Controlled, Randomized, Open-Label Trial of Risk-Stratified Corticosteroid Prevention of Acute Graft-Versus-Host Disease After Haploidentical Transplantation.
This study evaluated whether a prophylaxis strategy directed by the graft-versus-host disease (GVHD) biomarker might reduce the 100-day incidence of acute GVHD grades II to IV.



This controlled, open-label, randomized trial included 228 patients who underwent haploidentical transplantation. On the basis of bone marrow allogeneic graft CD4:CD8 ratios, patients were categorized as low risk (n = 83; group A) or high risk (n = 145). Patients at high risk were randomly assigned to either receive (n = 72; group B) or not receive (n = 73; group C) low-dose corticosteroid prophylaxis.



The incidence in group B was 21% (95% CI, 11% to 31%) compared with 26% (95% CI, 16%to 36%; P = .43) in group A and 48% (95% CI, 32% to 60%; P < .001) in group C. Low-dose corticosteroid prophylaxis was significantly associated with a relatively low risk of acute GVHD grades II to IV (hazard ratio, 0.66; 95% CI, 0.49 to 0.89; P = .007) and rapid platelet recovery (hazard ratio, 0.30; 95% CI, 0.23 to 0.47; P < .001). The incidence of moderate-to-severe chronic GVHD in group B (21%) was lower than that in both group A (50%; P = .025) and group C (36%; P = .066). The 100-day corticosteroid doses were 205 ± 111 mg in group B, 229 ± 149 mg in group A (P = .256), and 286.54 ± 259.67 mg in group C (P = .016). Compared with group C, group B showed significantly lower incidences of femoral head necrosis (P = .034) and hypertension (P = .015). Infection rates were comparable among these groups.



Our results suggest that risk stratification-directed, low-dose corticosteroid prophylaxis significantly decreased the incidence of acute GVHD grades II to IV, accelerated platelet recovery, and reduced adverse events without increasing infections.
Ying-Jun Chang, Lan-Ping Xu, Yu Wang, Xiao-Hui Zhang, Huan Chen, Yu-Hong Chen, Feng-Rong Wang, Wei Han, Yu-Qian Sun, Chen-Hua Yan, Fei-Fei Tang, Xiao-Dong Mo, Kai-Yan Liu, and Xiao-Jun Huang, Peking University People's Hospital and Peking University Institute of Hematology; Xiao-Jun Huang, Peking-Tsinghua Center for Life Sciences; and Ying-Jun Chang and Xiao-Jun Huang, Collaborative Innovation Center of Hematology, Peking University, Beijing, People's Republic of China.
Ying-Jun
YJ
Chang
Ying-Jun Chang, Lan-Ping Xu, Yu Wang, Xiao-Hui Zhang, Huan Chen, Yu-Hong Chen, Feng-Rong Wang, Wei Han, Yu-Qian Sun, Chen-Hua Yan, Fei-Fei Tang, Xiao-Dong Mo, Kai-Yan Liu, and Xiao-Jun Huang, Peking University People's Hospital and Peking University Institute of Hematology; Xiao-Jun Huang, Peking-Tsinghua Center for Life Sciences; and Ying-Jun Chang and Xiao-Jun Huang, Collaborative Innovation Center of Hematology, Peking University, Beijing, People's Republic of China.
Lan-Ping
LP
Xu
Ying-Jun Chang, Lan-Ping Xu, Yu Wang, Xiao-Hui Zhang, Huan Chen, Yu-Hong Chen, Feng-Rong Wang, Wei Han, Yu-Qian Sun, Chen-Hua Yan, Fei-Fei Tang, Xiao-Dong Mo, Kai-Yan Liu, and Xiao-Jun Huang, Peking University People's Hospital and Peking University Institute of Hematology; Xiao-Jun Huang, Peking-Tsinghua Center for Life Sciences; and Ying-Jun Chang and Xiao-Jun Huang, Collaborative Innovation Center of Hematology, Peking University, Beijing, People's Republic of China.
Yu
Y
Wang
Ying-Jun Chang, Lan-Ping Xu, Yu Wang, Xiao-Hui Zhang, Huan Chen, Yu-Hong Chen, Feng-Rong Wang, Wei Han, Yu-Qian Sun, Chen-Hua Yan, Fei-Fei Tang, Xiao-Dong Mo, Kai-Yan Liu, and Xiao-Jun Huang, Peking University People's Hospital and Peking University Institute of Hematology; Xiao-Jun Huang, Peking-Tsinghua Center for Life Sciences; and Ying-Jun Chang and Xiao-Jun Huang, Collaborative Innovation Center of Hematology, Peking University, Beijing, People's Republic of China.
Xiao-Hui
XH
Zhang
Ying-Jun Chang, Lan-Ping Xu, Yu Wang, Xiao-Hui Zhang, Huan Chen, Yu-Hong Chen, Feng-Rong Wang, Wei Han, Yu-Qian Sun, Chen-Hua Yan, Fei-Fei Tang, Xiao-Dong Mo, Kai-Yan Liu, and Xiao-Jun Huang, Peking University People's Hospital and Peking University Institute of Hematology; Xiao-Jun Huang, Peking-Tsinghua Center for Life Sciences; and Ying-Jun Chang and Xiao-Jun Huang, Collaborative Innovation Center of Hematology, Peking University, Beijing, People's Republic of China.
Huan
H
Chen
Ying-Jun Chang, Lan-Ping Xu, Yu Wang, Xiao-Hui Zhang, Huan Chen, Yu-Hong Chen, Feng-Rong Wang, Wei Han, Yu-Qian Sun, Chen-Hua Yan, Fei-Fei Tang, Xiao-Dong Mo, Kai-Yan Liu, and Xiao-Jun Huang, Peking University People's Hospital and Peking University Institute of Hematology; Xiao-Jun Huang, Peking-Tsinghua Center for Life Sciences; and Ying-Jun Chang and Xiao-Jun Huang, Collaborative Innovation Center of Hematology, Peking University, Beijing, People's Republic of China.
Yu-Hong
YH
Chen
Ying-Jun Chang, Lan-Ping Xu, Yu Wang, Xiao-Hui Zhang, Huan Chen, Yu-Hong Chen, Feng-Rong Wang, Wei Han, Yu-Qian Sun, Chen-Hua Yan, Fei-Fei Tang, Xiao-Dong Mo, Kai-Yan Liu, and Xiao-Jun Huang, Peking University People's Hospital and Peking University Institute of Hematology; Xiao-Jun Huang, Peking-Tsinghua Center for Life Sciences; and Ying-Jun Chang and Xiao-Jun Huang, Collaborative Innovation Center of Hematology, Peking University, Beijing, People's Republic of China.
Feng-Rong
FR
Wang
Ying-Jun Chang, Lan-Ping Xu, Yu Wang, Xiao-Hui Zhang, Huan Chen, Yu-Hong Chen, Feng-Rong Wang, Wei Han, Yu-Qian Sun, Chen-Hua Yan, Fei-Fei Tang, Xiao-Dong Mo, Kai-Yan Liu, and Xiao-Jun Huang, Peking University People's Hospital and Peking University Institute of Hematology; Xiao-Jun Huang, Peking-Tsinghua Center for Life Sciences; and Ying-Jun Chang and Xiao-Jun Huang, Collaborative Innovation Center of Hematology, Peking University, Beijing, People's Republic of China.
Wei
W
Han
Ying-Jun Chang, Lan-Ping Xu, Yu Wang, Xiao-Hui Zhang, Huan Chen, Yu-Hong Chen, Feng-Rong Wang, Wei Han, Yu-Qian Sun, Chen-Hua Yan, Fei-Fei Tang, Xiao-Dong Mo, Kai-Yan Liu, and Xiao-Jun Huang, Peking University People's Hospital and Peking University Institute of Hematology; Xiao-Jun Huang, Peking-Tsinghua Center for Life Sciences; and Ying-Jun Chang and Xiao-Jun Huang, Collaborative Innovation Center of Hematology, Peking University, Beijing, People's Republic of China.
Yu-Qian
YQ
Sun
Ying-Jun Chang, Lan-Ping Xu, Yu Wang, Xiao-Hui Zhang, Huan Chen, Yu-Hong Chen, Feng-Rong Wang, Wei Han, Yu-Qian Sun, Chen-Hua Yan, Fei-Fei Tang, Xiao-Dong Mo, Kai-Yan Liu, and Xiao-Jun Huang, Peking University People's Hospital and Peking University Institute of Hematology; Xiao-Jun Huang, Peking-Tsinghua Center for Life Sciences; and Ying-Jun Chang and Xiao-Jun Huang, Collaborative Innovation Center of Hematology, Peking University, Beijing, People's Republic of China.
Chen-Hua
CH
Yan
Ying-Jun Chang, Lan-Ping Xu, Yu Wang, Xiao-Hui Zhang, Huan Chen, Yu-Hong Chen, Feng-Rong Wang, Wei Han, Yu-Qian Sun, Chen-Hua Yan, Fei-Fei Tang, Xiao-Dong Mo, Kai-Yan Liu, and Xiao-Jun Huang, Peking University People's Hospital and Peking University Institute of Hematology; Xiao-Jun Huang, Peking-Tsinghua Center for Life Sciences; and Ying-Jun Chang and Xiao-Jun Huang, Collaborative Innovation Center of Hematology, Peking University, Beijing, People's Republic of China.
Fei-Fei
FF
Tang
Ying-Jun Chang, Lan-Ping Xu, Yu Wang, Xiao-Hui Zhang, Huan Chen, Yu-Hong Chen, Feng-Rong Wang, Wei Han, Yu-Qian Sun, Chen-Hua Yan, Fei-Fei Tang, Xiao-Dong Mo, Kai-Yan Liu, and Xiao-Jun Huang, Peking University People's Hospital and Peking University Institute of Hematology; Xiao-Jun Huang, Peking-Tsinghua Center for Life Sciences; and Ying-Jun Chang and Xiao-Jun Huang, Collaborative Innovation Center of Hematology, Peking University, Beijing, People's Republic of China.
Xiao-Dong
XD
Mo
Ying-Jun Chang, Lan-Ping Xu, Yu Wang, Xiao-Hui Zhang, Huan Chen, Yu-Hong Chen, Feng-Rong Wang, Wei Han, Yu-Qian Sun, Chen-Hua Yan, Fei-Fei Tang, Xiao-Dong Mo, Kai-Yan Liu, and Xiao-Jun Huang, Peking University People's Hospital and Peking University Institute of Hematology; Xiao-Jun Huang, Peking-Tsinghua Center for Life Sciences; and Ying-Jun Chang and Xiao-Jun Huang, Collaborative Innovation Center of Hematology, Peking University, Beijing, People's Republic of China.
Kai-Yan
KY
Liu
Ying-Jun Chang, Lan-Ping Xu, Yu Wang, Xiao-Hui Zhang, Huan Chen, Yu-Hong Chen, Feng-Rong Wang, Wei Han, Yu-Qian Sun, Chen-Hua Yan, Fei-Fei Tang, Xiao-Dong Mo, Kai-Yan Liu, and Xiao-Jun Huang, Peking University People's Hospital and Peking University Institute of Hematology; Xiao-Jun Huang, Peking-Tsinghua Center for Life Sciences; and Ying-Jun Chang and Xiao-Jun Huang, Collaborative Innovation Center of Hematology, Peking University, Beijing, People's Republic of China. xjhrm@medmail.com.cn.
Xiao-Jun
XJ
Huang
D000208:Acute Disease; D000293:Adolescent; D000305:Adrenal Cortex Hormones / Q000627:therapeutic use*; D000328:Adult; D016026:Bone Marrow Transplantation / Q000009:adverse effects*; D005260:Female; D006086:Graft vs Host Disease / Q000517:prevention & control*; D006410:Hematopoiesis; D006801:Humans; D008297:Male; D008875:Middle Aged; D014184:Transplantation, Homologous
D000305:Adrenal Cortex Hormones