First-line treatment for severe aplastic anemia in children: bone marrow transplantation from a matched family donor vs. immunosuppressive therapy | Aplastic Anemia and MDS International Foundation

First-line treatment for severe aplastic anemia in children: bone marrow transplantation from a matched family donor vs. immunosuppressive therapy

Journal Title: 
Haematologica
Author(s): 
Yoshida N, Kobayashi R, Yabe H, Kosaka Y, Yagasaki H, Watanabe KI, Kudo K, Morimoto A, Ohga S, Muramatsu H, Takahashi Y, Kato K, Suzuki R, Ohara A, Kojima S
Primary Author: 
Yoshida N
Original Publication Date: 
Friday, September 5, 2014

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 Japan Childhood Aplastic Anemia Study Group or who underwent bone marrow transplantation from an HLA-matched family donor registered in the Japan Society for Hematopoietic Cell Transplantation Registry. Between 1992 and 2009, 599 children (younger than 17 years) with severe aplastic anemia received bone marrow transplantation 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 immunosuppressive therapy and bone marrow transplantation (88% [95% confidence interval: 86-90] vs. 92% [90-94]), failure-free survival was significantly inferior in patients receiving immunosuppressive therapy than in those receiving bone marrow transplantation (56% [54-59] vs. 87% [85-90]; P<0.0001). There was no significant improvement in outcomes over the two time periods (1992-1999 vs. 2000-2009). In multivariate analysis, age <10 years was identified as a favorable factor for overall survival (P=0.007), and choice of first-line immunosuppressive therapy was the only unfavorable factor for failure-free survival (P<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.

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