy, Retention and Tolerability of Everolimus in Patients with Tuberous Sclerosis Complex: A Survey-Based Study on Patients' Perspectives.CNS drugs · 2021Article
- Effects of sirolimus in lymphangioleiomyomatosis patients on lung cysts and pulmonary function: long-term follow-up observational study.European radiology · 2020Observational
- Long-term efficacy and safety of sirolimus therapy in patients with lymphangioleiomyomatosis.Orphanet journal of rare diseases · 2019Article
- Recent advances in the management of lymphangioleiomyomatosis.F1000Research · 2018Review
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.
Funding
Abstract
backgroundSirolimus has been shown to be effective in patients with lymphangioleiomyomatosis (LAM). We wish to summarize our experience using sirolimus and its effectiveness in LAM patients.
methodsWe analyzed data from 98 patients who were diagnosed with definite or probable sporadic LAM based on the European Respiratory Society diagnosis criteria for LAM in 2010 at Peking Union Medical College Hospital and who had received sirolimus during January 2007 to June 2015. The data before and after the initiation of sirolimus therapy included pulmonary function tests, arterial blood gas analysis, 6-min walking distance (6MWD), size of chylous effusion and renal angiomyolipomas (AML), St. George's Respiratory Questionnaires (SGRQ) and vascular endothelial growth factor-D (VEGF-D) levels. Serum levels of sirolimus and adverse events were collected.
resultsMedian follow-up was 2.5 years. Most patients had forced expiratory volume in 1 s (FEV
conclusionLong-term use of sirolimus is safe in patients with LAM. LAM patients with FEV
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