@article{c8787643f92344d480eed983e23430c5,
title = "Long-term prevention of hereditary angioedema attacks with lanadelumab: The HELP OLE Study",
abstract = "Background: The aim was to evaluate long-term effectiveness and safety of lanadelumab in patients ≥12 y old with hereditary angioedema (HAE) 1/2 (NCT02741596). Methods: Rollover patients completing the HELP Study and continuing into HELP OLE received one lanadelumab 300 mg dose until first attack (dose-and-wait period), then 300 mg q2wks (regular dosing stage). Nonrollovers (newly enrolled) received lanadelumab 300 mg q2wks from day 0. Baseline attack rate for rollovers: ≥1 attack/4 weeks (based on run-in period attack rate during HELP Study); for nonrollovers: historical attack rate ≥1 attack/12 weeks. The planned treatment period was 33 months. Results: 212 patients participated (109 rollovers, 103 nonrollovers); 81.6% completed ≥30 months on study (mean [SD], 29.6 [8.2] months). Lanadelumab markedly reduced mean HAE attack rate (reduction vs baseline: 87.4% overall). Patients were attack free for a mean of 97.7% of days during treatment; 81.8% and 68.9% of patients were attack free for ≥6 and ≥12 months, respectively. Angioedema Quality-of-Life total and domain scores improved from day 0 to end of study. Treatment-emergent adverse events (TEAEs) (excluding HAE attacks) were reported by 97.2% of patients; most commonly injection site pain (47.2%) and viral upper respiratory tract infection (42.0%). Treatment-related TEAEs were reported by 54.7% of patients. Most injection site reactions resolved within 1 hour (70.2%) or 1 day (92.6%). Six (2.8%) patients discontinued due to TEAEs. No treatment-related serious TEAEs or deaths were reported. Eleven treatment-related TEAEs of special interest were reported by seven (3.3%) patients. Conclusion: Lanadelumab demonstrated sustained efficacy and acceptable tolerability with long-term use in HAE patients.",
keywords = "HAE, HAE attacks, HELP OLE, hereditary angioedema, lanadelumab, long-term prophylaxis",
author = "{for the HELP OLE Investigators} and Aleena Banerji and Bernstein, {Jonathan A.} and Johnston, {Douglas T.} and Lumry, {William R.} and Markus Magerl and Marcus Maurer and Inmaculada Martinez-Saguer and Andrea Zanichelli and James Hao and Neil Inhaber and Ming Yu and Riedl, {Marc A.} and J. H{\'e}bert and B. Ritchie and G. Sussman and Yang, {W. H.} and E. Ayg{\"o}ren-P{\"u}rs{\"u}n and M. Magerl and I. Martinez-Saguer and P. Staubach and M. Cicardi and M. Shennak and Zaragoza-Urdaz, {R. H.} and S. Kiani-Alikhan and J. Anderson and A. Banerji and Baptist, {A. P.} and Bernstein, {J. A.} and Busse, {P. J.} and T. Craig and M. Davis-Lorton and S. Gierer and Gower, {R. G.} and D. Harris and J. Jacobs and Johnston, {D. T.} and Li, {H. H.} and Lockey, {R. F.} and P. Lugar and Lumry, {W. R.} and Manning, {M. E.} and McNeil, {D. L.} and I. Melamed and Otto, {W. R.} and Rehman, {S. M.} and Riedl, {M. A.} and Schwartz, {L. B.} and R. Shapiro and E. Sher and Smith, {A. M.}",
note = "Funding Information: This study was sponsored by Shire Human Genetic Therapies, Inc., a Takeda company. Under the direction of the authors, Sophia Shumyatsky, PharmD, CMPP, employee of Excel Medical Affairs, provided writing assistance for this manuscript. Editorial assistance in formatting, proofreading, copyediting, and fact‐checking also was provided by Excel Medical Affairs. Takeda Development Center Americas, Inc. 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. Funding Information: This study was sponsored by Shire Human Genetic Therapies, Inc., a Takeda company. Under the direction of the authors, Sophia Shumyatsky, PharmD, CMPP, employee of Excel Medical Affairs, provided writing assistance for this manuscript. Editorial assistance in formatting, proofreading, copyediting, and fact-checking also was provided by Excel Medical Affairs. Takeda Development Center Americas, Inc. 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. We thank Kim Paes and Peng Lu (former employees of Takeda Pharmaceutical Company Limited) for providing clinical development support for this study. HELP OLE Principal Investigators: Canada: J. H?bert, B. Ritchie, G. Sussman, W.H. Yang; Germany: E. Ayg?ren-P?rs?n, M. Magerl, I. Martinez-Saguer, P. Staubach; Italy: M. Cicardi*; Jordan: M. Shennak; Puerto Rico: R.H. Zaragoza-Urdaz; United Kingdom: S. Kiani-Alikhan; United States: J. Anderson, A. Banerji, A.P. Baptist, J.A. Bernstein, P.J. Busse, T. Craig, M. Davis-Lorton, S. Gierer, R.G. Gower, D. Harris, J. Jacobs, D.T. Johnston, H.H. Li, R.F. Lockey, P. Lugar, W.R. Lumry, M.E. Manning, D.L. McNeil, I. Melamed, W.R. Otto, S.M. Rehman, M.A. Riedl, L.B. Schwartz, R. Shapiro, E. Sher, A.M. Smith, D. Soteres, R. Tachdjian, H.J. Wedner, M.E. Weinstein, H. Zafra. *Deceased. Funding Information: A. Banerji has received institutional research/study support from BioCryst and Takeda and/or honoraria for consulting from BioCryst, CSL Behring, KalVista, Pharming, Pharvaris, and Takeda. J.A. Bernstein has been or is currently a clinical investigator for BioCryst, CSL Behring, Ionis, KalVista, Pharming, and Takeda; a speaker for CSL Behring, Pharming, and Takeda; a consultant for BioCryst, CSL Behring, Fresenius Kabi, Ionis, KalVista, Pharming, and Takeda; and a member of the hereditary angioedema medical advisory board. D.T. Johnston has received consulting/speaker fees from CSL Behring, Pharming, and Takeda, and consulting fees from BioCryst and REGENXBIO. 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; has received consulting fees from BioCryst, CSL Behring, Fresenius Kabi, Pharming, and Takeda; payments for lectures from CSL Behring, Pharming, and Takeda; and is an advisory board member of the US Hereditary Angioedema Association. M. Magerl has received research grant support and/or speaker/consultancy fees from BioCryst, CSL Behring, KalVista, Pharming, and Takeda. M. Maurer has received research grant support and/or speaker/consultancy fees from Adverum, BioCryst, CSL Behring, KalVista, Pharming, Pharvaris, and Takeda. I. Martinez‐Saguer has received research grant support and/or speaker/consultancy fees from BioCryst, CSL Behring, KalVista, Pharming, and Takeda. A. Zanichelli has received speaker/consultancy fees from BioCryst, CSL Behring, Pharming, and Takeda. J. Hao, N. Inhaber, and M. Yu are full‐time employees of and hold stock/stock options in Takeda. M.A. Riedl has received research grants from BioCryst, CSL Behring, Pharming, and Takeda; has received consulting fees from Adverum, Attune, BioCryst, CSL Behring, Ionis, KalVista, Pharming, and Takeda; payments for lectures from CSL Behring, Pharming, and Takeda; and is an advisory board member of the US Hereditary Angioedema Association. Publisher Copyright: {\textcopyright} 2021 Takeda Development Center Americas, Inc. Allergy published by European Academy of Allergy and Clinical Immunology and John Wiley & Sons Ltd.",
year = "2022",
month = mar,
doi = "10.1111/all.15011",
language = "English",
volume = "77",
pages = "979--990",
journal = "Allergy: European Journal of Allergy and Clinical Immunology",
issn = "0105-4538",
publisher = "Wiley-Blackwell Publishing Ltd",
number = "3",
}