Neonatal erythroderma-autoinflammation-inflammatory bowel disease syndrome

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ORPHA:294023OMIM:614328D84.8
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Overview

Neonatal erythroderma-autoinflammation-inflammatory bowel disease syndrome, also known as ADAM17 deficiency or ADAM17-related immunodeficiency, is an extremely rare genetic condition that affects the skin, immune system, and digestive tract from birth. The disease is caused by mutations in the ADAM17 gene, which plays an important role in processing proteins on cell surfaces that are involved in immune regulation, skin barrier function, and gut health. Babies born with this condition typically present with erythroderma, which means widespread redness and scaling of the skin shortly after birth. The immune system becomes overactive, leading to autoinflammation — the body attacks its own tissues without an infection being present. Children also develop inflammatory bowel disease, which causes chronic diarrhea, bloody stools, poor weight gain, and abdominal pain. Skin infections are common because the skin barrier is compromised, and patients may also be prone to other infections due to immune dysfunction. Treatment is mainly supportive and focuses on managing symptoms. This includes skin care to protect the barrier, medications to control inflammation in the gut, nutritional support, and infection prevention. Because the condition is so rare, there is no established cure, and management requires a team of specialists working together. Early diagnosis through genetic testing is important to guide treatment and monitor for complications.

Key symptoms:

Widespread red, scaly skin at birth (erythroderma)Chronic diarrheaBloody stoolsPoor weight gain and failure to thriveFrequent skin infectionsRecurrent infectionsAbdominal pain and crampingHair abnormalities or hair lossSkin thickening and peelingInflammation of the intestinesFever episodes without infectionGrowth delayNutritional deficiencies

Clinical phenotype terms (18)— hover any for plain English
BlepharitisHP:0000498OnychogryphosisHP:0001805Psoriasiform dermatitisHP:0003765Recurrent bacterial skin infectionsHP:0005406Chronic monilial nail infectionHP:0008396Perianal dermatitisHP:0011131Horizontal eyebrowHP:0011228Anal fissureHP:0012390Bloody diarrheaHP:0025085Recurrent gastroenteritisHP:0031123Chapped lipHP:0040181Slow-growing scalp hairHP:0100038Otitis externaHP:0410017
Inheritance

Autosomal recessive

Passed on when both parents carry the same gene change; often skips generations

Age of Onset

Neonatal

Begins at or shortly after birth (first 4 weeks)

Orphanet ↗OMIM ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for Neonatal erythroderma-autoinflammation-inflammatory bowel disease syndrome.

View clinical trials →

No actively recruiting trials found for Neonatal erythroderma-autoinflammation-inflammatory bowel disease syndrome at this time.

New trials open frequently. Follow this disease to get notified.

Search ClinicalTrials.gov ↗Join the Neonatal erythroderma-autoinflammation-inflammatory bowel disease syndrome community →

No specialists are currently listed for Neonatal erythroderma-autoinflammation-inflammatory bowel disease syndrome.

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 Neonatal erythroderma-autoinflammation-inflammatory bowel disease syndrome.

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about Neonatal erythroderma-autoinflammation-inflammatory bowel disease syndrome

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Caregiver Resources

NORD Caregiver Resources

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

Mental Health Support

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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 is the severity of my child's condition, and what complications should I watch for?,What skin care routine do you recommend, and how can I prevent skin infections?,What treatments are available for the inflammatory bowel disease, and what are the side effects?,Does my child need special nutrition or feeding support?,Should we consider genetic counseling for future pregnancies?,Is bone marrow transplant an option for my child?,Are there any clinical trials or research studies we could participate in?

Common questions about Neonatal erythroderma-autoinflammation-inflammatory bowel disease syndrome

What is Neonatal erythroderma-autoinflammation-inflammatory bowel disease syndrome?

Neonatal erythroderma-autoinflammation-inflammatory bowel disease syndrome, also known as ADAM17 deficiency or ADAM17-related immunodeficiency, is an extremely rare genetic condition that affects the skin, immune system, and digestive tract from birth. The disease is caused by mutations in the ADAM17 gene, which plays an important role in processing proteins on cell surfaces that are involved in immune regulation, skin barrier function, and gut health. Babies born with this condition typically present with erythroderma, which means widespread redness and scaling of the skin shortly after birt

How is Neonatal erythroderma-autoinflammation-inflammatory bowel disease syndrome inherited?

Neonatal erythroderma-autoinflammation-inflammatory bowel disease syndrome follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Neonatal erythroderma-autoinflammation-inflammatory bowel disease syndrome typically begin?

Typical onset of Neonatal erythroderma-autoinflammation-inflammatory bowel disease syndrome is neonatal. Age of onset can vary across affected individuals.