PMID- 34461049
OWN - NLM
STAT- MEDLINE
DCOM- 20220322
LR  - 20230303
IS  - 2213-2619 (Electronic)
IS  - 2213-2600 (Linking)
VI  - 9
IP  - 11
DP  - 2021 Nov
TI  - Lymphangioleiomyomatosis: pathogenesis, clinical features, diagnosis, and 
      management.
PG  - 1313-1327
LID - S2213-2600(21)00228-9 [pii]
LID - 10.1016/S2213-2600(21)00228-9 [doi]
AB  - Lymphangioleiomyomatosis (LAM) is a slowly progressive, low-grade, metastasising 
      neoplasm of women, characterised by infiltration of the lung parenchyma with 
      abnormal smooth muscle-like cells, resulting in cystic lung destruction. The 
      invading cell in LAM arises from an unknown source and harbours mutations in 
      tuberous sclerosis complex (TSC) genes that result in constitutive activation of 
      the mechanistic target of rapamycin (mTOR) pathway, dysregulated cellular 
      proliferation, and a programme of frustrated lymphangiogenesis, culminating in 
      disordered lung remodelling and respiratory failure. Over the past two decades, 
      all facets of LAM basic and clinical science have seen important advances, 
      including improved understanding of molecular mechanisms, novel diagnostic and 
      prognostic biomarkers, effective treatment strategies, and comprehensive clinical 
      practice guidelines. Further research is needed to better understand the natural 
      history of LAM; develop more powerful diagnostic, prognostic, and predictive 
      biomarkers; optimise the use of inhibitors of mTOR complex 1 in the treatment of 
      LAM; and explore novel approaches to the development of remission-inducing 
      therapies.
CI  - Copyright (c) 2021 Elsevier Ltd. All rights reserved.
FAU - McCarthy, Cormac
AU  - McCarthy C
AD  - Department of Respiratory Medicine, St Vincent's University Hospital, University 
      College Dublin, Dublin, Ireland. Electronic address: cormac.mccarthy@ucd.ie.
FAU - Gupta, Nishant
AU  - Gupta N
AD  - Division of Pulmonary, Critical Care, and Sleep Medicine, University of 
      Cincinnati, Cincinnati, OH, USA.
FAU - Johnson, Simon R
AU  - Johnson SR
AD  - Division of Respiratory Medicine, University of Nottingham, NIHR Respiratory 
      Biomedical Research Centre, Nottingham, UK.
FAU - Yu, Jane J
AU  - Yu JJ
AD  - Division of Pulmonary, Critical Care, and Sleep Medicine, University of 
      Cincinnati, Cincinnati, OH, USA.
FAU - McCormack, Francis X
AU  - McCormack FX
AD  - Division of Pulmonary, Critical Care, and Sleep Medicine, University of 
      Cincinnati, Cincinnati, OH, USA.
LA  - eng
GR  - R34 HL138235/HL/NHLBI NIH HHS/United States
GR  - U01 HL131755/HL/NHLBI NIH HHS/United States
GR  - U01 HL131022/HL/NHLBI NIH HHS/United States
PT  - Journal Article
PT  - Research Support, N.I.H., Extramural
PT  - Research Support, Non-U.S. Gov't
PT  - Research Support, U.S. Gov't, Non-P.H.S.
PT  - Review
DEP - 20210827
PL  - England
TA  - Lancet Respir Med
JT  - The Lancet. Respiratory medicine
JID - 101605555
RN  - W36ZG6FT64 (Sirolimus)
SB  - IM
MH  - Female
MH  - Humans
MH  - Lung/pathology
MH  - *Lung Neoplasms/diagnosis/genetics/therapy
MH  - *Lymphangioleiomyomatosis/etiology/genetics
MH  - Mutation
MH  - Sirolimus/therapeutic use
COIS- Declaration of interests CM, NG, SRJ, and FXM are scientific advisory board 
      members of the LAM Foundation. CM reports grants (LAM0144SG01-20) from the LAM 
      Foundation, which did not influence the content of this Review. SRJ reports 
      grants from the UK Medical Research Council, LAM Action, the LAM Foundation, and 
      the Tuberous Sclerosis Association; and a grant from Pfizer for post-marketing 
      surveillance on rapamycin, outside the submitted work. FXM reports grants from 
      National Institutes of Health (NIH; U01HL131755, U01HL131022). JJY reports grants 
      from NIH National Heart, Lung, and Blood Institute (RO1HL153045, R01HL138481), 
      the US Department of Defense (W81XWH1910474, 81XWH2010736), and The LAM 
      Foundation (LAM0141E01-20). NG reports grants from NIH (U01HL131755, R34HL138235) 
      and the LAM Foundation.
EDAT- 2021/08/31 06:00
MHDA- 2022/03/23 06:00
CRDT- 2021/08/30 20:10
PHST- 2020/12/15 00:00 [received]
PHST- 2021/05/02 00:00 [revised]
PHST- 2021/05/07 00:00 [accepted]
PHST- 2021/08/31 06:00 [pubmed]
PHST- 2022/03/23 06:00 [medline]
PHST- 2021/08/30 20:10 [entrez]
AID - S2213-2600(21)00228-9 [pii]
AID - 10.1016/S2213-2600(21)00228-9 [doi]
PST - ppublish
SO  - Lancet Respir Med. 2021 Nov;9(11):1313-1327. doi: 10.1016/S2213-2600(21)00228-9. 
      Epub 2021 Aug 27.