TY - JOUR
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
Y1 - 2011/6
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.
UR - http://www.scopus.com/inward/record.url?scp=79958021197&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=79958021197&partnerID=8YFLogxK
U2 - 10.3324/haematol.2010.035600
DO - 10.3324/haematol.2010.035600
M3 - Article
C2 - 21422115
AN - SCOPUS:79958021197
SN - 0390-6078
VL - 96
SP - 814
EP - 819
JO - Haematologica
JF - Haematologica
IS - 6
ER -