Larue Marion, Labopin Myriam, Brissot Eolia, Alaskar Ahmed S, Aljurf Mahmoud, Arat Mutlu, Baron Frederic, Bazarbachi Ali, Ciceri Fabio, Corbacioglu Selim, Dignan Fiona L, Kenyon Michelle, Malard Florent, Nagler Arnon, Pagliuca Antonio, Ruggeri Annalisa, Yakoub-Agha Ibrahim, Ye Yishan, Duarte Rafael F, Ruutu Tapani, Carreras Enric, Peric Zinaida, Mohty Mohamad
Sorbonne Université, Centre de Recherche Saint-Antoine INSERM UMRs938, Service d'Hématologie Clinique et de Thérapie Cellulaire, Hôpital Saint Antoine, AP-HP, Paris, France.
King Abdullah International Medical Research Center, Ministry of National Guard Health Affairs, Riyadh, Saudi Arabia.
Bone Marrow Transplant. 2025 Jan;60(1):28-31. doi: 10.1038/s41409-024-02434-9. Epub 2024 Oct 7.
This international questionnaire survey aimed to explore the current incidence, diagnostic policies, management, and outcomes of veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS) among healthcare providers involved in the management of these patients. A questionnaire was e-mailed to practitioners with an interest in allogeneic hematopoietic cell transplantation (allo-HCT). Of the respondents, 144 of 215 (67.0%) felt that early detection or diagnosis of VOD/SOS was difficult. Regarding diagnostic criteria, 142 (66.1%) already declared using the 2023 EBMT refined criteria. Most respondents (163/215, 75.8%) found these recent refined EBMT criteria useful for diagnosis, and 193 (89.8%) found the severity criteria easy to use. The major risk factors identified for VOD/SOS were a second allo-HCT (41.4%), pre-existing liver disease (54.9%), and prior use of antibody-drug conjugates (49.8%). Preferences for starting VOD/SOS treatment varied, with 61 (28.4%) preferring initiating therapy at a mild stage, and 122 (56.7%) preferring the moderate stage. In summary, this survey provided valuable insight into the challenges and opportunities of the identification and management of VOD/SOS. By improving current knowledge and increasing collaboration among healthcare professionals, early detection, management, and clinical outcomes for patients with this potentially serious complication can also be improved.
这项国际问卷调查旨在探讨参与这些患者管理的医疗服务提供者中,静脉闭塞性疾病/窦性阻塞综合征(VOD/SOS)的当前发病率、诊断策略、管理方法及治疗结果。一份问卷通过电子邮件发送给了对异基因造血细胞移植(allo-HCT)感兴趣的从业者。在受访者中,215人中有144人(67.0%)认为VOD/SOS的早期检测或诊断困难。关于诊断标准,142人(66.1%)已声明使用2023年欧洲血液和骨髓移植学会(EBMT)完善的标准。大多数受访者(163/215,75.8%)认为这些最新完善的EBMT标准对诊断有用,193人(89.8%)认为严重程度标准易于使用。确定的VOD/SOS主要危险因素包括二次allo-HCT(41.4%)、既往存在的肝脏疾病(54.9%)和既往使用抗体药物偶联物(49.8%)。开始VOD/SOS治疗的偏好各不相同,61人(28.4%)倾向于在轻度阶段开始治疗,122人(56.7%)倾向于在中度阶段开始治疗。总之,这项调查为VOD/SOS的识别和管理所面临的挑战与机遇提供了宝贵见解。通过提高当前的知识水平并加强医疗专业人员之间的合作,这种潜在严重并发症患者的早期检测、管理及临床结果也能够得到改善。