速尿对预防依赖输血的地中海贫血儿童和青年成人容量超负荷的临床效用：一项随机、开放标签、交叉研究。
Clinical Usefulness of Furosemide to Prevent Volume Overload Among Children and Young Adults with Transfusion-Dependent Thalassemia: A Randomized, Open-Label, Crossover Study.
作者信息
Photia Apichat, Traivaree Chanchai, Monsereenusorn Chalinee, Simthamnimit Piyarat, Rujkijyanont Piya
机构信息
Division of Hematology-Oncology, Department of Pediatrics, Phramongkutklao Hospital and College of Medicine, Bangkok, Thailand.
Department of Pediatrics, Fort Prachaksilapakhom Hospital, Udonthani, Thailand.
出版信息
J Blood Med. 2020 Dec 29;11:503-513. doi: 10.2147/JBM.S285647. eCollection 2020.
PURPOSE
Red blood cell transfusion is a key element of treatment among patients with transfusion-dependent thalassemia (TDT). Volume overload and HCC syndrome (hypertension, convulsion, and intracranial hemorrhage) are fatal complications related to transfusion. Furosemide has been widely used to prevent hypertension secondary to volume overload with unclear supportive evidence. This study aimed to evaluate the efficacy of furosemide to prevent volume overload among children and young adults diagnosed with TDT.
METHODS
Patients diagnosed with TDT were enrolled and randomized to receive either furosemide pretransfusion or no furosemide pretransfusion. After 3 weeks to 4 months of wash-out periods, those patients underwent the alternate regimens as per crossover design of the study. Clinical and laboratory parameters including blood pressure and NT-proBNP levels were measured before and after each transfusion. The difference of those parameters between two randomized groups and their potential associated factors were analyzed.
RESULTS
In all, 30 patients undergoing 60 red blood cell transfusions were enrolled in the study. All were randomized and crossover was designed as receiving and not receiving furosemide pretransfusion. No transfusion reactions, symptoms of volume overload and HCC syndrome were observed. No statistically significant correlation was found between pretransfusion furosemide and the difference between pre- and posttransfusion systolic blood pressure (2 mmHg systolic blood pressure difference in pretransfusion furosemide and 1.5 mmHg in no pretransfusion furosemide; -value = 0.721), as well as between pretransfusion furosemide and the difference between pre- and posttransfusion NT-proBNP levels (-3.8 pg/mL NT-proBNP level difference in pretransfusion furosemide and -2.4 pg/mL in no pretransfusion furosemide; -value = 0.490). No significant correlation was also observed even in selected patients with high NT-proBNP levels (-value = 0.262). Associated factors affecting the difference between pre- and posttransfusion NT-proBNP levels were analyzed, and none of those were affected concerning the difference in the levels.
CONCLUSION
Furosemide has been included in standard transfusion guidelines in many institutions. Our study provided important evidence of the unnecessary use of the drug in preventing volume overload particularly in pediatric and young adult patients with TDT.
THAI CLINICAL TRIALS REGISTRY TCTR NUMBER
TCTR20180209001. Registered 6 February 2018, https://www.clinicaltrials.in.th/.
目的
红细胞输血是依赖输血的地中海贫血（TDT）患者治疗的关键要素。容量超负荷和HCC综合征（高血压、惊厥和颅内出血）是与输血相关的致命并发症。呋塞米已被广泛用于预防继发于容量超负荷的高血压，但支持证据尚不明确。本研究旨在评估呋塞米预防确诊为TDT的儿童和青年容量超负荷的疗效。
方法
招募确诊为TDT的患者并随机分为输血前接受呋塞米组或输血前不接受呋塞米组。经过3周至4个月的洗脱期后，这些患者根据研究的交叉设计接受交替治疗方案。在每次输血前后测量包括血压和NT-proBNP水平在内的临床和实验室参数。分析两个随机分组之间这些参数的差异及其潜在相关因素。
结果
总共30例接受60次红细胞输血的患者纳入本研究。所有患者均被随机分组，交叉设计为输血前接受或不接受呋塞米。未观察到输血反应、容量超负荷症状和HCC综合征。输血前使用呋塞米与输血前后收缩压差异之间未发现统计学显著相关性（输血前使用呋塞米时收缩压差异为2 mmHg，未输血前使用呋塞米时为1.5 mmHg；P值 = 0.721），以及输血前使用呋塞米与输血前后NT-proBNP水平差异之间也未发现相关性（输血前使用呋塞米时NT-proBNP水平差异为 -3.8 pg/mL，未输血前使用呋塞米时为 -2.4 pg/mL；P值 = 0.490）。即使在NT-proBNP水平较高的选定患者中也未观察到显著相关性（P值 = 0.262）。分析了影响输血前后NT-proBNP水平差异的相关因素，这些因素均未对水平差异产生影响。
结论
许多机构已将呋塞米纳入标准输血指南。我们的研究提供了重要证据，表明该药物在预防容量超负荷方面不必要使用，尤其是在儿科和青年TDT患者中。
泰国临床试验注册中心TCTR编号：TCTR20180209001。于2018年2月6日注册，https://www.clinicaltrials.in.th/ 。
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