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Clinical Trial
. 2021 Apr;76(4):1188-1198.
doi: 10.1111/all.14680. Epub 2020 Dec 24.

Impact of lanadelumab on health-related quality of life in patients with hereditary angioedema in the HELP study

William R Lumry  1 , Karsten Weller  2 , Markus Magerl  2 , Aleena Banerji  3 , Hilary J Longhurst  4 , Marc A Riedl  5 , Hannah B Lewis  6 , Peng Lu  7 , Giovanna Devercelli  7 , Gagan Jain  7 , Marcus Maurer  2 ; HELP Study Investigators
Collaborators, Affiliations
Clinical Trial

Impact of lanadelumab on health-related quality of life in patients with hereditary angioedema in the HELP study

William R Lumry et al. Allergy. 2021 Apr.

Abstract

Background: An objective of the phase 3 HELP Study was to investigate the effect of lanadelumab on health-related quality of life (HRQoL) in patients with hereditary angioedema (HAE).

Methods: Patients with HAE-1/2 received either lanadelumab 150 mg every 4 weeks (q4wks; n = 28), 300 mg q4wks (n = 29), 300 mg every 2 weeks (q2wks; n = 27), or placebo (n = 41) for 26 weeks (days 0-182). The Angioedema Quality of Life Questionnaire (AE-QoL) was administered monthly, consisting of four domain (functioning, fatigue/mood, fears/shame, nutrition) and total scores. The generic EQ-5D-5L questionnaire was administered on days 0, 98, and 182. Comparisons were made between placebo and (a) all lanadelumab-treated patients and (b) individual lanadelumab groups for changes in scores (day 0-182) and proportions achieving the minimal clinically important difference (MCID, -6) in AE-QoL total score.

Results: Compared with the placebo group, the lanadelumab total group demonstrated significantly greater improvements in AE-QoL total and domain scores (mean change, -13.0 to -29.3; p < 0.05 for all); the largest improvement was in functioning. A significantly greater proportion of the lanadelumab total group achieved the MCID (70% vs 37%; p = 0.001). The lanadelumab 300 mg q2wks group had the highest proportion (81%; p = 0.001) and was 7.2 times more likely to achieve the MCID than the placebo group. Mean EQ-5D-5L scores at day 0 were high in all groups, indicating low impairment, with no significant changes at day 182.

Conclusion: Patients with HAE-1/2 experienced significant and clinically meaningful improvements in HRQoL measured by AE-QoL following lanadelumab treatment in the HELP Study.

Keywords: AE-QoL; hereditary angioedema; lanadelumab; long-term prophylaxis; quality of life.

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Conflict of interest statement

W. R. Lumry is a member of advisory boards for BioCryst, CSL Behring, and Takeda; has received research grants from BioCryst, CSL Behring, Ionis, and Takeda, consultant fees from BioCryst, CSL Behring, Fresenius Kabi, Pharming, and Takeda, and payments for lectures from CSL Behring, Pharming, and Takeda; and is a medical advisory board member of the US Hereditary Angioedema Association. K. Weller has received research grant support and/or honoraria for educational lectures and/or consultant fees from BioCryst, CSL Behring, Moxie, and Takeda. M. Magerl has received research grant support and/or speaker/consultancy fees from BioCryst, CSL Behring, KalVista, Octapharma, Pharming, and Takeda. A. Banerji has received institutional research/study support from BioCryst and Takeda; and/or honoraria for consulting from Alnylam, BioCryst, CSL Behring, KalVista, Pharming, Pharvaris, and Takeda, and is a medical advisory board member of the US Hereditary Angioedema Association. H. J. Longhurst has received research grant support and/or speaker/consultancy fees from Adverum, BioCryst, CSL Behring, GlaxoSmithKline, Octapharma, Pharming, Pharvaris, and Takeda. M. A. Riedl has received research grants from BioCryst, CSL Behring, Ionis, Pharming, and Takeda; consulting fees from Adverum, Attune, BioCryst, CSL Behring, Ionis, KalVista, Pharming, Pharvaris, and Takeda; and speaker honoraria from CSL Behring, Pharming, and Takeda; and is a medical advisory board member of the US Hereditary Angioedema Association. H. B. Lewis is a full‐time employee of ICON plc. P. Lu was a full‐time employee of Takeda at the time of the study and holds stock/stock options in Takeda; her current affiliation is Pharvaris B.V. G. Devercelli and G. Jain are full‐time employees of and hold stock/stock options in Takeda. M. Maurer is or recently was a speaker and/or advisor for BioCryst, CSL Behring, KalVista, Moxie, Pharming, Pharvaris, and Takeda, and has received research funding from BioCryst, CSL Behring, Moxie, Pharming, and Takeda.

Figures

FIGURE 1
FIGURE 1
Mean change from baseline in AE‐QoL total and domain scores from day 0 to 182 for the (A) lanadelumab total group and (B) individual lanadelumab treatment groups vs the placebo group. *p < 0.05; **p < 0.01. The lanadelumab total group and placebo group were compared using analysis of covariance, adjusting for baseline scores. The dotted line indicates the minimal clinically important difference (−6) in AE‐QoL total score. AE‐QoL, Angioedema Quality of Life Questionnaire; LSM, least squares mean; q2wks, every 2 weeks; q4wks, every 4 weeks
FIGURE 2
FIGURE 2
Proportions of patients with functioning‐related restrictions at day 0 and 182, measured using responses to items in the AE‐QoL functioning domain (by treatment group). Items 1–4 of the AE‐QoL comprise the functioning domain. Percentages reflect combined responses for patients who reported experiencing symptoms “occasionally,” “often,” and “very often.” AE‐QoL, Angioedema Quality of Life Questionnaire; q2wks, every 2 weeks; q4wks, every 4 weeks
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References

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