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对异基因干细胞移植后静脉闭塞性疾病/窦性阻塞综合征(VOD/SOS)进行系统筛查和重点评估,有助于早期诊断并及时开始使用去纤苷治疗。

Systematic screening and focused evaluation for veno-occlusive disease/sinusoidal obstructive syndrome (VOD/SOS) following allogeneic stem cell transplant is associated with earlier diagnosis and prompt institution of defibrotide treatment.

作者信息

Avenoso Daniele, Kenyon Michelle, Mehra Varun, Krishnamurthy Pramila, Kulasekararaj Austin, Gandhi Shreyans, Dazzi Francesco, Naresh Shah Mili, Wood Henry, Leung Ye Ting, Eaton Alicia, Anteh Sandra, Cuadrado Maria, Correia de Farias Madson, Bourlon Christienne, Dragoi Diana Oana, Hardefeldt Prudence, Pagliuca Antonio, Potter Victoria

机构信息

Department of Haematological Medicine, King's College Hospital NHS Foundation Trust, London, United Kingdom.

出版信息

Front Transplant. 2022 Nov 8;1:996003. doi: 10.3389/frtra.2022.996003. eCollection 2022.

DOI:10.3389/frtra.2022.996003
PMID:38994385
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC11235283/
Abstract

Sinusoidal obstructive syndrome (SOS), also known as hepatic veno-occlusive disease (VOD), is a potentially life-threatening complication following haemopoietic stem cell transplantation (HSCT). The availability of new drugs for malignant hematological conditions has allowed more patients to be eligible for allogeneic haematopoietic stem cell transplants, which has translated into a significant proportion of transplant patients having multiple risk factors for VOD/SOS. Based on these considerations, we undertook a dedicated weekly VOD/SOS ward round, aiming to facilitate early diagnosis of VOD/SOS and pre-emptively identify patients at risk, where a careful evaluation of differential diagnosis is essential. Herein, we present the results of our VOD/SOS ward round; between September 2020 and April 2022, 110 consecutive patients were evaluated in a focused VOD/SOS ward round. From the 110 patients, 108 had undergone HSCT and had at least one known risk factor for developing VOD/SOS. The median number of risk factors present in the VOD/SOS group and non-VOD/SOS group was five (range: three to six) and three (range: zero to seven), respectively. Late-onset VOD/SOS was diagnosed in 45% of our patients. The early identification of patients with multiple risk factors for VOD/SOS allowed an earlier diagnosis and the administration of defibrotide on the same day of diagnosis, which was two days earlier than our previous experience prior to the implementation of this protocol.

摘要

窦性阻塞综合征(SOS),也称为肝静脉闭塞病(VOD),是造血干细胞移植(HSCT)后一种潜在的危及生命的并发症。用于恶性血液系统疾病的新药的出现使更多患者有资格接受异基因造血干细胞移植,这导致相当一部分移植患者具有发生VOD/SOS的多种危险因素。基于这些考虑,我们专门开展了每周一次的VOD/SOS病房查房,旨在促进VOD/SOS的早期诊断并预先识别有风险的患者,在此过程中对鉴别诊断进行仔细评估至关重要。在此,我们展示我们VOD/SOS病房查房的结果;在2020年9月至2022年4月期间,在一次重点VOD/SOS病房查房中对110例连续患者进行了评估。在这110例患者中,108例接受了HSCT,并且至少有一个已知的发生VOD/SOS的危险因素。VOD/SOS组和非VOD/SOS组中存在的危险因素的中位数分别为5个(范围:3至6个)和3个(范围:0至7个)。45%的患者被诊断为迟发性VOD/SOS。对具有VOD/SOS多种危险因素的患者进行早期识别,使得能够更早诊断并在诊断当天给予去纤苷,这比我们在实施该方案之前的经验提前了两天。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/14a3/11235283/d982904b4e74/frtra-01-996003-g0001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/14a3/11235283/d982904b4e74/frtra-01-996003-g0001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/14a3/11235283/d982904b4e74/frtra-01-996003-g0001.jpg

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Endothelial cell dysfunction: a key determinant for the outcome of allogeneic stem cell transplantation.内皮细胞功能障碍:异基因干细胞移植结局的关键决定因素。
Bone Marrow Transplant. 2021 Oct;56(10):2326-2335. doi: 10.1038/s41409-021-01390-y. Epub 2021 Jul 12.
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A multicentre, multinational, prospective, observational registry study of defibrotide in patients diagnosed with veno-occlusive disease/sinusoidal obstruction syndrome after haematopoietic cell transplantation: an EBMT study.一项多中心、多国、前瞻性、观察性登记研究,评估在造血细胞移植后诊断为静脉闭塞病/肝窦阻塞综合征的患者中使用地塞米松的疗效:一项 EBMT 研究。
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