Safety, effectiveness, and impact on quality of life of self-administration with plasma-derived nanofiltered C1 inhibitor (Berinert®) in patients with hereditary angioedema: the SABHA study

被引:25
作者
Zanichelli, Andrea [1 ]
Azin, Giulia Maria [1 ]
Cristina, Federico [1 ]
Vacchini, Romualdo [1 ]
Caballero, Teresa [2 ]
机构
[1] Univ Milan, ASST Fatebenefratelli Sacco, Dept Biomed & Clin Sci Luigi Sacco, Milan, Italy
[2] Hosp La Paz, Allergy Dept, Inst Hlth Res, Madrid, Spain
来源
ORPHANET JOURNAL OF RARE DISEASES | 2018年 / 13卷
关键词
Hereditary angioedema; C1 esterase inhibitor; Self-administration; Survey; Quality of life; TSQM; CONCENTRATE; MANAGEMENT; DEFICIENCY; DIAGNOSIS; THERAPY;
D O I
10.1186/s13023-018-0797-3
中图分类号
Q3 [遗传学];
学科分类号
071007 ; 090102 ;
摘要
Background: Hereditary angioedema with C1 inhibitor deficiency is a disabling, potentially fatal condition characterized by recurrent episodes of swelling. Self-treatment is recommended, in order to reduce admissions to the Emergency Room and the time between the onset of the attack and the treatment, resulting in a better treatment outcome and an improved quality of life (QoL). The purpose of this study is to assess the safety, tolerability, and effect on QoL of self-administration of pnf C1-INH for IV use (Berinert (R)). Methods: An observational, monocenter, prospective study was designed. Patients referring to a center for angioedema that attended two sessions of self-infusion training course in the period March 2014-July 2015 were enrolled in the study. The primary endpoint was to monitor the safety and feasibility of pnf C1-INH self-infusion. The secondary endpoint was to evaluate the effect of self-infusion on the QoL, by means of the HAE-QoL questionnaire and the need for access to Emergency Room for infusion of Berinert (R). Patients' medical history data were collected upon the first visit and questionnaires were filled after each attack treated with Berinert (R) (diary and Treatment Satisfaction Questionnaire for Medication) and upon the first visit and the follow-ups (HAE-QoL). Results: Twenty patients were enrolled (median age = 42, IQR: 39-49; 60% females). Fifteen patients completed the study. A total of 189 attacks were recorded (annual median rate of 4 attacks/patient). Patients waited a median of 2 h (IQR: 1-4) before self-administration, and the resolution of the attack occurred after a median of 6 h (IQR: 4-11). Most attacks were abdominal (39%) and peripheral (22%). 92% of the attacks were treated through self-/caregiver-administration. In most attacks no side effects were reported. The number of attacks with side effects decreased over time, from 37% to 13%. Global satisfaction grew over time during the study period, reaching statistical significance over the first 6 months. The median total HAE-QoL score at baseline was 86 (IQR: 76-103) and improved in a non-significant manner throughout the study period. 8% of the attacks treated with Berinert (R) required ER admission/healthcare professional help in the study period, compared with 100% in the 3 years before enrollment (p < 0.0001). Conclusions: Self-administration of pnf C1-INH is safe, and increases patients' confidence in the treatment, showing also a trend towards an improvement in QoL. It reduces the need for ER admission/healthcare professionals help for the acute attacks, as well as the related costs.
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页数:8
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