CAMPATH-1M T-cell depleted BMT for SCID: long-term follow-up of 19 children treated 1987-98 in a single center.
BACKGROUND
SCID can be cured by BMT. Depletion of mature T cells from BM has enabled HLA non-identical stem-cell transplantation. We report the outcome of 30 patients treated with 37 T-cell depleted BMT procedures using CAMPATH-1M in vitro between 1987-98 in a single center.


METHODS
Immune reconstitution and quality-of-life were assessed in 19 longterm survivors. All but two received pre-transplant conditioning. T- and B-cell chimerism, numbers and function were analyzed during a median follow-up of 5.3 years (range 1.33-12).


RESULTS
The overall engraftment rate was 59%, six children required repeated BMT and the survival rate was 63%. All have donor T cells, 58% normal T-cell numbers and 74% normal T-cell function. Of 17 evaluated, 16 patients (94%) have normal IgM and IgG levels, and production of specific Abs to protein Ags, but only 5/16 (31%) have a good response to pneumococcal polysaccharide. Early and late post-BMT complications were rare and there were no delayed deaths. Only one child continues on long-term i.v. Ig 4-years post-BMT. Eleven children died (37%).


CONCLUSIONS
CAMPATH-1M T-cell depleted BMT for SCID resulted in 63% survival. Deaths of 11 children were mainly due to pre-existing infections. Seventeen of 19 long-term survivors have normal immune function and good quality-of-life.
Gennery
A R
AR
Children's Bone Marrow Transplantation Unit, Newcastle upon Tyne Hospitals NHS Trust, UK.
Dickinson
A M
AM
Brigham
K
K
Barge
D
D
Spickett
G P
GP
Curtis
A
A
Spencer
V
V
Jackson
A
A
Cavanagh
G
G
Carter
V
V
Palmer
P
P
Flood
T J
TJ
Cant
A J
AJ
Abinun
M
M
D000074323:Alemtuzumab; D000911:Antibodies, Monoclonal / Q000009:adverse effects / Q000276:immunology / Q000627:therapeutic use*; D061067:Antibodies, Monoclonal, Humanized; D000912:Antibodies, Neoplasm; D001402:B-Lymphocytes / Q000166:cytology / Q000187:drug effects / Q000276:immunology; D016026:Bone Marrow Transplantation / Q000009:adverse effects / Q000379:methods*; D002648:Child; D002675:Child, Preschool; D005260:Female; D005500:Follow-Up Studies; D006085:Graft Survival / Q000187:drug effects / Q000276:immunology; D006801:Humans; D007107:Immune System / Q000166:cytology / Q000187:drug effects / Q000276:immunology; D007165:Immunosuppression Therapy / Q000009:adverse effects / Q000379:methods*; D007223:Infant; D007958:Leukocyte Count; D008297:Male; D011183:Postoperative Complications / Q000209:etiology / Q000276:immunology / Q000503:physiopathology; D012189:Retrospective Studies; D016511:Severe Combined Immunodeficiency / Q000188:drug therapy* / Q000276:immunology / Q000503:physiopathology; D015996:Survival Rate; D013601:T-Lymphocytes / Q000166:cytology / Q000187:drug effects* / Q000276:immunology; D018183:Transplantation Chimera / Q000276:immunology; D019172:Transplantation Conditioning / Q000009:adverse effects / Q000379:methods*; D016896:Treatment Outcome
D000911:Antibodies, Monoclonal; D061067:Antibodies, Monoclonal, Humanized; D000912:Antibodies, Neoplasm; D000074323:Alemtuzumab