title: Survival and disease progression in UK patients with lymphangioleiomyomatosis.
abstract: BACKGROUND
  Lymphangioleiomyomatosis (LAM) is a rare and progressive disease of young women with no effective treatment. Previous estimates of 10 year survival, based mostly on case series or patients from tertiary centres, have ranged from 40% to 79%; no data are available on the progression of respiratory disability. In order to provide data for patients and for planning intervention studies, we have looked at the time course of LAM using a national cohort.
  
  
  METHODS
  Time to death, time to MRC dyspnoea grades 2-5, and need for oxygen in patients on the UK LAM database were analysed using Kaplan-Meier analysis and Cox regression.
  
  
  RESULTS
  Fifty seven of 72 patients responded with a median duration of follow up of 12.6 years (range 2.3-37) from the onset of symptoms. Ten year survival was 91% from onset of symptoms but varied widely with 11 patients alive after 20 years. Median time to MRC grade 3 dyspnoea (breathless walking on the flat) was 9.3 years (95% CI 5.1 to 13.4) from onset of symptoms.
  
  
  CONCLUSIONS
  Survival from LAM appears to be better than that reported in early studies. These data should be helpful for patients and for planning clinical trials.
authors.affiliation: Division of Therapeutics and Molecular Medicine, Queens Medical Centre, University of Nottingham, Nottingham, UK. simon.johnson@nottingham.ac.uk
authors.forename: S R
authors.initials: SR
authors.lastname: Johnson
authors.forename: C I
authors.initials: CI
authors.lastname: Whale
authors.forename: R B
authors.initials: RB
authors.lastname: Hubbard
authors.forename: S A
authors.initials: SA
authors.lastname: Lewis
authors.forename: A E
authors.initials: AE
authors.lastname: Tattersfield
mesh_terms: D015331:Cohort Studies; D018450:Disease Progression; D004417:Dyspnea / Q000453:epidemiology / Q000401:mortality; D006801:Humans; D008175:Lung Neoplasms / Q000401:mortality*; D018192:Lymphangioleiomyomatosis / Q000401:mortality*; D011379:Prognosis; D012044:Regression Analysis; D016019:Survival Analysis; D006113:United Kingdom / Q000453:epidemiology