Livingston Michael H, Mahant Sanjay, Ratjen Felix, Connolly Bairbre L, Thorpe Kevin, Mamdani Muhammad, Maclusky Ian, Laberge Sophie, Giglia Lucy, Walton J Mark, Yang Connie L, Roberts Ashley, Shawyer Anna C, Brindle Mary, Parsons Simon J, Stoian Cristina A, Cohen Eyal
McMaster Children's Hospital, McMaster University, 1280 Main Street West, Hamilton, ON, L8S 4K1, Canada.
The Hospital for Sick Children, Department of Pediatrics, University of Toronto, 555 University Avenue, Toronto, ON, M5G 1X8, Canada.
Trials. 2017 Jun 24;18(1):293. doi: 10.1186/s13063-017-2026-0.
A randomized controlled trial of adults with empyema recently demonstrated decreased length of stay in hospital in patients treated with intrapleurally administered dornase alfa and fibrinolytics compared to fibrinolytics alone. Whether this treatment strategy is safe and effective in children remains unknown.
METHODS/DESIGN: This study protocol is for a superiority, placebo-controlled, parallel-design, multicenter randomized controlled trial. The participants are previously well children admitted to a children's hospital with a diagnosis of empyema requiring chest tube insertion and fibrinolytics administered intrapleurally. Children will be randomized after the treating physician has decided that pleural drainage is required but prior to chest tube insertion. After chest tube insertion, participants in the treatment group will receive intrapleurally administered tissue plasminogen activator (tPA) 4 mg followed by dornase alfa 5 mg. Participants in the placebo group will receive tPA 4 mg followed by normal saline. Study treatments will be administered once daily for 3 days. All participants, parents or caregivers, clinicians, and research personnel will remain blinded. The primary outcome is length of stay from chest tube insertion to discharge from hospital. Secondary outcomes include time to meeting discharge criteria, chest tube duration, fever duration, need for additional procedures, adverse events, hospital readmission, cost of hospitalization, and mortality.
This multicenter randomized controlled trial will assess the safety, effectiveness, and cost-effectiveness of combined treatment with dornase alfa and fibrinolytics compared to fibrinolytics alone for the treatment of empyema in children.
ClinicalTrials.gov: NCT01717742 . Registered on 8 October 2012.
一项针对成人脓胸患者的随机对照试验最近表明，与单独使用纤维蛋白溶解剂相比，胸膜内注射多奈哌齐和纤维蛋白溶解剂治疗的患者住院时间缩短。这种治疗策略在儿童中是否安全有效尚不清楚。
方法/设计：本研究方案为一项优效性、安慰剂对照、平行设计、多中心随机对照试验。参与者为之前健康的儿童，因诊断为脓胸入住儿童医院，需要插入胸管并胸膜内注射纤维蛋白溶解剂。在主治医生决定需要进行胸膜引流但在插入胸管之前，儿童将被随机分组。插入胸管后，治疗组的参与者将接受胸膜内注射组织纤溶酶原激活剂（tPA）4mg，随后注射多奈哌齐5mg。安慰剂组的参与者将接受tPA 4mg，随后注射生理盐水。研究治疗将每日给药一次，持续3天。所有参与者、家长或护理人员、临床医生和研究人员都将保持盲态。主要结局是从插入胸管到出院的住院时间。次要结局包括达到出院标准的时间、胸管留置时间、发热持续时间、是否需要额外的操作、不良事件、再次住院、住院费用和死亡率。
这项多中心随机对照试验将评估与单独使用纤维蛋白溶解剂相比，联合使用多奈哌齐和纤维蛋白溶解剂治疗儿童脓胸的安全性、有效性和成本效益。
ClinicalTrials.gov：NCT01717742。于2012年10月8日注册。
