PTCY 50 mg/kg/day on days +3/+4 is an excellent strategy to prevent GVHD. However, its use is associated with adverse outcomes such as delayed engraftment, increased risk of infection, and cardiac complications. This pilot study evaluates the efficacy and toxicity of a reduced dose of PTCY (40 mg/kg/day) combined with tacrolimus in 22 peripheral blood HLA-matched alloHSCT patients. At day +100, the cumulative incidences of grade II–IV and III–IV acute GVHD were 18.2% and 4.5%, respectively. No grade IV acute GVHD or steroid-refractory disease was observed. The cumulative incidences of all-grade and moderate-severe chronic GVHD at 1-year were 11.4% and 6.4%, respectively. No patient died from transplant-related complications. Two-year OS and RFS were 77.1% and 58.3%, respectively. All patients engrafted, with neutrophil and platelet recovery occurring at a median of 15 (IQR 14–16) and 16 days (IQR 12–23), respectively. The cumulative incidences of bloodstream bacterial infections, polyomavirus BK hemorrhagic cystitis, HHV6 reactivation, CMV reactivation, and fungal infections were 13.6%, 9.1%, 9.1%, 4.6%, and 6%, respectively. Only one early cardiac event was observed. These results suggest that PTCY 40 mg/kg/day on a +3/+4 schedule provides adequate immunosuppression to allow for engraftment and prevent clinically significant GVHD with a low toxicity profile.
在移植后第+3/+4天使用50 mg/kg/天的PTCY是预防移植物抗宿主病（GVHD）的优良策略，但其应用常伴随移植物延迟植入、感染风险增加及心脏并发症等不良结局。本项先导研究评估了22例外周血HLA相合同种异基因造血干细胞移植患者中，降低剂量的PTCY（40 mg/kg/天）联合他克莫司方案的疗效与毒性。至移植后第+100天，II–IV级与III–IV级急性GVHD累积发生率分别为18.2%和4.5%，未观察到IV级急性GVHD或激素难治性疾病。1年时全级别与中重度慢性GVHD累积发生率分别为11.4%和6.4%。无患者死于移植相关并发症。2年总生存率与无复发生存率分别为77.1%和58.3%。所有患者均成功植入，中性粒细胞与血小板恢复的中位时间分别为15天（四分位距14-16天）和16天（四分位距12-23天）。血流细菌感染、多瘤病毒BK出血性膀胱炎、HHV6再激活、CMV再激活及真菌感染的累积发生率分别为13.6%、9.1%、9.1%、4.6%和6%。仅观察到1例早期心脏事件。这些结果表明，采用+3/+4给药方案的40 mg/kg/天PTCY能提供足够的免疫抑制，在实现移植物植入的同时有效预防具有临床意义的GVHD，且具有较低的毒性特征。
肿瘤(癌症)患者之家