Autosomal systemic lupus erythematosus

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ORPHA:300345OMIM:614420M32.8
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Overview

Autosomal systemic lupus erythematosus (also called monogenic lupus or Mendelian lupus) is a rare genetic form of systemic lupus erythematosus (SLE) that is caused by a single gene mutation inherited from one or both parents. Unlike the more common form of lupus, which results from a complex mix of many genes and environmental factors, this type follows a clear inheritance pattern and often begins in childhood or early adolescence. In this condition, the immune system mistakenly attacks the body's own healthy tissues, causing widespread inflammation. This can affect many organs including the skin, joints, kidneys, blood cells, brain, heart, and lungs. Common symptoms include skin rashes (especially a butterfly-shaped rash across the cheeks), joint pain and swelling, extreme fatigue, fevers, mouth sores, hair loss, and kidney problems. Because the disease can affect so many parts of the body, it is considered a multi-system autoimmune disorder. Treatment focuses on controlling the overactive immune system and managing symptoms. Medications may include hydroxychloroquine, corticosteroids, and stronger immunosuppressive drugs such as mycophenolate mofetil, azathioprine, or biologics like belimumab. Because this form of lupus is driven by specific genetic defects, some patients may respond to targeted therapies depending on the gene involved. Early diagnosis through genetic testing is important because it can guide treatment choices and help predict disease course. Patients typically need lifelong monitoring and care from multiple specialists.

Also known as:

Key symptoms:

Butterfly-shaped rash across the cheeks and noseJoint pain and swellingExtreme tiredness and fatigueRecurring feversMouth or nose soresHair lossKidney problems including protein in the urineSensitivity to sunlight causing skin rashesSwollen lymph nodesChest pain when breathing deeplyFingers turning white or blue in the cold (Raynaud's phenomenon)Low blood cell counts (anemia, low white cells, low platelets)Seizures or other brain-related symptomsSkin lesions that worsen with sun exposureChilblain-like skin lesions on fingers and toes

Inheritance

Variable

Can be inherited in different ways depending on the underlying gene

Age of Onset

Childhood

Begins in childhood, roughly ages 1 to 12

Orphanet ↗OMIM ↗NORD ↗

FDA & Trial Timeline

1 event
May 2024Intergenerational Study of War-Affected Youth

Boston College

TrialACTIVE NOT RECRUITING

Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

Treatments

No FDA-approved treatments are currently listed for Autosomal systemic lupus erythematosus.

View clinical trials →

No actively recruiting trials found for Autosomal systemic lupus erythematosus at this time.

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Specialists

1 foundView all specialists →
PG
Peter K Gregersen
MANHASSET, NY
Specialist
PI on 1 active trial

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 Autosomal systemic lupus erythematosus.

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about Autosomal systemic lupus erythematosus

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Caregiver Resources

NORD Caregiver Resources

Support, advocacy, and financial assistance for caregivers of rare disease patients.

Mental Health Support

Rare disease caregiving can be isolating. Connect with counseling and peer support.

Family & Caregiver Grants

Financial assistance programs specifically for caregivers of rare disease patients.

Social Security Disability

Learn how rare disease patients may qualify for SSDI/SSI benefits.

Questions for your doctor

Bring these to your next appointment

  • Q1.What specific gene mutation is causing my lupus, and how does it affect my treatment options?,Which organs are currently affected, and how will you monitor them over time?,What medications do you recommend, and what are their potential side effects?,Should my family members be tested for this genetic mutation?,Are there any clinical trials or new treatments that might be appropriate for my specific type of lupus?,What signs of a flare should I watch for, and when should I seek emergency care?,How can I protect myself from sun exposure and infections while on treatment?

Common questions about Autosomal systemic lupus erythematosus

What is Autosomal systemic lupus erythematosus?

Autosomal systemic lupus erythematosus (also called monogenic lupus or Mendelian lupus) is a rare genetic form of systemic lupus erythematosus (SLE) that is caused by a single gene mutation inherited from one or both parents. Unlike the more common form of lupus, which results from a complex mix of many genes and environmental factors, this type follows a clear inheritance pattern and often begins in childhood or early adolescence. In this condition, the immune system mistakenly attacks the body's own healthy tissues, causing widespread inflammation. This can affect many organs including the

At what age does Autosomal systemic lupus erythematosus typically begin?

Typical onset of Autosomal systemic lupus erythematosus is childhood. Age of onset can vary across affected individuals.

Which specialists treat Autosomal systemic lupus erythematosus?

1 specialists and care centers treating Autosomal systemic lupus erythematosus are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.