Disease Directory Medullary Cystic Kidney Disease
Renal

Medullary Cystic Kidney Disease

Also known as: MCKD, UMOD-related nephropathy, uromodulin kidney disease

Prevalence

Rare; fewer than 1 in 100,000 estimated

Onset

Adult (ESRD typically in 3rd–7th decade)

Type

Rare autosomal dominant tubulointerstitial kidney disease

Gene

UMOD

About Medullary Cystic Kidney Disease

Medullary cystic kidney disease type 2 (now classified as autosomal dominant tubulointerstitial kidney disease-UMOD) is caused by mutations in UMOD encoding uromodulin (Tamm-Horsfall protein), leading to abnormal protein accumulation in tubular cells, progressive interstitial fibrosis, and slowly progressive renal failure. Medullary cysts are an inconstant finding and may not be visible on routine imaging, making UMOD genetic testing essential for diagnosis. Hyperuricaemia and gout are characteristic early features that may precede renal impairment by years.

Common Clinical Features

Hyperuricaemia and gout (often presenting manifestation) Progressive chronic kidney disease Anaemia disproportionate to CKD stage Normal or mildly reduced urine concentrating ability Medullary cysts (not always present on imaging) Hypertension in advanced disease Family history of renal failure and gout across generations

Clinical Trial Eligibility Tips

What to know before applying to Medullary Cystic Kidney Disease trials.

UMOD mutation confirmation is essential since medullary cysts may be absent; genetic testing should precede renal biopsy as histological findings are non-specific.

Serum uric acid levels and gout history are characteristic early markers; document the timeline of gout onset relative to renal impairment as this aids diagnosis and trial stratification.

Rate of eGFR decline over several years is the most critical eligibility variable for trials targeting disease progression; compile annual creatinine records from your GP or hospital.

Patient Resources

Patient Organization

NephCure Kidney International

Visit website ↗

Natural History Registry

Rare Kidney Disease Registry (RKD Registry)

Join registry ↗

Orphanet

European reference resource for rare diseases (ORPHA:488)

View on Orphanet ↗

NORD

National Organization for Rare Disorders

Search NORD ↗

Find recruiting Medullary Cystic Kidney Disease trials

Search 500,000+ studies from ClinicalTrials.gov, filtered for Medullary Cystic Kidney Disease. Updated daily.

Related Rare Diseases