@article{858c0c2a38ca4a5a9767efa6d79e6c95,
title = "Impact of lanadelumab on health-related quality of life in patients with hereditary angioedema in the HELP study",
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.",
author = "{on behalf of the HELP Study Investigators} and Lumry, {William R.} and Karsten Weller and Markus Magerl and Aleena Banerji and Longhurst, {Hilary J.} and Riedl, {Marc A.} and Lewis, {Hannah B.} and Peng Lu and Giovanna Devercelli and Gagan Jain and Marcus Maurer and J. H{\'e}bert and B. Ritchie and G. Sussman and Yang, {W. H.} and I. Martinez-Saguer and P. Staubach and M. Cicardi and M. Shennak and Zaragoza-Urdaz, {R. H.} and J. Anderson and Baptist, {A. P.} and Bernstein, {J. A.} and Boggs, {P. B.} and Busse, {P. J.} and T. Craig and M. Davis-Lorton and S. Gierer and Gower, {R. G.} and D. Harris and Hong, {D. I.} and J. Jacobs and Johnston, {D. T.} and Li, {H. H.} and Lockey, {R. F.} and P. Lugar and Manning, {M. E.} and McNeil, {D. L.} and I. Melamed and T. Mostofi and T. Nickel and Otto, {W. R.} and Petrov, {A. A.} and C. Radojicic and Rehman, {S. M.} and Schwartz, {L. B.} and R. Shapiro and E. Sher and Smith, {A. M.} and D. Soteres",
note = "Funding Information: The HELP Study was sponsored by Shire Human Genetic Therapies, Inc, a Takeda company. We thank Yi Wang, Senior Director, Clinical Pharmacology and Pharmacokinetics, Takeda Pharmaceutical Company Limited, for data collection on exposure-response relationships, and James Hao, Project Lead Biostatistician, Takeda Pharmaceutical Company Limited, for reviewing the manuscript. Under the direction of the authors, Alpa Parmar, PhD, CMPP, employee of Excel Medical Affairs, provided writing assistance for this manuscript. Editorial assistance in formatting, proofreading, copy editing, and fact checking also were provided by Excel Medical Affairs. Shire Human Genetic Therapies, Inc, a Takeda company, provided funding to Excel Medical Affairs for support in writing and editing this manuscript. The interpretation of the data was made by the authors independently. HELP Study Principal Investigators: Canada: J H{\'e}bert, B Ritchie, G Sussman, and WH Yang; Germany: M Magerl, I Martinez-Saguer, P Staubach; Italy: M Cicardi†, Jordan: M Shennak; Puerto Rico: RH Zaragoza-Urdaz; United Kingdom: HJ Longhurst; United States: J Anderson, A Banerji, AP Baptist, JA Bernstein, PB Boggs, PJ Busse, T Craig, M Davis-Lorton, S Gierer, RG Gower, D Harris, DI Hong, J Jacobs, DT Johnston, HH Li, RF Lockey, P Lugar, WR Lumry, ME Manning, DL McNeil, I Melamed, T Nickel, WR Otto, AA Petrov, C Radojicic, SM Rehman, MA Riedl, LB Schwartz, R Shapiro, E Sher, AM Smith, D Soteres, R Tachdjian, HJ Wedner, ME Weinstein, H Zafra. †Deceased Funding Information: The HELP Study was sponsored by Shire Human Genetic Therapies, Inc, a Takeda company. We thank Yi Wang, Senior Director, Clinical Pharmacology and Pharmacokinetics, Takeda Pharmaceutical Company Limited, for data collection on exposure‐response relationships, and James Hao, Project Lead Biostatistician, Takeda Pharmaceutical Company Limited, for reviewing the manuscript. Under the direction of the authors, Alpa Parmar, PhD, CMPP, employee of Excel Medical Affairs, provided writing assistance for this manuscript. Editorial assistance in formatting, proofreading, copy editing, and fact checking also were provided by Excel Medical Affairs. Shire Human Genetic Therapies, Inc, a Takeda company, provided funding to Excel Medical Affairs for support in writing and editing this manuscript. The interpretation of the data was made by the authors independently. Publisher Copyright: {\textcopyright} 2020 Takeda Pharmaceuticals Company Limited. Allergy published by European Academy of Allergy and Clinical Immunology and John Wiley & Sons Ltd",
year = "2021",
month = apr,
doi = "10.1111/all.14680",
language = "English (US)",
volume = "76",
pages = "1188--1198",
journal = "Allergy: European Journal of Allergy and Clinical Immunology",
issn = "0105-4538",
publisher = "Wiley-Blackwell",
number = "4",
}