CAMPATH-1M T-cell depleted BMT for SCID: long-term follow-up of 19 children treated 1987-98 in a single center

Cytotherapy. 2001;3(3):221-32. doi: 10.1080/146532401753174052.

Abstract

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%).

Discussion: 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.

Publication types

  • Clinical Trial

MeSH terms

  • Alemtuzumab
  • Antibodies, Monoclonal / adverse effects
  • Antibodies, Monoclonal / immunology
  • Antibodies, Monoclonal / therapeutic use*
  • Antibodies, Monoclonal, Humanized
  • Antibodies, Neoplasm
  • B-Lymphocytes / cytology
  • B-Lymphocytes / drug effects
  • B-Lymphocytes / immunology
  • Bone Marrow Transplantation / adverse effects
  • Bone Marrow Transplantation / methods*
  • Child
  • Child, Preschool
  • Female
  • Follow-Up Studies
  • Graft Survival / drug effects
  • Graft Survival / immunology
  • Humans
  • Immune System / cytology
  • Immune System / drug effects
  • Immune System / immunology
  • Immunosuppression / adverse effects
  • Immunosuppression / methods*
  • Infant
  • Leukocyte Count
  • Male
  • Postoperative Complications / etiology
  • Postoperative Complications / immunology
  • Postoperative Complications / physiopathology
  • Retrospective Studies
  • Severe Combined Immunodeficiency / drug therapy*
  • Severe Combined Immunodeficiency / immunology
  • Severe Combined Immunodeficiency / physiopathology
  • Survival Rate
  • T-Lymphocytes / cytology
  • T-Lymphocytes / drug effects*
  • T-Lymphocytes / immunology
  • Transplantation Chimera / immunology
  • Transplantation Conditioning / adverse effects
  • Transplantation Conditioning / methods*
  • Treatment Outcome

Substances

  • Antibodies, Monoclonal
  • Antibodies, Monoclonal, Humanized
  • Antibodies, Neoplasm
  • Alemtuzumab