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Blood, 15 September 2007, Vol. 110, No. 6, pp. 1756-1761.
Prepublished online as a Blood First Edition Paper on May 25, 2007; DOI 10.1182/blood-2006-11-050526.
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Submitted November 28, 2006
Accepted May 9, 2007
Treatment of severe aplastic anemia with antithymocyte globulin and cyclosporin A with or without G-CSF in adults; a multicenter randomized study in Japan
Masanao Teramura*, Akiro Kimura, Satsuki Iwase, Yuji Yonemura, Shinji Nakao, Akio Urabe, Mitsuhiro Omine, and Hideaki Mizoguchi
Department of Hematology, Tokyo Women's Medical University, Tokyo, Japan
Department of Hematology and Oncology, Research Institute for Radiation Biology and Medicine, Hiroshima University, Hiroshima, Japan
Department of Hematology/Oncology, Aoto Hospital, Tokyo, Japan
Blood transfusion Service, Kumamoto University school of Medicine, Kumamoto, Japan
Cellular Transplantation Biology, Division of Cancer Medicine, Kanazawa University Graduate School of Medical Science, Kanazawa, Japan
Division of Hematology, NTT Kanto Medical Center, Tokyo, Japan
Division of Hematology, Internal Medicine, Showa University Fujigaoka Hospital, Yokohama, Japan
* Corresponding author; email: teramura{at}dh.twmu.ac.jp.
We report the results of a randomized study to elucidate whether addition of granulocyte colony-stimulating factor (G-CSF) to immunosuppressive therapy is valuable for the treatment of severe aplastic anemia (SAA) in adults. One hundred and one previously untreated patients (median age; 54, range; 19 to 75) were randomized to receive antithymocyte globulin (ATG) and cyclosporin A (CyA) (G-CSF- group) or ATG, CyA and G-CSF (G-CSF+ group). In the G-CSF+ group, the hematological response rate at 6 months was higher (77% vs 57%; P=.03) than in the G-CSF- group. No differences were observed between the groups in terms of the incidence of infections and febrile episodes. There were no differences between the G-CSF- group and the G-CSF+ group in terms of survival (88% vs 94% at 4 years), and the development of myelodysplastic syndrome (MDS)/acute leukemia (AL) (1 patient vs 2 patients). However, the relapse rate was lower in the G-CSF+ group compared with the G-CSF- group (42% vs 15% at 4 years; P=.01). Further follow-up is required to elucidate the role of G-CSF in immunosuppressive therapy for adult SAA.

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