• Liping Wan, Yicheng Zhang, Yongrong Lai, Ming Jiang, Yongping Song, Jianfeng Zhou, Zhongming Zhang, Xianlin Duan, Yuewen Fu, Lianming Liao, and Chun Wang.
• Liping Wan and Chun Wang, Shanghai Jiao Tong University Affiliated First People's Hospital, Shanghai; Yicheng Zhang and Jianfeng Zhou, Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan; Yongrong Lai and Zhongming Zhang, First Affiliated Hospital of Guangxi Medical University, Nanning; Ming Jiang and Xianlin Duan, Hematologic Disease Center, the First Affiliated Hospital of Xinjiang Medical University, Urumqi; Yongping Song and Yuewen Fu, Henan Cancer Hospital, Zhengzhou; and Lianming Liao, Fujian Academy of Integrative Medicine, Fujian University of Traditional Chinese Medicine, Fuzhou, People's Republic of China.
• J. Clin. Oncol. 2015 Dec 1; 33 (34): 3999-4006.

PurposeFor recipients of allogeneic hematopoietic stem-cell transplantation (alloHSCT), we hypothesized that prophylactic therapy during neutropenia with granulocyte-macrophage colony-stimulating factor (GM-CSF) decreases invasive fungal disease (IFD).Patients And MethodsWe randomly assigned 206 patients undergoing alloHSCT to receive once-daily subcutaneous GM-CSF (5 to 7 μg/kg per day), granulocyte colony-stimulating factor (G-CSF; 5 to 7 μg/kg per day), or a combination of G-CSF and GM-CSF (2 to 3 μg/kg per day each). Treatment was started on day 5 after transplantation and was continued until the absolute neutrophil count was ≥ 1.5 × 10(9)/L for 2 consecutive days. The primary outcomes were 100-day incidence of proven and probable IFD and response rate of antifungal treatment.ResultsFor the intent-to-treat population, there was no significant difference in 100-day incidences of proven and probable IFD among the three groups. The antifungal treatment response was better in the GM-CSF group and G-CSF+GM-CSF group than in G-CSF group from day 22 to day 100 (P = .009). The 100-day cumulative mortality after transplantation was lower in the GM-CSF group than in the G-CSF group (10.3% v 24.6%, respectively; P = .037). The GM-CSF and G-CSF+GM-CSF groups had lower 100-day transplantation-related mortality than the G-CSF group (8.8%, 8.7%, and 21.7%, respectively; P = .034). After a median follow-up of 600 days, IFD-related mortality was lower in the groups that received GM-CSF or G-CSF+GM-CSF compared with G-CSF (1.47%, 1.45%, and 11.59%, respectively; P = .016). There were no significant differences in relapse, graft-versus-host disease, or hemorrhage-related mortality among the three groups of patients.ConclusionFor recipients of alloHSCT, compared with G-CSF, prophylactic GM-CSF was associated with lower 100-day transplantation-related mortality, lower 100-day cumulative mortality, and lower 600-day IFD-related mortality.© 2015 by American Society of Clinical Oncology.

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