Preterm neonates benefit from low prophylactic platelet transfusion threshold despite varying risk of bleeding or death
Preterm neonates benefit from low prophylactic platelet transfusion threshold despite varying risk of bleeding or death
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DOI：
10.1182/blood.2019000899
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发表时间：
2019-12-26
期刊：
影响因子：
20.3
通讯作者：
van der Bom, Johanna G.
中科院分区：
文献类型：
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作者：
Fustolo-Gunnink, Susanna F.;Fijnvandraat, Karin;van der Bom, Johanna G.
新生儿血小板减少症(PLANET-2)试验报告称，在早产儿大出血和/或死亡率方面，预防性血小板输注阈值为25×10(9)/L与50×10(9)/L相比，总体效果出人意料(绝对风险降低7%)。然而，试验中的一些新生儿可能从25x10(9)/L的阈值中获益甚微，甚至受到伤害。我们想在PLANET-2试验中评估这种治疗效果的异质性，以调查是否所有早产儿都受益于低阈值。我们在PLANET-2数据中开发了一个多变量Logistic回归模型，以预测所有653名新生儿的大出血和/或死亡的基线风险。然后，我们根据他们预测的基线风险对新生儿进行排名，并将他们分为四个风险四分位数。在这些四分位数内，我们评估了50×10(9)/L阈值组和25×10(9)/L阈值组之间的绝对风险差异。共有146名新生儿死亡或出现大出血。模型的内部验证C统计量为0.63(95%可信区间为0.58~0.68)。在所有风险组中，25×10(9)/L阈值与绝对风险降低相关，从最低风险组的4.9%到最高风险组的12.3%不等。这些结果表明，所有早产儿都可以采用25×10(9)/L的预防血小板计数阈值，而不考虑预测的基线结局风险。需要进一步的研究来提高基线风险模型的预测准确性。
The Platelets for Neonatal Thrombocytopenia (PlaNeT-2) trial reported an unexpected overall benefit of a prophylactic platelet transfusion threshold of 25 x 10(9)/L compared with 50 x 10(9)/L for major bleeding and/or mortality in preterm neonates (7% absolute-risk reduction). However, some neonates in the trial may have experienced little benefit or even harm from the 25 x 10(9)/L threshold. We wanted to assess this heterogeneity of treatment effect in the PlaNet-2 trial, to investigate whether all preterm neonates benefit from the low threshold. We developed a multivariate logistic regression model in the PlaNet-2 data to predict baseline risk of major bleeding and/or mortality for all 653 neonates. We then ranked the neonates based on their predicted baseline risk and categorized them into 4 risk quartiles. Within these quartiles, we assessed absolute-risk difference between the 50 x 10(9)/L- and 25 x 10(9)/L-threshold groups. A total of 146 neonates died or developed major bleeding. The internally validated C-statistic of the model was 0.63 (95% confidence interval, 0.58-0.68). The 25 x 10(9)/L threshold was associated with absolute-risk reduction in all risk groups, varying from 4.9% in the lowest risk group to 12.3% in the highest risk group. These results suggest that a 25 x 10(9)/L prophylactic platelet count threshold can be adopted in all preterm neonates, irrespective of predicted baseline outcome risk. Future studies are needed to improve the predictive accuracy of the baseline risk model.