Back to Search
Start Over
Long-term outcomes of nonconditioned patients with severe combined immunodeficiency transplanted with HLA-identical or haploidentical bone marrow depleted of T cells with anti-CD6 mAb
- Source :
- Journal of Allergy and Clinical Immunology. 122:1185-1193
- Publication Year :
- 2008
- Publisher :
- Elsevier BV, 2008.
-
Abstract
- Background Between 1981 and 1995, 20 children with severe combined immunodeficiency (SCID; median age at transplant, 6.5 [range, 0.5-145] mo, 12 with serious infection) were treated with haploidentical T cell–depleted (anti-CD6 antibody) bone marrow (median number of 5.7 [0.8-18.8] × 10 8 nucleated cells/kg) from mismatched related donors (MMRDs), and 5 children with SCID (median age at transplant, 1.8 [0.5-5.0] mo, 1 with serious infection) were given unmanipulated bone marrow from matched related donors (MRDs). No conditioning or graft-versus-host disease (GvHD) prophylaxis was used. Objective To assess the outcomes of patients with SCID who received bone marrow from MMRDs or MRDs. Methods We reviewed the medical records of these 25 consecutive patients with SCID (4 with Omenn syndrome). Results Of the 20 patients who received bone marrow from MMRDs, 12 engrafted, 10 survived at a median age of 15.2 [10.0-19.1] years, 4 had chronic GvHD (lung, intestine, skin), 5 required intravenous immunoglobulin, and 8 attended school or college. Two of 5 patients who died had chronic GvHD, and 2 developed lymphoproliferative disease. Of the 5 patients who received bone marrow from MRDs, 5 engrafted, 5 survived at a median age of 23.3 [18.5-26] years, 1 had chronic GvHD (lung, skin), 2 required intravenous immunoglobulin, and 4 attended school or college. Conclusions Treatment of critically ill patients with SCID with anti-CD6 antibody T cell–depleted MMRD marrow resulted in an overall 50% long-term survival of patients (83% survival of those engrafted). The principal barriers to long-term survival were delay in diagnosis, life-threatening infection, failure to engraft, and chronic GvHD. Educational goals were achieved in most of the survivors.
- Subjects :
- Adult
Antigens, Differentiation, T-Lymphocyte
Graft Rejection
Male
medicine.medical_specialty
Time Factors
Adolescent
T-Lymphocytes
medicine.medical_treatment
Immunology
Graft vs Host Disease
Hematopoietic stem cell transplantation
Gastroenterology
Disease-Free Survival
Lymphocyte Depletion
Antigens, CD
Internal medicine
medicine
Humans
Immunology and Allergy
Child
Survival rate
Immunodeficiency
Bone Marrow Transplantation
Retrospective Studies
Severe combined immunodeficiency
business.industry
Graft Survival
Antibodies, Monoclonal
Infant
medicine.disease
Lymphoproliferative Disorders
Omenn syndrome
Survival Rate
Transplantation
Graft-versus-host disease
medicine.anatomical_structure
Child, Preschool
Female
Severe Combined Immunodeficiency
Bone marrow
business
Subjects
Details
- ISSN :
- 00916749
- Volume :
- 122
- Database :
- OpenAIRE
- Journal :
- Journal of Allergy and Clinical Immunology
- Accession number :
- edsair.doi.dedup.....f13ce22536056a13d826c653ffc8f502
- Full Text :
- https://doi.org/10.1016/j.jaci.2008.10.030