We compared the incidence of refractory thrombocytopenia (RT) and platelet transfusion requirements (PTR) in 35 children who developed veno-occlusive disease (VOD) with 35 matched control subjects who underwent hematopoietic stem cell transplant but did not develop VOD. RT developed in 100% of the VOD patients, at a median of 8 days before VOD diagnosis, as compared with 71.5% of the control group. VOD patients required more platelet transfusions than control subjects (median PTR, 6.9 mL/kg [range, .57 to 17.59] versus 3.57 mL/kg [range, 0 to 14.63], respectively) with a statistically significant difference (P < .0001). The number of days with platelet requirements was significantly higher for VOD patients as compared with control subjects (median 68% versus 39%, P =< .0001). The PTR peaked at ~12 mL/kg/day, 2 days before VOD diagnosis, whereas the PTR in the control population was 5 mL/kg/day. The positive predictive value of developing VOD was 88.9% (95% confidence interval, 66.5% to 97%) in patients who were given >7 mL/kg/day of platelets during the at-risk period of days +3 to +13 after transplant. For patients who received >8 mL/kg/day of platelets, the positive predictive value of developing VOD was 86.7% (95% confidence interval, 61.2% to 96.4%). There was no difference in the PTR in patients with mild to moderate VOD as compared with severe VOD; however, the PTR was higher in patients whose VOD did not resolve. The median daily PTR after the diagnosis of VOD in 17 patients who got defibrotide as compared with those who did not get defibrotide was 6.04 mL/kg and 5.72 mL/kg, respectively, but the difference was not statistically significant (P = .56). On univariate and multivariate analysis use of intravenous immunoglobulin was significantly associated with VOD (P = .0088) but was not significantly associated with fatal VOD. In conclusion, RT occurs in 100% of patients at a median of 8 days before VOD diagnosis. VOD should be suspected in any patient with RT after the exclusion of other causes of consumptive thrombocytopenia, especially if they require >7 mL/kg/day of platelets.

中文翻译：

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难治性血小板减少症是儿童肝静脉阻塞性疾病的有效早期诊断标准。

我们比较了35例发生静脉闭塞性疾病（VOD）的儿童与35例进行了造血干细胞移植但未发生VOD的对照受试者的难治性血小板减少症（RT）和血小板输注需求（PTR）的发生率。在VOD诊断之前的中位数为8天，在100％的VOD患者中出现了RT，而对照组的这一比例为71.5％。VOD患者比对照组需要更多的血小板输注（中位PTR，6.9 mL / kg [范围，0.57-17.59]与3.57 mL / kg [范围，0-14.63]），具有统计学上的显着差异（P <.0001 ）。与对照组相比，VOD患者的血小板需求天数显着更高（中位数为68％对39％，P = <.0001）。在VOD诊断之前2天，PTR达到约12 mL / kg /天的峰值，对照人群的PTR为5 mL / kg /天。在高危天数后+3至+13天给予> 7 mL / kg /天血小板的患者，发展VOD的阳性预测值为88.9％（95％置信区间，为66.5％至97％）移植。对于接受> 8 mL / kg /天血小板的患者，发展中的VOD的阳性预测值为86.7％（95％置信区间，61.2％至96.4％）。与重度VOD相比，轻度至中度VOD患者的PTR没有差异。但是，VOD不能解决的患者的PTR较高。诊断为VOD的17例除颤患者与非除颤患者的每日PTR中位数分别为6.04 mL / kg和5.72 mL / kg，但差异无统计学意义（P = .56） 。在单变量和多变量分析中，静脉内免疫球蛋白的使用与VOD显着相关（P = .0088），但与致命的VOD没有显着相关。总之，RT在VOD诊断之前的8天中位数发生在100％的患者中。在排除其他引起性血小板减少的原因后，尤其是如果他们需要> 7 mL / kg /天的血小板，在排除其他原因引起的血小板减少症后，应该怀疑任何RT患者的VOD。