title: Prospective multicenter trial comparing repeated immunosuppressive therapy with stem-cell transplantation from an alternative donor as second-line treatment for children with severe and very severe aplastic anemia.
abstract: We conducted a prospective multicenter study to compare the efficacy of repeated immunosuppressive therapy (IST) with stem-cell transplantation (SCT) from an alternative donor in children with acquired aplastic anemia (AA) who failed to respond to an initial course of IST. Patients with severe (n = 86) and very severe disease (n = 119) received initial IST consisting of antithymocyte globulin (ATG) and cyclosporine. Sixty patients failed to respond to IST after 6 months from the initial IST and were eligible for second-line treatment. Among them, 21 patients lacking suitable donors received a second course of IST. Three patients developed an anaphylactoid reaction to ATG and could not complete the second IST. A trilineage response was seen in only 2 of 18 (11%) evaluable patients after 6 months. Thirty-one patients received SCT from an alternative donor. At 5 years from the initiation of second-line therapy, the estimated failure-free survival (FFS), defined as survival with response, was 83.9% (+/- 16.1%, SD) in the SCT group compared with 9.5% (+/- 9.0%) in the IST group (P = .001). These results suggest that SCT from an alternative donor offers a better chance of FFS than a second IST in patients not responding to an initial IST.
authors.affiliation: Department of Pediatrics, Hyogo Children Hospital, Hyogo, Japan.
authors.forename: Yoshiyuki
authors.initials: Y
authors.lastname: Kosaka
authors.forename: Hiroshi
authors.initials: H
authors.lastname: Yagasaki
authors.forename: Kimihiko
authors.initials: K
authors.lastname: Sano
authors.forename: Ryoji
authors.initials: R
authors.lastname: Kobayashi
authors.forename: Hiroshi
authors.initials: H
authors.lastname: Ayukawa
authors.forename: Takashi
authors.initials: T
authors.lastname: Kaneko
authors.forename: Hiromasa
authors.initials: H
authors.lastname: Yabe
authors.forename: Masahiro
authors.initials: M
authors.lastname: Tsuchida
authors.forename: Hideo
authors.initials: H
authors.lastname: Mugishima
authors.forename: Akira
authors.initials: A
authors.lastname: Ohara
authors.forename: Akira
authors.initials: A
authors.lastname: Morimoto
authors.forename: Yoshitoshi
authors.initials: Y
authors.lastname: Otsuka
authors.forename: Shouichi
authors.initials: S
authors.lastname: Ohga
authors.forename: Fumio
authors.initials: F
authors.lastname: Bessho
authors.forename: Tatsutoshi
authors.initials: T
authors.lastname: Nakahata
authors.forename: Ichiro
authors.initials: I
authors.lastname: Tsukimoto
authors.forename: Seiji
authors.initials: S
authors.lastname: Kojima
mesh_terms: D000293:Adolescent; D000741:Anemia, Aplastic / Q000188:drug therapy* / Q000473:pathology / Q000601:surgery*; D002648:Child; D002675:Child, Preschool; D005260:Female; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use*; D007223:Infant; D008297:Male; D011446:Prospective Studies; D033581:Stem Cell Transplantation; D014019:Tissue Donors; D016896:Treatment Outcome
chemical_list: D007166:Immunosuppressive Agents