一种用于评估多囊肝病患者报告症状的疾病特异性问卷的开发与验证

Development and Validation of a Disease-Specific Questionnaire to Assess Patient-Reported Symptoms in Polycystic Liver Disease.

作者信息

Neijenhuis Myrte K, Gevers Tom J G, Hogan Marie C, Kamath Patrick S, Wijnands Titus F M, van den Ouweland Ralf C P M, Edwards Marie E, Sloan Jeff A, Kievit Wietske, Drenth Joost P H

机构信息

Department of Gastroenterology and Hepatology, Radboud University Nijmegen Medical Center, Nijmegen, The Netherlands.

Division of Nephrology and Hypertension, Department of Internal Medicine, Mayo Clinic, Rochester, Minnesota.

出版信息

Hepatology. 2016 Jul;64(1):151-60. doi: 10.1002/hep.28545. Epub 2016 Apr 15.

DOI:10.1002/hep.28545

PMID:26970415

原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC4917464/

Abstract

UNLABELLED

Treatment of polycystic liver disease (PLD) focuses on symptom improvement. Generic questionnaires lack sensitivity to capture PLD-related symptoms, a prerequisite to determine effectiveness of therapy. We developed and validated a disease-specific questionnaire that assesses symptoms in PLD (PLD-Q). We identified 16 PLD-related symptoms (total score 0-100 points) by literature review and interviews with patients and clinicians. The developed PLD-Q was validated in Dutch (n = 200) and United States (US; n = 203) PLD patients. We assessed the correlation of PLD-Q total score with European Organization for Research and Treatment of Cancer (EORTC) symptom scale, global health visual analogue scale (VAS) of EQ-5D, and liver volume. To test discriminative validity, we compared PLD-Q total scores of patients with different PLD severity stages (Gigot classification) and PLD-Q total scores of PLD patients with general controls and polycystic kidney disease patients without PLD. Reproducibility was tested by comparing original test scores with 2-week retest scores. In total, 167 Dutch and 124 US patients returned the questionnaire. Correlation between PLD-Q total score and EORTC symptom scale (The Netherlands [NL], r = 0.788; US, r = 0.811) and global health VAS (NL, r = -0.517; US, r = -0.593) was good. There was no correlation of PLD-Q total score with liver volume (NL, r = 0.138; P = 0.236; US, r = 0.254; P = 0.052). Gigot type III individuals scored numerically higher than type II patients (NL, 46 vs. 40; P = 0.089; US, 48 vs. 36; P = 0.055). PLD patients scored higher on the PLD-Q total score than general controls (NL, 42 vs. 17; US, 40 vs. 13 points) and polycystic kidney disease patients without PLD (22 points). Reproducibility of PLD-Q was excellent (NL, r = 0.94; US, 0.96).

CONCLUSION

PLD-Q is a valid, reproducible, and sensitive disease-specific questionnaire that can be used to assess PLD-related symptoms in clinical care and future research. (Hepatology 2016;64:151-160).

摘要

未标注

多囊肝病（PLD）的治疗重点在于改善症状。通用问卷缺乏捕捉PLD相关症状的敏感性，而这是确定治疗效果的先决条件。我们开发并验证了一种评估PLD症状的疾病特异性问卷（PLD-Q）。我们通过文献综述以及与患者和临床医生的访谈，确定了16种PLD相关症状（总分0 - 100分）。所开发的PLD-Q在荷兰（n = 200）和美国（n = 203）的PLD患者中进行了验证。我们评估了PLD-Q总分与欧洲癌症研究与治疗组织（EORTC）症状量表、EQ-5D的整体健康视觉模拟量表（VAS）以及肝脏体积之间的相关性。为了测试区分效度，我们比较了不同PLD严重程度阶段（吉戈分类）患者的PLD-Q总分，以及PLD患者与一般对照和无PLD的多囊肾病患者的PLD-Q总分。通过比较原始测试分数与两周后复测分数来测试可重复性。总共有167名荷兰患者和124名美国患者返回了问卷。PLD-Q总分与EORTC症状量表（荷兰[r = 0.788]；美国[r = 0.811]）以及整体健康VAS（荷兰[r = -0.517]；美国[r = -0.593]）之间的相关性良好。PLD-Q总分与肝脏体积无相关性（荷兰[r = 0.138]；P = 0.236；美国[r = 0.254]；P = 0.052）。吉戈III型个体的得分在数值上高于II型患者（荷兰，46对40；P = 0.089；美国，48对36；P = 0.055）。PLD患者在PLD-Q总分上的得分高于一般对照（荷兰，42对17；美国，40对13分）和无PLD的多囊肾病患者（22分）。PLD-Q的可重复性极佳（荷兰，r = 0.94；美国，0.96）。