@article{Nao Yoshida_Ryoji Kobayashi_Hiromasa Yabe_Yoshiyuki Kosaka_Hiroshi Yagasaki_Ken-ichiro Watanabe_Kazuko Kudo_Akira Morimoto_Shouichi Ohga_Hideki Muramatsu_Yoshiyuki Takahashi_Koji Kato_Ritsuro Suzuki_Akira Ohara_Seiji Kojima_2014, place={Pavia, Italy}, title={First-line treatment for severe aplastic anemia in children: bone marrow transplantation from a matched family donor versus immunosuppressive therapy}, volume={99}, url={https://haematologica.org/article/view/7218}, DOI={10.3324/haematol.2014.109355}, abstractNote={The current treatment approach for severe aplastic anemia in children is based on studies performed in the 1980s, and updated evidence is required. We retrospectively compared the outcomes of children with acquired severe aplastic anemia who received immunosuppressive therapy within prospective trials conducted by the Japanese Childhood Aplastic Anemia Study Group or who underwent bone marrow transplantation from an HLA-matched family donor registered in the Japanese Society for Hematopoietic Cell Transplantation Registry. Between 1992 and 2009, 599 children (younger than 17 years) with severe aplastic anemia received a bone marrow transplant from an HLA-matched family donor (n=213) or immunosuppressive therapy (n=386) as first-line treatment. While the overall survival did not differ between patients treated with immunosuppressive therapy or bone marrow transplantation [88% (95% confidence interval: 86–90) <em>versus</em> 92% (90–94)], failure-free survival was significantly inferior in patients receiving immunosuppressive therapy than in those undergoing bone marrow transplantation [56% (54–59) <em>versus</em> 87% (85–90); <em>P</em><0.0001]. There was no significant improvement in outcomes over the two time periods (1992–1999 <em>versus</em> 2000–2009). In multivariate analysis, age <10 years was identified as a favorable factor for overall survival (<em>P</em>=0.007), and choice of first-line immunosuppressive therapy was the only unfavorable factor for failure-free survival (<em>P</em>&lt;0.0001). These support the current algorithm for treatment decisions, which recommends bone marrow transplantation when an HLA-matched family donor is available in pediatric severe aplastic anemia.}, number={12}, journal={Haematologica}, author={Nao Yoshida and Ryoji Kobayashi and Hiromasa Yabe and Yoshiyuki Kosaka and Hiroshi Yagasaki and Ken-ichiro Watanabe and Kazuko Kudo and Akira Morimoto and Shouichi Ohga and Hideki Muramatsu and Yoshiyuki Takahashi and Koji Kato and Ritsuro Suzuki and Akira Ohara and Seiji Kojima}, year={2014}, month={Dec.}, pages={1784-1791} }