Welcome and HAE in Asia-Pacific: Country perspectives

The scientific track opened with HAEi’s Tony Castaldo spending a few minutes to set the scene. He thanked the organizing team at HAEi before introducing and thanking the Scientific Committee Co-Chairs: Professor Connie Katelaris, Professor Philip Li, and Dr Jovila Abong. He also thanked the Committee members: Dr Hilary Longhurst, Dr Hiroshi Chantaphakul, Dr Michihiro Hide, Dr Ankur Jindal, Professor Yuxiang Zhi, and Dr Felicia Racquel Salvador-Tayag. Unfortunately, Dr Chantaphakul and Professor Zhi were unable to be in Manila.

“It’s telling that none of the countries in the Asia-Pacific region have access to all the modern HAE medicines.”

Tony explained that this lack of therapeutic access is core to the mission of HAEi. “We are a fierce advocacy organization,” he told those present. He went on to outline some of the key activities of HAEi and showcased HAEi’s contribution to the science of HAE via our research programs to assist in patient identification, pharmaco-economic evaluation of therapies and quality of life, and burden of illness. Tony also drew attention to the recent HAEi and United States HAE Association Symposium and the resulting publication on HAE with normal C1 inhibitor (see our scientific publications section for more information).

Tony reminded the audience that, as clinicians, they held the key to diagnosis and treatment. He asked them to work with HAEi and their local patient advocacy organization to get access to and reimbursement for modern medicine. “It can be done,” he concluded.

On behalf of the Scientific Committee, Co-Chair Professor Connie Katelaris welcomed more than 100 participants to the scientific track and, during the opening slides, presented the Young Researcher/Investigator award to Dr Jane Wong from Hong Kong. You can read about her award-winning research in the oral abstract presentations.

The scientific program started with a session dedicated to country perspectives on HAE. The 3 countries represented were the Philippines, Malaysia, and Nepal. Dr Ankur Jindal chaired the session, and the first presenter was Dr Jovila Abong, who spoke on the state of diagnosis and management of HAE in the Philippines.

HAE in the Philippines

Dr Abong opened by stressing that a lack of data in the Philippines hinders proper diagnosis and management of HAE and that reported cases are clustered around diagnostic testing sites. In 2024, a survey was conducted amongst members of the Philippine Society of Allergy, Asthma and Immunology (PSAAI). The 88 respondents reported 14 confirmed cases and 28 suspected cases. The initially prescribed medications were mainly allergy medication, and most suspected cases were treated as allergies. Dr Abong described the geography of the Philippines, with 7,641 islands divided into 3 regions: Luzon, Visayas, and Mindanao. Dr Abong indicated limited access to HAE testing facilities in Visayas and Mindanao, but more in Luzon where Manila is located.

Moving on to treatment, Dr Abong explained, that in the Philippines, on-demand treatment is fresh frozen plasma. Additionally, tranexamic acid and danazol are available. Patients with relatives and friends outside the Philippines can acquire other drugs such as C1-inhibitor infusions.

Looking to the future, Dr Abong indicated that PSAAI is working to include HAE in the Philippine’s Rare Disease Act. It would unlock access to government funding for diagnostic tests and treatment if accepted. Dr Abong mentioned ongoing dialogue with pharmaceutical partners, with an application for compassionate access being developed. Dr Abong felt hope in collaboration with patients and caregivers on activities to improve awareness, diagnostics, and access to treatment.

HAE in Malaysia

Malaysia, according to Associate Professor Adli Ali, was the youngest member of the HAE community in the Asia-Pacific region. The first reported HAE cases were in 1996, but in the subsequent years, treatment remained limited to ‘medieval treatments’ such as fresh frozen plasma. Today, Associate Professor Ali indicated that on-demand medication with icatibant is available through emergency protocols.

Currently, Malaysia has 22 diagnosed patients, most with HAE Type 1. Associate Professor Ali reported that there is a pool of interesting novel mutations amongst Malaysian HAE patients. There are immunologists with knowledge of HAE, but most of these are pediatric specialists. C4 complement testing is available at all centers in Malaysia. Quantitative and functional tests are available but require patients to pay.

Associate Professor Ali highlighted that there is now an HAE working group, with clinicians, mainly immunologists and dermatologists, and they are developing a national guideline to give a framework for diagnosis and management. Hopefully, this will create awareness and conversations among the HAE community in Malaysia.

Overall, Associate Professor Ali told the audience there is a need to improve awareness and understanding, so an education module is in development. Doctors in Malaysia are also actively developing a national guideline. Finally, Associate Professor Ali confirmed that centers in Malaysia look forward to being a greater part of the ACARE community.

HAE in Nepal

The last country perspective was given by Dr Dharmagat Bhattarai, who is from the beautiful and mountainous country of Nepal. Based on the prevalence studies, there should be more than 50 HAE patients, but this number has not been diagnosed, and the lack of affordable testing facilities hampers diagnosis.

Dr Bhattarai recounted how he established his center without support from the Government or other organizations, and it is dedicated to awareness and advocacy for immunological diseases such as HAE.

Testing facilities are an issue, as there are no genetic testing facilities in Nepal. All genetic testing is sent to a commercial lab in India. In terms of management, Dr Bhattarai referenced that the situation in Nepal is similar to Malaysia and the Philippines, in that most historically diagnosed patients were treated as allergy cases with antihistamines and steroids.

The current landscape of treatment is primarily based on the use of tranexamic acid and steroids such as danazol or stanazol. Acute attack patients are treated with fresh frozen plasma. Dr Bhattarai noted that 2 of his more fortunate patients can purchase modern medicine from India. Some medications have also been donated from institutes in Europe. Dr Bhattarai characterized the situation as ‘people are pleading for treatment.’ He has worked hard to raise awareness and advocate for the disease. Positive recent steps have included the creation of HAE Nepal as a patient organization. There are also plans to register C1-inhibitor concentrate and ensure genetic testing facilities, and he thanked all those who have helped Nepal to date.

Panel discussion

Following the third perspective, there was a panel discussion and audience Q&A. The first question was on the use of tranexamic acid. The panel believed that tranexamic acid should be used only in the absence of alternatives. After this, there was a discussion about problems with registering C1-inhibitor infusions in Nepal. Dr Bhattarai suggested the situation was due to a cumbersome registration process and a lack of engagement with the scientific literature. There is also a lack of awareness and understanding of the need for treatment, as HAE is a life-threatening disease. He also suggested that there is a focus on medicines for more common diseases. Ultimately, he suggested that the onus for completing a registration fell to him.

Having heard about a national database in Malaysia, a doctor from India asked about avoiding data duplication in the actual number of HAE patients. Associate Professor Ali responded that there is inevitable overlap but that this can be helped by making someone the gatekeeper of the data. Duplication is not such a problem in Malaysia right now as there are few patients, and Associate Professor Ali knows the names and ages of many of them. Dr Hide gave a Japanese perspective, as the country has 2 registries. For the patients themselves, there is no duplication, but there may be multiple entries by different physicians, which requires addressing.

The final question was about the most important things to do to make the medication accessible in a country. The panel highlighted the value of an orphan drug registration pathway where it exists. The next step was to focus on need. The panel commented that on-demand therapy may be most desired when speaking to patients, and, therefore, the focus should be there.

The advocacy of patient organizations, locally and through HAEi, was also seen as a positive step. The importance of increasing diagnosis was stressed, as having a defined patient population demonstrates the need for treatment.