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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.
CLINICAL TRIALS AND OBSERVATIONS Treatment of severe aplastic anemia with antithymocyte globulin and cyclosporin A with or without G-CSF in adults: a multicenter randomized study in Japan1 Department of Hematology, Tokyo Women's Medical University, Tokyo; 2 Department of Hematology and Oncology, Research Institute for Radiation Biology and Medicine, Hiroshima University, Hiroshima; 3 Department of Hematology/Oncology, Aoto Hospital, Tokyo; 4 Blood Transfusion Service, Kumamoto University School of Medicine, Kumamoto; 5 Cellular Transplantation Biology, Division of Cancer Medicine, Kanazawa University Graduate School of Medical Science, Kanazawa; 6 Division of Hematology, Nippon Telegraph and Telephone (NTT) Kanto Medical Center, Tokyo; 7 Division of Hematology, Internal Medicine, Showa University Fujigaoka Hospital, Yokohama, Japan 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. A total of 101 previously untreated patients (median age, 54 years; range, 19 to 75 years) 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 hematologic 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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