Thymus Transplantation With Immunosuppression (884)

This is an interventional treatment trial for DiGeorge Syndrome focused on measuring Thymus Transplantation, DiGeorge Anomaly, DiGeorge Syndrome, Athymia, Low T cell numbers, Immunoreconstitution, Immunodeficiency, Complete DiGeorge, Typical DiGeorge, Atypical DiGeorge, Complete DiGeorge Anomaly Read More

Transplant Inclusion

• No age limit
• Thyroid studies must be done and if abnormal, must be on therapy

DiGeorge diagnosis - must have 1 symptom from the following list:

• Heart defect
• Hypocalcemia requiring replacement
• 22q11 hemizygosity
• 10p13 hemizygosity
• CHARGE association
• Abnormal ears plus mother with diabetes (type I, type II, or gestational)

Atypical Diagnosis:

• Must have, or have had, a rash. If rash present, biopsy of rash must show T cells in skin. If rash & adenopathy resolved, must still have oligoclonal T cells.
• Within 1 month of tx must have PHA response >20 fold above background or >5,000 cpm, whichever is higher, or response can be < this.
• Circulating CD3+ T cells >50/mm3 but CD45RA+CD62L+CD3+ T cells <50/mm or <5% of CD3 count, whichever is higher (must be done 2x)
• Immunoscope with >40% oligoclonal TCRBV families. A 2nd test per sponsor discretion if T cell numbers increase or activation status changes.
• If TREC done pre-tx must have TRECs <100 per 100,000 CD3+ cells.

Typical Diagnosis:

• Circulating CD3+ CD45RA+ CD62L+ T cells and <50/mm3 or <5% of total T cells
• PHA response >20 fold above background or >5,000 cpm, whichever is higher.
• 2 studies must show similar immunological findings qualify for this study.
• TRECs, if done, should be <100/100,000 CD3 cells

Transplant Exclusion:

• Heart surgery <4 weeks pre-tx date
• Heart surgery anticipated w/in 3 months of proposed tx
• Rejection by surgeon or anesthesiologist as surgical candidates
• Lack of sufficient muscle tissue to accept 0.2 grams/kg transplant