Division of Hematology, University Hospital Basel,, Basel, Switzerland.
University of Basel, Basel, Switzerland.
Bone Marrow Transplant. 2022 Mar;57(3):391-398. doi: 10.1038/s41409-021-01546-w. Epub 2022 Jan 4.
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.
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。