Autoinflammation-PLCG2-associated antibody deficiency-immune dysregulation

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ORPHA:324530OMIM:614878D89.8
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Overview

Autoinflammation with PLCG2-associated antibody deficiency and immune dysregulation, often called APLAID, is an extremely rare genetic condition that affects the immune system in multiple ways. It is caused by a change (mutation) in the PLCG2 gene, which plays an important role in how immune cells communicate and respond to threats. In APLAID, the immune system is both overactive and underactive at the same time — it triggers too much inflammation in certain tissues while also failing to produce enough protective antibodies, leaving the body vulnerable to infections. People with APLAID typically experience recurrent skin blistering and rashes, eye inflammation (such as uveitis or conjunctivitis), joint pain, and frequent infections — especially of the lungs and sinuses. Some patients also develop inflammatory bowel symptoms, including abdominal pain and diarrhea. The condition can also cause granuloma-like skin lesions and problems with wound healing. Symptoms usually begin in infancy or early childhood and can vary in severity from person to person. Treatment for APLAID focuses on managing symptoms and preventing complications. This may include immunoglobulin replacement therapy to compensate for the antibody deficiency, anti-inflammatory medications, and sometimes immunosuppressive drugs. Because the disease affects multiple organ systems, patients typically need care from a team of specialists. Research into targeted therapies is ongoing, and some patients have been treated with medications that block specific inflammatory pathways.

Also known as:

Key symptoms:

Recurrent skin blistering and rashesEye inflammation (red, painful eyes)Frequent sinus and lung infectionsJoint pain and swellingAbdominal pain and diarrheaInflammatory bowel symptomsGranuloma-like skin nodulesPoor wound healingLow antibody levels in the bloodAllergic-type reactionsFever episodes without infectionSkin ulcersSensitivity to cold temperatures

Inheritance

Autosomal dominant

Passed on from just one parent; each child has about a 50% chance of inheriting it

Age of Onset

Infantile

Begins in infancy, roughly 1 month to 2 years old

Orphanet ↗OMIM ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for Autoinflammation-PLCG2-associated antibody deficiency-immune dysregulation.

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No actively recruiting trials found for Autoinflammation-PLCG2-associated antibody deficiency-immune dysregulation at this time.

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No specialists are currently listed for Autoinflammation-PLCG2-associated antibody deficiency-immune dysregulation.

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 Autoinflammation-PLCG2-associated antibody deficiency-immune dysregulation.

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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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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.How severe is my (or my child's) antibody deficiency, and will immunoglobulin replacement therapy be needed?,What signs of infection or inflammation should I watch for at home?,Are there any targeted therapies or clinical trials available for APLAID?,How often should we have eye exams to monitor for inflammation?,What vaccinations are safe, and which ones should be avoided?,How can we best manage skin symptoms and prevent scarring?,Should other family members be tested for the PLCG2 gene mutation?

Common questions about Autoinflammation-PLCG2-associated antibody deficiency-immune dysregulation

What is Autoinflammation-PLCG2-associated antibody deficiency-immune dysregulation?

Autoinflammation with PLCG2-associated antibody deficiency and immune dysregulation, often called APLAID, is an extremely rare genetic condition that affects the immune system in multiple ways. It is caused by a change (mutation) in the PLCG2 gene, which plays an important role in how immune cells communicate and respond to threats. In APLAID, the immune system is both overactive and underactive at the same time — it triggers too much inflammation in certain tissues while also failing to produce enough protective antibodies, leaving the body vulnerable to infections. People with APLAID typica

How is Autoinflammation-PLCG2-associated antibody deficiency-immune dysregulation inherited?

Autoinflammation-PLCG2-associated antibody deficiency-immune dysregulation follows a autosomal dominant inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Autoinflammation-PLCG2-associated antibody deficiency-immune dysregulation typically begin?

Typical onset of Autoinflammation-PLCG2-associated antibody deficiency-immune dysregulation is infantile. Age of onset can vary across affected individuals.