Young children with HAE face hurdles with injectable attack treatments
Caregiver reports show anxiety, pain, and delays; claims data show high ER use
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Injectable on-demand treatments for children with hereditary angioedema (HAE) are often associated with anxiety, pain, treatment delays, and high use of emergency healthcare services, highlighting the need for a simpler treatment option, according to data from Kalvista Pharmaceuticals, the company that markets Ekterly (sebetralstat), an oral on-demand treatment approved for people with HAE ages 12 and older.
“These data highlight the significant unmet needs that exist for children living with HAE and the families who care for them,” Ben Palleiko, CEO of Kalvista, said in a company press release announcing three recent studies presented at this year’s International Society for Pharmacoeconomics and Outcomes Research (ISPOR) and Eastern Allergy conference.
The studies examined treatment patterns, caregiver experiences, and healthcare use among children with HAE ages 2–11 who were included in insurance claims data or caregiver surveys. Injectable treatments can be challenging for many families. In the U.S., the only on-demand treatment approved by the U.S. Food and Drug Administration (FDA) for this age group is Berinert, an intravenous (into-the-vein) plasma-derived C1 inhibitor.
Injectable treatments may burden families
“Currently, the only FDA-approved on-demand treatment for children ages 2–11 requires intravenous administration, which can place a considerable burden on both children and caregivers during already stressful attacks,” Palleiko said. “As the first and only oral on-demand treatment for HAE, EKTERLY has the potential to meaningfully improve the treatment experience for this pediatric population and serve as a family-wide solution across generations.”
One study, titled “On-Demand Treatments for Hereditary Angioedema and Healthcare Resource Utilization in Pediatric (2–11 Years) Patients: A US Claims Database Analysis,” analyzed real-world insurance claims data. It found that about two-thirds of children had claims for or were prescribed intravenous plasma-derived C1 inhibitor. However, most of the dose claims were for off-label treatments, mainly icatibant, an injectable treatment available under the brand name Firazyr. This treatment is available in the U.S. for adults with HAE, but not for children. In Canada and Europe, however, it is approved for patients ages 2 and older.
Claims data showed a mean of 0.43 on-demand treatment dose claims per patient per month. The rate was lower for Berinert, at 0.28 dose claims per month, while icatibant had a higher rate, at 0.72 dose claims per month.
The study also showed high use of healthcare services. Nearly half of the children (47%) had at least one emergency department visit, while fewer (17%) had at least one home healthcare visit, where medical professionals help administer treatment outside a hospital.
“Undertreatment with prescribed on-demand therapy, substantial off-label use of on-demand treatments, and high emergency department utilization in pediatric patients with HAE suggest that new, less invasive on-demand treatment options are needed,” the researchers wrote.
Caregivers report treatment anxiety and delays
A caregiver survey, titled “Burden of Injectable On-Demand Treatment for Hereditary Angioedema Attacks in Children,” explored the burden of injectable treatments. Almost half of the children did not receive treatment during their last HAE attack. Among those who were treated, most (93.7%) experienced anxiety about receiving injections, and more than two-thirds described their anxiety as extreme.
Reasons for not treating included thinking the attack would be mild, uncertainty about whether it was an actual attack, and concerns about needle pain or burning, stinging, or pain from the medicine. Reasons for treatment anxiety included fear of needles and anticipated burning, stinging, or pain. These concerns may contribute to delays in treatment, even though medical guidelines recommend treating attacks as early as possible to help prevent swelling from worsening and shorten the time to attack resolution.
Side effects from injections were also common. Three-quarters of children experienced at least one side effect, most often pain from the needle itself or discomfort such as burning, stinging, or pain while the medicine was being injected.
A caregiver-focused analysis of the same survey data, presented in a separate poster titled “Caregiver Burden Associated with Injectable On-Demand Treatment of Hereditary Angioedema Attacks in Children,” also highlighted practical difficulties. These included swelling attacks that started when children were away from home (43%) or when the caregiver was not with the child (37%). As a result, giving treatment quickly was often difficult. Nearly two-thirds of caregivers reported difficulty administering treatment.
Anxiety among caregivers was also very high. Ninety percent felt anxious when deciding whether to treat an attack, and 60% reported extreme anxiety. Among treated attacks, the mean time from attack onset to treatment was 2.8 hours, and only about 31% were treated within the first hour after symptoms began.
Caregivers most often ranked an oral treatment as the top improvement, and 96% included it among their top choices. “Across real-world treatment patterns, healthcare resource utilization, and caregiver experiences, these findings reinforce the need for new treatment options that are easy to use and support early, consistent treatment of attacks,” Palleiko said.
