Faculty of Medicine, Hebrew University of Jerusalem, Jerusalem, Israel.
Department of Bone Marrow Transplantation and Cancer Immunotherapy, Hadassah-Hebrew University Medical Center, Jerusalem, Israel.
Clin Transplant. 2024 Dec;38(12):e70037. doi: 10.1111/ctr.70037.
Hepatic veno-occlusive disease (VOD) is a life-threatening complication of hematopoietic cell transplantation (HCT) and is categorized as a transplant-related, systemic endothelial disease. Severe VOD can lead to multi-organ dysfunction (MOF) and is associated with a high mortality rate.
To evaluate the incidence of VOD in children after HCT and analyze the outcomes and risk factors associated with increased mortality.
A retrospective cohort study of 1243 children with malignant and non-malignant diseases who underwent HCT at two large pediatric centers over 20 years.
One hundred one patients (8%) developed VOD post HCT. Most patients developed VOD post allogeneic HCT (76%) versus autologous (24%). The incidence of VOD was twice as high in children with malignant diseases compared to non-malignant (68% vs. 32%). A much higher incidence of VOD occurred in patients after a busulfan-based regimen versus total body irradiation-based and treosulfan-based, 73%, 18%, and 1%, respectively. The 100-day survival rate of HCT patients with VOD was 69%. The overall survival rate of the entire group was 50%, showing improvement over the span of the study years, from 40% between 2000 and 2009 to 63% between 2010 and 2021 (p = 0.022). Factors associated with increased mortality included infections before transplant (p = 0.013), conditioning regimen (p = 0.01), abnormal liver function (p = 0.019), presence of ascites (p = 0.008), MOF (p < 0.001), and the need for admission to a pediatric intensive care unit (p < 0.001). There was no significant difference in survival rates between children treated with defibrotide alone or with those treated with defibrotide and steroids (61% and 65%, respectively; p = 0.685).
Severe VOD in pediatric patients following HCT remains a life-threatening complication with a high mortality rate. Early diagnosis and treatment with defibrotide are critical for managing this condition. In our cohort, the addition of steroids to defibrotide was not associated with improved outcomes.
肝静脉闭塞病（VOD）是造血细胞移植（HCT）的一种危及生命的并发症，属于移植相关的系统性内皮疾病。严重的 VOD 可导致多器官功能障碍（MOF），并与高死亡率相关。
评估儿童 HCT 后 VOD 的发生率，并分析与死亡率增加相关的结局和危险因素。
对 20 年来在两个大型儿科中心接受 HCT 的 1243 例恶性和非恶性疾病儿童进行回顾性队列研究。
101 例（8%）患者在 HCT 后发生 VOD。大多数患者在接受异基因 HCT 后发生 VOD（76%），而非自体（24%）。与非恶性疾病相比，恶性疾病患者 VOD 的发生率几乎翻了一番（68%对 32%）。与基于全身照射和替莫唑胺的方案相比，基于白消安的方案发生 VOD 的发生率高得多，分别为 73%、18%和 1%。VOD 患儿 HCT 的 100 天生存率为 69%。整个组的总生存率为 50%，显示出随着研究年份的推移而有所改善，从 2000 年至 2009 年的 40%提高到 2010 年至 2021 年的 63%（p = 0.022）。与死亡率增加相关的因素包括移植前感染（p = 0.013）、预处理方案（p = 0.01）、肝功能异常（p = 0.019）、腹水存在（p = 0.008）、MOF（p < 0.001）和需要入住儿科重症监护病房（p < 0.001）。单独使用或联合使用地昔他滨和皮质类固醇治疗的患儿的生存率无显著差异（分别为 61%和 65%；p = 0.685）。
儿童 HCT 后严重的 VOD 仍然是一种危及生命的并发症，死亡率高。早期诊断和治疗用地昔他滨是管理这种疾病的关键。在我们的队列中，地昔他滨加用皮质类固醇并未改善结局。
