PDF(Pubmed)
Abstract：
Hereditary angioedema due to C1 inhibitor deficiency (HAE) is a rare inborn error of immunity that presents with episodic swelling. Management is multifaceted and includes on-demand treatment of swelling episodes, short-term prophylaxis to prevent swelling episodes from procedures, and long-term prophylaxis (LTP) to prevent angioedema on an ongoing basis. All approved on-demand therapies are parenteral, necessitating patient training for home administration, particularly intravenous C1 inhibitor. These complexities can result in care gaps for rural HAE patients. We conducted a cross-sectional study at our Angioedema Center of Reference and Excellence to assess the care provided to urban and rural patients. The proportion of patients receiving LTP, proportion of patients diagnosed as children, and disease control measured using the Angioedema Control Test (AECT) were collected. Logistic and Poisson regression models adjusted for age and sex were used to compare the two groups. The proportion using LTP was similar at 62% and 61% in urban and rural patients, respectively (odds ratio [OR] 1.01 (CI 95% 0.34-2.99)). Among urban patients, 52% were diagnosed as children compared to 60% among rural residents (1.43 (0.37-5.56)). The mean (IQR) AECT score was 14.0 (8.5-15.5) in urban patients and 13.0 (10.0-14.0) in rural patients (Poisson β -0.001 (-0.23-0.23). These data indicate that rural patients received similar high-quality care. We attribute these findings to the centralized care model employed in which HAE patients in the region are seen at a single comprehensive care clinic.
摘要：
由于C1抑制剂缺乏症（HAE）引起的遗传性血管性水肿是一种罕见的先天性免疫错误，表现为偶发性肿胀。管理是多方面的，包括按需治疗肿胀发作，短期预防以防止手术引起的肿胀发作，和长期预防（LTP）以持续预防血管性水肿。所有批准的按需治疗都是肠胃外治疗，需要对病人进行家庭管理培训，特别是静脉内C1抑制剂。这些复杂性可能导致农村HAE患者的护理差距。我们在我们的血管性水肿参考和卓越中心进行了一项横断面研究，以评估为城市和农村患者提供的护理。接受LTP的患者比例，被诊断为儿童的患者比例，收集使用血管性水肿控制测试(AECT)测量的疾病控制。采用经年龄和性别调整的Logistic和泊松回归模型比较两组。城乡患者使用LTP的比例相似，分别为62%和61%，分别（比值比[OR]1.01（CI95%0.34-2.99））。在城市患者中，52％被诊断为儿童，而农村居民为60％（1.43（0.37-5.56））。城市患者的平均（IQR）AECT评分为14.0（8.5-15.5），农村患者为13.0（10.0-14.0）（泊松β-0.001（-0.23-0.23）。这些数据表明，农村患者接受了类似的高质量护理。我们将这些发现归因于所采用的集中护理模式，该模式在单个综合护理诊所中看到该地区的HAE患者。
