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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Abstract

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
JournalHaematologica
Volume96
Issue number6
DOIs
Publication statusPublished - Jun 1 2011

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Aplastic Anemia
Immunosuppressive Agents
Japan
Recurrence
Antilymphocyte Serum
Therapeutics
Cyclosporine
Hematopoietic Stem Cell Transplantation
Multicenter Studies
Danazol
Cohort Studies
Multivariate Analysis
Prospective Studies

All Science Journal Classification (ASJC) codes

  • Hematology

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Relapse of aplastic anemia in children after immunosuppressive therapy : A report from the Japan childhood aplastic anemia study group. / Kamio, Takuya; Ito, Etsuro; Ohara, Akira; Kosaka, Yoshiyuki; Tsuchida, Masahiro; Yagasaki, Hiroshi; Mugishima, Hideo; Yabe, Hiromasa; Morimoto, Akira; Ohga, Shouichi; Muramatsu, Hideki; Hama, Asahito; Kaneko, Takashi; Nagasawa, Masayuki; Kikuta, Atsushi; Osugi, Yuko; Bessho, Fumio; Nakahata, Tatsutoshi; Tsukimoto, Ichiro; Kojima, Seiji.

In: Haematologica, Vol. 96, No. 6, 01.06.2011, p. 814-819.

Research output: Contribution to journalArticle

Kamio, T, Ito, E, Ohara, A, Kosaka, Y, Tsuchida, M, Yagasaki, H, Mugishima, H, Yabe, H, Morimoto, A, Ohga, S, Muramatsu, H, Hama, A, Kaneko, T, Nagasawa, M, Kikuta, A, Osugi, Y, Bessho, F, Nakahata, T, Tsukimoto, I & Kojima, S 2011, 'Relapse of aplastic anemia in children after immunosuppressive therapy: A report from the Japan childhood aplastic anemia study group', Haematologica, vol. 96, no. 6, pp. 814-819. https://doi.org/10.3324/haematol.2010.035600
Kamio, Takuya ; Ito, Etsuro ; Ohara, Akira ; Kosaka, Yoshiyuki ; Tsuchida, Masahiro ; Yagasaki, Hiroshi ; Mugishima, Hideo ; Yabe, Hiromasa ; Morimoto, Akira ; Ohga, Shouichi ; Muramatsu, Hideki ; Hama, Asahito ; Kaneko, Takashi ; Nagasawa, Masayuki ; Kikuta, Atsushi ; Osugi, Yuko ; Bessho, Fumio ; Nakahata, Tatsutoshi ; Tsukimoto, Ichiro ; Kojima, Seiji. / Relapse of aplastic anemia in children after immunosuppressive therapy : A report from the Japan childhood aplastic anemia study group. In: Haematologica. 2011 ; Vol. 96, No. 6. pp. 814-819.
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abstract = "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.",
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T1 - Relapse of aplastic anemia in children after immunosuppressive therapy

T2 - A report from the Japan childhood aplastic anemia study group

AU - Kamio, Takuya

AU - Ito, Etsuro

AU - Ohara, Akira

AU - Kosaka, Yoshiyuki

AU - Tsuchida, Masahiro

AU - Yagasaki, Hiroshi

AU - Mugishima, Hideo

AU - Yabe, Hiromasa

AU - Morimoto, Akira

AU - Ohga, Shouichi

AU - Muramatsu, Hideki

AU - Hama, Asahito

AU - Kaneko, Takashi

AU - Nagasawa, Masayuki

AU - Kikuta, Atsushi

AU - Osugi, Yuko

AU - Bessho, Fumio

AU - Nakahata, Tatsutoshi

AU - Tsukimoto, Ichiro

AU - Kojima, Seiji

PY - 2011/6/1

Y1 - 2011/6/1

N2 - 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.

AB - 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.

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