title: Effects of conditioning regimens and T cell depletion in hematopoietic cell transplantation for primary immune deficiency.
abstract: This study analyzes the hematopoietic cell transplantation experience in patients with immune deficiency at a single institution. The objective is to comprehensively evaluate the short-term and long-term outcomes with various preparative regimens, donor grafts, and ex vivo manipulations to identify transplantation approaches that most likely favor early donor immune competency without generating excessive toxicity. Clinical outcomes were evaluated in 52 consecutive patients with immune deficiencies. Thirty-seven of the 52 patients (71%) survived with attenuation of their underlying disease. The use of a melphalan-based reduced-intensity conditioning preparative regimen and immunomagnetic CD3(+) T cell depletion techniques (when T cell depletion was indicated) were associated with improved event-free survival. Survivors who received a preparative regimen other than a melphalan-based reduced-intensity regimen suffered from therapy-related morbidities or chronic/recurrent infections. Our findings indicate that melphalan-based reduced-intensity conditioning regimens and immunomagnetic CD3(+) T cell depletion limit therapy-related toxicity, and demonstrate promising results for the early establishment of donor immune competency.
authors: Department of Bone Marrow Transplantation and Cellular Therapy, St. Jude Children's Research Hospital, Memphis, Tennessee, USA. Brandon.Triplett@stjude.org
authors: Brandon M
authors: BM
authors: Triplett
authors: Chong
authors: C
authors: Wang
authors: Jie
authors: J
authors: Yang
authors: Mari
authors: M
authors: Dallas
authors: Christine
authors: C
authors: Hartford
authors: Vanessa
authors: V
authors: Howard
authors: Asha
authors: A
authors: Pillai
authors: David
authors: D
authors: Shook
authors: Ashok
authors: A
authors: Srinivasan
authors: Joseph
authors: J
authors: Laver
authors: Wing
authors: W
authors: Leung
mesh_terms: D000293:Adolescent; D000328:Adult; D002648:Child; D002675:Child, Preschool; D018572:Disease-Free Survival; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods*; D006801:Humans; D007223:Infant; D007231:Infant, Newborn; D008212:Lymphocyte Depletion / Q000379:methods*; D008297:Male; D012189:Retrospective Studies; D016511:Severe Combined Immunodeficiency / Q000276:immunology / Q000601:surgery*; D013601:T-Lymphocytes / Q000166:cytology / Q000276:immunology*; D014184:Transplantation, Homologous; D055815:Young Adult