ArticleOrphanet journal of rare diseases2018
Efficacy and safety of low-dose Sirolimus in Lymphangioleiomyomatosis.
Article 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. It is linked to 2 registered trials, which are not on this map. Cited by 14 papers, 1 of them a synthesis 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.
Safety and Efficacy of Low-dose Sirolimus to Kaposiform Hemangioendothelioma:A Prospective, Randomized Open Trial
Microsampling Assays for Immunosuppressive Drugs in Children
Who cites it
14 citing papers in PubMed, 1 synthesis or guideline pooled it.
- Brazilian Thoracic Association recommendations for the management of lymphangioleiomyomatosis.Jornal brasileiro de pneumologia : publicacao oficial da Sociedade Brasileira de Pneumologia e Tisilogia · 2025Guideline
- Determinants of adherence to sirolimus among lymphangioleiomyomatosis patients.BMC pulmonary medicine · 2026Article
- Review
- Lymphangioleiomyomatosis and mTOR inhibitors in real world-a narrative review.Journal of thoracic disease · 2023Review
- Refractory systemic lupus erythematosus with chylous effusion successfully treated with sirolimus: a case report and literature review.Rheumatology international · 2023Review
- An Evidence-Based Update on the Potential Association between Rheumatoid Arthritis and Lymphangioleiomyomatosis.Journal of personalized medicine · 2023Review
- Long-term clinical course and progression of lymphangioleiomyomatosis in a single lung transplant referral centre in Korea.Scientific reports · 2022Article
- Rapamycin in Cerebral Cavernous Malformations: What Doses to Test in Mice and Humans.ACS pharmacology & translational science · 2022Review
- Rapamycin Promotes the Expansion of Myeloid Cells by Increasing G-CSF Expression in Mesenchymal Stem Cells.Frontiers in cell and developmental biology · 2022Article
- Lung Diseases Unique to Women.Clinics in chest medicine · 2021Review
- Development and Validation of a Bioanalytical LC-MS/MS Method for Simultaneous Determination of Sirolimus in Porcine Whole Blood and Lung Tissue and Pharmacokinetic Application with Coronary Stents.Molecules (Basel, Switzerland) · 2021Article
- A Retrospective Study of Lung Transplantation in Patients With Lymphangioleiomyomatosis: Challenges and Outcomes.Frontiers in medicine · 2021Article
- Review
- mTOR in Lung Neoplasms.Pathology oncology research : POR · 2020Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
4 authors.
Funding
Abstract
backgroundLymphangioleiomyomatosis is a rare disease caused by unregulated activation of mammalian target of rapamycin (mTOR) signalling pathway. Sirolimus showed efficacy in a phase 3 trial of patients with lymphangioleiomyomatosis, but the optimal dose remains unclear.
methodsWe investigated the efficacy and safety of low-dose compared with conventional-dose sirolimus. Clinical data of 39 patients with lymphangioleiomyomatosis (mean age, 34.8 years; median treatment period, 29.6 months) who received sirolimus were retrospectively reviewed. Low-dose sirolimus was defined as any dose that maintained mean blood trough levels lower than those maintained with conventional doses (5-15 ng/mL).
resultsFifty-one percent of patients received low-dose therapy. The rate of decline in lung function decreased after treatment in the whole group (forced expiratory volume in 1 s [FEV
conclusionsLow-dose sirolimus may stabilise lung function decline in lymphangioleiomyomatosis patients, but its efficacy appears to be inferior to that of conventional-dose sirolimus.
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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.