Ith C1-INH concentrate (Berinert? CSL Behring; or Cinryze? Shire; administered
Ith C1-INH concentrate (Berinert? CSL Behring; or Cinryze? Shire; administered by intravenous infusion); home-based therapy with icatibant (Firazyr? Shire; administered by subcutaneous injection); hospital-based therapy with C1-INH concentrate (Berinert?. In all groups, treatments were administered according to the manufacturer’s indications and local guidelines. The objectives of the study were: 1) to compare compliance with therapy and quality of life in patients treated at home vs. hospital at the end of the 6 month-observation period and; 2) to identify factors associated with the decision to adopt home therapy. The study was approved by the local institutional review board (Comitato Etico Universit?”Federico II”, Naples, Italy). All patients, or parents/legal guardians for minors, gave their informed consent to data collection and analysis.Self-administration trainingIcatibant is administered subcutaneously and patients are taught self-administration during regular visits to their physician, at the first prescription of this medication [22]. pdC1-INH concentrates are administered by intravenous infusion and patients require more extensive training before feeling comfortable with this administration route. As previously described, in 2010, our center initiated a training program to instruct patients on selfadministration of pdC1-INH concentrates by intravenous infusion [21]. The training course consisted of a theory session and a practical session during which participants could practice intravenous injection on a simulator arm. Participants were also instructed on how to behave in case of a laryngeal attack, or if unable to administer treatment, and were invited to keep a diary documenting attack characteristics, treatment administered, time from symptom onset to treatment administration, time from treatment administration to symptom resolution, and other relevant features of home therapy.AssessmentsPatients were visited at baseline (beginning of observation) and followed up for 6 months, and were interviewed once monthly during a phone call conducted by trained personnel. At baseline, demographic data were collected and recorded in a patient PubMed ID:https://www.ncbi.nlm.nih.gov/pubmed/28381880 chart designed for the study. Other data recorded in the patient chart wereSqueglia et al. Orphanet Journal of Rare Diseases (2016) 11:Page 3 ofthe number of attacks in the previous month, the number of treated attacks, the time from symptom onset to treatment administration, and the time from treatment administration to symptom resolution for each attack. These data were extracted from patients’ diaries. Disease severity was also established. To this purpose we used the general disease severity score developed by Bygum and coworkers [23]. This score ranges from 0 to 10 (10 corresponds to the highest disease severity) and is based on the age at disease onset, number of organs ever affected, and need for long-term prophylaxis (age at onset 0? years, 3 points; age at onset 6?0 years, 2 points; age at onset, 11?0 years, 1 point; age at onset > 20 years, 0 points; skin edema ever, 1 point; painful abdominal edema ever, 2 points; laryngeal edema ever, 2 points; other clinical manifestations, 1 point; long-term prophylaxis ever, 1 point). In contrast with other disease severity assessment tools, this score does not consider a specific time frame, and is based on medical records and a patient interview JWH-133 site thereby reducing recall bias and subjective interpretation. Compliance with therapy PubMed ID:https://www.ncbi.nlm.nih.gov/pubmed/28242652 was defined as.