DISEASE SCANNER

Global Incurable Diseases Tracker

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Pulmonary Disease

Lymphangioleiomyomatosis (LAM)

HIGH SEVERITY

A rare lung disease characterized by proliferation of abnormal smooth muscle-like cells (LAM cells) causing cystic destruction of lung tissue. Almost exclusively affects women. Associated with tuberous sclerosis complex or sporadic. Can cause pneumothorax and chylous effusions.

Global Affected

200.0K

Countries

15

Symptoms

Dyspnea
Recurrent pneumothorax
Chylous pleural effusion
Cough
Hemoptysis
Fatigue
Wheezing
Renal angiomyolipomas

Treatment Options

Sirolimus (rapamycin)
Everolimus
Hormone therapy
Pleurodesis (for pneumothorax)
Oxygen therapy
Lung transplantation
Management of complications

Risk Factors

1Female gender
2TSC1/TSC2 mutations
3Tuberous sclerosis complex
4Sporadic LAM (TSC2 mutations)

Diagnostic Methods

  • 1High-resolution CT (characteristic cysts)
  • 2Pulmonary function tests
  • 3Vascular endothelial growth factor-D (VEGF-D)
  • 4Biopsy (if needed)
  • 5Genetic testing
  • 6Abdominal CT/MRI

Prognosis

Variable; 10-year survival ~85% with modern therapy. Progressive decline in lung function. Sirolimus stabilizes disease.

Prevention

  • No known prevention
  • Avoid estrogen exposure
  • Smoking cessation
  • Regular monitoring
  • Genetic counseling (TSC)

Research Status

Sirolimus (rapamycin) and Everolimus are primary treatments. Research focuses on improved therapies and outcomes.

Sources

  • https://www.lung.org/lung-health-diseases/lung-disease-lookup/lymphangioleiomyomatosis
  • https://www.ncbi.nlm.nih.gov/books/NBK448131
  • https://www.hematology.org/education/patients
  • https://www.ncbi.nlm.nih.gov/books
  • https://rarediseases.org/rare-diseases

Medical Disclaimer

This information is for educational purposes only. Always consult healthcare professionals for medical advice, diagnosis, and treatment.