The HAEi Central Eastern European region is a massive one consisting of 12 countries with a population close to 300 million people – and with only around 15 per cent of all HAE patients diagnosed. Therefore, this region requires an individual approach and hands-on assistance in activities related to advocacy, gaining access to and reimbursement of modern therapies.
“It all starts and finishes with motivated HAE patients, caregivers, and physicians who desire to improve quality of life”, says Michal Rutkowski, Vice President of HAEi and President of the Polish HAE organization Pięknie Puchnę.
27-28 October 2018 HAEi and Pięknie Puchnę once more teamed up for the Central Eastern Europe Conference and Workshop in Warsaw, Poland. This third event of its kind in this region brought comprehensive assistance, guidance, education, and awareness programs to HAE patients as well as support and motivation to physicians to serve as expert medical advisors.
Among many other things the 221 delegates heard Professor Marc Riedl from the US HAEA Angioedema Center at UCSD in USA talk about “HAE background and Treatment Landscape (now and the future)”. Based on the acute treatment recommendations from the most recent World Allergy Organization/International Association of Allergy and Clinical Immunology guidelines (2017) Professor Riedl said that all attacks should be considered for on-demand treatment and any attack affecting or potentially affecting the upper airway should be treated. Furthermore, HAE attacks should be treated as early as possible – either with C1-INH, ecallantide, or icatibant. Also, he said that all patients should have sufficient medication for on-demand treatment of two attacks and carry on-demand medicines at all times and that any patient who is provided with on-demand treatment licensed to self-administer should be taught to self-administer.
Regarding prophylactic treatment recommendations, Professor Riedl said that patients should be evaluated for long-term prophylaxis at every visit and that disease burden, as well as patient preference, should be taken into consideration. Regarding medication, he suggested the use of C1-INH for first-line long-term prophylaxis and androgens as second-line long-term prophylaxis. Also, he suggested the adaptation of long-term prophylaxis in terms of dosage and/or treatment interval as needed to minimize the burden of the disease.
Article continued below images
On the topic of progress in HAE the Professor spoke about the educational and diagnostic efforts, not least improved recognition and testing for HAE, family testing, and development of specialized centers for HAE care. Regarding treatment advances, he said that there is improved global access to effective HAE medications and that he sees a shift towards increased use of preventative strategies.
The program also included an orientation on “HAE during pregnancy”, delivered by Professor Henriette Farkas from the Hungarian HAE Center at Semmelweis University in Budapest. The Professor asked why women with C1-INH-HAE are unique – and answered the question herself: Women have more frequent attacks than men, women have more severe attacks than men, hormonal factors play a significant role in the worsening of the condition, and female sex hormones affect the synthesis of many proteins.
Both HAEi President Anthony J. Castaldo and HAEi Executive Director Henrik Balle Boysen spoke at the conference. While Mr Balle Boysen gave an orientation on HAEi advocacy tools one of the topics for Mr Castaldo was “Advancing HAE Advocacy”. Among other things he said that the advocacy key to success is always to be ready to demonstrate how modern treatments result in dramatic improvements in patient quality of life, never take no for an answer, and to stay vigilant, patient, and creative even though the path to success is a long one with unexpected turns. Most importantly, Mr Castaldo urged the delegates to call upon HAEi Regional Patient Advocate Michal Rutkowski to bring his experience and resources. Furthermore, the HAEi President said:
“You must build your national HAE organization as there is strength in numbers. And you must forge partnerships to sharpen advocacy effects – not least by finding and working with healthcare professionals, seeking out industry representatives and exploring ways to collaborate on raising awareness and broadening access to life-saving medicines, and working with government at all levels.”
Mr Castaldo also gave an overview of the extensive ongoing research for new HAE treatments:
- Ongoing trials on an oral treatment for prophylaxis and acute attacks (BioCryst)
- Ruconest recombinant C1-INH for prophylaxis (Pharming)
- Long-acting subcutaneous humanized monoclonal antibody against factor XIIa (CSL Behring)
- Developing oral treatment (KalVista)
- Adeno virus assisted gene therapy (Adverum)
- A number of companies with compounds in early stage development
Among many other items, the program included a talk on “How Patients’ Organization can help Stakeholders (Physicians and Pharmaceuticals Companies) improve patients’ quality of life” by Paweł Przewięźlikowski, CEO of Selvita SA. Furthermore, at the welcome evening on the first conference day, the organizers premiered a film on living with HAE from the Polish patients’ perspective. As previously the program included a Q&A session as well as presentations from the majority of the countries in the HAEi Central Eastern European region.