移植后环磷酰胺对儿童急性白血病患者行非亲缘单份脐带血移植结局的影响。
Impact of posttransplant cyclophosphamide on the outcome of patients undergoing unrelated single-unit umbilical cord blood transplantation for pediatric acute leukemia.
机构信息
Department of Pediatrics, Sun Yat-Sen Memorial Hospital, Sun Yat-Sen University, Guangzhou, 510120, China.
Guangdong Provincial Key Laboratory of Malignant Tumor Epigenetics and Gene Regulation, Sun Yat-Sen Memorial Hospital, Sun Yat-Sen University, Guangzhou, 510120, China.
出版信息
BMC Cancer. 2022 Nov 18;22(1):1190. doi: 10.1186/s12885-022-10309-9.
BACKGROUND
Umbilical cord blood transplantation (UCBT) from unrelated donors is one of the successful treatments for acute leukemia in childhood. The most frequent side effect of UCBT is peri-engraftment syndrome (PES), which is directly associated with the greater prevalence of acute and chronic graft-versus-host-disease (aGvHD and cGvHD). In haploidentical stem cell transplantation, posttransplant cyclophosphamide (PTCY) has been demonstrated to be an effective method against GvHD. However, the effects of PTCY as a GvHD prophylactic in UCBT had not been investigated. This study aimed to evaluate the effects of PTCY on the outcomes of UCBT for pediatric acute leukemia.
METHODS
This retrospective study included 52 children with acute leukemia who underwent unrelated single-unit UCBT after myeloablative conditioning regimens. The results from the PTCY and non-PTCY groups were compared.
RESULTS
The incidence of transplantation-related mortality in non-PTCY and PTCY were 5% and 10% (p = 0.525), respectively. The incidence of relapse in non-PTCY and PTCY were 5% and 23% (p = 0.095), respectively. Second complete remission status (CR2) was an independent risk factor for relapse-free survival (hazard ratio = 9.782, p = 0.001). The odds ratio for sepsis or bacteremia incidence was significantly greater in the PTCY group (9.524, p = 0.017). PTCY group had increased rates of cytomegalovirus activity and fungal infection. The incidence of PES, aGvHD, cGvHD, and hemorrhagic cystitis in the PTCY group was lower than that in the non-PTCY group, although it was not significantly different. Additionally, higher doses of PTCY (29 mg/kg and 40 mg/kg) were associated with lower incidences of aGvHD and severe GvHD (65% and 29%, respectively) than lower doses (93% and 57%, respectively). Engraftment time and graft failure incidence were similar across groups.
CONCLUSION
The results support the safety and efficiency of PTCY as part of PES controlling and GvHD prophylaxis in single-unit UCBT for children with acute leukemia. A PTCY dosage of 29 mg/kg to 40 mg/kg appears to be more effective in GvHD prophylaxis for UCBT patients.
背景
非血缘脐带血移植（UCBT）是儿童急性白血病的成功治疗方法之一。UCBT 最常见的副作用是植入前综合征（PES），这与急性和慢性移植物抗宿主病（aGvHD 和 cGvHD）的更高发生率直接相关。在单倍体造血干细胞移植中，移植后环磷酰胺（PTCY）已被证明是一种对抗 GvHD 的有效方法。然而，PTCY 作为 UCBT 中 GvHD 预防药物的效果尚未得到研究。本研究旨在评估 PTCY 对儿童急性白血病 UCBT 结果的影响。
方法
本回顾性研究纳入了 52 例接受清髓性预处理方案后行非血缘单份 UCBT 的急性白血病患儿。比较了 PTCY 组和非 PTCY 组的结果。
结果
非 PTCY 和 PTCY 组的移植相关死亡率分别为 5%和 10%（p=0.525）。非 PTCY 和 PTCY 组的复发率分别为 5%和 23%（p=0.095）。第二次完全缓解状态（CR2）是无复发生存的独立危险因素（危险比=9.782，p=0.001）。PTCY 组败血症或菌血症的发生率明显更高（优势比=9.524，p=0.017）。PTCY 组巨细胞病毒活性和真菌感染的发生率增加。PTCY 组 PES、aGvHD、cGvHD 和出血性膀胱炎的发生率低于非 PTCY 组，但差异无统计学意义。此外，较高剂量的 PTCY（29mg/kg 和 40mg/kg）与较低剂量的 PTCY（65%和 29%）相比，aGvHD 和重度 GvHD 的发生率较低（分别为 93%和 57%）。各组的植入时间和移植物失败发生率相似。
结论
结果支持 PTCY 作为 PES 控制和儿童急性白血病 UCBT 中 GvHD 预防的一部分的安全性和有效性。PTCY 剂量为 29mg/kg 至 40mg/kg 似乎在 UCBT 患者的 GvHD 预防中更有效。
Zotero 插件