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First-line treatment for severe aplastic anemia in children: bone marrow transplantation from a matched family donor versus immunosuppressive therapy.
Haematologica. 2014 Dec; 99(12):1784-91.H

Abstract

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) versus 92% (90-94)], failure-free survival was significantly inferior in patients receiving immunosuppressive therapy than in those undergoing bone marrow transplantation [56% (54-59) versus 87% (85-90); P<0.0001]. There was no significant improvement in outcomes over the two time periods (1992-1999 versus 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.

Authors+Show Affiliations

Department of Hematology and Oncology, Children's Medical Center, Japanese Red Cross Nagoya First Hospital, Nagoya;Department of Pediatrics, Sapporo Hokuyu Hospital, Isehara;Department of Cell Transplantation and Regenerative Medicine, Tokai University School of Medicine, Isehara;Department of Pediatrics, Hyogo Children's Hospital, Kobe;Department of Pediatrics, Nihon University School of Medicine, Tokyo;Division of Hematology and Oncology, Shizuoka Children's Hospital, Toyoake;Department of Pediatrics, Fujita Health University School of Medicine, Toyoake;Department of Pediatrics, Jichi Medical University School of Medicine, Shimotsuke;Department of Perinatal and Pediatric Medicine, Kyushu University Graduate School of Medical Sciences, Fukuoka;Department of Pediatrics, Nagoya University Graduate School of Medicine, Tokyo, Japan;Department of Pediatrics, Nagoya University Graduate School of Medicine, Tokyo, Japan;Department of Hematology and Oncology, Children's Medical Center, Japanese Red Cross Nagoya First Hospital, Nagoya;Department of HSCT Data Management & Biostatistics, Nagoya University Graduate School of Medicine, Tokyo, Japan;Department of Pediatrics, Toho University School of Medicine, Tokyo, Japan.Department of Pediatrics, Nagoya University Graduate School of Medicine, Tokyo, Japan; kojimas@med.nagoya-u.ac.jp.

Pub Type(s)

Comparative Study
Journal Article
Multicenter Study

Language

eng

PubMed ID

25193958

Citation

Yoshida, Nao, et al. "First-line Treatment for Severe Aplastic Anemia in Children: Bone Marrow Transplantation From a Matched Family Donor Versus Immunosuppressive Therapy." Haematologica, vol. 99, no. 12, 2014, pp. 1784-91.
Yoshida N, Kobayashi R, Yabe H, et al. First-line treatment for severe aplastic anemia in children: bone marrow transplantation from a matched family donor versus immunosuppressive therapy. Haematologica. 2014;99(12):1784-91.
Yoshida, N., Kobayashi, R., Yabe, H., Kosaka, Y., Yagasaki, H., Watanabe, K., Kudo, K., Morimoto, A., Ohga, S., Muramatsu, H., Takahashi, Y., Kato, K., Suzuki, R., Ohara, A., & Kojima, S. (2014). First-line treatment for severe aplastic anemia in children: bone marrow transplantation from a matched family donor versus immunosuppressive therapy. Haematologica, 99(12), 1784-91. https://doi.org/10.3324/haematol.2014.109355
Yoshida N, et al. First-line Treatment for Severe Aplastic Anemia in Children: Bone Marrow Transplantation From a Matched Family Donor Versus Immunosuppressive Therapy. Haematologica. 2014;99(12):1784-91. PubMed PMID: 25193958.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - First-line treatment for severe aplastic anemia in children: bone marrow transplantation from a matched family donor versus immunosuppressive therapy. AU - Yoshida,Nao, AU - Kobayashi,Ryoji, AU - Yabe,Hiromasa, AU - Kosaka,Yoshiyuki, AU - Yagasaki,Hiroshi, AU - Watanabe,Ken-Ichiro, AU - Kudo,Kazuko, AU - Morimoto,Akira, AU - Ohga,Shouichi, AU - Muramatsu,Hideki, AU - Takahashi,Yoshiyuki, AU - Kato,Koji, AU - Suzuki,Ritsuro, AU - Ohara,Akira, AU - Kojima,Seiji, Y1 - 2014/09/05/ PY - 2014/9/7/entrez PY - 2014/9/7/pubmed PY - 2015/10/16/medline SP - 1784 EP - 91 JF - Haematologica JO - Haematologica VL - 99 IS - 12 N2 - 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) versus 92% (90-94)], failure-free survival was significantly inferior in patients receiving immunosuppressive therapy than in those undergoing bone marrow transplantation [56% (54-59) versus 87% (85-90); P<0.0001]. There was no significant improvement in outcomes over the two time periods (1992-1999 versus 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. SN - 1592-8721 UR - https://www.unboundmedicine.com/medline/citation/25193958/First_line_treatment_for_severe_aplastic_anemia_in_children:_bone_marrow_transplantation_from_a_matched_family_donor_versus_immunosuppressive_therapy_ L2 - https://doi.org/10.3324/haematol.2014.109355 DB - PRIME DP - Unbound Medicine ER -