Patient-important outcomes in randomized controlled trials in critically ill patients: a systematic review

Abstract

BackgroundIntensivists’ clinical decision making pursues two main goals for patients: to decrease mortality and to improve quality of life and functional status in survivors. Patient-important outcomes are gaining wide acceptance in most fields of clinical research. We sought to systematically review how well patient-important outcomes are reported in published randomized controlled trials (RCTs) in critically ill patients.MethodsLiterature search was conducted to identify eligible trials indexed from January to December 2013. Articles were eligible if they reported an RCT involving critically ill adult patients. We excluded phase II, pilot and physiological crossover studies. We assessed study characteristics. All primary and secondary outcomes were collected, described and classified using six categories of outcomes including patient-important outcomes (involving mortality at any time on the one hand and quality of life, functional/cognitive/neurological outcomes assessed after ICU discharge on the other).ResultsOf the 716 articles retrieved in 2013, 112 RCTs met the inclusion criteria. Most common topics were mechanical ventilation (27%), sepsis (19%) and nutrition (17%). Among the 112 primary outcomes, 27 (24%) were patient-important outcomes (mainly mortality, 21/27) but only six (5%) were patient-important outcomes besides mortality assessed after ICU discharge (functional disability = 4; quality of life = 2). Among the 598 secondary outcomes, 133 (22%) were patient-important outcomes (mainly mortality, 92/133) but only 41 (7%) were patient-important outcomes besides mortality assessed after ICU discharge (quality of life = 20, functional disability = 14; neurological/cognitive performance = 5; handicap = 1; post-traumatic stress = 1). Seventy-three RCTs (65%) reported at least one patient-important outcome but only 11 (10%) reported at least one patient-important outcome besides mortality assessed after ICU discharge.ConclusionPatient-important outcomes are rarely primary outcomes in RCTs in critically ill patients published in 2013. Among them, mortality accounted for the majority. We promote the use of patient-important outcomes in critical care trials.Electronic supplementary materialThe online version of this article (doi:10.1186/s13613-017-0243-z) contains supplementary material, which is available to authorized users.

Tracheostomy in patients with acute respiratory distress syndrome is not related to quality of life, symptoms of psychiatric disorders or return-to-work: the prospective DACAPO cohort study

“…For all 1205 patients, the median duration of mechanical ventilation was 16 days (IQR 10-27) and that of ICU length of stay 22 days (IQR 14-36). The median duration of ventilation (20 days [IQR [13][14][15][16][17][18][19][20][21][22][23][24][25][26][27][28][29] vs. 11 days [IQR 6-16], p < 0.001) and ICU stay (p < 0.001, Table 1) was twice as long for tracheotomised patients. Primary 877 ARDS patients survived and discharged from ICU.…”

“…A current systematic review of patient-important outcomes for critically ill patients in randomised controlled clinical studies has shown ICU mortality to be the most frequently measured clinical outcome. Only 10% of studies have included at least one patient-important outcome in addition to mortality after ICU discharge [26]. It should be noted that, in contrast to mortality, HRQoL is a complex construct that contains individual aspects with multiple dimensions, often operationalised as social, somatic and psychological variables [4].…”

The present and future of cardiac arrest care: international experts reach out to caregivers and healthcare authorities

“…Outcomes preferred by investigators may not be what is important to patients. Trials involving critically ill patients rarely measure patient-reported outcomes as the primary or secondary outcome measure (5 vs. 22%, respectively) [96]. Patients and their family members involved in the Core Outcomes Set for Cardiac Arrest (COSCA) process identified survival, neurological function and HRQoL as the preferred outcome measures [97].…”

Understanding and Enhancing Sepsis Survivorship. Priorities for Research and Practice

“…However, guidelines have traditionally focused on early recognition and management, not mitigation of longer-term sequelae 19 . Likewise, clinical trials typically use shorter-term mortality endpoints and only rarely collect data on functional outcomes or quality of life 20,21 . Perhaps not surprisingly given the lack of attention to sepsis survivorship, many patients report dissatisfaction with follow-up care after hospitalization 22 .…”