Dupilumab Improves Quality of Life in Caregivers of Children with Uncontrolled Moderate-to-severe Asthma: LIBERTY ASTHMA VOYAGE Study

Background: Asthma is the most common chronic childhood disease. Children with uncontrolled treatment-refractory asthma have poor quality of life (QoL), as do their caregivers. Dupilumab, a fully human monoclonal antibody, blocks the shared receptor component for IL-4/13, key and central drivers of type 2 inflammation in multiple diseases. VOYAGE, a 52-week, randomized, double-blind, placebo-controlled phase 3 study (NCT02948959), demonstrated dupilumab efficacy/safety in children aged 6-11 years with uncontrolled moderate-to-severe asthma. We assessed QoL improvement in caregivers of children who participated in VOYAGE.

Methods: Children aged 6-11 years were randomized 2:1 to receive add-on dupilumab or matched placebo at weight-tiered dose 100 or 200 mg (≤30 vs >30 kg) every 2 weeks. Caregivers of patients aged 7-11 years completed the pediatric asthma caregiver QoL questionnaire (PACQLQ), with a one-week recall period, at study baseline and up to Week 52. PACQLQ global and domain scores (activity limitation and emotional function scores, ranging from 1-7 with a higher score indicating better QoL) were analyzed in patients with a type 2 asthma phenotype (baseline blood eosinophils ≥150 cells/µL or FeNO ≥20 ppb; n=318) and patients with baseline eosinophils ≥300 cells/µL (n=239).

Results: At baseline, 1 caregiver in 4 reported that the impact of the child’s asthma on the caregiver’s work was ‘a lot’. The activity and emotional domains were impacted, and 1 caregiver in 3 reported that they were at least ‘very worried or concerned’ about their child leading a normal life (Figure 1), reflecting high burden on caregiver QoL. Compared to placebo, global PACQLQ scores of caregivers of dupilumab patients significantly improved by Week 36 and onwards in the type 2 population (LS mean [95% CI] difference in change from baseline vs placebo: 0.40 [0.16−0.64, P=0.0013]) and by Week 12 and onwards in the population with eosinophils ≥300 cells/µL (LS mean [95% CI] difference vs placebo: 0.34 [0.01−0.67, P=0.0445]; Figure 2). Activity limitation domain scores significantly improved by Week 24 and onwards in caregivers of dupilumab patients in the type 2 population (LS mean [95% CI] difference vs placebo: 0.35 [0.10−0.60, P=0.0072]) and in the population with eosinophils ≥300 cells/µL (LS mean [95% CI] difference vs placebo: 0.40 [0.10−0.71, P=0.0100]). Emotional function domain scores significantly improved by Week 36 and onwards in caregivers of dupilumab patients in the type 2 population (LS mean [95% CI] difference vs placebo: 0.41 [0.16−0.67, P=0.0017]) and by Week 12 and onwards in the population with eosinophils ≥300 cells/µL (LS mean [95% CI] difference vs placebo: 0.39 [0.04−0.73, P=0.0292]).

Conclusion: The benefits of dupilumab for patients aged 7-11 years with uncontrolled asthma included improvements in caregiver QoL, as reflected by PACQLQ scores. Improvements were seen in global scores, and in activity limitations and emotional function domain scores.

Presenting Author(s)

AF
Alessandro G. Fiocchi, MD
Director of Allergy
Bambino Gesù Children’s Hospital IRCCS, Rome, Italy
Rome, Lazio, Italy
Disclosure: Abbott (Products/Services: Advisory board member) (Consultant, Advisory board member)Danone (Products/Services: Advisory board member; currently sponsoring research) (Consultant, Advisory board member; currently sponsoring research)DBV Technologies (Products/Services: advisory board member) (Consultant, Advisory board member)Ferrero (Products/Services: Currently sponsoring research) (Consultant, Currently sponsoring research)HiPP Organic (Products/Services: Advisory board member; currently sponsoring research) (Consultant, Advisory board member; currently sponsoring research)Novartis (Products/Services: Advisory board member) (Consultant, Advisory board member)Sanofi (Products/Services: Currently sponsoring research) (Consultant, Currently sponsoring research)Stallergenes Greer (Products/Services: Advisory board member) (Consultant, Advisory board member)

Co-Author(s)

WP
Wanda Phipatanakul, Professor
Professor of Pediatrics
Boston Children’s Hospital, Harvard Medical School, MA, USA
Boston, Massachusetts
Disclosure: Genentech (Products/Services: Consulting and clinical trial support/medication support) (Consultant)GSK for Asthma Therapeutics (Products/Services: Consulting and clinical trial support/medication support) (Consultant)Merck (Products/Services: Consulting and clinical trial support/medication support) (Consultant)Regeneron Pharmaceuticals, Inc. (Products/Services: Consulting and clinical trial support/medication support) (Consultant)Sanofi (Products/Services: Consulting and clinical trial support/medication support) (Consultant)
SD
Sandy R. Durrani, MD
Faculty Member
Cincinnati Children’s Hospital Medical Center, University of Cincinnati College of Medicine, Cincinnati, OH, USA
Cincinnati, Ohio
Disclosure: Regeneron Pharmaceuticals, Inc. (Products/Services: Contracted research) (Contracted Research (Principal Investigators must provide information, even if received by the institution))Sanofi-aventis (Products/Services: Contracted research) (Contracted Research (Principal Investigators must provide information, even if received by the institution))
JC
Jeremy Cole, MD
Pulmonologist
OK Clinical Research, Edmond, OK, USA
Edmond, Oklahoma
Disclosure: No Financial Relationships Disclosed
DL
Dongfang Liu, MSc
Statistician
Sanofi, Beijing, China;
Beijing, Beijing, China (People's Republic)
Disclosure: Sanofi (Products/Services: employees, may hold stock and/or stock options in the company.) (Employee)
JM
Jérôme Msihid, MSc
Post-hoc lead Statistician
Sanofi, Chilly-Mazarin, France
Chilly-Mazarin, Ile-de-France, France
Disclosure: Sanofi (Products/Services: employees, may hold stock and/or stock options in the company) (Employee)
DL
David J. Lederer, MD
Medical Director, Clinical Sciences, Respiratory and Inflammation
Regeneron Pharmaceuticals, Inc., Tarrytown, NY, USA
Tarrytown, New York
Disclosure: Regeneron Pharmaceuticals, Inc. (Products/Services: employees and shareholders) (Employee)
MH
Megan Hardin, n/a
Employee
Sanofi
Cambridge, Massachusetts
Disclosure: Sanofi (Products/Services: employee and shareholder) (Employee, Stockholder/Ownership Interest (excluding diversified mutual funds))
YZ
Yi Zhang, PhD. MPH
Health Economics and Outcomes Research, Medical Affairs
Regeneron Pharmaceuticals, Inc., Tarrytown, NY, USA
Tarrytown, New York
Disclosure: Regeneron Pharmaceuticals, Inc. (Products/Services: employees and shareholders.) (Employee)
AK
Asif H. Khan, MBBS, MPH
Senior Director, Global Medical, Respiratory
Sanofi, Chilly-Mazarin, France
Chilly-Mazarin, Ile-de-France, France
Disclosure: Sanofi (Products/Services: employees, may hold stock and/or stock options in the company) (Employee)