Lanadelumab for the treatment of hereditary angioedema

References

• Aygoren-Pursun E, Magerl M, Maetzel A, et al. Epidemiology of Bradykinin-mediated angioedema: a systematic investigation of epidemiological studies. Orphanet J Rare Dis. 2018;13:73.
   PubMed Web of Science ®Google Scholar
• Lachmann PJ, Rosen FS. The catabolism of C1(-)-inhibitor and the pathogenesis of hereditary angio-edema. Acta Pathol Microbiol Immunol Scand Suppl. 1984;284:35–39.
   PubMedGoogle Scholar
• Longhurst H, Cicardi M. Hereditary angio-oedema. Lancet. 2012;379:474–481.
   PubMed Web of Science ®Google Scholar
• Pappalardo E, Cicardi M, Duponchel C, et al. Frequent de novo mutations and exon deletions in the C1inhibitor gene of patients with angioedema. J Allergy Clin Immunol. 2000;106:1147–1154.
   PubMed Web of Science ®Google Scholar
• Wu MA, Perego F, Zanichelli A, et al. Angioedema Phenotypes: disease Expression and Classification. Clin Rev Allergy Immunol. 2016;51:162–169.
   PubMed Web of Science ®Google Scholar
• Cicardi M, Aberer W, Banerji A, et al. Classification, diagnosis, and approach to treatment for angioedema: consensus report from the hereditary angioedema international working group. Allergy. 2014;69:602–616.
   PubMed Web of Science ®Google Scholar
• Dewald G, Bork K. Missense mutations in the coagulation factor XII (Hageman factor) gene in hereditary angioedema with normal C1 inhibitor. Biochem Biophys Res Commun. 2006;343:1286–1289.
   PubMed Web of Science ®Google Scholar
• Bjorkqvist J, de Maat S, Lewandrowski U, et al. Defective glycosylation of coagulation factor XII underlies hereditary angioedema type III. J Clin Invest. 2015;125:3132–3146.
   PubMed Web of Science ®Google Scholar
• Zuraw BL, Bork K, Binkley KE, et al. Hereditary angioedema with normal C1 inhibitor function: consensus of an international expert panel. Allergy Asthma Proc. 2012;33(Suppl 1):S145–156.
   PubMed Web of Science ®Google Scholar
• Bafunno V, Firinu D, D’Apolito M, et al. Mutation of the angiopoietin-1 gene (ANGPT1) associates with a new type of hereditary angioedema. J Allergy Clin Immunol. 2018;141:1009–1017.
   PubMedGoogle Scholar
• Bork K, Wulff K, Steinmuller-Magin L, et al. Hereditary angioedema with a mutation in the plasminogen gene. Allergy. 2018;73:442–450.
   PubMed Web of Science ®Google Scholar
• Bork K, Wulff K, Rossmann H, et al. Hereditary angioedema cosegregating with a novel kininogen 1 gene mutation changing the N-terminal cleavage site of bradykinin. Allergy. 2019. cited 2019 May 14. Epub ahead of print. doi:10.1111/all.13869.
   Web of Science ®Google Scholar
• Bock SC, Skriver K, Nielsen E, et al. Human C1 inhibitor: primary structure, cDNA cloning, and chromosomal localization. Biochemistry. 1986;25:4292–4301.
   PubMed Web of Science ®Google Scholar
• Davis AE 3rd, Mejia P, Lu F. Biological activities of C1 inhibitor. Mol Immunol. 2008;45:4057–4063.
   PubMed Web of Science ®Google Scholar
• Leeb-Lundberg LM, Marceau F, Müller-Esterl W, et al. International union of pharmacology. XLV. Classification of the kinin receptor family: from molecular mechanisms to pathophysiological consequences. Pharmacol Rev. 2005;57:27–77.
   PubMed Web of Science ®Google Scholar
• Zuraw BL, Christiansen SC. HAE pathophysiology and underlying mechanisms. Clin Rev Allergy Immunol. 2016;51:216–229. Review.
   PubMed Web of Science ®Google Scholar
• Zanichelli A, Magerl M, Longhurst HJ, et al.; Group IOSS. Improvement in diagnostic delays over time in patients with hereditary angioedema: findings from the icatibant outcome survey. Clin Transl Allergy. 2018;8:42. eCollection 2018. .
   PubMed Web of Science ®Google Scholar
• Banerji A, Li Y, Busse P, et al. Hereditary angioedema from the patient’s perspective: A follow-up patient survey. Allergy Asthma Proc. 2018;39:212–223.
   PubMed Web of Science ®Google Scholar
• Bygum A, Aygoren-Pursun E, Beusterien K, et al. Burden of illness in hereditary angioedema: a conceptual model. Acta Derm Venereol. 2015;95:706–710.
   PubMed Web of Science ®Google Scholar
• Longhurst H, Bygum A. The humanistic, societal, and pharmaco-economic burden of angioedema. Clin Rev Allergy Immunol. 2016;51:230–239.
   PubMed Web of Science ®Google Scholar
• Caballero T, Aygoren-Pursun E, Bygum A, et al. The humanistic burden of hereditary angioedema: results from the burden of illness study in Europe. Allergy Asthma Proc. 2014;35:47–53.
   PubMed Web of Science ®Google Scholar
• Busse PJ, Christiansen SC, Birmingham JM, et al. Development of a health-related quality of life instrument for patients with hereditary angioedema living in the United States. J Allergy Clin Immunol Pract. 2019;7(1679–1683):e1677.
   Google Scholar
• Cicardi M, Levy RJ, McNeil DL, et al. Ecallantide for the treatment of acute attacks in hereditary angioedema. N Engl J Med. 2010;363:523–531.
   PubMed Web of Science ®Google Scholar
• Levy JH, O’Donnell PS. The therapeutic potential of a kallikrein inhibitor for treating hereditary angioedema. Expert Opin Investig Drugs. 2006;15:1077–1090.
   PubMed Web of Science ®Google Scholar
• Schneider L, Lumry W, Vegh A, et al. Critical role of kallikrein in hereditary angioedema pathogenesis: a clinical trial of ecallantide, a novel kallikrein inhibitor. J Allergy Clin Immunol. 2007;120:416–422.
   PubMed Web of Science ®Google Scholar
• Levy RJ, Lumry WR, McNeil DL, et al. EDEMA4: a phase 3, double-blind study of subcutaneous ecallantide treatment for acute attacks of hereditary angioedema. Ann Allergy Asthma Immunol. 2010;104:523–529.
   PubMed Web of Science ®Google Scholar
• Lumry WR, Bernstein JA, Li HH, et al. Efficacy and safety of ecallantide in treatment of recurrent attacks of hereditary angioedema: open-label continuation study. Allergy Asthma Proc. 2013;34:155–161.
   PubMed Web of Science ®Google Scholar
• Riedl MA. Critical appraisal of androgen use in hereditary angioedema: a systematic review. Ann Allergy Asthma Immunol. 2015;114(281–288):e287.
   Google Scholar
• Bouillet L, Boccon-Gibod I, Launay D, et al. Shortage in France of plasma derived C1-INH concentrates: state of play and consequences for patients. Allergy Asthma Clin Immunol. 2019;15(Suppl4): P40. from the CREAK.
   PubMedGoogle Scholar
• Craig T, Zuraw B, Longhurst H, et al. Long-term outcomes with subcutaneous C1-inhibitor replacement therapy for prevention of hereditary angioedema attacks. J Allergy Clin Immunol Pract. 2019;7(1793–1802):e1792.
   Google Scholar
• Craig T, Lumry W, Cicardi M, et al. Treatment effect of switching from intravenous to subcutaneous C1-inhibitor for prevention of hereditary angioedema attacks: COMPACT subgroup findings. J Allergy Clin Immunol Pract. 2019;7:2035–2038.
   PubMed Web of Science ®Google Scholar
• Reshef A, Moldovan D, Obtulowicz K, et al. Recombinant human C1 inhibitor for the prophylaxis of hereditary angioedema attacks: a pilot study. Allergy. 2013;68:118–124.
   PubMed Web of Science ®Google Scholar
• Riedl MA, Grivcheva-Panovska V, Moldovan D, et al. Recombinant human C1 esterase inhibitor for prophylaxis of hereditary angio-oedema: a phase 2, multicentre, randomised, double-blind, placebo-controlled crossover trial. Lancet. 2017;390:1595–1602.
   PubMed Web of Science ®Google Scholar
• Riedl MA, Aygoren-Pursun E, Baker J, et al. Evaluation of avoralstat, an oral kallikrein inhibitor, in a Phase 3 hereditary angioedema prophylaxis trial: the OPuS-2 study. Allergy. 2018;73:1871–1880.
   PubMed Web of Science ®Google Scholar
• Aygoren-Pursun E, Bygum A, Grivcheva-Panovska V, et al. Oral plasma kallikrein inhibitor for prophylaxis in hereditary angioedema. N Engl J Med. 2018;379:352–362.
   PubMed Web of Science ®Google Scholar
• https://www.epgonline.org/uk/news/initialresults- from-zenith-1-trial-of-bcx-7353-or-both-prophylactic and- acute-treatment-of-hae-attacks—biocryst-pharma-.html Data from Pharmawand – Curated by EPG Health – Date added 6 September 2018
   Google Scholar
• Maetzel A, Smith MS, Duckworth EJ, et al. KVD900, a new oral on-demand treatment of hereditary angioedema attacks achieves complete plasma kallikrein suppression: safety, tolerability, pharmacokinetic and pharmacodynamic results from a phase 1 first-in-human study. Allergy Asthma Clin Immunol. 2019;15(Suppl 4):O25.
   PubMedGoogle Scholar
• Kalfus I, Offman E, McDonald A. Pharmacokinetics, safety, and potency of ATN-249, a novel oral plasma kallikrein inhibitor for hereditary angioedema. Allergy Asthma Clin Immunol. 2019;15(Suppl 4):O26.
   PubMedGoogle Scholar
• Lesage ASJ, Knolle J, Marceau F, et al. PHA-022121, the first in class orally active bradykinin receptor B2 antagonist for on-demand and prophylactic treatment of HAE. Allergy Asthma Clin Immnunol. 2019;15(suppl 4):45.
   Google Scholar
• Liu J, Qin J, Borodovsky A, et al. An investigational RNAi therapeutic targeting Factor XII (ALN-F12) for the treatment of hereditary angioedema. RNA. 2019;25:255–263.
   PubMed Web of Science ®Google Scholar
• Kenniston JA, Faucette RR, Martik D, et al. Inhibition of plasma kallikrein by a highly specific active site blocking antibody. J Biol Chem. 2014;289:23596–23608.
   PubMed Web of Science ®Google Scholar
• https://www.fda.gov/Drugs/DrugSafety/ucm618261.htm
   Google Scholar
• Kaplon H, Reichert JM. Antibodies to watch in 2019. MAbs. 2019;11:219–238.
   PubMed Web of Science ®Google Scholar
• Hoet RM, Cohen EH, Kent RB, et al. Generation of high-affinity human antibodies by combining donor-derived and synthetic complementarity-determining-region diversity. Nat Biotechnol. 2005;23:344–348.
   PubMed Web of Science ®Google Scholar
• Alhenc-Gelas F, Bouby N, Girolami JP. Kallikrein/K1, Kinins, and ACE/Kininase II in homeostasis and in disease insight from human and experimental genetic studies, therapeutic implication. Front Med (Lausanne). 2019;6:136. eCollection 2019. Review.
   PubMed Web of Science ®Google Scholar
• Chyung Y, Vince B, Iarrobino R, et al. A phase 1 study investigating DX-2930 in healthy subjects. Ann Allergy Asthma Immunol. 2014;113:460–466 e462.
   PubMed Web of Science ®Google Scholar
• clinical trial.gov NCT03401671
   Google Scholar
• Banerji A, Busse P, Shennak M, et al. Inhibiting plasma kallikrein for hereditary angioedema prophylaxis. N Engl J Med. 2017;376:717–728.
   PubMed Web of Science ®Google Scholar
• Banerji A, Riedl MA, Bernstein JA, et al. Effect of lanadelumab compared with placebo on prevention of hereditary angioedema attacks: a randomized clinical trial. JAMA. 2018;320:2108–2121.
   PubMed Web of Science ®Google Scholar
• on behalf of the HELP Study Investigators., Maurer M, Riedl MA, Bernstein JA, et al. Lanadelumab demonstrates rapid and sustained prevention of hereditary Angioedema (HAE) attacks in the phase 3 HELP study: findings for days 0-69 and at steady state. Allergy. 2019;74(Suppl. 106):209.
   PubMedGoogle Scholar
• Banerji A, Manning ME, Martinez-Saguer I, et al. Subgroup analyses from the phase 3 HELP study of lanadelumab for the prevention of hereditary angioedema attacks. J Allergy Clin Immunol. 2019;143(2):AB42.
   Web of Science ®Google Scholar
• Inhaber N, Zuraw BL, Marier J, et al. Relationship between body weight, PK/ PD,and attack responses following subcutaneous administration of lanadelumab in patients with hereditary angioedema. J Allergy Clin Immunol. 2019;143(2):AB38.
   Web of Science ®Google Scholar
• Wang Y, Marier J, Kassir N, et al. Exposure-response analyses of lanadelumab in patients with hereditary angioedema. J Allergy Clin Immunol. 2019;143(2):AB40.
   Web of Science ®Google Scholar
• on behalf of the HELP Study Investigators, Cicardi M, Maurer M, Longhurst HJ, et al. Lanadelumab prophylaxis reduces breakthrough hereditary angioedema attacks and the need for rescue medication: exploratory findings from the phase 3 HELP study. Allergy. 2019;74(Suppl. 106):212.
   Google Scholar
• Riedl MA, Bernstein JA, Craig T, et al. An open-label study to evaluate the long-term safety and efficacy of lanadelumab for prevention of attacks in hereditary angioedema: design of the HELP study extension. Clin Transl Allergy. 2017;7:36.
   PubMed Web of Science ®Google Scholar
• ClinicalTrials.gov. Long-term Safety and Efficacy Study of DX-2930 to Prevent Acute Angioedema Attacks in Patients with Type I and Type II HAE.https://clinicaltrials.gov/ct2/show/NCT02741596
   Google Scholar
• Riedl MA, Anderson JT, Cicardi M, et al. Efficacy of lanadelumab in hereditary angioedema patients switching from C1 inhibitor long-term prophylaxis: interim results from the HELP open-label extension study. J Allergy Clin Immunol. 2019;143(2):AB37.
   Web of Science ®Google Scholar
• Jacobs JS, Bernstein J, Davis-Lorton M, et al. Increased response to higher dose lanadelumab in hereditary angioedema patients: exploratory findings from the HELP and HELP OLE studies. J Allergy Clin Immunol. 2019;143(2):AB38.
   Web of Science ®Google Scholar
• Busse PJ, Tachdjian R, Martinez-Saguer I, et al. Efficacy and safety of lanadelumab for prophylactic treatment in adolescents with hereditary angioedema (HAE). J Allergy Clin Immunol. 2019;143(2):AB43.
   Web of Science ®Google Scholar
• on behalf of the HELP OLE Study Investigators., Magerl M, Kiani-Alikhan S, Riedl MA, et al. Efficacy and safety outcomes for switching to lanadelumab from prior LTP: findings from an interim analysis of the HELP OLE study. Allergy. 2019;74(Suppl. 106):755.
   Google Scholar
• On behalf of the HELP and HELP OLE Study Investigators, Staubach P, Soteres D, Aygören-Pürsün E, et al. Use of short term prophylaxis during treatment with lanadelumab does not impact the risk of an attack: analysis of data from the HELP study and HELP OLE. Allergy. 2019;74(Suppl. 106):211–212.
   Google Scholar
• Bork K, Witzke G. Shortened Activated Partial Thromboplastin Time May Help in Diagnosing Hereditary and Acquired Angioedema. Int Arch Allergy Immunol. 2016;170:101–107.
   PubMed Web of Science ®Google Scholar
• Schmaier AH, Bauer KA, Cicardi M, et al. Effect of lanadelumab on coagulation parameters in patients with hereditary angioedema: findings from the phase 3 HELP study. J Allergy Clin Immunol. 2019;143(2):AB41.
   Web of Science ®Google Scholar
• Sexton DJ, Brown NJ, Lumry WR, et al. Lanadelumab And Cardiovascular Risk: findings From The Phase 3 HELP Study. J Allergy Clin Immunol. 2019;143(2):AB45.
   Web of Science ®Google Scholar
• [cited 2018 Nov]. https://www.int.takhzyro.com/downloads/pdf/TAKHZYRO_International_EU_SmPC_November_2018.pdf
   Google Scholar
• Koenig A, de Albuquerque Diniz EM, Barbosa SF, et al. Immunologic factors in human milk: the effects of gestational age and pasteurization. J Hum Lact. 2005;21:439–443.
   PubMed Web of Science ®Google Scholar
• Adhisivam B, Vishnu Bhat B, Rao K, et al. Effect of Holder pasteurization on macronutrients and immunoglobulin profile of pooled donor human milk. J Matern Fetal Neonatal Med. 2019;32:3016–3019.
   PubMed Web of Science ®Google Scholar
• Rodriguez-Camejo C, Puyol A, Fazio L, et al. Antibody profile of colostrum and the effect of processing in human milk banks: implications in immunoregulatory properties. J Hum Lact. 2018;34:137–147.
   PubMed Web of Science ®Google Scholar
• Maurer M, Magerl M, Ansotegui I, et al. The international WAO/EAACI guideline for the management of hereditary angioedema-The 2017 revision and update. Allergy. 2018;73:1575–1596.
   PubMed Web of Science ®Google Scholar
• Agboola F, Lubinga S, Carlson J, et al. The effectiveness and value of lanadelumab and C1 esterase inhibitors for prophylaxis of hereditary angioedema attacks. J Manag Care Spec Pharm. 2019;25:143–148.
   PubMed Web of Science ®Google Scholar