Totadri Sidharth, Trehan Amita, Bansal Deepak, Jain Richa
Department of Pediatrics, Advanced Pediatrics Center, Pediatric Hematology-Oncology Unit, Postgraduate Institute of Medical Education and Research, Chandigarh, India.
Indian J Med Paediatr Oncol. 2017 Oct-Dec;38(4):447-451. doi: 10.4103/ijmpo.ijmpo_188_16.
Survival rates exceed 90% in Wilms' tumor (WT). Actinomycin-D (ACT-D) which is indispensable in the management of WT is associated with the development of sinusoidal obstruction syndrome (SOS), a potentially fatal complication.
The aim is to study the presentation, management, and outcome of SOS complicating ACT-D administration in WT.
Retrospective file review conducted in a Pediatric Hematology-Oncology unit.
Patients diagnosed and treated for WT from January 2012 to December 2015 were analyzed. SOS was diagnosed clinically, based on McDonalds criteria, requiring two of the following: jaundice, hepatomegaly and/or right upper quadrant pain, weight gain with or without ascites.
Of 104 patients treated, SOS occurred in 5 (4.8%). Age: 6 months to 5 years, 3 were girls. Tumor involved left kidney in 3, right in 1 and a horseshoe kidney in 1. Histopathology was consistent with WT in 4 and clear cell sarcoma kidney in 1. One had pulmonary metastases. Three developed SOS preoperatively and two during adjuvant chemotherapy. None received radiotherapy. Clinical manifestations comprised of jaundice, hepatomegaly, ascites/weight gain, respiratory distress, hypotension, and encephalopathy. Laboratory findings included thrombocytopenia, elevated serum transaminases, and coagulopathy. Treatment included fluid restriction, broad spectrum antibiotics, and transfusional support. Two children received N-acetyl cysteine infusion. Defibrotide was administered to two patients. Four recovered and one succumbed to multi-organ failure. Two patients were safely re-challenged with 50% doses of ACT-D.
SOS is a clinical diagnosis. Systematic supportive care can enable complete recovery. Under close monitoring, re-challenge of ACT-D can be performed in gradually escalating doses.
肾母细胞瘤（WT）的生存率超过90%。放线菌素D（ACT-D）在WT的治疗中不可或缺，但与窦性阻塞综合征（SOS）的发生有关，SOS是一种潜在的致命并发症。
研究WT患者在接受ACT-D治疗时并发SOS的表现、治疗及预后。
在儿科血液肿瘤病房进行回顾性病历审查。
分析2012年1月至2015年12月期间诊断并接受WT治疗的患者。根据麦当劳标准进行SOS的临床诊断，需要满足以下两项：黄疸、肝肿大和/或右上腹疼痛、伴有或不伴有腹水的体重增加。
在接受治疗的104例患者中，5例（4.8%）发生SOS。年龄为6个月至5岁，3例为女孩。肿瘤累及左肾3例，右肾1例，马蹄肾1例。4例组织病理学与WT一致，1例为肾透明细胞肉瘤。1例有肺转移。3例在术前发生SOS，2例在辅助化疗期间发生。均未接受放疗。临床表现包括黄疸、肝肿大、腹水/体重增加、呼吸窘迫、低血压和脑病。实验室检查结果包括血小板减少、血清转氨酶升高和凝血功能障碍。治疗包括液体限制、广谱抗生素和输血支持。2例儿童接受N-乙酰半胱氨酸输注。2例患者使用了去纤苷。4例康复，1例死于多器官功能衰竭。2例患者以50%的ACT-D剂量安全地再次接受挑战。
SOS是一种临床诊断。系统的支持治疗可实现完全康复。在密切监测下，可逐渐增加剂量对ACT-D进行再次挑战。
