title: Unrelated donor bone marrow transplantation for children with severe aplastic anemia: minimal GVHD and durable engraftment with partial T cell depletion.
abstract: Both increased graft rejection and increased graft vs host disease (GVHD) remain obstacles to success for unrelated donor (URD) BMT for patients with SAA. Partial T cell depletion (PTCD) may decrease the risk of severe GVHD, while still maintaining sufficient donor T lymphocytes to ensure engraftment. We report on 12 patients with SAA who underwent PTCD URD BMT. All patients had failed medical therapy or relapsed following initial responses, and were transfusion dependent. The median age was 6 years, and there were five males. Donors were matched for four patients, and mismatched for eight. All patients received total body irradiation with either Ara-C or thiotepa and cyclophosphamide. PTCD was accomplished using monoclonal antibody T10B9 or OKT3 and complement. All patients engrafted, with a median time of 18 days to ANC >500. Only one patient had greater than grade II acute GVHD; two patients had limited and one patient extensive chronic GVHD. Nine patients are alive and transfusion independent at a median months post BMT. Three patients died from infection or renal failure. This series suggests that an aggressive immunosuppressive conditioning regimen with PTCD results in successful engraftment and minimal GVHD in pediatric patients with SAA, even with HLA mismatched donors.
authors.affiliation: The Children's Hospital of Philadelphia, Philadelphia, PA, USA. buninn@email.chop.edu
authors.forename: N
authors.initials: N
authors.lastname: Bunin
authors.forename: R
authors.initials: R
authors.lastname: Aplenc
authors.forename: R
authors.initials: R
authors.lastname: Iannone
authors.forename: A
authors.initials: A
authors.lastname: Leahey
authors.forename: S
authors.initials: S
authors.lastname: Grupp
authors.forename: D
authors.initials: D
authors.lastname: Monos
authors.forename: G
authors.initials: G
authors.lastname: Pierson
mesh_terms: D000293:Adolescent; D000328:Adult; D000741:Anemia, Aplastic / Q000401:mortality / Q000628:therapy*; D000970:Antineoplastic Agents / Q000008:administration & dosage; D016026:Bone Marrow Transplantation; D002648:Child; D002675:Child, Preschool; D005260:Female; D006085:Graft Survival; D006086:Graft vs Host Disease / Q000401:mortality / Q000517:prevention & control*; D006650:Histocompatibility Testing; D006801:Humans; D008212:Lymphocyte Depletion / Q000379:methods; D008297:Male; D019172:Transplantation Conditioning / Q000379:methods; D014916:Whole-Body Irradiation
chemical_list: D000970:Antineoplastic Agents