Physician and patient perspectives on the management of hereditary angioedema: a survey on treatment burden and needs

Abstract

Hereditary angioedema (HAE) is a rare disorder caused by genetic mutations that lead to recurrent episodes of swelling in various parts of the body. Prophylactic treatment is common for patients with HAE, and the therapeutic options have expanded in recent years. The current standard of care for prophylactic HAE therapies is subcutaneous treatment, which can be self-administered at home, greatly improving patient quality of life. As new therapies emerge, it is important for patients and physicians to discuss the risks and benefits associated with each treatment to develop an individualized approach to HAE management. We conducted surveys of patients with HAE and physicians who treat patients with HAE to identify prescribing trends for prophylactic HAE treatments and the impact that such treatments has on patients. Our results confirmed that newer, subcutaneous therapies are prescribed for HAE prophylaxis more frequently than other therapies in the United States and that treatment burdens still exist for patients with HAE. We found that physicians and patients were not always aligned on how treatment choices affect patients’ lives, which may mean that there are opportunities for enhanced patient‐physician dialog and shared decision-making in HAE management in the United States.

Berotralstat (BCX7353): Structure-Guided Design of a Potent, Selective, and Oral Plasma Kallikrein Inhibitor to Prevent Attacks of Hereditary Angioedema (HAE)

“…Prophylactic therapeutic options have grown in the past two decades to include lanadelumab (Takhzyro), SC C1-INH (Haegarda), and IV C1-INH (Cinryze). All these treatment options are either large biologics or peptides that must be administered via injection or infusion leading to a significant treatment burden; therefore, the development of an oral long-term prophylactic therapy could increase convenience and improve the ease of administration, reducing treatment burden and improving the quality of life (QoL) in patients with HAE . No oral drugs have been approved for the prevention or treatment of HAE, though a few small molecule PKal inhibitors have been reported. − We report the discovery of Berotralstat (BCX7353), an orally bioavailable small molecule PKal inhibitor that has been effective in preventing HAE attacks and has recently been approved by the U.S. Food and Drug Administration (FDA), Japan’s Ministry of Health, Labor, and Welfare (MHLW), and the European Commission for the prophylactic treatment of HAE in patients 12 years and older.…”

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

“…Conversely, the studies from United States reported established use of first‐line HAE treatment options, with the use of C1‐INH reported in up to 60.7% of patients and the use of lanadelumab (since 2018) reported in up to 53.3% of patients in separate studies 37–42 . Widespread use of C1‐INH was also reported in Canada (93.5% of physicians reported prescribing C1‐INH for LTP), 64 Germany (ongoing LTP and/or STP with C1‐INH reported in up to 65.0% of patients), 65 and Puerto Rico (LTP with C1‐INH reported in up to 68.7% patients who were taking prophylaxis) 66 .…”

“…Despite the adoption of first‐line LTP options, the frequent use of second‐line LTP therapies persisted even in these countries, with 27.2% of patients from the United States reporting the use of androgens for LTP 39 and 40.0% of patients from Germany reporting the use of androgens for LTP and/or STP 65 . In a survey of physicians from Canada, 41.9% of physicians reported prescribing androgens and 19.4% reported prescribing antifibrinolytics 64 ; in two similar surveys of physicians from the United States, up to 38% of physicians reported currently prescribing androgens and up to 11% of physicians reported currently prescribing antifibrinolytics 37,38 . These trends are similar to the findings of multinational (Australia, Austria, Canada, France, Germany, Spain, Switzerland, and United Kingdom) study from 2018 which reported that 45.7% of patients overall received LTP with C1‐INH, 34.4% with androgens, and 17.9% with tranexamic acid 62 …”

“…In line with the emphasis that current treatment guidelines and consensus statements have placed on shared decision‐making, QoL assessments should be individualized by including patient input on how disease control and QoL are measured 4,7,9,27 . The importance of shared decision‐making can be highlighted with a recent survey of patients with HAE and physicians, in which the physicians placed significantly more importance on some of the treatment burdens (eg, HAE medication interfering with daily activities) compared with the patients 38 …”

“…States included data from up to 1429 patients with HAE and up to 156 physicians treating HAE per study. [36][37][38][39][40][41][42][43] In contrast, we have found only one study from the continent of Africa reporting on HAE treatment patterns; this study included 43 patients with HAE from South Africa. 44 The low number of patients reported in some country-specific studies suggests that many patients with HAE remain undiagnosed in these countries, taking into the account the estimated HAE prevalence rates and the populations of these countries.…”