KalVista Pharmaceuticals, Inc. presented significant real-world data derived from surveys of Hereditary Angioedema (HAE) patients at the European Academy of Allergy and Clinical Immunology Congress 2024 in Valencia, Spain. The insights provided valuable perspectives on patient experiences, challenges, and treatment approaches for HAE.
Patient Perspectives on On-Demand Treatments
A notable presentation by Cristine Radojicic from Duke University revealed that 50% of surveyed HAE patients in the US experienced untreated attacks that worsened, while 25% saw these attacks migrate to other parts of the body, including the throat. Patients often refrained from using on-demand treatments for reasons such as saving medication for severe attacks, expecting the attack to remain mild, and avoiding the discomfort of needle pain or burns.
Rashmi Jain of Oxford University Hospital Trust highlighted that among UK survey participants, 63% perceived their treatment timing as early, yet only 14% treated within an hour. Barriers to timely treatment included beliefs that attacks would stay mild and a desire to conserve medication for more severe episodes.
Anxiety and Treatment Timing
Research by Patrick Yong from Frimley Health NHS Foundation Trust found that nearly half of UK survey participants experienced moderate to extreme anxiety over using injectable on-demand therapies. Primary causes for this anxiety included fears of wasting treatment, uncertainty about the time needed for the therapy to take effect, and concerns about finding a vein for IV infusion.
Conversely, Hilary Longhurst from Auckland City Hospital highlighted that HAE patients who treated attacks early, within an hour, were more likely to carry their medication and experienced quicker recovery times. Early treatment led to a faster return to normalcy and reduced anxiety about future treatment.
Future Oral On-Demand Treatments
Anna Valerieva from the Medical University of Sofia presented findings that patients were more likely to carry and use oral on-demand treatments compared to injectable options. Oral treatments were preferred by 95.1% of respondents, who anticipated treating 88.5% of their attacks with oral medication, compared to 80.3% with injectable treatments. An impressive 80% of respondents indicated they would experience less anxiety using a pill rather than an injection.
Breakthrough Attacks and Long-Term Prophylaxis
William Lumry from Allergy and Asthma Research Associates reported that patients receiving non-androgen long-term prophylaxis (LTP) still experienced breakthrough attacks, with 68% describing their recent attack as moderate to very severe. Laryngeal swelling occurred in 19.6% of these cases, and 12% required emergency room visits or hospitalization. Only 55% of these patients reported all their attacks to their physicians, potentially leading to an underestimation of the frequency and severity of attacks.
Challenges and Unmet Needs with Current Therapies
Lumry also pointed out that despite the availability of non-androgen LTP treatments, patients faced significant treatment burdens. Issues such as lack of efficacy, gastrointestinal problems for oral LTP, and administration discomfort and frequent dosing for injectable LTP were commonly reported by physicians.
Advancements in Diagnostic Tools
Daniel Lee from KalVista Pharmaceuticals discussed advancements in a plasma kallikrein activity assay, which could be pivotal in diagnosing and understanding PKa-mediated diseases, including hereditary angioedema with normal C1 esterase inhibitor (nC1-INH-HAE).
Overall, the findings presented at EAACI 2024 underscore the ongoing challenges faced by HAE patients and highlight the potential benefits of early and alternative treatment options.
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