Long-term outcomes of sirolimus therapy in sporadic and tuberous sclerosis complex–associated lymphangioleiomyomatosis: a retrospective cohort study
Rattachement africain : ir, ge, pl, es. Niveau de preuve : code pays fourni par la source.
Le résumé fourni par la source
INTRODUCTION: Sirolimus is a standard disease‑modifying therapy for lymphangioleiomyomatosis (LAM), but long‑term routine‑care data integrating pulmonary, lymphatic, extrapulmonary, and biomarker outcomes remain limited. OBJECTIVES: Our aim was to assess long‑term effectiveness and safety of sirolimus in routine clinical practice. PATIENTS AND METHODS: We retrospectively analyzed consecutive adult patients with definite LAM treated with sirolimus at a national tertiary referral center in Poland between 2010 and 2020. RESULTS: Seventy‑one patients were included (70 women; 57 [80%] with sporadic LAM and 14 [20%] with tuberous sclerosis complex-associated disease). Median (interquartile range) duration of available functional follow‑up was 5 (2-5) years, and mean (SD) trough sirolimus concentration was 7.85 (2.36) ng/ml. Baseline chylous pleural and / or peritoneal effusions were present in 15 patients (21%), renal angiomyolipomas in 33 (46%), and lymphangioleiomyomas in 28 individuals (39%). Forced expiratory volume in 1 second increased at 12 months by a median of 0.03 (-0.1 to 0.3) l from baseline (n = 66; P = 0.03). Forced vital capacity and 6‑minute walk distance also improved during early follow‑up, while transfer factor of the lung for carbon monoxide remained generally stable. Chylous effusions resolved in all affected patients by 12 months without recurrence, and renal angiomyolipoma and lymphangioleiomyoma volumes decreased significantly in patients who underwent paired magnetic resonance imaging. Higher baseline level of vascular endothelial growth factor D (VEGF‑D) was associated with lymphatic involvement, and decreased during treatment. Adverse events were mostly mild; permanent discontinuation occurred in approximately 6% of the patients. CONCLUSIONS: In routine practice, sirolimus treatment was associated with long‑term stabilization of lung function, marked improvement of lymphatic disease, reduction of angiomyolipoma burden, and acceptable tolerability. VEGF‑D level aligned with lymphatic phenotype and declined with treatment, thus supporting its role as a monitoring biomarker.
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Le contrôle bibliographique ouvert
DOI retrouvé dans Crossref DOI retrouvé ; titre concordant.
- Titre Crossref
- Long-term outcomes of sirolimus therapy in sporadic and tuberous sclerosis complex–associated lymphangioleiomyomatosis: a retrospective cohort study
- Date Crossref
- 12/06/2026
- Éditeur
- "Medycyna Praktyczna" Spolka Jawna
- Type
- journal-article
Ce recoupement confirme des métadonnées liées au DOI. Il ne confirme ni la méthode ni les conclusions de l’étude, et il ne compte pas comme une seconde source scientifique indépendante.
Où se fait cette recherche
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Masih Daneshvari Hospital pays non établi dans la noticeÉtablissement de santé
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National Center for Tuberculosis and Lung Disease pays non établi dans la noticeOrganisation à but non lucratif
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Medical University of Silesia pays non établi dans la noticeUniversité ou école supérieure
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Medical University of Warsaw pays non établi dans la noticeUniversité ou école supérieure
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Bellvitge University Hospital pays non établi dans la noticeÉtablissement de santé
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Institut d'Investigació Biomédica de Bellvitge pays non établi dans la noticeStructure de recherche
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Medical University of Lodz pays non établi dans la noticeUniversité ou école supérieure
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Third Department of Lung Diseases and Oncology National Tuberculosis and Lung Disease Research Institute Radiology Department pays non établi dans la noticeStructure de recherche
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Medical University of Łódź Department of Pneumology pays non établi dans la noticeUniversité ou école supérieure
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Department of Genetics and Clinical Immunology pays non établi dans la noticeÉtablissement de santé
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Catalan Institute of Oncology ProCURE pays non établi dans la noticeStructure de recherche
Masih Daneshvari Hospital, National Center for Tuberculosis and Lung Disease et Medical University of Silesia, avec 8 autres affiliations.
Une affiliation ne permet pas de déduire la nationalité d’un auteur.