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）婴儿通常会发生早产贫血，并且在新生儿重症监护期间经常需要多次红细胞输注（RBCT）。目前用于表明在这一人群中进行RBCT的标准是基于专家意见，而不是证据和结论性数据，不存在RBCT做法的长期影响。为了提高携氧能力而给予RBCT，以及为了避免RBCT相关的风险和成本而限制RBCT，这两种情况都可能损害RBCT的长期发展。提出的盲法随机对照试验旨在比较限制性和自由红细胞输血指南对ELBW婴儿长期神经发育结局的影响。ELBW婴儿将根据自由或限制性RBCT指南随机接受RBCT，这既反映了德国目前的做法，又旨在平均血红蛋白浓度的临床相关差异。主要结局指标是在24月龄校正早产后确定的死亡或主要神经发育障碍发生率。关键的次要结局是复合主要结局的个别组成部分的发生率、Bayley婴儿发育量表（II版）的身心发育指数得分和生长。安全性分析将评估所有主要早产疾病的发生率。这项试验的结果可能有助于改善这些患者的生活质量，降低长期医疗保健费用。

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.