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Blood, 1 February 2008, Vol. 111, No. 3, pp. 1054-1059. Prepublished online as a Blood First Edition Paper on November 7, 2007; DOI 10.1182/blood-2007-08-099168. This Article Full Text Full Text (PDF) Supplemental Appendix All Versions of this Article: blood-2007-08-099168v1 111/3/1054    most recent Alert me when this article is cited Alert me if a correction is posted Citation Map Services Email this article to a friend Similar articles in this journal Similar articles in PubMed Alert me to new issues of the journal Download to citation manager Rights and Permissions Citing Articles Citing Articles via CrossRef Citing Articles via Google Scholar Google Scholar Articles by Kosaka, Y. Articles by Kojima, S. Search for Related Content PubMed PubMed Citation Articles by Kosaka, Y. Articles by Kojima, S. Related Collections Clinical Trials and Observations Social Bookmarking                   What's this?  Previous Article  |  Table of Contents  |  Next Article  CLINICAL TRIALS AND OBSERVATIONS 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 Yoshiyuki Kosaka1, Hiroshi Yagasaki2, Kimihiko Sano3, Ryoji Kobayashi4, Hiroshi Ayukawa5, Takashi Kaneko6, Hiromasa Yabe7, Masahiro Tsuchida8, Hideo Mugishima9, Akira Ohara10, Akira Morimoto11, Yoshitoshi Otsuka12, Shouichi Ohga13, Fumio Bessho14, Tatsutoshi Nakahata15, Ichiro Tsukimoto16, Seiji Kojima, on behalf of the Japan Childhood Aplastic Anemia Study Group2 1 Department of Pediatrics, Hyogo Children Hospital, Hyogo; 2 Department of Pediatrics, Nagoya University Graduate School of Medicine, Aichi; 3 Department of Pediatrics, Kobe University Graduate School of Medicine, Hyogo; 4 Department of Pediatrics, Hokkaido University Graduate School of Medicine, Hokkaido; 5 Department of Pediatrics, Yamaguchi University School of Medicine, Yamaguchi; 6 Department of Pediatrics, Kiyose Children's Hospital, Tokyo; 7 Specialized Clinical Science, Pediatrics, Tokai University School of Medicine, Kanagawa; 8 Department of Pediatrics, Ibaraki Children's Hospital, Ibaraki; 9 Department of Pediatrics, Nihon University School of Medicine, Tokyo; 10 Department of Transfusion, Toho University School of Medicine, Tokyo; 11 Department of Pediatrics, Kyoto Prefectural University of Medicine, Kyoto; 12 Department of Pediatrics, Hyogo College of Medicine, Hyogo; 13 Department of Pediatrics, Kyushu University Graduate School of Medicine, Fukuoka; 14 Department of Pediatrics, Kyorin University School of Medicine, Tokyo; 15 Department of Pediatrics, Kyoto University Graduate School of Medicine, Kyoto; and 16 Department of Pediatrics, Toho University School of Medicine, Tokyo, Japan 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. CiteULike    Connotea    Del.icio.us    Digg    Reddit    Technorati    What's this?

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