Menon Kusum, McNally James Dayre, Zimmerman Jerry J, Agus Michael S D, O'Hearn Katie, Watson R Scott, Wong Hector R, Duffett Mark, Wypij David, Choong Karen
1Division of Pediatric Critical Care, Children's Hospital of Eastern Ontario, University of Ottawa, Ottawa, ON. 2Division of Critical Care, Seattle Children's Hospital, University of Washington, Seattle, WA. 3Division of Critical Care Medicine, Boston Children's Hospital, Harvard Medical School, Boston, MA. 4Division of Critical Care Medicine, Cincinnati Children's Hospital Medical Center, University of Cincinnati, Cincinnati, OH. 5Department of Pediatrics, McMaster Children's Hospital, McMaster University, Hamilton, ON.
Pediatr Crit Care Med. 2017 Mar;18(3):e146-e154. doi: 10.1097/PCC.0000000000001078.
To evaluate all published pediatric randomized controlled trials of patients with septic shock from any cause to examine the outcome measures used, the strengths and limitations of these measurements and whether the trial outcomes met feasibility criteria.
We used a previously published database of pediatric critical care randomized controlled trials (PICUtrials.net) derived from searches of MEDLINE, EMBASE, LILACS, and CENTRAL.
We included randomized controlled trials of interventions to children admitted to a PICU with septic or dengue hemorrhagic shock which were published in English.
Study characteristics and outcomes were retrieved by two independent reviewers with disagreement being resolved by a third reviewer. We defined feasibility as 1) recruitment of at least 90% of the targeted sample size and agreement of the observed outcome rate in the control group with the rate used for the sample size calculation to within 10% or 2) finding of a statistically significant difference in an interim or final analysis.
Nineteen of 321 identified articles were selected for review. Fourteen of 19 studies (74%) provided an a priori definition of their primary outcome measure in their "Methods section." Mortality rate was the most commonly reported primary outcome (8/14; 57%), followed by duration of shock (4/14; 29%) followed by organ failure (1/14; 7%). Only three of 19 included trials met feasibility criteria.
Our review found that use of mortality alone as a primary outcome in pediatric septic shock trials was associated with significant limitations and that long-term patient-centered outcomes were not used in this setting. Composite outcomes incorporating mortality and long-term outcomes should be explored for use in future pediatric septic shock trials.
评估所有已发表的关于任何病因所致感染性休克患儿的儿科随机对照试验，以检查所使用的结局指标、这些测量方法的优缺点，以及试验结局是否符合可行性标准。
我们使用了先前发表的儿科重症监护随机对照试验数据库（PICUtrials.net），该数据库来自对MEDLINE、EMBASE、LILACS和CENTRAL的检索。
我们纳入了以英文发表的、针对入住儿科重症监护病房（PICU）的感染性或登革出血热休克患儿进行干预的随机对照试验。
由两名独立的审阅者检索研究特征和结局，如有分歧则由第三名审阅者解决。我们将可行性定义为：1）招募至少90%的目标样本量，且对照组观察到的结局发生率与样本量计算所使用的发生率相差在10%以内；或2）在中期或最终分析中发现具有统计学意义的差异。
从321篇已识别的文章中选择了19篇进行综述。19项研究中的14项（74%）在其“方法部分”对主要结局指标进行了预先定义。死亡率是最常报告的主要结局（8/14；57%），其次是休克持续时间（4/14；29%），然后是器官衰竭（1/14；7%）。19项纳入试验中只有3项符合可行性标准。
我们的综述发现，在儿科感染性休克试验中仅将死亡率作为主要结局存在显著局限性，且在这种情况下未使用以患者为中心的长期结局。未来的儿科感染性休克试验应探索使用包含死亡率和长期结局的综合结局指标。
