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    • Retrospective Analysis of Veno-Occlusive Disease/sinusoidal Obstruction Syndrome in Paediatric Patients Undergoing Hematopoietic Cell Transplantation -a Multicentre Study

    Dristhi Ragoonanan,Hisham Abdel-Azim,Aditya Sharma,Saleh Bhar,Jennifer Mcarthur,Renee Madden,April Rahrig,Rajinder Bajwa,Jian Wang,Victoria Sun,Mariah Wright,Rebekah Lassiter,Basirat Shoberu,Jitesh Kawedia,Sajad Jawad Khazal,Kris Michael Mahadeo

    LANCET REGIONAL HEALTH-AMERICAS（2024）

    Department of Pediatrics | Loma Linda Univ | Pediatric Blood and Marrow Transplant | Texas Childrens Hosp | St Jude Childrens Res Hosp | Nationwide Childrens Hosp | Univ Texas MD Anderson Canc Ctr | Division of Hematology/Oncology/BMT | Univ Tennessee | Division of Transplant and Cell Therapy | Duke Univ

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    Abstract
    BackgroundSinusoidal obstruction syndrome is a potentially fatal complication following hematopoietic cell transplantation, high-intensity chemotherapies and increasingly seen with calicheamicin based leukemia therapies. Paediatric specific European Society for Blood and Marrow Transplantation (pEBMT) diagnostic criteria have demonstrated benefit in single center studies compared to historic criteria. Yet, the extent to which they have been universally implemented remains unclear.MethodsWe conducted a retrospective multi-centre study to examine the potential impact of the Baltimore, modified Seattle and pEBMT criteria on the incidence, severity, and outcomes of sinusoidal obstruction syndrome among paediatric hematopoietic cell transplantation patients.FindingsThe incidence of sinusoidal obstruction syndrome in this cohort (n = 488) was higher by pEBMT (21.5%) vs historic modified Seattle (15.6%) and Baltimore (7.0%) criteria (p < 0.001). Application of pEBMT criteria identified 44 patients who were not previously diagnosed with sinusoidal obstruction syndrome. Overall, 70.5% of all patients diagnosed with sinusoidal obstruction syndrome ultimately developed very severe disease and almost half of diagnosed patients required critical care support. Overall survival was significantly lower in patients who were diagnosed with sinusoidal obstruction syndrome vs those who were not.InterpretationTaken together, pEBMT criteria may be a sensitive method for prompter diagnosis of patients who subsequently develop severe/very severe sinusoidal obstruction syndrome. To our knowledge, this is the first multi-centre study in the United States (US) to demonstrate that pEBMT guidelines are associated with earlier detection of sinusoidal obstruction syndrome. Since early initiation of definitive treatment for sinusoidal obstruction syndrome has been associated with improved survival in paediatric patients and implementation of pEBMT criteria appears feasible in the US, universal adoption should facilitate prompter diagnosis and lead to improved outcomes of children with sinusoidal obstruction syndrome.FundingNone.
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    Key words
    Veno-occlusive disease,Hematopoietic cell transplantation,Paediatrics,Sinusoidal obstruction syndrome
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    【要点】：本研究通过回顾性多中心分析，证明了儿童造血细胞移植后使用欧洲血液和骨髓移植协会儿童特定诊断标准（pEBMT）可更敏感地诊断出肝窦阻塞综合征，提高了生存率。
    

    【方法】：研究采用了回顾性多中心研究方法，比较了巴尔的摩标准、改良西雅图标准和pEBMT标准在儿童造血细胞移植患者中诊断肝窦阻塞综合征的影响。
    

    【实验】：研究共纳入488名儿童患者，发现使用pEBMT标准时，肝窦阻塞综合征的发病率（21.5%）显著高于改良西雅图标准（15.6%）和巴尔的摩标准（7.0%）。应用pEBMT标准诊断出44名之前未被诊断出肝窦阻塞综合征的患者。约70.5%的确诊患者病情发展为非常严重，近一半的患者需要重症监护支持，确诊患者的总生存率显著低于未确诊患者。