title: Successful sustained engraftment after reduced-intensity umbilical cord blood transplantation for adult patients with severe aplastic anemia.
abstract: We retrospectively analyzed 12 consecutive adult severe aplastic anemia patients who received unrelated umbilical cord blood transplantation after a reduced-intensity conditioning regimen (RI-UCBT). The conditioning regimen consisted of 125 mg/m² fludarabine, 80 mg/m² melphalan, and 4 Gy of total body irradiation. The median infused total nucleated cell number and CD34(+) cell number were 2.50 × 10⁷/kg and 0.76 × 10⁵/kg, respectively. Eleven of the 12 patients achieved primary neutrophil and platelet engraftment. All patients who achieved engraftment had complete hematologic recovery with complete donor chimerism, except for one patient who developed late graft failure 3 years after RI-UCBT. Two of the 12 patients died of idiopathic pneumonia syndrome, and the remaining 10 patients are alive, having survived for a median of 36 months. Our encouraging results indicate that RI-UCBT may become a viable therapeutic option for adult severe aplastic anemia patients who lack suitable human leukocyte antigen-matched donors and fail immunosuppressive therapy.
authors.affiliation: Department of Hematology, Toranomon Hospital, Tokyo, Japan.
authors.forename: Hisashi
authors.initials: H
authors.lastname: Yamamoto
authors.forename: Daisuke
authors.initials: D
authors.lastname: Kato
authors.forename: Naoyuki
authors.initials: N
authors.lastname: Uchida
authors.forename: Kazuya
authors.initials: K
authors.lastname: Ishiwata
authors.forename: Hideki
authors.initials: H
authors.lastname: Araoka
authors.forename: Shinsuke
authors.initials: S
authors.lastname: Takagi
authors.forename: Nobuaki
authors.initials: N
authors.lastname: Nakano
authors.forename: Masanori
authors.initials: M
authors.lastname: Tsuji
authors.forename: Yuki
authors.initials: Y
authors.lastname: Asano-Mori
authors.forename: Naofumi
authors.initials: N
authors.lastname: Matsuno
authors.forename: Kazuhiro
authors.initials: K
authors.lastname: Masuoka
authors.forename: Koji
authors.initials: K
authors.lastname: Izutsu
authors.forename: Atsushi
authors.initials: A
authors.lastname: Wake
authors.forename: Akiko
authors.initials: A
authors.lastname: Yoneyama
authors.forename: Shigeyoshi
authors.initials: S
authors.lastname: Makino
authors.forename: Shuichi
authors.initials: S
authors.lastname: Taniguchi
mesh_terms: D000328:Adult; D000368:Aged; D000741:Anemia, Aplastic / Q000628:therapy*; D036101:Cord Blood Stem Cell Transplantation; D006801:Humans; D008875:Middle Aged; D016019:Survival Analysis; D019172:Transplantation Conditioning; D016896:Treatment Outcome; D055815:Young Adult