Combined immunodeficiency due to TFRC deficiency

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ORPHA:476113OMIM:616740D81.8
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Overview

Combined immunodeficiency due to TFRC deficiency is a very rare inherited disease that affects the immune system. TFRC stands for Transferrin Receptor 1, a protein that helps cells absorb iron. When this protein does not work properly, certain immune cells — especially T cells and B cells, which are the body's infection-fighting soldiers — cannot develop or function normally. This leaves the body poorly equipped to fight off bacteria, viruses, and fungi. People with this condition typically experience serious, repeated infections starting in infancy or early childhood. These infections can affect the lungs, gut, skin, and other organs. Because the immune system is so weakened, even germs that would not normally cause illness in healthy people can become life-threatening. The condition is sometimes grouped under the broader category of combined immunodeficiencies (CID), meaning both T cells and B cells are affected. Treatment focuses on protecting the patient from infections using preventive antibiotics and antifungal medicines, replacing missing antibodies through infusions (immunoglobulin therapy), and potentially curing the disease through a bone marrow or stem cell transplant. Early diagnosis and treatment are critical to improving outcomes and survival.

Also known as:

Key symptoms:

Repeated and severe infections (lung, gut, skin, blood)Infections caused by unusual or opportunistic germsFailure to thrive or poor weight gain in infancyChronic diarrheaSkin rashes or infectionsSwollen lymph nodesLow levels of protective antibodies in the bloodReduced number or function of T cells and B cellsFever that keeps coming backLung infections such as pneumonia

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 Combined immunodeficiency due to TFRC deficiency.

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No actively recruiting trials found for Combined immunodeficiency due to TFRC deficiency at this time.

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No specialists are currently listed for Combined immunodeficiency due to TFRC deficiency.

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 Combined immunodeficiency due to TFRC deficiency.

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Community

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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.Is my child a candidate for a stem cell transplant, and how soon should we consider it?,What infections should I watch for most closely, and when should I go to the emergency room?,How often does my child need immunoglobulin infusions, and can these be done at home?,Are there any clinical trials or research studies we could join?,Should other family members be tested for the TFRC gene change?,What vaccinations are safe for my child, and which ones should be avoided?,What support services are available to help our family manage day-to-day care?

Common questions about Combined immunodeficiency due to TFRC deficiency

What is Combined immunodeficiency due to TFRC deficiency?

Combined immunodeficiency due to TFRC deficiency is a very rare inherited disease that affects the immune system. TFRC stands for Transferrin Receptor 1, a protein that helps cells absorb iron. When this protein does not work properly, certain immune cells — especially T cells and B cells, which are the body's infection-fighting soldiers — cannot develop or function normally. This leaves the body poorly equipped to fight off bacteria, viruses, and fungi. People with this condition typically experience serious, repeated infections starting in infancy or early childhood. These infections can af

How is Combined immunodeficiency due to TFRC deficiency inherited?

Combined immunodeficiency due to TFRC deficiency follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Combined immunodeficiency due to TFRC deficiency typically begin?

Typical onset of Combined immunodeficiency due to TFRC deficiency is infantile. Age of onset can vary across affected individuals.