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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 (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 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. Social Bookmarking                   What's this?  Previous Article  |  Next Article  Submitted August 8, 2007 Accepted October 8, 2007 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 Kosaka, Hiroshi Yagasaki, Kimihiko Sano, Ryoji Kobayashi, Hiroshi Ayukawa, Takashi Kaneko, Hiromasa Yabe, Masahiro Tsuchida, Hideo Mugishima, Akira Ohara, Akira Morimoto, Yoshitoshi Otsuka, Shyoichi Oga, Fumio Bessho, Tatsutoshi Nakahata, Ichiro Tsukimoto, and Seiji Kojima* Department of Pediatrics, Hyogo Children's Hospital, Hyogo, Japan Department of Pediatrics, Nagoya University Graduate School of Medicine, Aichi, Japan Department of Pediatrics, Kobe University Graduate School of Medicine, Hyogo, Japan Department of Pediatrics, Hokkaido University Graduate School of Medicine, Hokkaido, Japan Department of Pediatrics, Yamaguchi University School of Medicine, Yamaguchi, Japan Department of Pediatrics, Kiyose Childrens' Hospital, Tokyo, Japan Specialized Clinical Science, Pediatrics, Tokai University School of Medicine, Kanagawa, Japan Department of Pediatrics, Ibaraki Childrens' Hospital, Ibaraki, Japan Department of Pediatrics, Nihon University School of Medicine, Tokyo, Japan Department of Transfusion, Toho University School of Medicine, Tokyo, Japan Department of Pediatrics, Kyoto Prefectural University of Medicine, Kyoto, Japan Department of Pediatrics, Hyogo College of Medical, Hyogo, Japan Department of Pediatrics, Kyushu University Graduate School of Medicine, Fukuoka, Japan Department of Pediatrics, Kyorin University School of Medicine, Tokyo, Japan Department of Pediatrics, Kyoto University Graduate School of Medicine, Kyoto, Japan Department of Pediatrics, Toho University School of Medicine, Tokyo, Japan * Corresponding author; email: kojimas{at}med.nagoya-u.ac.jp. 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% in the SCT group compared with 9.5%±9.0% in the IST group (p=0.0001). 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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