TCR-alpha-beta-positive T-cell deficiency

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ORPHA:397959OMIM:615387D81.8
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1Specialists8Treatment centers

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Overview

TCR-alpha-beta-positive T-cell deficiency is a rare primary immunodeficiency disorder that affects the immune system's ability to fight infections. In this condition, the body has a specific problem with certain types of immune cells called T cells. Normally, the immune system produces different types of T cells, including those that carry a receptor called TCR-alpha-beta and others that carry TCR-gamma-delta. In this disease, the TCR-alpha-beta-positive T cells are either absent or severely reduced in number, while other immune cell populations may be relatively preserved or abnormally expanded. This imbalance leaves the body vulnerable to frequent and severe infections, particularly from bacteria, viruses, and fungi. Patients typically present in infancy or early childhood with recurrent infections, failure to thrive, and sometimes autoimmune problems where the immune system mistakenly attacks the body's own tissues. Skin rashes, enlarged lymph nodes, and an enlarged spleen are also commonly seen. Some patients may develop inflammatory complications affecting multiple organs. Treatment depends on the severity of the condition. Supportive care includes antibiotics and antifungal medications to prevent and treat infections, as well as immunoglobulin replacement therapy to boost the immune system. For severe cases, hematopoietic stem cell transplantation (bone marrow transplant) may be considered as a potentially curative option. Early diagnosis and management by an experienced immunology team are critical for improving outcomes.

Also known as:

Key symptoms:

Frequent and severe infectionsRecurrent pneumonia or lung infectionsChronic diarrheaFailure to thrive or poor weight gainSkin rashes or eczema-like skin problemsEnlarged lymph nodesEnlarged spleenEnlarged liverAutoimmune problems such as low blood cell countsMouth sores or thrushRecurrent ear infectionsDelayed growthFever episodes without clear causeInflammatory complications in various organs

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 TCR-alpha-beta-positive T-cell deficiency.

View clinical trials →

No actively recruiting trials found for TCR-alpha-beta-positive T-cell deficiency at this time.

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

Search ClinicalTrials.gov ↗Join the TCR-alpha-beta-positive T-cell deficiency community →

Specialists

1 foundView all specialists →
EM
Erin Morales, MD
Houston, Texas
Specialist

Rare Disease Specialist

PI on 1 active trial

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 TCR-alpha-beta-positive T-cell deficiency.

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about TCR-alpha-beta-positive T-cell deficiency

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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.How severe is my child's immune deficiency, and which specific immune cells are affected?,Is bone marrow transplant recommended for my child, and what are the risks and benefits?,What infections should we be most concerned about, and what preventive medications are needed?,Will my child need lifelong immunoglobulin replacement therapy?,Are there any clinical trials or new treatments being studied for this condition?,What genetic testing should other family members consider?,What precautions should we take at home, school, and in public to reduce infection risk?

Common questions about TCR-alpha-beta-positive T-cell deficiency

What is TCR-alpha-beta-positive T-cell deficiency?

TCR-alpha-beta-positive T-cell deficiency is a rare primary immunodeficiency disorder that affects the immune system's ability to fight infections. In this condition, the body has a specific problem with certain types of immune cells called T cells. Normally, the immune system produces different types of T cells, including those that carry a receptor called TCR-alpha-beta and others that carry TCR-gamma-delta. In this disease, the TCR-alpha-beta-positive T cells are either absent or severely reduced in number, while other immune cell populations may be relatively preserved or abnormally expand

How is TCR-alpha-beta-positive T-cell deficiency inherited?

TCR-alpha-beta-positive T-cell deficiency follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does TCR-alpha-beta-positive T-cell deficiency typically begin?

Typical onset of TCR-alpha-beta-positive T-cell deficiency is infantile. Age of onset can vary across affected individuals.

Which specialists treat TCR-alpha-beta-positive T-cell deficiency?

1 specialists and care centers treating TCR-alpha-beta-positive T-cell deficiency are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.