title: Feasibility of unmanipulated haploidentical stem cell transplantation using standard GVHD prophylaxis for HLA-homozygous patients.
abstract: HLA-haploidentical hematopoietic stem cell transplantation (haplo-SCT) in HLA-homozygous patients is accompanied by HLA mismatches only in the host-versus-graft vector, and thus theoretically could be performed with standard graft-versus-host disease (GVHD) prophylaxis. However, the risk of GVHD remains uncertain, and graft failure could be a problem. In this study, we assessed nine HLA-homozygous patients who underwent haplo-SCT. Preparative treatment was cyclophosphamide/total body irradiation-based regimen in five patients, fludarabine/busulfan-based regimen in two, and other regimens in two. GVHD prophylaxis consisted of cyclosporine and methotrexate in seven patients, cyclosporine and mycophenolate mofetil in one, and cyclosporine alone in one. Seven patients achieved neutrophil engraftment and platelet recovery. The median times to neutrophil engraftment and platelet recovery were 15 and 44 days, respectively. Two patients developed graft failure, including one who achieved engraftment with a second SCT from the same donor. Grade II GVHD was observed in half of the evaluable patients; grades III and IV were not observed. Two patients died from treatment-related causes. Five patients were alive after a median follow-up period of 563 days. The probability of overall survival at 5 years was 65 %. These findings may serve as a rationale for considering haplo-SCT as a treatment option for HLA-homozygous patients.
authors.affiliation: Division of Hematology, Department of Internal Medicine, Hyogo College of Medicine, 1-1 Mukogawa-cho, Nishinomiya, Hyogo, Japan.
authors.forename: Kazuhiro
authors.initials: K
authors.lastname: Ikegame
authors.forename: Katsuji
authors.initials: K
authors.lastname: Kaida
authors.forename: Satoshi
authors.initials: S
authors.lastname: Yoshihara
authors.forename: Masayuki
authors.initials: M
authors.lastname: Fujiwara
authors.forename: Kyoko
authors.initials: K
authors.lastname: Taniguchi
authors.forename: Ruri
authors.initials: R
authors.lastname: Kato
authors.forename: Takayuki
authors.initials: T
authors.lastname: Inoue
authors.forename: Tatsuya
authors.initials: T
authors.lastname: Fujioka
authors.forename: Hiroya
authors.initials: H
authors.lastname: Tamaki
authors.forename: Masaya
authors.initials: M
authors.lastname: Okada
authors.forename: Toshihiro
authors.initials: T
authors.lastname: Soma
authors.forename: Norihiko
authors.initials: N
authors.lastname: Kamikonya
authors.forename: Hiroh
authors.initials: H
authors.lastname: Saji
authors.forename: Shozo
authors.initials: S
authors.lastname: Hirota
authors.forename: Hiroyasu
authors.initials: H
authors.lastname: Ogawa
mesh_terms: D000328:Adult; D019072:Antibiotic Prophylaxis; D044466:Asian People; D005260:Female; D006085:Graft Survival / Q000235:genetics / Q000276:immunology; D006086:Graft vs Host Disease / Q000188:drug therapy / Q000517:prevention & control*; D006680:HLA Antigens / Q000235:genetics* / Q000276:immunology; D006239:Haplotypes; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects*; D006648:Histocompatibility; D006720:Homozygote; D006801:Humans; D007564:Japan; D008024:Ligands; D008297:Male; D008875:Middle Aged; D054340:Receptors, KIR / Q000276:immunology; D016896:Treatment Outcome
chemical_list: D006680:HLA Antigens; D008024:Ligands; D054340:Receptors, KIR