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 nature 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 long-term 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 B-If T 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 5116 (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-BAIT Eleven children died (37%). Discussion CAMPATH-1M T-cell depleted BMT for SCID resulted in 63% survival. Deaths of I I children were mainly due to pre-existing infections Seventeen of 19 long- term survivors have normal immune function and good quality-of-life.