Petraroli Angelica, Squeglia Veronica, Di Paola Nadia, Barbarino Alessandro, Bova Maria, Spanò Rosanna, Marone Gianni, Triggiani Massimo
Department of Translational Medical Sciences, University of Naples Federico II, Naples, Italy.
Int Arch Allergy Immunol. 2015;166(4):259-66. doi: 10.1159/000381341. Epub 2015 Apr 28.
Attacks of hereditary angioedema with C1 inhibitor deficiency (C1-INH-HAE) are commonly treated in the emergency department. Self-administration is emerging as an effective treatment option. In this study, we assessed the impact of home therapy with plasma-derived C1 esterase inhibitor (pdC1-INH) concentrate on treatment outcomes and costs.
This is an observational study in C1-INH-HAE patients who switched to home therapy with pdC1-INH (Berinert®) after learning intravenous self-infusion in a training course at a center in Southern Italy. Before starting home therapy and after the first year of home therapy, patients were interviewed about their treatment and outcomes during the prior 12 months. Annual costs were analyzed by cost minimization from the Italian health care payer perspective and the societal perspective. Outcomes and costs before and after the switch to home therapy were compared.
The training course was attended by 36 participants, 17 (47.2%) of whom decided to switch to home therapy. This therapy was associated with a significant decrease in the mean annual number of hospitalizations (16.8 vs. 2.1, p = 0.003) and missed work/school days (20.3 vs. 7.1, p = 0.037) compared to conventional treatment. The times from symptom onset to treatment administration and from treatment administration to symptom improvement/resolution were not significantly different between the two strategies. The mean annual per-patient costs decreased with home therapy from EUR 30,010.57 to EUR 26,621.16 (11.3% saving) and from EUR 29,309.34 to EUR 26,522.04 (9.5% saving) from the societal and payer perspective, respectively.
Home therapy with pdC1-INH is a feasible strategy for the management of C1-INH-HAE and may result in cost savings.
C1 抑制剂缺乏型遗传性血管性水肿（C1-INH-HAE）发作通常在急诊科进行治疗。自我给药正成为一种有效的治疗选择。在本研究中，我们评估了使用血浆源性 C1 酯酶抑制剂（pdC1-INH）浓缩物进行家庭治疗对治疗结果和成本的影响。
这是一项针对 C1-INH-HAE 患者的观察性研究，这些患者在意大利南部一个中心参加培训课程学习静脉自我输注后，改用 pdC1-INH（Berinert®）进行家庭治疗。在开始家庭治疗前以及家庭治疗的第一年结束后，对患者进行访谈，了解他们在之前 12 个月的治疗情况和结果。从意大利医疗保健支付方和社会角度，通过成本最小化分析年度成本。比较改用家庭治疗前后的结果和成本。
36 名参与者参加了培训课程，其中 17 名（47.2%）决定改用家庭治疗。与传统治疗相比，这种治疗使平均每年住院次数显著减少（16.8 次对 2.1 次，p = 0.003），误工/缺课天数也显著减少（20.3 天对 7.1 天，p = 0.037）。两种治疗策略在症状发作到开始治疗的时间以及治疗开始到症状改善/缓解的时间方面无显著差异。从社会和支付方角度来看，家庭治疗使每位患者的平均年度成本分别从 30,010.57 欧元降至 26,621.16 欧元（节省 11.3%）和从 29,309.34 欧元降至 26,522.04 欧元（节省 9.5%）。
使用 pdC1-INH 进行家庭治疗是管理 C1-INH-HAE 的一种可行策略，且可能节省成本。
