title: Outcome of non-T-cell-depleted HLA-haploidentical hematopoietic stem cell transplantation from family donors in children and adolescents.
abstract: Non-T-cell-depleted HLA-haploidentical hematopoietic stem cell transplantation (SCT) from family members has been reported, but its effectiveness and safety are not fully known. In this study, we examined the outcomes of 83 children and adolescents with nonmalignant (n = 11) or malignant (n = 72) disorders who underwent SCT mismatched at 2 or 3 HLA loci, either from the mother (n = 56), a noninherited maternal antigen (NIMA)-mismatched sibling (n = 14), or the father/a noninherited paternal antigen (NIPA)-mismatched sibling (n = 13). Engraftment was satisfactory. Severe (grade III-IV) acute graft-versushost disease (GVHD) was noted only in malignant disease, with an incidence of 21 of 64 evaluable patients. GVHD prophylaxis with a combination of tacrolimus and methotrexate was significantly associated with a lower risk of severe acute GVHD, compared with other types of prophylaxis (P = .04). Nine of 11 patients with nonmalignant disease and 29 of 72 patients with malignant disease were alive at a median follow-up of 26 months (range, 4-57 months). Outcomes were not significantly different among the 3 donor groups (mother versus NIMA-mismatched sibling versus father/NIPA-mismatched sibling) for the malignancy disorders. Our results indicate that non-T-cell-depleted HLA-haploidentical SCT may be feasible, with appropriate GVHD prophylaxis, for young recipients who lack immediate access to a conventional stem cell source.
authors.affiliation: Department of Pediatrics, Matsushita Memorial Hospital, Moriguchi, Japan. yoshihara.takao@jp.panasonic.com
authors.forename: Takao
authors.initials: T
authors.lastname: Yoshihara
authors.forename: Keiko
authors.initials: K
authors.lastname: Okada
authors.forename: Michihiro
authors.initials: M
authors.lastname: Kobayashi
authors.forename: Atsushi
authors.initials: A
authors.lastname: Kikuta
authors.forename: Koji
authors.initials: K
authors.lastname: Kato
authors.forename: Naoto
authors.initials: N
authors.lastname: Adachi
authors.forename: Akira
authors.initials: A
authors.lastname: Kikuchi
authors.forename: Hiroyuki
authors.initials: H
authors.lastname: Ishida
authors.forename: Yasuzou
authors.initials: Y
authors.lastname: Hirota
authors.forename: Hiroshi
authors.initials: H
authors.lastname: Kuroda
authors.forename: Yoshihisa
authors.initials: Y
authors.lastname: Nagatoshi
authors.forename: Takeshi
authors.initials: T
authors.lastname: Inukai
authors.forename: Kazutoshi
authors.initials: K
authors.lastname: Koike
authors.forename: Hisato
authors.initials: H
authors.lastname: Kigasawa
authors.forename: Hiroshi
authors.initials: H
authors.lastname: Yagasaki
authors.forename: Kiriko
authors.initials: K
authors.lastname: Tokuda
authors.forename: Tomoko
authors.initials: T
authors.lastname: Kishimoto
authors.forename: Takahide
authors.initials: T
authors.lastname: Nakano
authors.forename: Naoto
authors.initials: N
authors.lastname: Fujita
authors.forename: Hiroaki
authors.initials: H
authors.lastname: Goto
authors.forename: Yozo
authors.initials: Y
authors.lastname: Nakazawa
authors.forename: Hirokazu
authors.initials: H
authors.lastname: Kanegane
authors.forename: Akinobu
authors.initials: A
authors.lastname: Matsuzaki
authors.forename: Yuko
authors.initials: Y
authors.lastname: Osugi
authors.forename: Daiichiro
authors.initials: D
authors.lastname: Hasegawa
authors.forename: Nobuhiko
authors.initials: N
authors.lastname: Uoshima
authors.forename: Kazuhiro
authors.initials: K
authors.lastname: Nakamura
authors.forename: Masahiro
authors.initials: M
authors.lastname: Tsuchida
authors.forename: Ryuhei
authors.initials: R
authors.lastname: Tanaka
authors.forename: Arata
authors.initials: A
authors.lastname: Watanabe
authors.forename: Hiromasa
authors.initials: H
authors.lastname: Yabe
mesh_terms: D000293:Adolescent; D016026:Bone Marrow Transplantation / Q000379:methods; D002648:Child; D002675:Child, Preschool; D046528:Chimerism; D004359:Drug Therapy, Combination; D030342:Genetic Diseases, Inborn / Q000628:therapy; D006086:Graft vs Host Disease / Q000517:prevention & control; D019337:Hematologic Neoplasms / Q000628:therapy; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects / Q000379:methods*; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use*; D007223:Infant; D053208:Kaplan-Meier Estimate; D008727:Methotrexate / Q000627:therapeutic use*; D036102:Peripheral Blood Stem Cell Transplantation / Q000379:methods; D012189:Retrospective Studies; D016559:Tacrolimus / Q000627:therapeutic use*; D014184:Transplantation, Homologous / Q000009:adverse effects; D016896:Treatment Outcome
chemical_list: D007166:Immunosuppressive Agents; D016559:Tacrolimus; D008727:Methotrexate