Shamaa Omar, Ahmed Abdul, Rupp Lora, Trudeau Sheri, Gordon Stuart C
Gastroenterology and Hepatology, Henry Ford Health System, Detroit, USA.
Internal Medicine, Northwestern Medicine McHenry Hospital, McHenry, USA.
Cureus. 2024 Jul 17;16(7):e64753. doi: 10.7759/cureus.64753. eCollection 2024 Jul.
Ursodeoxycholic acid (UDCA) slows disease progression among patients with primary biliary cholangitis (PBC), yet not all patients receive this standard-of-care medication. Our study aims to identify reasons why PBC patients did not receive the recommended UDCA treatment.
Using medical record data collected by the Fibrotic Liver Disease (FOLD) Consortium for 2006-2016, we identified PBC patients from a single site with no UDCA therapy record. Two independent reviewers used a structured data collection instrument to systematically confirm and record the reasons for the lack of treatment.
Among 494 PBC patients (11% men and 13.2% Black patients) with a median follow-up of 5.2 years, 35 (7%) had never received UDCA (16% men and 24% Black patients). Of these, 18 (51%) had laboratory indications of PBC but were not formally diagnosed. Among the remaining 17 patients with recognized PBC, six were never offered UDCA, seven declined treatment, and four remained untreated despite being offered treatment. We did not find a statistically significant association between the lack of PBC diagnosis and treatment and patients' age (p = 0.139), gender (p = 0.222), race (p = 0.081), or insurance coverage (p = 0.456), perhaps due to our small sample size.
Multiple factors influencing the lack of evaluation and treatment in PBC patients were identified at the provider and patient levels. The most common reasons included financial barriers, loss to follow-up, severe decompensated disease at diagnosis, and lack of referral to specialists for further evaluation. Future interventions targeting modifiable provider and patient barriers may improve rates and timeliness of PBC diagnosis and treatment.
熊去氧胆酸（UDCA）可减缓原发性胆汁性胆管炎（PBC）患者的疾病进展，但并非所有患者都接受这种标准治疗药物。我们的研究旨在确定PBC患者未接受推荐的UDCA治疗的原因。
利用纤维化肝病（FOLD）联盟在2006年至2016年收集的病历数据，我们从单一地点识别出没有UDCA治疗记录的PBC患者。两名独立的评审员使用结构化数据收集工具系统地确认并记录未治疗的原因。
在494例PBC患者（11%为男性，13.2%为黑人患者）中，中位随访时间为5.2年，35例（7%）从未接受过UDCA治疗（16%为男性，24%为黑人患者）。其中，18例（51%）有PBC的实验室指标，但未被正式诊断。在其余17例确诊为PBC的患者中，6例从未被提供UDCA，7例拒绝治疗，4例尽管被提供了治疗但仍未接受治疗。我们没有发现PBC诊断和治疗的缺失与患者的年龄（p = 0.139）、性别（p = 0.222）、种族（p = 0.081）或保险覆盖情况（p = 0.456）之间存在统计学上的显著关联，这可能是由于我们的样本量较小。
在医疗服务提供者和患者层面发现了多种影响PBC患者缺乏评估和治疗的因素。最常见的原因包括经济障碍、失访、诊断时严重失代偿性疾病以及缺乏转诊至专科医生进行进一步评估。针对可改变的医疗服务提供者和患者障碍的未来干预措施可能会提高PBC诊断和治疗的比例及及时性。
