Autosomal non-syndromic agammaglobulinemia

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ORPHA:33110OMIM:619707D80.0
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Overview

Autosomal non-syndromic agammaglobulinemia is a rare inherited immune system disorder where the body cannot make enough antibodies — the proteins that fight off infections. Unlike the more well-known X-linked form of agammaglobulinemia (called Bruton's disease, which mainly affects boys), this autosomal form can affect both males and females equally. The condition is sometimes called autosomal agammaglobulinemia or non-Bruton agammaglobulinemia. In this disease, a key type of immune cell called B cells either fails to develop properly or is present in very low numbers. B cells are responsible for making antibodies, so without them, the immune system struggles to defend the body against bacteria, viruses, and other germs. This leaves affected people highly vulnerable to repeated and serious infections, especially of the lungs, sinuses, ears, and digestive system. The main treatment is regular infusions of immunoglobulin (antibodies collected from healthy donors), which helps replace the missing antibodies and greatly reduces the risk of serious infections. With consistent treatment and careful monitoring, many people with this condition can live relatively normal lives, though lifelong therapy is required. Early diagnosis is very important to prevent organ damage from repeated infections.

Also known as:

Key symptoms:

Repeated bacterial infections, especially pneumonia and sinus infectionsFrequent ear infectionsChronic diarrhea or stomach infectionsVery low or absent levels of antibodies in the bloodFew or no B cells (a type of immune cell) in the bloodSlow recovery from infections that most people fight off easilyInfections caused by unusual germs that healthy immune systems normally handleSwollen lymph nodes or spleen in some casesFailure to thrive or poor growth in infantsJoint pain or swelling due to infections spreading to joints

Clinical phenotype terms (29)— hover any for plain English
Inheritance

Autosomal recessive

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

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 Autosomal non-syndromic agammaglobulinemia.

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No actively recruiting trials found for Autosomal non-syndromic agammaglobulinemia at this time.

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No specialists are currently listed for Autosomal non-syndromic agammaglobulinemia.

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 Autosomal non-syndromic agammaglobulinemia.

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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 gene is causing my condition, and what does that mean for my family members?,Should my siblings or parents be tested for this condition?,What type of immunoglobulin therapy is best for me — intravenous or subcutaneous — and can I do it at home?,How often should I have my lung function and antibody levels checked?,Are there any vaccines I should or should not receive?,What infections should prompt me to go to the emergency room right away?,Are there any clinical trials or new treatments I should know about?

Common questions about Autosomal non-syndromic agammaglobulinemia

What is Autosomal non-syndromic agammaglobulinemia?

Autosomal non-syndromic agammaglobulinemia is a rare inherited immune system disorder where the body cannot make enough antibodies — the proteins that fight off infections. Unlike the more well-known X-linked form of agammaglobulinemia (called Bruton's disease, which mainly affects boys), this autosomal form can affect both males and females equally. The condition is sometimes called autosomal agammaglobulinemia or non-Bruton agammaglobulinemia. In this disease, a key type of immune cell called B cells either fails to develop properly or is present in very low numbers. B cells are responsible

How is Autosomal non-syndromic agammaglobulinemia inherited?

Autosomal non-syndromic agammaglobulinemia follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Autosomal non-syndromic agammaglobulinemia typically begin?

Typical onset of Autosomal non-syndromic agammaglobulinemia is infantile. Age of onset can vary across affected individuals.