title: A trial of alemtuzumab adjunctive therapy in allogeneic hematopoietic cell transplantation with minimal conditioning for severe combined immunodeficiency.
abstract: For infants with SCID the ideal conditioning regimen before allogeneic HCT would omit cytotoxic chemotherapy to minimize short- and long-term complications. We performed a prospective pilot trial with alemtuzumab monotherapy to overcome NK-cell mediated immunologic barriers to engraftment. We enrolled four patients who received CD34-selected haploidentical cells, two of whom failed to engraft donor T cells. The two patients who engrafted had delayed T-cell reconstitution, despite rapid clearance of circulating alemtuzumab. Although well-tolerated, alemtuzumab failed to overcome immunologic barriers to donor engraftment. Furthermore, alemtuzumab may slow T-cell development in patients with SCID in the setting of a T-cell depleted graft.
authors: Division of Pediatric Allergy, Immunology, and Blood and Marrow Transplant, University of California San Francisco, Benioff Children's Hospital, San Francisco, CA, USA.
authors: Christopher C
authors: CC
authors: Dvorak
authors: Biljana N
authors: BN
authors: Horn
authors: Jennifer M
authors: JM
authors: Puck
authors: Stuart
authors: S
authors: Adams
authors: Paul
authors: P
authors: Veys
authors: Agnieszka
authors: A
authors: Czechowicz
authors: Morton J
authors: MJ
authors: Cowan
mesh_terms: D000074323:Alemtuzumab; D061067:Antibodies, Monoclonal, Humanized / Q000009:adverse effects / Q000627:therapeutic use*; D002140:California; D017024:Chemotherapy, Adjuvant; D002648:Child; D018380:Hematopoietic Stem Cell Transplantation; D006650:Histocompatibility Testing; D006801:Humans; D007223:Infant; D008297:Male; D010865:Pilot Projects; D011446:Prospective Studies; D016511:Severe Combined Immunodeficiency / Q000188:drug therapy / Q000276:immunology / Q000628:therapy*; D019172:Transplantation Conditioning; D014184:Transplantation, Homologous; D016896:Treatment Outcome
chemical_list: D061067:Antibodies, Monoclonal, Humanized; D000074323:Alemtuzumab
keywords: alemtuzumab; engraftment; haploidentical; severe combined immunodeficiency