Children and Young People's Unit, The Royal Marsden Hospital, Sutton, United Kingdom.
Department of Pharmacy, The Royal Marsden Hospital, Sutton, United Kingdom.
Pediatr Blood Cancer. 2020 Nov;67(11):e28677. doi: 10.1002/pbc.28677. Epub 2020 Aug 31.
Hepatic sinusoidal obstruction syndrome (SOS) is a serious complication of autologous stem cell transplant (ASCT) in children with historically high mortality rates. Defibrotide has shown proven benefit in its treatment and may have a modest role in prevention. We report our experience with SOS in children undergoing autologous transplant.
Case records of 82 consecutive patients undergoing ASCT following high-dose chemotherapy between 2010 and 2017 were reviewed. Defibrotide was used for treatment of all with SOS and prophylactically in patients receiving busulfan-based conditioning until 2014.
Fourteen of the 82 children (17%) were diagnosed with SOS. The incidence was higher in those receiving busulfan-based conditioning (13/42 vs 1/40, P = 0.008). Mean (±SD) time to diagnosis of SOS was 19 (±5.6) days following stem cell rescue. Bilirubin levels and ultrasound were normal in 7/14 and 3/14 patients. Coagulopathy was noted in 10/14; one child developed multiorgan involvement. Nine children had mild SOS, whereas two and three had moderate and severe SOS, respectively. Intensive care was required for four of five non-mild cases. Patients with SOS had significantly delayed platelet recovery, higher transfusion requirement, and longer hospital stay. Unavailability of defibrotide prophylaxis for 17/42 receiving busulfan did not change the incidence of SOS (7/25 with defibrotide vs 6 /17 without defibrotide, P = 0.74). There was no significant difference in the severity of SOS between these groups.
Hepatic SOS was more commonly seen in children receiving busulfan-based conditioning. Stopping the use of prophylactic defibrotide did not increase incidence or severity of SOS. Overall outcome was excellent with supportive care and timely treatment with defibrotide.
肝窦阻塞综合征（SOS）是儿童自体干细胞移植（ASCT）后的一种严重并发症，其死亡率历来较高。已证实去纤维肽在其治疗中有明显益处，且可能在预防方面有一定作用。我们报告了接受自体移植的儿童 SOS 的经验。
回顾了 2010 年至 2017 年间接受大剂量化疗后进行 ASCT 的 82 例连续患者的病历。所有 SOS 患者均使用去纤维肽治疗，在 2014 年之前，接受以白消安为基础的预处理方案的患者也预防性使用去纤维肽。
82 例儿童中有 14 例（17%）被诊断为 SOS。接受以白消安为基础的预处理方案的患者发病率更高（13/42 比 1/40，P=0.008）。SOS 诊断后的平均（±SD）时间为干细胞挽救后 19（±5.6）天。14 例患者中 7 例胆红素水平和超声检查正常，3 例正常。10 例患者出现凝血功能障碍，1 例患儿出现多器官受累。9 例患儿为轻度 SOS，2 例和 3 例分别为中度和重度 SOS。4/5 例非轻度病例需要重症监护。SOS 患者血小板恢复明显延迟，输血需求较高，住院时间较长。因白消安的 17/42 例患者未使用去纤维肽预防，故发病率并未改变（使用去纤维肽者 7/25 例，未使用者 6/17 例，P=0.74）。两组间 SOS 严重程度无显著差异。
接受以白消安为基础的预处理方案的患儿更常出现肝 SOS。停止使用预防性去纤维肽并未增加 SOS 的发生率或严重程度。通过支持性治疗和及时使用去纤维肽，总体预后良好。
