ArticleRespiratory research2011
Is sirolimus a therapeutic option for patients with progressive pulmonary lymphangioleiomyomatosis?
Article in Respiratory research, 2011. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 17 papers, 2 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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
17 citing papers in PubMed, 2 syntheses or guidelines pooled it, 38 citations in OpenAlex.
- The effect of mTOR inhibitors on respiratory infections in lymphangioleiomyomatosis.European respiratory review : an official journal of the European Respiratory Society · 2017Pooled it
- Official American Thoracic Society/Japanese Respiratory Society Clinical Practice Guidelines: Lymphangioleiomyomatosis Diagnosis and Management.American journal of respiratory and critical care medicine · 2016Guideline
- Effects of Sirolimus on Lung function in patients with Lymphangioleiomyomatosis.Caspian journal of internal medicine · 2019Article
- Efficacy and safety of low-dose Sirolimus in Lymphangioleiomyomatosis.Orphanet journal of rare diseases · 2018Article
- Functional improvements in patients with lymphangioleiomyomatosis after sirolimus: an observational study.Orphanet journal of rare diseases · 2018Observational
- Lymphangioleiomyomatosis: a case report and review of diagnosis and treatment.OncoTargets and therapy · 2018Article
- Recent advances in the management of lymphangioleiomyomatosis.F1000Research · 2018Review
- Pelvic lymphangioleiomyomatosis treated successfully with everolimus: Two case reports with literature review.Medicine · 2017Review
- Review
- Diffuse Cystic Lung Disease. Part I.American journal of respiratory and critical care medicine · 2015Review
- Management of lymphangioleiomyomatosis.F1000prime reports · 2014Review
- Is mTOR inhibition a systemic treatment for tuberous sclerosis?Italian journal of pediatrics · 2013Review
- Lymphangioleiomyomatosis: current and future.Journal of thoracic disease · 2013Article
- [Chylothorax in an 87-year-old patient].Der Internist · 2013Article
- Optimizing treatments for lymphangioleiomyomatosis.Expert review of respiratory medicine · 2012Review
- Use of sirolimus in the treatment of lymphangioleiomyomatosis: favorable responses in patients with different extrapulmonary manifestations.Jornal brasileiro de pneumologia : publicacao oficial da Sociedade Brasileira de Pneumologia e TisilogiaArticle
- Sirolimus in lymphangioleiomyomatosis: A case in point for research in 'orphan' diseases.Lung India : official organ of Indian Chest SocietyArticle
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
13 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundLymphangioleiomyomatosis (LAM) is a rare lung disease characterised by progressive airflow obstruction. No effective medical treatment is available but therapy with sirolimus has shown some promise. The aim of this observational study was to evaluate sirolimus in progressive LAM.
methodsSirolimus (trough level 5 - 10 ng/ml) was administered to ten female patients (42.4 ± 11.9 years) with documented progression. Serial pulmonary function tests and six-minute-walk-distance (6-MWD) assessments were performed.
resultsThe mean loss of FEV1 was -2.30 ± 0.52 ml/day before therapy and a significant mean gain of FEV1 of 1.19 ± 0.26 ml/day was detected during treatment (p = 0.001). Mean FEV1 and FVC at baseline were 1.12 ± 0.15 l (36.1 ± 4.5%pred.) and 2.47 ± 0.25 l (69.2 ± 6.5%pred.), respectively. At three and six months during follow-up a significant increase of FEV1 and FVC was demonstrated (3 months ΔFEV1: 220 ± 82 ml, p = 0.024; 6 months ΔFEV1: 345 ± 58 ml, p = 0.001); (3 months ΔFVC: 360 ± 141 ml, p = 0.031; 6 months ΔFVC: 488 ± 138 ml, p = 0.006). Sirolimus was discontinued in 3 patients because of serious recurrent lower respiratory tract infection or sirolimus-induced pneumonitis. No deaths and no pneumothoraces occurred during therapy.
conclusionsOur data suggest that sirolimus might be considered as a therapeutic option in rapidly declining LAM patients. However, sirolimus administration may be associated with severe respiratory adverse events requiring treatment cessation in some patients. Moreover, discontinuation of sirolimus is mandatory prior to lung transplantation.
Indexed as
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.