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Caregiver-rated QoL rose from 1.9 to 4.5 on a 5-point scale with lanadelumab prophylaxis in a pediatric HAE registry analysis, William Lumry, MD, said.
Lanadelumab (Takhzyro) prophylaxis was associated with improved quality of life (QoL) for children with hereditary angioedema (HAE) and their caregivers, according to a retrospective registry analysis presented at the American College of Allergy, Asthma and Immunology (ACAAI) 2026 Annual Scientific Meeting in Phoenix.¹
The US Food and Drug Administration (FDA) expanded the lanadelumab label to include patients aged 2 to <12 years in February 2023, based on the open-label phase 3 SPRING study and efficacy data extrapolated from the phase 3 HELP study.²˒³ SPRING reported large gains in caregiver-reported Pediatric Quality of Life Inventory (PedsQL) scores across 52 weeks of treatment.² The new analysis, presented as a late-breaker by William R. Lumry, MD, examines how long-term prophylaxis affects QoL and caregiving burden in routine practice.¹
“It was just a complete flip in what the patients and the caregivers perceived as far as their quality of life with treatment,” said Lumry, a clinical professor of internal medicine at the University of Texas Southwestern Medical School in Dallas, in an interview with HCPLive.
Investigators identified patients aged 2 to < 18 years at lanadelumab initiation, along with their caregivers, through the TRIO Health Consortium of Independent Immunology Clinics (CIIC) PIONEER HAE registry.¹ Caregivers completed a cross-sectional survey including the PedsQL and the PedsQL Family Impact Module (FIM), each scored 0 to 100, with greater scores indicating better QoL. The survey captured the 12 months before lanadelumab initiation and the most recent 3 months of treatment.¹
Eleven caregivers (mean age, 42.3 years; 64% female) reported on 13 pediatric patients (mean age, 12.2 years; 62% male).¹ Patients started lanadelumab at a mean age of 8.9 years and received treatment for a mean of 3.7 years.
On a 5-point scale (1 = poor; 5 = excellent), caregiver-perceived patient QoL improved from a mean of 2.2 before lanadelumab to 4.7 during the current period.¹ Caregiver-perceived caregiver QoL improved from 1.9 to 4.5. During treatment, mean PedsQL total scores were 68.5 for patients aged 5 to 7 years, 91 for those aged 8 to 12 years, and 84 for those aged 13 to 18 years.¹
During the current treatment period, 69% of caregivers reported no impact of their child’s HAE on work or daily activities.¹ Before lanadelumab, only 23% reported no work impact and 8% reported no daily activity impact. Lumry noted 68% of caregivers had to request flexibility, stop working, or make other adjustments before their child began prophylaxis.
PedsQL-FIM scores during treatment were 92 (SD, 13) for family functioning and 90 (SD, 20) for daily activities.¹ According to Lumry, the proportion of caregivers reporting impaired sleep fell from 85% before treatment to 31% during treatment. Reported impact on recreational activities dropped from 62% to 8%, with an identical shift for work responsibilities.
Errands, household chores, and family time affected 30% to 45% of caregivers before lanadelumab, with no reported impact during treatment, Lumry said. He noted 2 FIM domains, communication and worry about unpredictable attacks, did not change significantly with treatment.
“The study provides a really great multidimensional assessment of the broader impact of this very effective treatment in pediatric patients and in their caregivers,” Lumry said.
Watch part 1 of our interview with Lumry here: Lanadelumab Reduces Pediatric HAE Attacks 95% in Real-World Analysis
Editor’s note: Reported disclosures for Lumry include Novartis Pharmaceuticals Corporation, CSL Behring, Takeda Pharmaceuticals U.S.A., Inc., BioCryst US Sales Co., LLC, and more.
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