Pyogenic autoinflammatory syndrome of childhood

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Overview

Pyogenic autoinflammatory syndrome of childhood, also known as pyogenic sterile arthritis, pyoderma gangrenosum, and acne syndrome (PAPA syndrome), is a very rare inherited condition that causes the body's immune system to trigger inflammation without an actual infection. The word "pyogenic" means pus-forming, which describes the type of inflammation seen in this disease. It mainly affects the skin and joints. Children with this condition typically develop painful, swollen joints (sterile arthritis) that can flare up after minor injuries or even without a clear trigger. The joint inflammation can be severe and destructive if not treated. Skin problems are also common and include pyoderma gangrenosum, which causes deep, painful skin ulcers that heal slowly, and severe cystic acne that usually appears during adolescence. These symptoms come and go in episodes or flares. Treatment focuses on controlling inflammation and preventing flares. Corticosteroids are often used during acute episodes. Biologic medications that block specific inflammatory signals, particularly interleukin-1 (IL-1) inhibitors like anakinra and canakinumab, and tumor necrosis factor (TNF) blockers like infliximab and etanercept, have shown benefit in managing symptoms. There is currently no cure, so treatment is lifelong and aimed at reducing the frequency and severity of flares while preventing joint damage and skin scarring.

Key symptoms:

Painful swollen joints without infection (sterile arthritis)Deep painful skin ulcers (pyoderma gangrenosum)Severe cystic acne, especially during teenage yearsJoint flares triggered by minor injuriesPus-like fluid in the jointsSkin wounds that heal very slowlyScarring of the skinJoint damage and stiffness over timeRecurring fevers during flaresFatigue during episodes of inflammationMuscle aches during flares

Inheritance

Autosomal dominant

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

Age of Onset

Childhood

Begins in childhood, roughly ages 1 to 12

Orphanet ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for Pyogenic autoinflammatory syndrome of childhood.

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No actively recruiting trials found for Pyogenic autoinflammatory syndrome of childhood at this time.

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No specialists are currently listed for Pyogenic autoinflammatory syndrome of childhood.

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 Pyogenic autoinflammatory syndrome of childhood.

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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.Which biologic medication do you recommend for my child, and what are the potential side effects?,How can we tell the difference between a disease flare and a true joint infection?,What should we do at home when a flare starts — is there an action plan?,How often will my child need blood tests or other monitoring?,Are there any activities or triggers we should avoid to reduce flares?,Should other family members be tested for the PSTPIP1 gene mutation?,Are there any clinical trials or new treatments being studied for PAPA syndrome?

Common questions about Pyogenic autoinflammatory syndrome of childhood

What is Pyogenic autoinflammatory syndrome of childhood?

Pyogenic autoinflammatory syndrome of childhood, also known as pyogenic sterile arthritis, pyoderma gangrenosum, and acne syndrome (PAPA syndrome), is a very rare inherited condition that causes the body's immune system to trigger inflammation without an actual infection. The word "pyogenic" means pus-forming, which describes the type of inflammation seen in this disease. It mainly affects the skin and joints. Children with this condition typically develop painful, swollen joints (sterile arthritis) that can flare up after minor injuries or even without a clear trigger. The joint inflammation

How is Pyogenic autoinflammatory syndrome of childhood inherited?

Pyogenic autoinflammatory syndrome of childhood follows a autosomal dominant inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Pyogenic autoinflammatory syndrome of childhood typically begin?

Typical onset of Pyogenic autoinflammatory syndrome of childhood is childhood. Age of onset can vary across affected individuals.