Effects of transfusion thresholds on neurocognitive outcome of extremely low birth weight infants (ETTNO) a blinded randomized controlled multicenter trial

极低出生体重(ELBW)婴儿一致发展为早产儿贫血，在新生儿重症监护期间经常需要多次红细胞输注(RBCT)。目前应用的标准表明，这一人群中的有条件现金转移是基于专家意见，而不是证据，而且没有关于有风险转移做法的长期影响的确凿数据。给予RBCT以提高携氧能力，以及限制RBCT以避免与RBCT相关的风险和成本，都可能损害长期发展。这项拟议的随机随机对照试验旨在比较限制性和自由性红细胞输注指南对ELBW婴儿长期神经发育结果的影响。根据自由或限制性的RBCT指南，ELBW婴儿将被随机接受RBCT，这些指南既反映了德国目前的做法，又旨在实现临床上相关的平均血红蛋白浓度差异。主要的结果衡量标准是在24个月时确定的死亡或主要神经发育障碍的发生率，对早产进行了校正。关键的次要结果是综合主要结果的各个组成部分的发生率、贝利婴儿发育量表(II版)的智力和身体发育指数得分以及生长。安全分析将评估所有主要早产儿疾病的发病率。这项试验的结果可能有助于提高这些患者的生活质量，降低长期医疗成本。

Extremely low birth weight (ELBW) infants uniformly develop anemia of prematurity and frequently require multiple red blood cell transfusions (RBCT) during neonatal intensive care. The criteria currently applied to indicate RBCT in this population are based on expert opinion rather than evidence and conclusive data of long-term effects of RBCT practices do not exist. Both, giving RBCT to improve oxygen carrying capacity and restricting RBCT to avoid RBCT associated risks and costs potentially impair long-term development. The proposed blinded randomized controlled trial was designed and will be powered to compare the effect of restrictive versus liberal red blood cell transfusion guidelines on long-term neurodevelopmental outcome in ELBW infants. ELBW infants will be randomized to receive RBCT according to liberal or restrictive RBCT guidelines, which both reflect current practice in Germany and aim for a clinically relevant difference in mean hemoglobin concentrations. The primary outcome measure is the incidence of death or major neurodevelopmental impairment determined at 24 months of age corrected for prematurity. Key secondary outcomes are the incidences of individual components of the composite primary outcome, the mental and physical developmental index scores of the Bayley Scales of Infant Development (II edition), and growth. Safety analyses will assess the incidences of all major diseases of prematurity. The results of this trial may help to improve the quality of life of these patients and reduce long-term health care costs.