背景:许多临床试验使用系统方法来监测不良事件(AE)并确定等级(严重程度),期待,以及与临床医生确定的治疗的相关性。然而,在这个过程中,患者的观点仍然缺乏。
目的:在一项泌尿外科手术试验中,比较临床医生与患者的AE严重程度分级。次要目标是评估患者AE分级与决策和生活质量结果之间的关联,并确定患者观点是否随时间变化。
方法:这是一项计划中的补充研究,“不良事件报告中的患者观点”(PPAR),一项随机试验,比较3种手术方法治疗阴道顶端脱垂。在家长审判中,每6个月根据标准化方案收集患者经历的AE,其中临床医生对AE严重程度进行分级(轻度,中度,严重/危及生命)。在这项子研究中,我们获得了19个预定的“PPARAE”的额外纵向患者视角。患者提供了他们自己的严重程度分级(轻度,中度,严重/非常严重/危及生命)在初始评估以及术后12和36个月。临床医生和患者被掩盖了彼此的报告。主要结果是评分者之间的协议(kappa统计,κ)用于初始临床医生和患者评估之间的AE严重程度,结合轻度和中度的患者等级。不良事件严重程度与决策后悔量表(DRS)之间的关联对决策量表(SDS)的满意度,简短形式的健康调查-12(SF-12),使用连续量表的Spearman相关系数(ρ)评估患者-总体改善印象(PGI-I)评分,PGI-I的Mantel-Haenszel卡方检验,和T检验或卡方检验,比较严重与其他等级的评估。为了描述患者观点随时间的变化,使用加权kappa系数估算了观察者内的AE严重程度等级随时间的一致性.
结果:在360名随机分组的患者中,219例(61%)经历了总共527例PPAR不良事件(91%为中度,9%为严重/威胁生命的临床医生分级)。患者平均年龄为67岁,87%是白人,和12%的西班牙裔。报告任何PPAR事件的患者,最常见的是尿路感染(61%),从头尿失禁(35%),压力性尿失禁(22%),和大便失禁(13%)。临床医生和参与者严重程度分级之间的总体一致性较差(κ=0.24(95CI0.14,0.34)。在414名临床医生中,患者120级(29%)轻度,80(19%)严重。在临床医生评定为严重的39个不良事件中,患者15级(38%)轻度或中度。报告的最严重AE的初始患者分级与较差的DRS轻度相关(ρ=0.2,p=0.01),SF-12(ρ=-0.24,p<0.01)和PGI-I(p<0.01)。AE严重程度与SDS之间没有关联。最初分级为“严重”的患者更后悔,生活质量较低,与严重程度最差的人相比,全球对健康的印象较差(p<0.05)。在12个月(κ=0.48(95%CI0.39,0.58))和36个月(κ=0.45(95%CI0.37,0.53))时,患者的初始严重程度与后来的时间点之间的一致性相当。
结论:临床医生和患者对AE严重程度的看法是不一致的。从患者的角度来看,更严重的严重程度与以患者为中心的结果相关。包括患者视角提供了用于评估外科手术的附加信息。
Many clinical trials use systematic methodology to monitor adverse events and determine grade (severity), expectedness, and relatedness to treatments as determined by clinicians. However, patient perspectives are often not included in this process.
This study aimed to compare clinician vs patient grading of adverse event severity in a urogynecologic surgical trial. Secondary objectives were to estimate the association of patient grading of adverse events with decision-making and quality of life outcomes and to determine if patient perspective changes over time.
This was a planned supplementary study (Patient Perspectives in Adverse Event Reporting [PPAR]) to a randomized trial comparing 3 surgical approaches to vaginal apical prolapse. In the parent trial, adverse events experienced by patients were collected per a standardized protocol every 6 months during which clinicians graded adverse event severity (mild, moderate, severe/life-threatening). In this substudy, we obtained additional longitudinal patient perspectives for 19 predetermined \"PPAR adverse events.\" Patients provided their own severity grading (mild, moderate, severe/very severe/life-threatening) at initial assessment and at 12 and 36 months postoperatively. Clinicians and patients were masked to each other\'s reporting. The primary outcome was the interrater agreement (kappa statistic) for adverse event severity between the initial clinician and patient assessment, combining patient grades of mild and moderate. The association between adverse event severity and the Decision Regret Scale, Satisfaction with Decision Scale, the 12-Item Short-Form Health Survey, and Patient Global Impression of Improvement scores was assessed using the Spearman correlation coefficient (ρ) for continuous scales, the Mantel-Haenszel chi-square test for Patient Global Impression of Improvement, and t tests or chi-square tests comparing the assessments of patients who rated their adverse events or symptoms as severe with those who gave other ratings. To describe patient perspective changes over time, the intraobserver agreement was estimated for adverse event severity grade over time using weighted kappa coefficients.
Of the 360 randomly assigned patients, 219 (61%) experienced a total of 527 PPAR adverse events (91% moderate and 9% severe/life-threatening by clinician grading). Mean patient age was 67 years; 87% were White and 12% Hispanic. Among the patients reporting any PPAR event, the most common were urinary tract infection (61%), de novo urgency urinary incontinence (35%), stress urinary incontinence (22%), and fecal incontinence (13%). Overall agreement between clinician and participant grading of severity was poor (kappa=0.24 [95% confidence interval, 0.14-0.34]). Of the 414 adverse events that clinicians graded as moderate, patients graded 120 (29%) as mild and 80 (19%) as severe. Of the 39 adverse events graded as severe by clinicians, patients graded 15 (38%) as mild or moderate. Initial patient grading of the most severe reported adverse event was mildly correlated with worse Decision Regret Scale (ρ=0.2; P=.01), 12-Item Short-Form Health Survey (ρ=-0.24; P<.01), and Patient Global Impression of Improvement (P<.01) scores. There was no association between adverse event severity and Satisfaction with Decision Scale score. Patients with an initial grading of \"severe\" had more regret, lower quality of life, and poorer global impressions of health than those whose worst severity grade was mild (P<.05). Agreement between the patients\' initial severity ratings and their ratings at 12 months (kappa=0.48 [95% confidence interval, 0.39-0.58]) and 36 months (kappa=0.45 [95% confidence interval, 0.37-0.53]) was fair.
Clinician and patient perceptions of adverse event severity are discordant. Worse severity from the patient perspective was associated with patient-centered outcomes. Including the patient perspective provides additional information for evaluating surgical procedures.