Opinions of Turkish Hereditary Angioedema Patients Regarding the Use of C1 Inhibitor Concentrate in the Treatment of Acute Attacks

Abstract

Objective: No data exists about the perceptions of Turkish hereditary angioedema patients regarding the use of C1 inhibitor concentrate that has been used in the treatment of acute attacks for the last 7 years. Our aim was to evaluate patient opinions about the C1 inhibitor (C1-inh) concentrate and determine the level of satisfaction with this drug. Materials and Methods: Fifty-seven hereditary angioedema (HAE) patients who used C1-inh concentrate for their angioedema attacks were asked to complete a questionnaire which contained various questions related to demographic information, clinical features, and experiences with C1-inh concentrate use. Results: Sixty-five % of the patients were female and 94.7% of the patients had type 1 HAE. The mean curent age and age of diagnosis were 38.11±12.6 and 29.95±13.85 years, respectively. Thirteen patients did not have a family history. Forty patients were under prophylaxis (danazol n=39, tranexamic acid= 1). The mean duration of the C1-inh concentrate usage was 4.07±1.76 years. The patients had taken this drug 6 times over the past year on average. All patients stated that they experienced difficulties during injections with this medication in the emergency units due to a lack of awareness by health care professionals regarding both HAE and C1-inh concentrate. While 61.4% of the patients reported overall satisfaction about the efficacy of drug, 43.8% stated that this medication should have a more practical application; 52.6% proposed that the drug should be more accessible in hospital emergency rooms. Conclusion: Patients with HAE generally considered that C1-inh concentrate was effective in their attacks. However, this group of patients expressed concerns related to the medical personnel's awareness about HAE as well as access to the medication.

Current challenges and future opportunities in patient‐focused management of hereditary angioedema: A narrative review

“…For example, the estimated proportion of countries' total population of patients with HAE enrolled in the Hereditary Angioedema Global Registry ranged from 14.4% (Greece) to 91.2% (Hungary) 45 . This is perhaps unsurprising as long delays between the onset of HAE symptoms and HAE diagnosis are still reported by multiple recent studies, each from a different country: a mean of 7.75 years in Korea, 46 a median of 10 years in Sweden, 47 a mean of 14 years in Switzerland, 48 a mean of 15.6 years in Romania, 49 and 17 years in Turkey 50 …”

“… 45 This is perhaps unsurprising as long delays between the onset of HAE symptoms and HAE diagnosis are still reported by multiple recent studies, each from a different country: a mean of 7.75 years in Korea, 46 a median of 10 years in Sweden, 47 a mean of 14 years in Switzerland, 48 a mean of 15.6 years in Romania, 49 and 17 years in Turkey. 50 …”

“…45 This is perhaps unsurprising as long delays between the onset of HAE symptoms and HAE diagnosis are still reported by multiple recent studies, each from a different country: a mean of 7.75 years in Korea, 46 a median of 10 years in Sweden, 47 a mean of 14 years in Switzerland, 48 a mean of 15.6 years in Romania, 49 and 17 years in Turkey. 50 The available information on country-specific HAE treatment patterns since 2016 is summarized in Table 6. In most of the countries with available data, attenuated androgens and antifibrinolytics remain widely used for HAE prophylaxis, although these medications are no longer recommended as first-line treatment options for LTP in the international guidelines.…”

“…4,9,10,22 In line with lack of approvals and/or limited availability for HAE-specific medications, studies from multiple countries only reported the use of attenuated androgens and tranexamic acid for LTP in HAE. These included studies from Brazil, 51,52 China, 53,54 Israel, 55 Italy, 56 Korea, 46 Poland, 57 Romania, 49 South Africa, 44 Turkey, 50 and Switzerland. 48 Interestingly, 6/12 of these studies reported that >50% of the investigated patients were receiving LTP, suggesting that even second-line LTP options are considered useful in real-world practice when the first-line options are not available or affordable.…”