Relapse of aplastic anemia in children after immunosuppressive therapy: A report from the Japan childhood aplastic anemia study group

Takuya Kamio, Etsuro Ito, Akira Ohara, Yoshiyuki Kosaka, Masahiro Tsuchida, Hiroshi Yagasaki, Hideo Mugishima, Hiromasa Yabe, Akira Morimoto, Shouichi Ohga, Hideki Muramatsu, Asahito Hama, Takashi Kaneko, Masayuki Nagasawa, Atsushi Kikuta, Yuko Osugi, Fumio Bessho, Tatsutoshi Nakahata, Ichiro Tsukimoto, Seiji Kojima

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31 Citations (Scopus)


Background Although the therapeutic outcome of acquired aplastic anemia has improved markedly with the introduction of immunosuppressive therapy using antithymocyte globulin and cyclosporine, a significant proportion of patients subsequently relapse and require second-line therapy. However, detailed analyses of relapses in aplastic anemia children are limited. Design and Methods We previously conducted two prospective multicenter trials of immunosuppressive therapy for children with aplastic anemia: AA-92 and AA-97, which began in 1992 and 1997, respectively. In this study, we assessed the relapse rate, risk factors for relapse, and the response to secondline treatment in children with aplastic anemia treated with antithymocyte globulin and cyclosporine. Results From 1992 to 2007, we treated 441 children with aplastic anemia with standard immunosuppressive therapy. Among the 264 patients who responded to immunosuppressive therapy, 42 (15.9%) relapsed. The cumulative incidence of relapse was 11.9% at 10 years. Multivariate analysis revealed that relapse risk was significantly associated with an immunosuppressive therapy regimen using danazol (relative risk, 3.15; P=0.001) and non-severe aplastic anemia (relative risk, 2.51; P=0.02). Seventeen relapsed patients received additional immunosuppressive therapy with antithymocyte globulin and cyclosporine. Eight patients responded within 6 months. Seven of nine non-responders to second immunosuppressive therapy received hematopoietic stem cell transplantation and five are alive. Eleven patients underwent hematopoietic stem cell transplantation directly and seven are alive. Conclusions In the present study, the cumulative incidence of relapse at 10 years was relatively low compared to that in other studies mainly involving adult patients. A multicenter prospective study is warranted to establish optimal therapy for children with aplastic anemia.

Original languageEnglish
Pages (from-to)814-819
Number of pages6
Issue number6
Publication statusPublished - Jun 2011

All Science Journal Classification (ASJC) codes

  • Hematology


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