Institute of Cellular Medicine, Newcastle University, NIHR Biomedical Research Centre in Ageing-Liver Theme, Newcastle-upon-Tyne, UK.
Eur J Gastroenterol Hepatol. 2011 Jul;23(7):566-72. doi: 10.1097/MEG.0b013e3283470256.
To assess patient-reported functional ability and its relationship with symptoms in primary biliary cirrhosis (PBC).
Functional status was assessed in a representative cohort of 75 patients with PBC using the Patient-Reported Outcome Measure Information System Health-Assessment Questionnaire (PROMIS-HAQ) functional assessment tool and was related to both symptom severity at the point of assessment (assessed using the PBC-40 and Orthostatic Grading Scale) and symptom severity change over the previous 4 years. Functional status in the PBC group was compared with primary sclerosing cholangitis (cholestatic liver disease) and community controls.
Functional impairment at follow-up (PROMIS-HAQ) was substantial in PBC significantly higher than that in both primary sclerosing cholangitis and community controls. PROMIS-HAQ domain scores confirmed that patients with PBC had significant impairment in arising, eating, walking, reach and grip and activity, but not dressing or hygiene. Functional impairment correlated positively with greater PBC-40 Fatigue, Cognitive and Social and Emotional domains and higher orthostatic symptoms. Over 4 years, total symptom burden increased significantly (P=0.03). The predominant factor was rise in Cognitive domain scores indicating worsening cognitive symptoms (P<0.0001). Change in PBC-40 Cognitive, Social and Emotional scores (2005-2009) strongly predicted functional ability in 2009. Multivariate analysis confirmed that PROMIS-HAQ scores were predicted independently by PBC-40 Social and Emotional scores (P=0.02; β=0.3) and orthostatic symptoms (P=0.04; β=0.3).
PBC associates with substantial functional impairment. PBC symptom distribution evolves over time, with cognitive symptoms making ever-greater contribution to overall symptom burden. The major potentially modifiable determinant responsible for the functional impairment appears to be orthostatic symptoms.
评估原发性胆汁性肝硬化（PBC）患者的报告功能能力及其与症状的关系。
使用患者报告结局测量信息系统健康评估问卷（PROMIS-HAQ）功能评估工具，对 75 例 PBC 代表性队列患者的功能状态进行评估，并将其与评估时的症状严重程度（使用 PBC-40 和直立分级量表评估）以及过去 4 年的症状严重程度变化相关联。将 PBC 组的功能状态与原发性硬化性胆管炎（胆汁淤积性肝病）和社区对照进行比较。
PBC 患者在随访时（PROMIS-HAQ）的功能障碍明显，显著高于原发性硬化性胆管炎和社区对照组。PROMIS-HAQ 域评分证实，PBC 患者在起床、进食、行走、伸手和抓握以及活动方面存在显著障碍，但在穿衣或卫生方面没有障碍。功能障碍与 PBC-40 疲劳、认知和社会情感域以及更高的直立症状呈正相关。4 年来，总症状负担显著增加（P=0.03）。主要因素是认知域评分的升高，表明认知症状恶化（P<0.0001）。2005-2009 年 PBC-40 认知、社会和情感评分的变化强烈预测了 2009 年的功能能力。多变量分析证实，PROMIS-HAQ 评分独立地由 PBC-40 社会和情感评分（P=0.02；β=0.3）和直立症状（P=0.04；β=0.3）预测。
PBC 与严重的功能障碍相关。PBC 的症状分布随时间演变，认知症状对总症状负担的贡献越来越大。导致功能障碍的主要潜在可调节因素似乎是直立症状。
