ASSESSING THE ABILITY OF THE EQ-5D-5L TO CAPTURE LONG-TERM HEALTH-RELATED QUALITY OF LIFE IMPACTS OF HEREDITARY ANGIOEDEMA ATTACKS
Author(s)
Lena Hubig, PhD1, Adam Gordois, MSc2, Patrick Gillard, DPharm3, Sandra Nestler-Parr, MPhil, MSc, PhD4, Andrew Lloyd, DPhil5, Andrew Briggs, DPhil6, Tom Booth, PhD5;
1Acaster Lloyd Consulting, London, United Kingdom, 2Biocryst Pharmaceuticals, Leeds, United Kingdom, 3Biocryst Pharmaceuticals, Durham, NC, USA, 4Biocryst Pharmaceuticals, Weybridge, United Kingdom, 5Acaster Lloyd, London, United Kingdom, 6Occam Research, London, United Kingdom
1Acaster Lloyd Consulting, London, United Kingdom, 2Biocryst Pharmaceuticals, Leeds, United Kingdom, 3Biocryst Pharmaceuticals, Durham, NC, USA, 4Biocryst Pharmaceuticals, Weybridge, United Kingdom, 5Acaster Lloyd, London, United Kingdom, 6Occam Research, London, United Kingdom
OBJECTIVES: Hereditary angioedema (HAE) is a rare disorder characterised by recurrent swelling attacks varying in frequency and severity. This study assessed the ability of the EQ-5D-5L instrument to quantify long-term health-related quality of life (HRQoL) impacts of HAE attacks (beyond and between attacks).
METHODS: We conducted a secondary analysis of APeX-2 (NCT03485911), a phase 3 trial of berotralstat for long-term prophylaxis in HAE. Trial participants (n=121) completed the EQ-5D-5L at baseline and study visits every 4 to 12 weeks up to 144 weeks. HAE attacks were characterised based on monthly frequency (any or by location: abdominal, peripheral, mixed), laryngeal attack (i.e., life-threatening; yes/no), and days since last attack. Cross-sectional Spearman’s correlations between the EQ-5D-5L index score and attack characteristics were estimated. A longitudinal two-part model (TPM) was employed consisting of: (1) logistic mixed-effects model estimating the odds of being in full health on the EQ-5D-5L; (2) linear mixed-effects model estimating the EQ-5D-5L index score if not in full health. Models included baseline characteristics (age, sex, body mass index), treatment arm, and attack characteristics.
RESULTS: During the trial, the mean EQ-5D-5L index score was high (>0.91), and most (>54%) participants reported full health. Attack characteristics were weakly (r<0.3) correlated with EQ-5D-5L index scores. The TPM showed no relationships between attack characteristics and full health, and only mixed-location attacks indicated a significant, small disutility (-0.007) in part two of the model.
CONCLUSIONS: This study showed weak associations between HAE attack characteristics and EQ-5D-5L index scores. The EQ-5D-5L appears unable to capture the longer-term (between attack) HRQoL impacts of HAE. Likely contributing factors are the generic nature of the EQ-5D-5L being insensitive to HRQoL changes in HAE, and the short reference period (‘health today’) of the EQ-5D-5L, which is not optimal when HAE attacks occur sporadically and/or infrequently.
METHODS: We conducted a secondary analysis of APeX-2 (NCT03485911), a phase 3 trial of berotralstat for long-term prophylaxis in HAE. Trial participants (n=121) completed the EQ-5D-5L at baseline and study visits every 4 to 12 weeks up to 144 weeks. HAE attacks were characterised based on monthly frequency (any or by location: abdominal, peripheral, mixed), laryngeal attack (i.e., life-threatening; yes/no), and days since last attack. Cross-sectional Spearman’s correlations between the EQ-5D-5L index score and attack characteristics were estimated. A longitudinal two-part model (TPM) was employed consisting of: (1) logistic mixed-effects model estimating the odds of being in full health on the EQ-5D-5L; (2) linear mixed-effects model estimating the EQ-5D-5L index score if not in full health. Models included baseline characteristics (age, sex, body mass index), treatment arm, and attack characteristics.
RESULTS: During the trial, the mean EQ-5D-5L index score was high (>0.91), and most (>54%) participants reported full health. Attack characteristics were weakly (r<0.3) correlated with EQ-5D-5L index scores. The TPM showed no relationships between attack characteristics and full health, and only mixed-location attacks indicated a significant, small disutility (-0.007) in part two of the model.
CONCLUSIONS: This study showed weak associations between HAE attack characteristics and EQ-5D-5L index scores. The EQ-5D-5L appears unable to capture the longer-term (between attack) HRQoL impacts of HAE. Likely contributing factors are the generic nature of the EQ-5D-5L being insensitive to HRQoL changes in HAE, and the short reference period (‘health today’) of the EQ-5D-5L, which is not optimal when HAE attacks occur sporadically and/or infrequently.
Conference/Value in Health Info
2026-05, ISPOR 2026, Philadelphia, PA, USA
Value in Health, Volume 29, Issue S6
Code
PCR22
Topic
Patient-Centered Research
Topic Subcategory
Patient-reported Outcomes & Quality of Life Outcomes
Disease
SDC: Rare & Orphan Diseases