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熊去氧胆酸和低剂量肝素预防成人异基因干细胞移植后肝窦阻塞综合征/静脉闭塞性疾病的发生率低。

Low Incidence of hepatic sinusoidal obstruction syndrome/veno-occlusive disease in adults undergoing allogenic stem cell transplantation with prophylactic ursodiol and low-dose heparin.

机构信息

Division of Hematology, University Hospital Basel,, Basel, Switzerland.

University of Basel, Basel, Switzerland.

出版信息

Bone Marrow Transplant. 2022 Mar;57(3):391-398. doi: 10.1038/s41409-021-01546-w. Epub 2022 Jan 4.

Abstract

Hepatic sinusoidal obstruction syndrome (SOS)/veno-occlusive disease (VOD) is a complication after allogenic hematopoietic stem-cell transplantation (allo-HSCT) with high mortality. The purpose of this study was to assess the incidence and outcome of SOS in patients after allo-HSCT with the impact of ursodeoxycholic acid (UDCA) and low-dose heparin as SOS prophylaxis. Out of 1016 patients, 23 developed SOS, with a cumulative incidence of 2.3% (95% CI 1.3-3.3) 6 months after HSCT. Approximately one quarter of these patients (26.1%) had late-onset SOS. A high proportion were very severe SOS cases (74%), and 83% of the patients were treated with defibrotide (DF). In multivariate analysis, advanced disease (p = 0.003), previous HSCT (p = 0.025) and graft versus host disease (GvHD) prophylaxis by post-transplant cyclophosphamide (PTCy) (p = 0.055) were associated with the development of SOS. The 1-year overall survival (OS) was significantly lower in the SOS group compared to patients without SOS (13% versus 70%, p = 0.0001). In conclusion, we found a low incidence of SOS in patients receiving low-dose heparin and UDCA prophylactically, but among SOS patients, a high mortality. Low-dose heparin and UDCA might be a prophylactic approach for SOS.

摘要

肝窦阻塞综合征(SOS)/静脉阻塞病(VOD)是异基因造血干细胞移植(allo-HSCT)后的一种并发症,死亡率高。本研究旨在评估 allo-HSCT 后患者发生 SOS 的发生率和结局,以及熊去氧胆酸(UDCA)和低剂量肝素作为 SOS 预防的影响。在 1016 例患者中,有 23 例发生 SOS,HSCT 后 6 个月的累积发生率为 2.3%(95%CI 1.3-3.3)。这些患者中约有四分之一(26.1%)发生迟发性 SOS。很大一部分患者(74%)为严重 SOS 病例,83%的患者接受了地塞米松(DF)治疗。多变量分析显示,晚期疾病(p=0.003)、既往 HSCT(p=0.025)和移植后环磷酰胺(PTCy)预防移植物抗宿主病(GvHD)(p=0.055)与 SOS 的发生有关。与无 SOS 的患者相比,SOS 组的 1 年总生存率(OS)显著降低(13%比 70%,p=0.0001)。总之,我们发现接受低剂量肝素和 UDCA 预防性治疗的患者 SOS 发生率较低,但在 SOS 患者中死亡率较高。低剂量肝素和 UDCA 可能是 SOS 的一种预防方法。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/f587/8907064/69713111daf5/41409_2021_1546_Fig1_HTML.jpg

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