新生儿血小板输注阈值的随机试验。

Randomized Trial of Platelet-Transfusion Thresholds in Neonates.

机构信息

From the Department of Neonatology, National Maternity Hospital, Dublin (A.C.), and University Maternity Hospital Limerick, Limerick (R.K.) - both in Ireland; Department of Transfusion Medicine, National Health Service (NHS) Blood and Transplant, the Department of Haematology, John Radcliffe Hospital, Oxford University Hospitals NHS Foundation Trust, the Radcliffe Department of Medicine, University of Oxford, and Oxford Biomedical Research Centre Haematology Theme, Oxford (S.J.S.), the Department of Obstetrics and Gynaecology, University of Cambridge (K.W.), NHS Blood and Transplant, Clinical Trials Unit (K.W., C.H., A. Deary, R.H., V.H., A.M., C.L.), and the Neonatal Intensive Care Unit, Cambridge University Hospitals NHS Trust (A. D'Amore), Cambridge, the Neonatal Intensive Care Unit, Evelina London Children's Hospital, Guy's and St. Thomas' NHS Foundation Trust (B.L.S., T.W.), NHS Blood and Transplant (H.N.), and the Department of Hematology, Imperial College London (H.N.), London, and the Neonatal Intensive Care Unit, Norfolk and Norwich University Hospitals NHS Foundation Trust, and Norwich Medical School, University of East Anglia, Norwich Research Park, Norwich (P.C.) - all in the United Kingdom; the Center for Clinical Transfusion Research, Sanquin Blood Supply (S.F.F.-G., K.F.), and the Departments of Epidemiology (S.F.F.-G.) and Neonatology (E.L.), Leiden University Medical Center, Leiden, and the Departments of Pediatric Hematology (S.F.F.-G., K.F.) and Neonatology (W.O.), Emma Children's Hospital, Academic Medical Center, Amsterdam - both in the Netherlands; and the Neonatal Intensive Care Unit, Cloudnine Hospital, Bangalore, India (V.V.).

出版信息

N Engl J Med. 2019 Jan 17;380(3):242-251. doi: 10.1056/NEJMoa1807320. Epub 2018 Nov 2.

DOI:10.1056/NEJMoa1807320

PMID:30387697

Abstract

BACKGROUND

Platelet transfusions are commonly used to prevent bleeding in preterm infants with thrombocytopenia. Data are lacking to provide guidance regarding thresholds for prophylactic platelet transfusions in preterm neonates with severe thrombocytopenia.

METHODS

In this multicenter trial, we randomly assigned infants born at less than 34 weeks of gestation in whom severe thrombocytopenia developed to receive a platelet transfusion at platelet-count thresholds of 50,000 per cubic millimeter (high-threshold group) or 25,000 per cubic millimeter (low-threshold group). Bleeding was documented prospectively with the use of a validated bleeding-assessment tool. The primary outcome was death or new major bleeding within 28 days after randomization.

RESULTS

A total of 660 infants (median birth weight, 740 g; and median gestational age, 26.6 weeks) underwent randomization. In the high-threshold group, 90% of the infants (296 of 328 infants) received at least one platelet transfusion, as compared with 53% (177 of 331 infants) in the low-threshold group. A new major bleeding episode or death occurred in 26% of the infants (85 of 324) in the high-threshold group and in 19% (61 of 329) in the low-threshold group (odds ratio, 1.57; 95% confidence interval [CI], 1.06 to 2.32; P=0.02). There was no significant difference between the groups with respect to rates of serious adverse events (25% in the high-threshold group and 22% in the low-threshold group; odds ratio, 1.14; 95% CI, 0.78 to 1.67).

CONCLUSIONS

Among preterm infants with severe thrombocytopenia, those randomly assigned to receive platelet transfusions at a platelet-count threshold of 50,000 per cubic millimeter had a significantly higher rate of death or major bleeding within 28 days after randomization than those who received platelet transfusions at a platelet-count threshold of 25,000 per cubic millimeter. (Funded by the National Health Service Blood and Transplant Research and Development Committee and others; Current Controlled Trials number, ISRCTN87736839 .).

摘要

背景

血小板输注常用于预防伴有血小板减少症的早产儿出血。目前缺乏数据来指导伴有严重血小板减少症的早产儿预防性血小板输注的阈值。

方法

在这项多中心试验中，我们将出生胎龄小于 34 周且发生严重血小板减少症的婴儿随机分配，接受血小板输注的血小板计数阈值分别为 50,000/立方毫米（高阈值组）或 25,000/立方毫米（低阈值组）。前瞻性使用经验证的出血评估工具记录出血情况。主要结局为随机分组后 28 天内死亡或新发重大出血。

结果

共有 660 名婴儿（中位出生体重为 740 克，中位胎龄为 26.6 周）接受了随机分组。在高阈值组中，90%（296/328 名婴儿）的婴儿接受了至少一次血小板输注，而在低阈值组中，这一比例为 53%（177/331 名婴儿）。高阈值组中有 26%（85/324 名婴儿）的婴儿发生新的重大出血事件或死亡，而低阈值组中有 19%（61/329 名婴儿）（比值比，1.57；95%置信区间[CI]，1.06 至 2.32；P=0.02）。两组严重不良事件发生率无显著差异（高阈值组为 25%，低阈值组为 22%；比值比，1.14；95%CI，0.78 至 1.67）。

结论

在伴有严重血小板减少症的早产儿中，与接受血小板计数阈值为 25,000/立方毫米的血小板输注的婴儿相比，随机接受血小板计数阈值为 50,000/立方毫米的血小板输注的婴儿在随机分组后 28 天内死亡或发生重大出血的风险显著更高。（由英国国民保健服务血液与移植研究与开发委员会等资助；当前对照试验编号，ISRCTN87736839）。