Rare disease with adrenal Cushing syndrome as a major feature

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ORPHA:314749
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Overview

This condition is classified by Orphanet under code 314749 as a rare disease grouping where adrenal Cushing syndrome is a major clinical feature. Adrenal Cushing syndrome occurs when the adrenal glands, which sit on top of the kidneys, produce too much cortisol — a hormone that helps regulate blood pressure, blood sugar, and the body's stress response. When cortisol levels stay too high for a long time, it can cause a wide range of health problems throughout the body. Patients with this group of conditions typically develop weight gain (especially around the midsection and face), thinning skin that bruises easily, muscle weakness, high blood pressure, high blood sugar, and mood changes. The excess cortisol comes directly from the adrenal glands themselves, which may be affected by tumors (benign or cancerous), abnormal growth (hyperplasia), or genetic conditions that cause the adrenal glands to overproduce cortisol. Treatment depends on the specific underlying cause. Surgery to remove the affected adrenal gland is often the primary treatment. Medications that block cortisol production may be used when surgery is not possible or as a bridge before surgery. Because this is a grouping of several rare conditions, the exact genetic cause, inheritance pattern, and prognosis vary depending on the specific diagnosis within this category. Conditions in this group include primary pigmented nodular adrenocortical disease (PPNAD), ACTH-independent macronodular adrenal hyperplasia (AIMAH), and adrenocortical carcinoma, among others.

Key symptoms:

Unexplained weight gain, especially around the belly and face (moon face)Round, puffy faceFat buildup between the shoulders (buffalo hump)Thin skin that bruises easilyPurple or red stretch marks on the belly, thighs, or armsMuscle weakness, especially in the legsHigh blood pressureHigh blood sugar or diabetesBone thinning (osteoporosis) and fracturesMood changes, anxiety, or depressionFatigue and trouble sleepingSlow wound healingIncreased infectionsIrregular or absent menstrual periods in womenExcess body hair growth in women

Inheritance

Variable

Can be inherited in different ways depending on the underlying gene

Age of Onset

Variable

Can begin at different ages, from infancy through adulthood

Orphanet ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for Rare disease with adrenal Cushing syndrome as a major feature.

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No actively recruiting trials found for Rare disease with adrenal Cushing syndrome as a major feature at this time.

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No specialists are currently listed for Rare disease with adrenal Cushing syndrome as a major feature.

View NORD Rare Disease Centers ↗Undiagnosed Disease Network ↗

Treatment Centers

8 centers
🏥 NORD

Baylor College of Medicine Rare Disease Center

Baylor College of Medicine

📍 Houston, TX

🏥 NORD

Stanford Medicine Rare Disease Center

Stanford Medicine

📍 Stanford, CA

🔬 UDN

NIH Clinical Center Undiagnosed Diseases Program

National Institutes of Health

📍 Bethesda, MD

🔬 UDN

UCLA UDN Clinical Site

UCLA Health

📍 Los Angeles, CA

🔬 UDN

Baylor College of Medicine UDN Clinical Site

Baylor College of Medicine

📍 Houston, TX

🔬 UDN

Harvard/MGH UDN Clinical Site

Massachusetts General Hospital

📍 Boston, MA

🏥 NORD

Mayo Clinic Center for Individualized Medicine

Mayo Clinic

📍 Rochester, MN

👤 Mayo Clinic Center for Individualized Medicine

🏥 NORD

UCLA Rare Disease Day Program

UCLA Health

📍 Los Angeles, CA

Travel Grants

No travel grants are currently matched to Rare disease with adrenal Cushing syndrome as a major feature.

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Community

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Caregiver Resources

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Questions for your doctor

Bring these to your next appointment

  • Q1.What is the specific cause of my adrenal Cushing syndrome, and is it genetic?,Should my family members be tested for a genetic condition?,Is surgery recommended, and if so, will I need one or both adrenal glands removed?,Will I need lifelong hormone replacement therapy after treatment?,What are the signs of adrenal crisis, and what should I do in an emergency?,How long will it take for my symptoms to improve after treatment?,What monitoring and follow-up tests will I need long-term?

Common questions about Rare disease with adrenal Cushing syndrome as a major feature

What is Rare disease with adrenal Cushing syndrome as a major feature?

This condition is classified by Orphanet under code 314749 as a rare disease grouping where adrenal Cushing syndrome is a major clinical feature. Adrenal Cushing syndrome occurs when the adrenal glands, which sit on top of the kidneys, produce too much cortisol — a hormone that helps regulate blood pressure, blood sugar, and the body's stress response. When cortisol levels stay too high for a long time, it can cause a wide range of health problems throughout the body. Patients with this group of conditions typically develop weight gain (especially around the midsection and face), thinning ski