Women with TSC: Relationship between Clinical, Lung Function and Radiological Features in a Genotyped Population Investigated for Lymphangioleiomyomatosis

PLoS One. 2016 May 12;11(5):e0155331. doi: 10.1371/journal.pone.0155331. eCollection 2016.

Abstract

The advent of pharmacological therapies for lymphangioleiomyomatosis (LAM) has made early diagnosis important in women with tuberous sclerosis complex (TSC), although the lifelong cumulative radiation exposure caused by chest computer tomography (CT) should not be underestimated. We retrospectively investigated, in a cohort of TSC outpatients of San Paolo Hospital (Milan, Italy) 1) the role of pulmonary function tests (PFTs) for LAM diagnosis, 2) the association between LAM and other features of TSC (e.g. demography, extrapulmonary manifestations, genetic mutations, etc.), and 3) the characteristics of patients with multifocal micronodular pneumocyte hyperplasia (MMPH). Eighty-six women underwent chest CT scan; pulmonary involvement was found in 66 patients (77%; 49% LAM with or without MMPH, and 28% MMPH alone). LAM patients were older, with a higher rate of pneumothorax, presented more frequently with renal and hepatic angiomyolipomas, and tended to have a TSC2 mutation profile. PFTs, assessed in 64% of women unaffected by cognitive impairments, revealed a lower lung diffusion capacity in LAM patients. In multivariate analysis, age, but not PFTs, resulted independently associated with LAM diagnosis. Patients with MMPH alone did not show specific clinical, functional or genetic features. A mild respiratory impairment was most common in LAM-TSC patients: In conclusions, PFTs, even if indicated to assess impairment in lung function, are feasible in a limited number of patients, and are not significantly useful for LAM diagnosis in women with TSC.

MeSH terms

  • Adolescent
  • Adult
  • Age Factors
  • Aged
  • Child
  • Child, Preschool
  • Demography
  • Female
  • Genotype
  • Humans
  • Infant
  • Lymphangioleiomyomatosis / epidemiology
  • Lymphangioleiomyomatosis / genetics*
  • Male
  • Middle Aged
  • Multivariate Analysis
  • Odds Ratio
  • Prevalence
  • Respiratory Function Tests
  • Risk Factors
  • Tuberous Sclerosis / diagnostic imaging*
  • Tuberous Sclerosis / genetics
  • Tuberous Sclerosis / pathology
  • Tuberous Sclerosis / physiopathology*
  • Young Adult

Grants and funding

The study has been supported by grant from Regione Lombardia aimed to develop a database software for data collection. The funder had no role in study design, data collection and analysis, decision to publish, or preparation of the manuscript.