Outcome of non-T-cell-depleted HLA-haploidentical hematopoietic stem cell transplantation from family donors in children and adolescents.
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.
Yoshihara
Takao
T
Department of Pediatrics, Matsushita Memorial Hospital, Moriguchi, Japan. yoshihara.takao@jp.panasonic.com
Okada
Keiko
K
Kobayashi
Michihiro
M
Kikuta
Atsushi
A
Kato
Koji
K
Adachi
Naoto
N
Kikuchi
Akira
A
Ishida
Hiroyuki
H
Hirota
Yasuzou
Y
Kuroda
Hiroshi
H
Nagatoshi
Yoshihisa
Y
Inukai
Takeshi
T
Koike
Kazutoshi
K
Kigasawa
Hisato
H
Yagasaki
Hiroshi
H
Tokuda
Kiriko
K
Kishimoto
Tomoko
T
Nakano
Takahide
T
Fujita
Naoto
N
Goto
Hiroaki
H
Nakazawa
Yozo
Y
Kanegane
Hirokazu
H
Matsuzaki
Akinobu
A
Osugi
Yuko
Y
Hasegawa
Daiichiro
D
Uoshima
Nobuhiko
N
Nakamura
Kazuhiro
K
Tsuchida
Masahiro
M
Tanaka
Ryuhei
R
Watanabe
Arata
A
Yabe
Hiromasa
H
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
D007166:Immunosuppressive Agents; D016559:Tacrolimus; D008727:Methotrexate