近日，荷兰莱顿大学医学中心Camila Caram-Deelder团队研究了重度血小板减少症早产儿血小板输注结果的个体化预测。相关论文于2025年9月15日发表在《美国医学会杂志》上。

早产儿伴严重血小板减少症(血小板计数<；50 ×10⁹/L)频繁接受血小板输注。然而，目前尚不清楚在哪些情况下预防性输血确实能降低出血风险，或者它是否弊大于利。

为了建立并验证严重血小板减少症患儿是否给予预防性血小板输注时大出血或死亡率的动态预测模型，研究组在国际多中心队列（2017-2021）中开发了一个动态预测模型，该队列包括荷兰、瑞典和德国的14个新生儿重症监护病房。模型评估在一个国家多中心队列（2010-2014）中进行，包括7个荷兰新生儿重症监护病房。研究人群包括妊娠34周以内患有严重血小板减少症的婴儿。

在每个预测点对比两种输血策略：在6小时内接受血小板输注（预防）与3天不输血小板（不预防）。主要终点是严重血小板减少症发作后第一周内每2小时重新评估的3天大出血或死亡风险。预测因素包括胎龄和出生后年龄、胎龄小的婴儿、坏死性小肠结肠炎、败血症、机械通气、血管活性药物、血小板计数和既往血小板输注。结合克隆-审查器-权重方法实现了两种输血策略下的动态预测，考虑了时变的混杂。在外部验证队列中评估模型的性能。

在发展（n = 1042) 队列和验证（n = 637) 队列中，中位胎龄为28周，中位出生体重为900克；分别有613名（59%）和370名（58%）男性。 发展组235例（23%）和验证组135例（21%）发生大出血或死亡。在验证队列中，预防策略的受试者工作特征曲线下的时间依赖面积为0.69 (95% CI, 0.60-0.76)，无预防策略的受试者工作特征曲线下的时间依赖面积为0.85 (95% CI, 0.76-0.92)，校准图显示校准良好。根据预测时婴儿的临床状况，两种策略下的估计风险差异很大。

研究结果表明，在患有严重血小板减少症的早产儿中，该模型研究发现，基于他们当前的临床特征，预防性血小板输注的预测益处和危害在个体之间存在实质性差异。动态预测模型在验证队列中表现良好，其支持个性化决策的价值值得在未来的研究中进行评估。

附：英文原文

Title: Individualized Prediction of Platelet Transfusion Outcomes in Preterm Infants With Severe Thrombocytopenia

Author: Hilde van der Staaij, Ilaria Prosepe, Camila Caram-Deelder, Ruth H. Keogh, Emke Deschmann, Christof Dame, Wes Onland, Sandra A. Prins, Florian Cassel, Esther J. d’Haens, Elke van Westering-Kroon, Peter Andriessen, Sabine L. Vrancken, Christian V. Hulzebos, Daniel C. Vijlbrief, Suzanne F. Fustolo-Gunnink, Karin Fijnvandraat, Enrico Lopriore, Johanna G. van der Bom, Nan van Geloven

Issue&Volume: 2025-09-15

Abstract:

IMPORTANCE  Preterm infants with severe thrombocytopenia (platelet count <50×109/L) frequently receive platelet transfusions. However, it is unclear in what cases prophylactic transfusion truly reduces bleeding risk or whether it does more harm than good.

OBJECTIVE  To develop and validate a dynamic prediction model for major bleeding or mortality if prophylactic platelet transfusion were or were not to be given to infants with severe thrombocytopenia.

DESIGN, SETTING, AND PARTICIPANTS  The dynamic prediction model was developed in an international multicenter cohort (2017-2021) comprising 14 neonatal intensive care units in the Netherlands, Sweden, and Germany. Model evaluation was performed in a national multicenter cohort (2010-2014) including 7 Dutch neonatal intensive care units. The study population consisted of infants with severe thrombocytopenia less than 34 weeks’ gestation.

EXPOSURE  Two transfusion strategies were contrasted at each prediction point: receiving a platelet transfusion within 6 hours (prophylaxis) vs no platelet transfusion for 3 days (no prophylaxis).

MAIN OUTCOMES AND MEASURES  The primary outcome was the 3-day risk of major bleeding or mortality, reestimated every 2 hours during the first week after severe thrombocytopenia onset. Predictors included gestational and postnatal age, small-for-gestational-age infant, necrotizing enterocolitis, sepsis, mechanical ventilation, vasoactive agents, platelet count, and prior platelet transfusion(s). Landmarking combined with the clone-censor-weight approach enabled dynamic prediction under the 2 transfusion strategies, accounting for time-varying confounding. Model performance was evaluated in the external validation cohort.

RESULTS  In both the development (n=1042) and validation (n=637) cohorts, the median gestational age was 28 weeks and median birth weight was 900 g; there were 613 (59%) and 370 (58%) males, respectively. Major bleeding or death occurred in 235 infants (23%) in the development cohort and 135 (21%) in the validation cohort. In the validation cohort, the time-dependent area under the receiver operating characteristic curve was 0.69 (95% CI, 0.60-0.76) for the prophylaxis strategy and 0.85 (95% CI, 0.76-0.92) for the no prophylaxis strategy, with calibration plots showing good calibration. Estimated risks under both strategies varied considerably depending on the infant’s clinical condition at the time of prediction.

CONCLUSIONS AND RELEVANCE  Among preterm infants with severe thrombocytopenia, this modeling study found substantial variation among individuals in predicted benefits and harms of prophylactic platelet transfusion based on their current clinical characteristics. The dynamic prediction model performed well in a validation cohort, and its value to support individualized decisions warrants evaluation in future studies.

DOI: 10.1001/jama.2025.14194

Source: https://jamanetwork.com/journals/jama/fullarticle/2838997