Pediatric Hematology Oncology, Department of Pediatrics, University of Padova, Padova, Italy.
Pediatr Blood Cancer. 2011 Aug;57(2):258-61. doi: 10.1002/pbc.22841. Epub 2011 Jan 18.
Hepatic veno-occlusive disease (VOD) is a rare and potentially severe complication of chemotherapy. We describe five patients who developed VOD after chemotherapy for Wilms tumor (WT) and evaluate the role of plasminogen activator inhibitor-1 (PAI-1) and defibrotide for diagnosis and therapy of VOD, respectively.
Thirty-five patients treated from 2002 to 2009 for WT were eligible. Diagnosis of VOD was according McDonald's criteria that required two of the following: jaundice, hepatomegaly and/or right upper quadrant pain, weight gain with or without ascites.
VOD occurred in 5 of 35 patients (14%) after 21-105 days from starting chemotherapy. Two patients developed multiorgan failure (MOF). PAI-1 was high in four patients who were tested. Three patients were treated with defibrotide and no side effects were reported while two patients received supportive measures only. Four patients recovered and three of them received defibrotide. They are all alive and well after a median follow-up of 35 months. One of two patients not treated with defibrotide died of MOF.
PAI-1 levels were abnormal in WT-VOD. Defibrotide was a safe, well-tolerated, and potentially efficacious therapy in this group of patients. Further prospective study is needed in WT-VOD to confirm these data.
肝静脉闭塞病（VOD）是化疗后罕见且潜在严重的并发症。我们描述了 5 例因化疗治疗肾母细胞瘤（WT）而发生 VOD 的患者，并分别评估了纤溶酶原激活物抑制剂-1（PAI-1）和多西他赛在 VOD 诊断和治疗中的作用。
2002 年至 2009 年期间，35 例接受 WT 治疗的患者符合条件。VOD 的诊断依据是 McDonald 标准，需要满足以下两项中的两项：黄疸、肝肿大和/或右上腹疼痛、体重增加伴或不伴腹水。
化疗开始后 21-105 天，35 例患者中有 5 例（14%）发生 VOD。2 例患者发生多器官功能衰竭（MOF）。4 例检测到 PAI-1 升高。3 例患者接受了多西他赛治疗，未报告不良反应，而 2 例患者仅接受了支持性治疗。4 例患者恢复，其中 3 例接受了多西他赛治疗。在中位随访 35 个月后，他们均存活且状况良好。未接受多西他赛治疗的 2 例患者之一因 MOF 死亡。
WT-VOD 患者的 PAI-1 水平异常。多西他赛在该组患者中是一种安全、耐受良好且潜在有效的治疗方法。需要进一步的前瞻性研究来证实这些数据。
