T-B+ severe combined immunodeficiency due to CD45 deficiency

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ORPHA:169157OMIM:619924D81.2
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Overview

T-B+ severe combined immunodeficiency (SCID) due to CD45 deficiency is an extremely rare form of SCID caused by mutations in the PTPRC gene, which encodes the CD45 protein (also known as leukocyte common antigen or protein tyrosine phosphatase receptor type C). CD45 is a transmembrane phosphatase expressed on all nucleated hematopoietic cells and plays a critical role in T-cell and B-cell receptor signaling. Loss of functional CD45 leads to a profound defect in T-cell development and function, while B cells may be present in normal or near-normal numbers but are functionally impaired due to the lack of T-cell help and intrinsic B-cell signaling defects. Affected infants typically present in the first months of life with severe, recurrent, and life-threatening infections, including pneumonia, chronic diarrhea, oral candidiasis, and failure to thrive. The immunological phenotype is characterized by absent or profoundly reduced T cells with the presence of B cells (T-B+ SCID), though natural killer (NK) cell numbers may vary. Without treatment, the condition is fatal in early childhood due to overwhelming infections. The immune system is the primary body system affected, but secondary complications can involve the lungs, gastrointestinal tract, skin, and other organs due to opportunistic infections. The definitive treatment for CD45-deficient SCID is hematopoietic stem cell transplantation (HSCT), which can reconstitute the immune system if performed early, ideally before the onset of severe infections. Newborn screening programs that detect SCID through T-cell receptor excision circle (TREC) assays can identify affected infants before clinical symptoms develop, improving transplant outcomes. Gene therapy is not yet established for this specific form of SCID. Supportive care includes infection prophylaxis, immunoglobulin replacement therapy, and protective isolation until definitive treatment is achieved.

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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 CD45 deficiency.

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No actively recruiting trials found for T-B+ severe combined immunodeficiency due to CD45 deficiency at this time.

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No specialists are currently listed for T-B+ severe combined immunodeficiency due to CD45 deficiency.

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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 CD45 deficiency.

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Community

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Common questions about T-B+ severe combined immunodeficiency due to CD45 deficiency

What is T-B+ severe combined immunodeficiency due to CD45 deficiency?

T-B+ severe combined immunodeficiency (SCID) due to CD45 deficiency is an extremely rare form of SCID caused by mutations in the PTPRC gene, which encodes the CD45 protein (also known as leukocyte common antigen or protein tyrosine phosphatase receptor type C). CD45 is a transmembrane phosphatase expressed on all nucleated hematopoietic cells and plays a critical role in T-cell and B-cell receptor signaling. Loss of functional CD45 leads to a profound defect in T-cell development and function, while B cells may be present in normal or near-normal numbers but are functionally impaired due to th

How is T-B+ severe combined immunodeficiency due to CD45 deficiency inherited?

T-B+ severe combined immunodeficiency due to CD45 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 CD45 deficiency typically begin?

Typical onset of T-B+ severe combined immunodeficiency due to CD45 deficiency is infantile. Age of onset can vary across affected individuals.