Levi Marcel, Choi Goda, Picavet Charles, Hack C Erik
Department of Internal Medicine, Academic Medical Center, University of Amsterdam, The Netherlands.
J Allergy Clin Immunol. 2006 Apr;117(4):904-8. doi: 10.1016/j.jaci.2006.01.002. Epub 2006 Feb 14.
Administration of C1-inhibitor concentrate is effective for prophylaxis and treatment of severe angioedema attacks caused by C1-inhibitor deficiency. The concentrate should be administered intravenously and hence needs to be administered by health care professionals, which might cause considerable delay in treatment and inconvenience for patients.
The aim of this study was to investigate the feasibility, efficacy, and safety of on-demand and prophylactic self-administration of C1-inhibitor concentrate in patients with frequent attacks of angioedema.
Patients with hereditary or acquired C1-inhibitor deficiency who had very frequent angioedema attacks were trained to self-administer C1-inhibitor concentrate. The study consisted of 31 patients using on-demand treatment and 12 patients using prophylaxis with C1-inhibitor concentrate. Mean follow-up was 3.5 years.
All patients were capable of self-administering the concentrate, with technical failure rates of self-injection being less than 2%. Times between the onset of the attack and the initiation of relief or complete resolution of symptoms in the on-demand group were significantly shortened (2.2 hours and 7.9 hours, respectively) compared with the situation before the start of self-administration. In the prophylaxis group self-administration of C1-inhibitor concentrate decreased the angioedema attack rate from 4.0 to 0.3 attacks per month.
Intravenous self-administration of C1-inhibitor concentrate is a feasible and safe option and results in more rapid and more effective treatment or prevention of severe angioedema attacks in patients with C1-inhibitor deficiency.
Self-administration of C1-inhibitor concentrate could be a valuable and convenient treatment modality to prevent or treat angioedema attacks in patients with C1-inhibitor deficiency.
给予C1抑制剂浓缩物对预防和治疗由C1抑制剂缺乏引起的严重血管性水肿发作有效。该浓缩物应静脉给药，因此需要由医护人员进行给药，这可能会导致治疗的显著延迟并给患者带来不便。
本研究的目的是调查在血管性水肿频繁发作的患者中按需和预防性自我注射C1抑制剂浓缩物的可行性、有效性和安全性。
对患有遗传性或获得性C1抑制剂缺乏且血管性水肿发作非常频繁的患者进行培训，使其能够自我注射C1抑制剂浓缩物。该研究包括31例采用按需治疗的患者和12例采用C1抑制剂浓缩物进行预防的患者。平均随访时间为3.5年。
所有患者均能够自我注射浓缩物，自我注射的技术失败率低于2%。与开始自我给药前的情况相比，按需治疗组中发作开始至症状缓解或完全消退的时间显著缩短（分别为2.2小时和7.9小时）。在预防组中，自我注射C1抑制剂浓缩物使血管性水肿发作率从每月4.0次降至0.3次。
静脉自我注射C1抑制剂浓缩物是一种可行且安全的选择，可更快速有效地治疗或预防C1抑制剂缺乏患者的严重血管性水肿发作。
自我注射C1抑制剂浓缩物可能是预防或治疗C1抑制剂缺乏患者血管性水肿发作的一种有价值且方便的治疗方式。
