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

Autor: V. Spencer, Dawn Barge, Andrew J. Cant, G. Cavanagh, Terry Flood, K Brigham, P. Palmer, Andrew R. Gennery, A Jackson, Mario Abinun, A Curtis, Vaughan Carter, Anne M. Dickinson, Gavin P. Spickett
Rok vydání: 2001
Předmět:
Male
Cancer Research
medicine.medical_specialty
Transplantation Conditioning
Campath-1M
Antibodies
Neoplasm
Long term follow up
T-Lymphocytes
T cell
Immunology
Human leukocyte antigen
Antibodies
Monoclonal
Humanized
Single Center
Gastroenterology
Leukocyte Count
Postoperative Complications
Immune system
Internal medicine
Humans
Immunology and Allergy
Medicine
Child
Alemtuzumab
Survival rate
Genetics (clinical)
Bone Marrow Transplantation
Retrospective Studies
Immunosuppression Therapy
B-Lymphocytes
Transplantation Chimera
Transplantation
business.industry
Graft Survival
Antibodies
Monoclonal
Infant
Cell Biology
Survival Rate
Treatment Outcome
surgical procedures
operative
medicine.anatomical_structure
Oncology
Child
Preschool
Immune System
Female
Severe Combined Immunodeficiency
business
Follow-Up Studies
Zdroj: Cytotherapy. 3:221-232
ISSN: 1465-3249
DOI: 10.1080/146532401753174052
Popis: 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 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 BMT and the survival rate was 63%. All have donor T cells, 58% normal T-cell numbers and 14% 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 polysacchande. 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%sur-vival. 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.
Databáze: OpenAIRE
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