ArticleOrphanet journal of rare diseases2019
Long-term efficacy and safety of sirolimus therapy in patients with lymphangioleiomyomatosis.
Article in Orphanet journal of rare diseases, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 29 papers, 2 of them syntheses that pooled it.
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Who cites it
29 citing papers in PubMed, 2 syntheses or guidelines pooled it, 45 citations in OpenAlex.
- Brazilian Thoracic Association recommendations for the management of lymphangioleiomyomatosis.Jornal brasileiro de pneumologia : publicacao oficial da Sociedade Brasileira de Pneumologia e Tisilogia · 2025Guideline
- Diagnostic performance of VEGF-D for lymphangioleiomyomatosis: a meta-analysis.Jornal brasileiro de pneumologia : publicacao oficial da Sociedade Brasileira de Pneumologia e Tisilogia · 2022Pooled it
- Determinants of adherence to sirolimus among lymphangioleiomyomatosis patients.BMC pulmonary medicine · 2026Article
- Diagnostic Value of Serum VEGF-D in Lymphangioleiomyomatosis: Results of the First Prospective Study in the Russian Federation.Diagnostics (Basel, Switzerland) · 2026Article
- Six-minute walk distance predicting the risk of mortality in lymphangioleiomyomatosis patients.Orphanet journal of rare diseases · 2026Article
- Long-term low-dose sirolimus therapy and successful discontinuation in an adult with kaposiform lymphangiomatosis and disseminated intravascular coagulation: a case report.Frontiers in medicine · 2026Article
- Article
- A real-world study of sirolimus in the treatment of pediatric head and neck lymphatic malformations.Journal of vascular surgery. Venous and lymphatic disorders · 2025Article
- Nested Case-Control Study of Intervention in Early Infancy to Prevent Tuberous Sclerosis Complex-Associated Renal Angiomyolipoma.Kidney international reports · 2025Article
- Associations of VEGF-D levels with clinical manifestations in lymphangioleiomyomatosis: a cross-sectional analysis of 631 cases.Orphanet journal of rare diseases · 2025Article
- Illness cognition, illness perception and related factors in patients with lymphangioleiomyomatosis.Orphanet journal of rare diseases · 2025Article
- Rare genetic interstitial lung diseases: a pictorial essay.European respiratory review : an official journal of the European Respiratory Society · 2024Review
- Intestinal lymphangiectasia: Understanding the bigger picture.World journal of clinical cases · 2024Article
- Experience of primary intestinal lymphangiectasia in adults: Twelve case series from a tertiary referral hospital.World journal of clinical cases · 2024Article
- Pulmonary benign metastasizing leiomyoma in patients aged 45 years and younger: clinical features and novelty in treatment.BMC pulmonary medicine · 2023Article
- The impact of the use of immunosuppressive treatment after an embryo transfer in increasing the rate of live birth.Frontiers in medicine · 2023Review
- Monitoring of sirolimus in the whole blood samples from pediatric patients with lymphatic anomalies.Open medicine (Warsaw, Poland) · 2023Article
- Expression of γ-globin genes in β-thalassemia patients treated with sirolimus: results from a pilot clinical trial (Sirthalaclin).Therapeutic advances in hematology · 2022Article
- Soluble Immune-Related Proteins as New Candidate Serum Biomarkers for the Diagnosis and Progression of Lymphangioleiomyomatosis.Frontiers in immunology · 2022Article
- Tuberous sclerosis complex for the pulmonologist.European respiratory review : an official journal of the European Respiratory Society · 2021Article
Corrections and comments
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Authors and funding
9 authors at 3 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundSirolimus has been confirmed to be effective for lymphangioleiomyomatosis (LAM), a rare multisystem neoplastic disease in women. The long-term effects of sirolimus treatment for LAM, however, are largely unknown. We aimed to analyze the long-term efficacy and safety of sirolimus therapy for LAM with 4-year follow-up.
methodsIn total, 142 sporadic LAM patients who took sirolimus for 1-4 years were retrospectively enrolled for this analysis. The variables used for analysis included pulmonary function tests, arterial blood gas analysis, 6-min walking distance (6MWD), St. George's Respiratory Questionnaires (SGRQ) and serum vascular endothelial growth factor-D (VEGF-D) levels before and after the initiation of sirolimus therapy. The rates of change (slope) in those variables were calculated, and adverse events were also analyzed.
resultsIn total, 122, 83, 60 and 32 patients out of 142 were followed for 1, 2, 3 and 4 years respectively. Sirolimus treatment improved the change rate in forced expiratory volume in 1 second (FEV
conclusionSirolimus therapy is effective at improving or stabilizing pulmonary function, oxygen levels, exercise capacity, and quality of life in patients with LAM for up to 4 years. VEGF-D is maintained at a lower level for 4 years after treatment. Adverse events related to sirolimus were mild.
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