Lumry discusses routine quality-of-life assessment, the pediatric HAE prophylaxis pipeline, and open questions after real-world lanadelumab data.
Patient-reported quality-of-life (QoL) measures remain largely absent from routine pediatric
The late-breaking analysis drew on 13 pediatric patients aged 2 to under 18 years in the PIONEER HAE registry, with 11 caregivers reporting outcomes.¹ Caregiver-perceived patient QoL rose from 2.2 to 4.7 on a 5-point scale, and attack frequency fell by 95% from a 1-year baseline.²˒³ In the final part of an interview with HCPLive, Lumry discussed why QoL tools rarely reach routine practice.
“We have recommended [them], and it’s certainly part of the treatment guidelines, both nationally as well as internationally,” said Lumry, clinical professor of internal medicine at the University of Texas Southwestern Medical School. “I think [quality of life assessments during] patients’… follow-up visits with a busy physician… may be overlooked just for a matter of time.”
According to Lumry, clinicians typically look more closely at QoL only when a problem surfaces with a patient’s treatment or disease control. Outside the setting of a clinical research trial, he said, these instruments are not commonly used.
The ACAAI analysis shows the kind of data routine assessment can capture. Caregiver QoL improved from 1.9 to 4.5 on the same 5-point scale, impaired sleep fell from 85% to 31% of caregivers, and impact on recreational activities fell from 62% to 8%.³ Mean Pediatric Quality of Life Inventory (PedsQL) total scores during treatment were 68.5 for ages 5 to 7 years, 91 for ages 8 to 12 years, and 84 for ages 13 to 18 years.³
The FDA expanded the lanadelumab label to children aged ≥ 2 years in February 2023.² In December 2025, the agency approved once-daily oral berotralstat (Orladeyo) pellets for long-term prophylaxis in children aged 2 to 11 years, which Lumry described as the only other approved targeted prophylactic option for this age group.⁴
Trials are underway evaluating on-demand and long-term prophylactic therapies in patients younger than 12 years, according to Lumry. He expects new oral and subcutaneous options within 2 to 3 years, depending on pediatric safety results.
All current trials in this space include QoL measures, Lumry noted. He hopes clinically treated patients outside trials will also use patient-reported outcomes, building on the lanadelumab analysis as a first step for younger patients.
“Hopefully [this] will continue to improve our ability to pick the right drug for the right patient for control of their HAE or treatment of attacks when they occur,” Lumry said.
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