Prognostic Factors Associated With Increased Mortality in Pediatric Veno-Occlusive Disease Following Hematopoietic Cell Transplantation

IF 1.9 4区医学 Q2 SURGERY Clinical Transplantation Pub Date : 2024-11-27 DOI:10.1111/ctr.70037

Irina Zaidman, Natalie Barsoum, Ehud Even-Or, Miriam Daher, Adi Avniel Aran, Polina Stepensky, Aharon Gefen

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Abstract

Background

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.

Objective

To evaluate the incidence of VOD in children after HCT and analyze the outcomes and risk factors associated with increased mortality.

Study Design

A retrospective cohort study of 1243 children with malignant and non-malignant diseases who underwent HCT at two large pediatric centers over 20 years.

Results

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).

Conclusions

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.

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与小儿造血细胞移植后静脉闭塞症死亡率增加相关的预后因素

背景肝静脉闭塞症（VOD）是造血细胞移植（HCT）的一种危及生命的并发症，被归类为移植相关的全身性内皮疾病。严重的 VOD 可导致多器官功能障碍 (MOF)，并与高死亡率相关。目的评估儿童 HCT 后 VOD 的发病率，并分析与死亡率增加相关的结果和风险因素。研究设计对在两家大型儿科中心接受 HCT 的 1243 名患有恶性和非恶性疾病的儿童进行回顾性队列研究，历时 20 年。结果 101名患者（8%）在接受 HCT 后出现 VOD。大多数患者在接受异体造血干细胞移植（76%）和自体造血干细胞移植（24%）后出现 VOD。恶性疾病患儿的 VOD 发生率是非恶性疾病患儿的两倍（68% 对 32%）。以丁胺磺胺为基础的治疗方案与以全身照射为基础的治疗方案和以曲硫磺为基础的治疗方案相比，VOD发生率要高得多，分别为73%、18%和1%。有 VOD 的 HCT 患者的 100 天存活率为 69%。整组患者的总生存率为50%，在研究期间有所提高，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）、调理方案（p = 0.01）、肝功能异常（p = 0.019）、腹水（p = 0.008）、MOF（p < 0.001）和需要入住儿科重症监护室（p < 0.001）。单独使用去纤维化药物治疗的患儿与使用去纤维化药物和类固醇治疗的患儿在存活率上没有明显差异（分别为61%和65%；P = 0.685）。结论 HCT 后儿童患者的严重 VOD 仍是一种危及生命的并发症，死亡率很高。早期诊断和使用去纤维化药物治疗对控制病情至关重要。在我们的队列中，在使用去纤肽的同时使用类固醇与改善预后无关。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Clinical Transplantation 医学-外科

CiteScore

3.70

自引率

4.80%

发文量

286

审稿时长

2 months

期刊介绍： Clinical Transplantation: The Journal of Clinical and Translational Research aims to serve as a channel of rapid communication for all those involved in the care of patients who require, or have had, organ or tissue transplants, including: kidney, intestine, liver, pancreas, islets, heart, heart valves, lung, bone marrow, cornea, skin, bone, and cartilage, viable or stored. Published monthly, Clinical Transplantation’s scope is focused on the complete spectrum of present transplant therapies, as well as also those that are experimental or may become possible in future. Topics include: Immunology and immunosuppression; Patient preparation; Social, ethical, and psychological issues; Complications, short- and long-term results; Artificial organs; Donation and preservation of organ and tissue; Translational studies; Advances in tissue typing; Updates on transplant pathology;. Clinical and translational studies are particularly welcome, as well as focused reviews. Full-length papers and short communications are invited. Clinical reviews are encouraged, as well as seminal papers in basic science which might lead to immediate clinical application. Prominence is regularly given to the results of cooperative surveys conducted by the organ and tissue transplant registries. Clinical Transplantation: The Journal of Clinical and Translational Research is essential reading for clinicians and researchers in the diverse field of transplantation: surgeons; clinical immunologists; cryobiologists; hematologists; gastroenterologists; hepatologists; pulmonologists; nephrologists; cardiologists; and endocrinologists. It will also be of interest to sociologists, psychologists, research workers, and to all health professionals whose combined efforts will improve the prognosis of transplant recipients.