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Risk factors and mortality predictors of hepatic veno-occlusive disease after pediatric hematopoietic stem cell transplantation.

机译:小儿造血干细胞移植后肝静脉闭塞性疾病的危险因素和死亡率预测指标。

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A cohort of 138 children with 144 hematopoietic stem cell transplantation (HSCT) performed in 1997-2006 were analyzed to evaluate risk factors and mortality predictors of hepatic veno-occlusive disease (VOD). Nineteen patients (13.2%) developed VOD (nine boys, median age 3.5 years) at 1-21 days after HSCT (median 13 days). Age < or =2 years at transplant (odds ratio (OR)=5.25, P=0.011), BU-CY conditioning (OR=5.16, P=0.001), thalassemia major (OR=3.97, P=0.015), platelet engraftment beyond day +21 (OR=8.67, P=0.025) were univariate risk factors for VOD. The first two remained significant in multivariate regression. Seven patients (36.8%) with VOD died, at a median of 44 days post transplant (range, 30-421 days). The 5-year survival was 62%. All surviving patients had normal liver function on follow-up at 0.5-9 years. Patients with VOD had higher 100-day mortality (16.3 vs 9.6%, P=0.024). Mortality predictors included donors other than autologous or matched sibling (hazard ratio (HR)=23.6, P=0.006), hepatic and cutaneous GVHD (HR=8.15, P=0.038), maximal weight gain >9% (HR=6.81, P=0.023), pleural effusion, intensive care unit admission, peak bilirubin >300 micromol l(-1) (HR=13.6, P=0.016), day +21 bilirubin >200 micromol l(-1) (HR=33.9, P=0.001), and rise of bilirubin >15 micromol l(-1) per day within the first week (HR=19.8, P=0.006). Mortality was substantially higher if >3 predictors were present (HR=33.9, P=0.001). Meticulous monitoring in high-risk patients and early treatment should be considered before VOD progresses beyond salvage.
机译:分析了1997年至2006年进行的138例儿童的144例造血干细胞移植(HSCT)队列,以评估肝静脉闭塞性疾病(VOD)的危险因素和死亡率预测因子。 19例患者(13.2%)在HSCT(中位数13天)后1-21天出现了VOD(9名男孩,中位年龄3.5岁)。移植时年龄<或= 2岁(优势比(OR)= 5.25,P = 0.011),BU-CY调节(OR = 5.16,P = 0.001),重度地中海贫血(OR = 3.97,P = 0.015),血小板移植超过+21天(OR = 8.67,P = 0.025)是VOD的单因素风险因素。前两个变量在多元回归中仍然很显着。 7名(36.8%)的VOD患者在移植后的中位数为44天(范围为30-421天)中死亡。 5年生存率为62%。所有存活的患者在0.5-9年随访时肝功能均正常。 VOD患者的100天死亡率较高(16.3 vs 9.6%,P = 0.024)。死亡率预测指标包括自体或相配同胞(危险比(HR)= 23.6,P = 0.006),肝和皮肤GVHD(HR = 8.15,P = 0.038),最大体重增加> 9%(HR = 6.81,P)以外的供体= 0.023),胸腔积液,重症监护病房入院,峰值胆红素> 300 micromol l(-1)(HR = 13.6,P = 0.016),每天+21胆红素> 200 micromol l(-1)(HR = 33.9,P = 0.001),并且在第一周内每天胆红素的升高> 15 micromol l(-1)(HR = 19.8,P = 0.006)。如果存在> 3个预测因子,则死亡率会更高(HR = 33.9,P = 0.001)。在VOD进展超过救助之前,应考虑对高危患者进行细致的监测和早期治疗。

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