Objective: To investigate the impact of mycophenolate mofetil (MMF) prophylaxis duration on acute graft-versus-host disease (aGVHD) after haploidentical stem cell transplantation (haplo-HSCT) using 'Beijing Protocol'. Methods: Adult patients (≥14 years) received haplo-HSCT in Peking University Institute of Hematology from Sep, 2016 to Mar, 2017 were retrospectively reviewed if they fulfilled the criterias: ①diagnosed with hematological maligancies; ②standard-risk status at haplo-HSCT. A total of 237 patients [including 102 patients with long MMF duration (defined as started on day -9 with 100 mg/d, adjusted to 500 mg/d from day +30 and discontinued on day +45 to +60 or occurrence of CMV/EBV reactivation or late-onset hemorrhagic cytitis), and 135 patients with short MMF duration (defined as started on day -9 with 500 mg/d and discontinued on the day achieved neutrophil engraftment)] were reviewed. The incidence of aGVHD, virus infection and overall survival (OS) were compared between the two groups. Results: The median durations of MMF prophylaxis of long and short duration groups were 27(7-71) and 15(9-24) days, respectively after haplo-HSCT. There were no differences of baseline characteristics (including sex, patient age, disease, mismatched HLA loci, donor-recipient relation, donor-recipient sex and donor age) between the two groups. The incidences of the grade Ⅱ-Ⅳ and Ⅲ/Ⅳ aGVHD in long and short duration groups were 31.1% versus 17.6% (P=0.018) and 7.4% verus 7.8% (P=0.900), respectively. The duration of MMF prophylaxis was not found to be associated with gradeⅡ-Ⅳ aGVHD by the multivariate analysis. There were no significant differences in terms of CMV viremia, EBV viremia, hemorrhagic cytitis and OS between the two groups. Conclusion: Prophylaxis with short duration MMF in the setting of 'Beijing protocol' haplo-SCT was not associated with increased acute GVHD with no impact on OS, which indicated that short duration MMF might be a feasible GVHD prophylaxis regimen.
目的： 分析霉酚酸酯（MMF）预防疗程对于"北京方案"单倍体相合造血干细胞移植（haplo-HSCT）后急性移植物抗宿主病（GVHD）的影响。 方法： 回顾性分析2016年9月至2017年3月在北京大学血液病研究所接受haplo-HSCT的237例标危成人（≥14岁）血液肿瘤患者，男140例（59.1%），女97例（40.9%），中位年龄30（14~60）岁。根据MMF疗程分为长疗程组（MMF 500 mg/d口服，-9 d开始，+30 d减半，+45~+60 d停药，合并CMV血症、EBV血症或迟发出血性膀胱炎者提前停用）和短疗程组（MMF 500 mg/d口服，-9 d开始，中性粒细胞植活后停药）。 结果： 长疗程组（102例）、短疗程组（135例）MMF中位用药时间分别为27（7~71）、15（9~24）d。两组患者在性别、年龄、疾病类型、HLA不合位点、供受者关系、供受者血型、供者性别、供者年龄方面差异均无统计学意义。长疗程组、短疗程组Ⅱ~Ⅳ度急性GVHD发生率分别为31.1%、17.6%（P=0.018）、Ⅲ/Ⅳ度急性GVHD发生率分别为7.4%、7.8%（P=0.900）。多因素分析未发现MMF预防疗程长短对Ⅱ~Ⅳ度急性GVHD有显著影响。两组CMV血症、EBV血症、出血性膀胱炎发生率及总生存率差异均无统计学意义（P值均>0.05）。 结论： 短疗程MMF预防并未增加"北京方案"haplo-HSCT患者急性GVHD发生率，对总生存亦无显著影响，可能是一种安全可行的方案。.
Keywords: Graft vs host disease; Hematopoietic stem cell transplantation; Mycophenolate mofetil.