Penn State University, Hershey, Pennsylvania 17033, USA.
Allergy Asthma Proc. 2012 Jul-Aug;33(4):354-61. doi: 10.2500/aap.2012.33.3589.
Placebo-controlled studies established the efficacy of replacement therapy with C1 esterase inhibitor (C1-INH) concentrate for treating single acute hereditary angioedema (HAE) attacks, but only limited data from prospective studies are available on repeated treatment of successive HAE attacks. This study evaluates the association between repeated treatments with 20 U/kg of C1-INH concentrate (Berinert; CSL Behring, Marburg, Germany) for HAE attacks at any body location and treatment response. In a post hoc analysis of an open-label extension study (International Multicenter Prospective Angioedema C1-INH Trial [I.M.P.A.C.T.2]), the association between repeated treatment with C1-INH and times to onset of symptom relief and complete resolution of HAE symptoms was assessed in patients who were treated for at least 15 attacks by linear regression on the ordinal attack number. Eighteen patients received C1-INH concentrate for at least 15 HAE attacks over a mean duration of 34 months. Demographic and baseline characteristics of these patients were similar to those of all patients in the study. The distribution of body locations and the intensity of HAE attacks were similar for each of the first 15 attacks and subsequent attacks. The extent of previous use of C1-INH concentrate had no effect on the time to onset of symptom relief, the time to complete resolution of HAE symptoms, or the time between attacks treated with C1-INH concentrate; the median of individual linear regression coefficients was not statistically significantly different from 0. Treatment with 20 U/kg of C1-INH concentrate provided consistent treatment response in patients treated for multiple successive HAE attacks at any body location. (Clinicaltrials.gov identifier: NCT00292981).
安慰剂对照研究确立了 C1 酯酶抑制剂(C1-INH)浓缩物替代疗法治疗单一急性遗传性血管性水肿(HAE)发作的疗效,但只有有限的前瞻性研究数据可用于连续 HAE 发作的重复治疗。本研究评估了在任何身体部位接受 20 U/kg C1-INH 浓缩物(Berinert;CSL Behring,马尔堡,德国)重复治疗 HAE 发作与治疗反应之间的关联。在开放标签扩展研究(国际多中心前瞻性血管性水肿 C1-INH 试验[I.M.P.A.C.T.2])的事后分析中,通过线性回归对有序发作次数评估了在至少接受 15 次发作治疗的患者中,C1-INH 的重复治疗与症状缓解和 HAE 症状完全缓解的发作次数之间的关联。18 名患者接受 C1-INH 浓缩物治疗至少 15 次 HAE 发作,平均持续时间为 34 个月。这些患者的人口统计学和基线特征与研究中的所有患者相似。在每个前 15 次发作和随后的发作中,身体部位的分布和 HAE 发作的强度相似。先前使用 C1-INH 浓缩物的程度对症状缓解的发作时间、HAE 症状完全缓解的发作时间或接受 C1-INH 浓缩物治疗的发作时间均无影响;个体线性回归系数的中位数在统计学上与 0 无显著差异。在任何身体部位接受 20 U/kg C1-INH 浓缩物治疗的患者,在接受多次连续 HAE 发作治疗时,均能获得一致的治疗反应。(临床试验.gov 标识符:NCT00292981)。