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	Yoshiyuki Kosaka, Hiroshi Yagasaki, Kimihiko Sano, Ryoji Kobayashi, Hiroshi Ayukawa, Takashi Kaneko, Hiromasa Yabe, Masahiro Tsuchida, Hideo Mugishima, Akira Ohara, Akira Morimoto, Yoshitoshi Otsuka, Shouichi Ohga, Fumio Bessho, Tatsutoshi Nakahata, Ichiro Tsukimoto, Seiji Kojima, Japan Childhood Aplastic Anemia Study Group
Journal	Blood (Blood) Vol. 111 Issue 3 Pg. 1054-9 (Feb 01 2008) ISSN: 0006-4971 [Print] United States
PMID	17989314 (Publication Type: Clinical Trial, Comparative Study, Journal Article, Multicenter Study)
Chemical References	Immunosuppressive Agents
Topics	Adolescent Anemia, Aplastic (drug therapy, pathology, surgery) Child Child, Preschool Female Humans Immunosuppressive Agents (therapeutic use) Infant Male Prospective Studies Stem Cell Transplantation Tissue Donors Treatment Outcome

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