Injectable Preventive Treatments Weigh on Patients and Caregivers, Surveys Show

Routine injectable treatments to prevent hereditary angioedema (HAE) attacks place a large burden on patients and their caregivers, a survey study has found.

Surveyed patients and doctors also agreed that less burdensome and easier-to-administer preventive treatments are needed to improve the quality of life and independence of those with HAE.

The surveys and resulting work were funded by BioCryst Pharmaceuticals, which is developing berotralstat, an oral preventive therapy for HAE attacks now under review for approval in the U.S., Japan, and Europe.

“These data are consistent across HAE patients, caregivers and treating physicians showing many patients experience a significant treatment burden associated with current prophylactic HAE therapies. New therapies with easier routes of administration may meet a significant unmet need,” Cristine Radojicic, MD, an assistant professor of medicine at Duke University School of Medicine who led the study, said in a press release.

Study findings were presented across three posters featured at the 2020 Annual Scientific Meeting of the American College of Allergy, Asthma & Immunology (ACAAI), held virtually on Nov. 13–15.

HAE is a rare genetic disorder in which the lack of a functional blood protein called C1-inhibitor (C1-INH) leads to sudden and recurrent swelling episodes that can affect the deeper layers of the skin, as well as the patients’ upper airways and gastrointestinal tract.

The standard course of treatment to manage HAE consists on a combination of emergency medications for acute attacks, and long-term prophylactic therapies that aim to prevent these swelling episodes. Although several treatments have been approved to prevent and manage acute HAE attacks, nearly all are administered by intravenous or under the skin (subcutaneous) injections.

To evaluate the burden associated with the use of injectable prophylactic treatments, investigators at BioCryst and colleagues carried out a cross-sectional survey study, in which they gathered the opinions of patients, caregivers, and physicians on the use of these medications.

In the poster “Patient Perspectives on the Treatment Burden of Injectable Medication Administration for Hereditary Angioedema,” researchers described patients’ take on injectable preventive treatments, based on the answers provided by 75 HAE patients who completed the survey.

Nearly all (93%) reported routinely using prophylactic treatment via injections or infusions, and more than a third (38%) noted a need to avoid social activities due to their disease.

Almost half (47%) of the patients who recently started on a prophylactic for HAE attacks noted a need to rearrange life events to accommodate the medication, and more than half (58%) acknowledged being tired of repeat injections or infusions.

Still, almost nine in 10 said they had learned to tolerate difficult aspects of their treatment, and were satisfied with their current therapy regimen. A clear majority (86%), however, expressed interest in being able to move to a less burdensome treatment, and 84% agreed that alternative therapies for HAE are needed.

Sizable groups — from 76% to 90% of these patients — somewhat or strongly agreed that a daily oral pill treatment would make various aspects of their life more pleasant.

In the second poster, “Prophylactic Treatment Burden: Assessment by Caregivers of Patients with Hereditary Angioedema,” investigators reported the opinions of 30 caregivers who completed the survey.

Most (77%) had HAE themselves, and more than half (53%) were parents of a child with the disease. More than half of surveyed caregivers agreed it was challenging to learn to properly administer the medication, especially to themselves (55%) and to be comfortable using needles (61%).

Most (86%) also agreed the patient they cared for had learned to tolerate treatment and were satisfied with it, but felt they would be open to try more convenient options. More than half (71%) agreed those in their care were tired of repeat injections or infusions.

Nearly all agreed that a once-daily pill would allow patients to regain their freedom (86%) and be more independent (85%), while lowering their own care burden.

In the poster, “Understanding Differences in Perceptions of Hereditary Angioedema Treatment Burden May Improve Patient-Physician Treatment Care Dialogue,” investigators focused on the views of 109 physicians who completed the survey.

More than half (71%) were immunologists or allergists, treating an average of seven HAE patients each year.

Nearly all (94%) physicians agreed that alternative prophylactic treatment options are needed. More than half also believed their patients would tire of repeat injections or infusions (70%), and that this form of treatment would interfere with aspects of their lives (71%).

Survey findings also demonstrated doctors tend to underestimate the time needed to prepare and administer prophylactic treatments.

A strong discordance was found between physicians and patients regarding who initiated conversations on challenges with medication use: 72% of patients surveyed said they started these conversations, and 80% of physicians said the initiative was theirs.

This relevant piece of information “can lead to improvements in dialogue and an individualized approach to the management of HAE,” the researchers concluded.