Sinusoidal obstructive syndrome, also known as hepatic veno-occlusive disease, is a potentially life-threatening complication that occurs in children undergoing haemopoietic stem-cell transplantation (HSCT). Differences in the incidence of genetic predisposition and clinical presentation of sinusoidal obstructive syndrome between children and adults have rendered the historical Baltimore and Seattle diagnostic criteria insufficient for children. In 2017, the European Society for Blood and Marrow Transplantation (EBMT) proposed the first paediatric diagnostic and severity grading guidelines for sinusoidal obstructive syndrome, intended for implementation across European centres. However, universally accepted paediatric criteria are needed to ensure prompt diagnosis, definitive treatment, and improved outcomes for children, adolescents, and young adults with sinusoidal obstructive syndrome, and to facilitate international clinical research collaboration. We convened an international panel of multidisciplinary experts including physicians with expertise in HSCT, paediatric intensive care, nephrology, hepatology, radiology, pathology, and transfusion medicine; HSCT advanced-practice providers and medical trainees; pharmacists; and translational and basic science researchers from the Pediatric Acute Lung Injury and Sepsis Investigators Network, the EBMT, the Pediatric Blood and Marrow Transplant Consortia, and several other institutions with extensive experience in sinusoidal obstructive syndrome. Panellists convened at The University of Texas, MD Anderson Cancer Center (Houston, TX, USA) in February, 2019, to evaluate the available evidence. In this expert position statement paper, we provide consensus recommendations for the international implementation of guidelines for the diagnosis, severity grading, and treatment of sinusoidal obstructive syndrome among children, adolescents, and young adults. We endorse universal adoption of paediatric diagnostic guidelines for sinusoidal obstruction syndrome as proposed by the EBMT, and provide implementation guidance for standardisation across centres; we have further proposed adjunctive use of age-appropriate organ-specific toxicity criteria for severity grading and provided prophylaxis and treatment considerations among children and adolescent and young adult patients. Key recommendations include: (1) liver biopsy, portal venous wedge pressure, and reversal of portal venous flow on Doppler ultrasonography should not be used for the routine diagnosis of sinusoidal obstructive syndrome in children, adolescents, and young adults; (2) platelet refractoriness can be defined as a corrected count increment of less than 5000-7500 following at least two sequential ABO-compatible fresh platelet transfusions; (3) hepatomegaly is best defined as an absolute increase of at least 1 cm in liver length at the midclavicular line; and if a baseline measurement is not available, hepatomegaly can be defined as greater than 2 SDs above normal for age; and (4) the presence and volume of ascites can be categorised as mild (minimal fluid by liver, spleen, or pelvis), moderate (<1 cm fluid), or severe (fluid in all three regions with >1 cm fluid in at least two regions).

中文翻译：

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正弦波阻塞综合征儿童、青少年和年轻人的诊断、分级和治疗建议：国际专家立场声明。

肝窦阻塞综合征，也称为肝静脉闭塞性疾病，是一种可能危及生命的并发症，发生在接受造血干细胞移植 (HSCT) 的儿童中。儿童和成人之间正弦波阻塞综合征的遗传易感性和临床表现的差异使得巴尔的摩和西雅图的历史诊断标准不足以用于儿童。2017 年，欧洲血液和骨髓移植学会 (EBMT) 提出了第一个针对正弦波阻塞综合征的儿科诊断和严重程度分级指南，旨在在整个欧洲中心实施。然而，需要普遍接受的儿科标准来确保及时诊断、明确治疗和改善儿童、青少年、和患有正弦波阻塞综合征的年轻成人，并促进国际临床研究合作。我们召集了一个国际多学科专家小组，包括在 HSCT、儿科重症监护、肾脏病学、肝病学、放射学、病理学和输血医学方面具有专业知识的医生；HSCT 高级实践提供者和医疗实习生；药剂师；来自小儿急性肺损伤和脓毒症调查员网络、EBMT、小儿血液和骨髓移植联盟以及其他几个在正弦阻塞综合征方面具有丰富经验的机构的转化和基础科学研究人员。小组成员于 2019 年 2 月在德克萨斯大学 MD 安德森癌症中心（美国德克萨斯州休斯顿）召开会议，评估现有证据。在这份专家立场声明文件中，我们为国际实施儿童、青少年和年轻成人正弦波阻塞综合征的诊断、严重程度分级和治疗指南提供了共识建议。我们赞同普遍采用 EBMT 提议的儿科正弦阻塞综合征诊断指南，并为跨中心标准化提供实施指南；我们进一步建议辅助使用适合年龄的器官特异性毒性标准进行严重程度分级，并提供儿童、青少年和年轻成人患者的预防和治疗考虑。主要建议包括：(1) 肝活检、门静脉楔压、多普勒超声显示的门静脉血流逆转不应用于儿童、青少年和年轻成人的肝窦阻塞综合征的常规诊断；(2) 血小板不应性可定义为在至少连续两次连续输注 ABO 相容的新鲜血小板后，校正计数增量小于 5000-7500；(3) 肝肿大最好定义为锁骨中线处肝脏长度绝对增加至少 1 厘米；如果无法获得基线测量值，则肝肿大可定义为比年龄正常值高出 2 个标准差以上；(4) 腹水的存在和体积可分为轻度（肝脏、脾脏或骨盆的少量液体）、中度（<1 cm 液体）或重度（所有三个区域的液体 >1 cm 液体在至少两个区域）。和年轻人；(2) 血小板不应性可定义为在至少连续两次连续输注 ABO 相容的新鲜血小板后，校正计数增量小于 5000-7500；(3) 肝肿大最好定义为锁骨中线处肝脏长度绝对增加至少 1 厘米；如果无法获得基线测量值，则肝肿大可定义为比年龄正常值高出 2 个标准差以上；(4) 腹水的存在和体积可分为轻度（肝脏、脾脏或骨盆的少量液体）、中度（<1 cm 液体）或重度（所有三个区域的液体 >1 cm 液体在至少两个区域）。和年轻人；(2) 血小板不应性可定义为在至少连续两次连续输注 ABO 相容的新鲜血小板后，校正计数增量小于 5000-7500；(3) 肝肿大最好定义为锁骨中线处肝脏长度绝对增加至少 1 厘米；如果无法获得基线测量值，则肝肿大可定义为比年龄正常值高出 2 个标准差以上；(4) 腹水的存在和体积可分为轻度（肝脏、脾脏或骨盆的少量液体）、中度（<1 cm 液体）或重度（所有三个区域的液体 >1 cm 液体在至少两个区域）。(3) 肝肿大最好定义为锁骨中线处肝脏长度绝对增加至少 1 厘米；如果无法获得基线测量值，则肝肿大可定义为比年龄正常值高出 2 个标准差以上；(4) 腹水的存在和体积可分为轻度（肝脏、脾脏或骨盆的少量液体）、中度（<1 cm 液体）或重度（所有三个区域的液体 >1 cm 液体在至少两个区域）。(3) 肝肿大最好定义为锁骨中线处肝脏长度绝对增加至少 1 厘米；如果无法获得基线测量值，则肝肿大可定义为比年龄正常值高出 2 个标准差以上；(4) 腹水的存在和体积可分为轻度（肝脏、脾脏或骨盆的少量液体）、中度（<1 cm 液体）或重度（所有三个区域的液体 >1 cm 液体在至少两个区域）。