DISEASE SCANNER

Global Incurable Diseases Tracker

Back to Globe
Nephrologic Disease

Alport Syndrome

HIGH SEVERITY

A genetic disorder affecting the collagen network of the glomerular basement membrane, causing progressive kidney disease, hearing loss, and eye abnormalities. Caused by mutations in COL4A3, COL4A4, or COL4A5 genes. X-linked form most common and severe in males.

Global Affected

60.0K

Countries

15

Symptoms

Hematuria (blood in urine)
Proteinuria
Progressive kidney failure
Sensorineural hearing loss
Eye abnormalities (lenticonus, cataracts)
Hypertension
Edema

Treatment Options

ACE inhibitors/ARBs
Blood pressure control
Diuretics
Erythropoietin for anemia
Kidney transplantation
Hearing aids/cochlear implants
Cataract surgery

Risk Factors

1X-linked inheritance (most common)
2Autosomal recessive inheritance
3Autosomal dominant inheritance (milder)
4Family history
5Male sex (X-linked form)

Diagnostic Methods

  • 1Urinalysis (persistent hematuria)
  • 2Genetic testing
  • 3Skin biopsy (type IV collagen)
  • 4Kidney biopsy with immunofluorescence
  • 5Hearing tests
  • 6Ophthalmologic examination

Prognosis

X-linked: males develop ESRD by age 16-35; females have milder disease. Autosomal recessive: similar to X-linked males. Early ACE inhibitor therapy delays ESRD by 10+ years.

Prevention

  • Genetic counseling
  • Family screening
  • Early ACE inhibitor therapy
  • Avoid nephrotoxic drugs
  • Regular monitoring

Research Status

No cure; ACE inhibitors/ARBs slow progression. Renin-angiotensin-aldosterone system blockade standard care. Kidney transplantation effective; anti-GBM disease rare post-transplant. Gene therapy research ongoing.

Sources

  • https://www.kidney.org/atoz/content/alport-syndrome
  • https://www.ncbi.nlm.nih.gov/books/NBK1207
  • 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.