Matched unrelated bone marrow transplant for T+ combined immunodeficiency.
Little information is currently available on the outcome and the long-term restoration of immune function in infants with combined immunodeficiency and residual T cells (T+ CID) treated by BMT. We prospectively followed patients with T+ CID who received matched unrelated donor BMT at our center. Engraftment, immune reconstitution and transplant-related complications were recorded. Humoral and cellular immunity were evaluated. Ten patients with combined immune deficiency who had more than 1,000 circulating T cells/mul were designated as having T+ CID. They were diagnosed at a mean age of 9.7 months and received a matched unrelated donor BMT at the mean age of 17.4 months. All 10 patients are alive and well at a mean of 110 months after transplant. All patients have evidence of full hemopoietic engraftment and robust immune function. We have shown here that matched unrelated donor BMT is highly effective in curing patients with T+ CID. This mode of treatment should be preferred for patients with T+ CID when a related identical donor is not available.
Roifman
C M
CM
Division of Immunology and Allergy, The Hospital for Sick Children, Ontario, Canada. chaim.roifman@s ickkids.ca
Somech
R
R
Grunebaum
E
E
D016026:Bone Marrow Transplantation / Q000379:methods*; D002675:Child, Preschool; D018572:Disease-Free Survival; D005260:Female; D006085:Graft Survival; D006650:Histocompatibility Testing; D006801:Humans; D007223:Infant; D008297:Male; D011446:Prospective Studies; D016511:Severe Combined Immunodeficiency / Q000276:immunology / Q000628:therapy*; D016176:T-Lymphocyte Subsets; D014184:Transplantation, Homologous / Q000276:immunology / Q000379:methods*