Valerieva Anna, Staevska Maria T, Grivcheva-Panovska Vesna, Jesenak Milos, Kőhalmi Kinga Viktória, Hrubiskova Katarina, Zanichelli Andrea, Bellizzi Luca, Relan Anurag, Hakl Roman, Farkas Henriette
Department of Allergology, Medical University of Sofia, Sofia, Bulgaria.
PHI University Clinic of Dermatology, School of Medicine, University Saints Cyril and Methodius, Skopje, Macedonia.
World Allergy Organ J. 2021 Apr 22;14(4):100535. doi: 10.1016/j.waojou.2021.100535. eCollection 2021 Apr.
Hereditary angioedema (HAE) due to C1 esterase inhibitor deficiency (C1-INH-HAE) is characterized by recurrent swelling attacks. A European treatment registry was established to review the adverse event profile and efficacy of recombinant human C1 esterase inhibitor (rhC1-INH) for HAE attacks.
Individuals with C1-INH-HAE were enrolled following a decision to treat with rhC1-INH and provision of written informed consent. Medical history and baseline HAE information were collected at screening. Healthcare providers entered data on HAE attacks, response to treatment, and adverse events using a web-based questionnaire.
From July 1, 2011, through December 1, 2019, 71 patients with C1-INH-HAE (30 male/41 female; mean age, 47.3 years; age range, 19-78 years) in 9 countries reported 2356 attacks and were treated with rhC1-INH. Before registry entry, patients, including 20 (28.2%) who were on maintenance therapy/prophylaxis at registry enrollment, experienced a mean of 25 HAE attacks per year (median, 16 [range, 0-185]). Most treated HAE attacks were abdominal (46.1%), followed by peripheral (38.3%), oro-facial-pharyngeal (14.8%), urogenital (3.2%), and laryngeal (2.6%). The mean rhC1-INH dose was 3307 U (43.3 U/kg). Patients reported symptom improvement within 4 h for 97.8% of attacks (2305/2356) with rhC1-INH; most attacks (99.8%; 2351/2356) required only 1 dose. Five attacks were treated with a second dose (total rhC1-INH dose administered for attack, 4200 U). No hypersensitivity, thrombotic/thromboembolic events, or drug-related serious adverse events were reported.
The rhC1-INH treatment registry provided real-world data on the treatment of 2356 HAE attacks that were consistent with clinical trial data of rhC1-INH in patients with C1-INH-HAE.
由于C1酯酶抑制剂缺乏导致的遗传性血管性水肿(HAE)(C1-INH-HAE)的特征是反复出现肿胀发作。建立了一个欧洲治疗登记处,以审查重组人C1酯酶抑制剂(rhC1-INH)治疗HAE发作的不良事件情况和疗效。
决定使用rhC1-INH治疗并提供书面知情同意书后,招募C1-INH-HAE患者。在筛查时收集病史和HAE基线信息。医疗保健提供者使用基于网络的问卷输入有关HAE发作、治疗反应和不良事件的数据。
从2011年7月1日至2019年12月1日,9个国家的71例C1-INH-HAE患者(30例男性/41例女性;平均年龄47.3岁;年龄范围19 - 78岁)报告了2356次发作,并接受了rhC1-INH治疗。在进入登记处之前,患者,包括登记入组时正在接受维持治疗/预防的20例(28.2%),每年平均经历25次HAE发作(中位数为16次[范围0 - 185次])。大多数接受治疗的HAE发作是腹部发作(46.1%),其次是外周发作(38.3%)、口面部-咽部发作(14.8%)、泌尿生殖系统发作(3.2%)和喉部发作(2.6%)。rhC1-INH的平均剂量为3307 U(43.3 U/kg)。患者报告rhC1-INH治疗的发作中有97.8%(2305/2356)在4小时内症状改善;大多数发作(99.8%;2351/2356)仅需1剂。5次发作接受了第二剂治疗(发作时rhC1-INH的总给药剂量为4200 U)。未报告过敏反应、血栓形成/血栓栓塞事件或与药物相关的严重不良事件。
rhC1-INH治疗登记处提供了关于2356次HAE发作治疗的真实世界数据,这些数据与rhC1-INH在C1-INH-HAE患者中的临床试验数据一致。