HAE Junior is a Czech-based patient organization aiming to improve the quality of life of children & youth with hereditary angioedema (HAE). It was set up in December 2019 in Prague by a group of parents of pediatric HAE patients.
One of the first projects of HAE Junior was a comparative patient survey, intended to map the experience, needs and perspectives of both adult and pediatric Czech HAE patients. The survey was conducted during the months of May – June 2020 and consisted of three questionnaires: one for adult HAE patients, one for parents of pediatric HAE patients, and a shorter one for school-aged children/ teenagers with HAE. Due to the pandemic. The survey was conducted fully online. The results of the survey were presented as part of a webinar held by HAE Junior in September 2020.
According to HAE Junior’s patient survey findings, hereditary angioedema most frequently limits patients in developing their full potential in the areas of study/career and leisure/hobby activities. These areas have a long-term impact on the quality of life and thus indicate that HAE is not only a health burden, but also can also become an economic and social burden. Although HAE limitations are similar in adult and pediatric patients, the survey found that the priorities of each respondent group are slightly different. This is most probably related to their different experiences with the disease and access to treatment.
All adult patients participating in this survey indicated improved access to effective preventive treatment as their top priority. As a second priority, all but one of the surveyed HAE adult patients mentioned that they would like to have the option to treat all HAE attacks at the first symptom on-set (currently only most severe episodes are eligible for reimbursement of the acute treatment in the Czech Republic).
The priorities of parents of HAE pediatric patients however seem to focus more on removing the existing barriers related to acute therapies, as this patient group is still highly dependent on emergency healthcare services. Based on HAE Junior’s survey findings, the top priorities of the pediatric patient population are:
- the need to raise awareness of HAE among healthcare professionals.
- better access to home therapy in the treatment of acute symptoms.
- the possibility to treat all HAE episodes at the onset of first symptoms.
To conclude, even though the limitations of HAE to the quality of life are quite similar for both adult and pediatric patient groups, given the existing differences in available treatment options, their top priorities are different. Adult patients have a wider and more convenient rage of acute treatment options and therefore their top priority is related to better access to efficient prophylactic therapies. On the other hand, the pediatric patients represented in this survey had fewer acute treatment options and minimal access to home therapy. Therefore, based on their experience, the top priorities of this patient group are primarily related to removing the existing barriers related to acute treatments and to enhance their convenience level.
Any questions related to this patient survey should be address to HAE Junior patient organization at firstname.lastname@example.org.
Written by Camelia Isaic
About the author:
Camelia Isaic is an international business consultant and passionate volunteer in rare disease patient advocacy. She is the founder and chairwoman of HAE Junior, a Czech patient organization seeking to improve the quality of life of children & youth with hereditary angioedema (HAE).