T-B+ severe combined immunodeficiency due to JAK3 deficiency

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ORPHA:35078OMIM:600802D81.2
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Overview

T-B+ severe combined immunodeficiency due to JAK3 deficiency (also called JAK3-SCID or JAK3-deficient SCID) is a rare and serious disorder of the immune system. In this condition, the body cannot make enough working T cells — the white blood cells that fight infections and coordinate the immune response. B cells are present in normal or even high numbers, but they do not work properly because they depend on T cells to function. Natural killer (NK) cells are also absent or very low. This pattern is called 'T-B+ SCID' because T cells are missing (T-) while B cells are present (B+). The disease is caused by changes (mutations) in the JAK3 gene, which provides instructions for making a protein that immune cells need to grow and survive. Without working JAK3 protein, the immune system cannot develop normally, leaving babies almost completely defenseless against infections. Babies with JAK3-SCID typically become very sick in the first months of life with severe, repeated, or unusual infections — including infections that would not normally cause serious illness in healthy children. Without treatment, this condition is life-threatening. The main curative treatment is a bone marrow transplant (also called a hematopoietic stem cell transplant), which can restore a working immune system. Gene therapy is also being studied as a future option.

Also known as:

Key symptoms:

Severe, repeated bacterial infectionsSerious viral infections that healthy children handle easilyFungal infections such as thrush (Candida) that keep coming backPneumonia, especially from a germ called Pneumocystis jiroveciiFailure to gain weight and grow normally (failure to thrive)Chronic diarrheaSkin rashesSwollen lymph nodes or, unusually, very small or absent lymph nodesInfections that do not get better with standard treatmentReactions to live vaccines (such as BCG or rotavirus) that cause serious illnessVery low or absent T cells and NK cells on blood testsB cells present but not working properly

Clinical phenotype terms (25)— hover any for plain English
Impaired lymphocyte transformation with phytohemagglutininHP:0003347Absent cellular immunityHP:0005354Decreased total T cell countHP:0005403Decreased mitogen-induced T-cell proliferationHP:0031381Recurrent opportunistic infectionsHP:0005390Chronic oral candidiasisHP:0009098Absent natural killer cellsHP:0040219Acute otitis mediaHP:0000371Decreased circulating total IgMHP:0002850Decreased circulating IgG concentrationHP:0004315Recurrent viral infectionsHP:0004429Recurrent infection of the gastrointestinal tractHP:0004798Decreased total B cell countHP:0010976Partial IgA deficiencyHP:0011837
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 T-B+ severe combined immunodeficiency due to JAK3 deficiency.

View clinical trials →

No actively recruiting trials found for T-B+ severe combined immunodeficiency due to JAK3 deficiency at this time.

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Search ClinicalTrials.gov ↗Join the T-B+ severe combined immunodeficiency due to JAK3 deficiency community →

No specialists are currently listed for T-B+ severe combined immunodeficiency due to JAK3 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 T-B+ severe combined immunodeficiency due to JAK3 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 bone marrow transplant, and how soon should it happen?,What type of donor is best for my child, and how do we find one?,What infections should I watch for, and when should I go to the emergency room?,Which vaccines are safe for my child and for other people in our household?,Are there any clinical trials for gene therapy that my child might qualify for?,What does immune recovery look like after transplant, and how will we monitor it?,Should other family members be tested to see if they are carriers of the JAK3 mutation?

Common questions about T-B+ severe combined immunodeficiency due to JAK3 deficiency

What is T-B+ severe combined immunodeficiency due to JAK3 deficiency?

T-B+ severe combined immunodeficiency due to JAK3 deficiency (also called JAK3-SCID or JAK3-deficient SCID) is a rare and serious disorder of the immune system. In this condition, the body cannot make enough working T cells — the white blood cells that fight infections and coordinate the immune response. B cells are present in normal or even high numbers, but they do not work properly because they depend on T cells to function. Natural killer (NK) cells are also absent or very low. This pattern is called 'T-B+ SCID' because T cells are missing (T-) while B cells are present (B+). The disease

How is T-B+ severe combined immunodeficiency due to JAK3 deficiency inherited?

T-B+ severe combined immunodeficiency due to JAK3 deficiency follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does T-B+ severe combined immunodeficiency due to JAK3 deficiency typically begin?

Typical onset of T-B+ severe combined immunodeficiency due to JAK3 deficiency is infantile. Age of onset can vary across affected individuals.