129
Views
0
CrossRef citations to date
0
Altmetric
Review

A safety review of prophylaxis drugs for adolescent patients with hereditary angioedema

ORCID Icon & ORCID Icon
Pages 549-561 | Received 31 Jan 2023, Accepted 14 Jun 2023, Published online: 21 Jun 2023

References

  • Aygören-Pürsün E, Magerl M, Maetzel A, et al. Epidemiology of Bradykinin-mediated angioedema: a systematic investigation of epidemiological studies. Orphanet J Rare Dis. 2018;13(1):1–9. doi: 10.1186/s13023-018-0815-5
  • Mansi M, Zanichelli A, Coerezza A, et al. Presentation, diagnosis and treatment of angioedema without wheals: a retrospective analysis of a cohort of 1058 patients. J Intern Med. 2015;277(5):585–593. doi: 10.1111/joim.12304
  • Bork K, Wulff K, Witzke G, et al. Hereditary angioedema with normal C1-INH with versus without specific F12 gene mutations. Allergy: Eur J Allergy Clin Immunol. 2015;70(8):1004–1012. doi: 10.1111/all.12648
  • Ponard D, Gaboriaud C, Charignon D, et al. SERPING1 mutation update: mutation spectrum and C1 Inhibitor phenotypes. Hum Mutat. 2020;41(1):38–57. doi: 10.1002/humu.23917
  • Davis AE III, Mejia P, Lu F. Biological activities of C1 inhibitor. Mol Immunol. 2008;45(16):4057–4063. doi: 10.1016/j.molimm.2008.06.028
  • Kaplan AP, Joseph K. Complement, kinins, and hereditary angioedema: mechanisms of plasma instability when C1 inhibitor is absent. Clin Rev Allergy Immunol. 2016;51:207–215. InternetAvailable from. doi: 10.1007/s12016-016-8555-6
  • Kaplan AP, Joseph K. The bradykinin-forming cascade and its role in hereditary angioedema. Ann Allergy Asthma Immunol. 2010;104:193–204. InternetAvailable from. doi: 10.1016/j.anai.2010.01.007
  • Reshef A, Zanichelli A, Longhurst H, et al. Elevated D-dimers in attacks of hereditary angioedema are not associated with increased thrombotic risk. Allergy: Eur J Allergy Clin Immunol. 2015;70(5):506–513. doi: 10.1111/all.12587
  • Caballero T. Angio-oedema due to hereditary C1 inhibitor deficiency in children. Allergol Immunopathol. 2013;41:45–53. InternetAvailable from. doi: 10.1016/j.aller.2012.01.002
  • Farkas H, Kőhalmi KV, Visy B, et al. Clinical characteristics and safety of plasma-derived C1-inhibitor therapy in children and adolescents with hereditary angioedema—A long-term survey. J Allergy Clin Immunol Pract. 2020;8(7):2379–2383. doi: 10.1016/j.jaip.2020.02.043
  • Martinez-Saguer I, Farkas H. Erythema marginatum as an early symptom of hereditary angioedema: case report of 2 newborns. Pediatrics. 2016;137(2):15–17. doi: 10.1542/peds.2015-2411
  • Bork K, Meng G, Staubach P, et al. Hereditary Angioedema: new findings concerning symptoms, affected organs, and course. Am j med. 2006;119(3):267–274. InternetAvailable from. doi: 10.1016/j.amjmed.2005.09.064
  • Farkas H, Martinez-Saguer I, Bork K, et al. International consensus on the diagnosis and management of pediatric patients with hereditary angioedema with C1 inhibitor deficiency. Allergy: Eur J Allergy Clin Immunol. 2017;72(2):300–313. doi: 10.1111/all.13001
  • Farkas H. Pediatric hereditary angioedema due to C1-inhibitor deficiency. Allergy, Asthma Clin Immunol. 2010;6(1):1–10. doi: 10.1186/1710-1492-6-18
  • Agostoni A, Cicardi M. Hereditary and acquired Cl-inhibitor deficiency: biological and clinical characteristics in 235 patients. Med. 1992;71:206–215. doi: 10.1097/00005792-199207000-00003
  • Zanichelli A, Longhurst HJ, Maurer M, et al. Misdiagnosis trends in patients with hereditary angioedema from the real-world clinical setting. Ann Allergy Asthma Immunol. 2016;117(4):394–398. doi: 10.1016/j.anai.2016.08.014
  • Bork K, Hardt J, Schicketanz KH, et al. Clinical studies of sudden upper airway obstruction in patients with hereditary angioedema due to C1 esterase inhibitor deficiency. Arch Intern Med. 2003;163(10):1229–1235. doi: 10.1001/archinte.163.10.1229
  • Farkas H. Management of upper airway edema caused by hereditary angioedema. Allergy, Asthma Clin Immunol. 2010;6(1):1–8. doi: 10.1186/1710-1492-6-19
  • Balla Z, Andrási N, Pólai Z, et al. The characteristics of upper airway edema in hereditary and acquired angioedema with C1-inhibitor deficiency. Clin Transl Allergy. 2021;11(10):1–10. doi: 10.1002/clt2.12083
  • Maurer M, Magerl M, Betschel S, et al. The international WAO/EAACI guideline for the management of hereditary angioedema—The 2021 revision and update. Allergy: Eur J Allergy Clin Immunol. 2022;77(7):1961–1990. doi: 10.1111/all.15214
  • Farkas H, Veszeli N, Kajdácsi E, et al. “Nuts and Bolts” of laboratory evaluation of angioedema. Clin Rev Allergy Immunol. 2016;51:140–151. InternetAvailable from. doi: 10.1007/s12016-016-8539-6
  • Germenis AE, Margaglione M, Pesquero JB, et al. International consensus on the use of genetics in the management of hereditary angioedema. J Allergy Clin Immunol Pract. 2020;8(3):901–911. doi: 10.1016/j.jaip.2019.10.004
  • FDA. Ruconest® Label. 2014. 1–22.
  • FDA. BERINERT [C1 Esterase Inhibitor (Human) Label. 2020. 0–20.
  • FDA. CINRYZE® Label. 2008.
  • Martelo JL, Woytowish MR, Chambers H. Ecallantide for treatment of acute attacks of hereditary angioedema. Am J Heal Pharm. 2012;69:651–657. doi: 10.2146/ajhp110227
  • FDA. KALBITOR (ecallantide) Label. 2020.
  • Cicardi M, Banerji A, Bracho F, et al. Icatibant, a new bradykinin-receptor antagonist, in hereditary angioedema. N Engl J Med. 2010;363(6):532–541. doi: 10.1056/NEJMoa0906393
  • Farkas H, Kőhalmi KV. Icatibant for the treatment of hereditary angioedema with C1-inhibitor deficiency in adolescents and in children aged over 2 years. Expert Rev Clin Immunol. 2018;14:447–460. InternetAvailable from. doi: 10.1080/1744666X.2018.1476851
  • Gelfand JA, Sherins RJ, Alling DW, et al. Treatment of hereditary angioedema with danazol. Reversal of clinical and biochemical abnormalities. N Engl J Med. 1976;295(26):1444–1448. doi: 10.1056/NEJM197612232952602
  • Pappalardo E, Zingale LC, Cicardi M. Increased expression of C1-inhibitor mRNA in patients with hereditary angioedema treated with Danazol. Immunol Lett. 2003;86(3):271–276. doi: 10.1016/S0165-2478(03)00029-4
  • Drouet C, Desormeaux A, Robillard J, et al. Metallopeptidase activities in hereditary angioedema: effect of androgen prophylaxis on plasma aminopeptidase P. J Allergy Clin Immunol. 2008;121(2):429–433. doi: 10.1016/j.jaci.2007.10.048
  • Frank MM, Sergent JS, Kane MA, et al. Epsilon aminocaproic acid therapy of hereditary angioneurotic edema. N Engl J Med. 1972;286(15):808–812. doi: 10.1056/NEJM197204132861503
  • Aygören-Pürsün E, Martinez Saguer I, Kreuz W, et al. Risk of angioedema following invasive or surgical procedures in HAE type i and II - The natural history. Allergy: Eur J Allergy Clin Immunol. 2013;68:1034–1039. doi: 10.1111/all.12186
  • Farkas H, Zotter Z, Csuka D, et al. Short-term prophylaxis in hereditary angioedema due to deficiency of the C1-inhibitor - a long-term survey. Allergy: Eur J Allergy Clin Immunol. 2012;67:1586–1593. doi: 10.1111/all.12032
  • Jurado-Palomo J, Muñoz-Caro J, López-Serrano MC, et al. Management of dental-oral procedures in patients with hereditary angioedema due to C1 inhibitor deficiency. J Investig Allergol Clin Immunol. 2013;23:1–6.
  • Bork K, Hardt J, Staubach-Renz P, et al. Risk of laryngeal edema and facial swellings after tooth extraction in patients with hereditary angioedema with and without prophylaxis with C1 inhibitor concentrate: a retrospective study. Oral Surg Oral Med Oral Pathol Oral Radiol Endodontol. 2011;112:58–64. InternetAvailable from. doi: 10.1016/j.tripleo.2011.02.034
  • Betschel S, Badiou J, Binkley K, et al. The international/Canadian hereditary angioedema guideline. Allergy, Asthma Clin Immunol. 2019;15:1–29. InternetAvailable from. doi: 10.1186/s13223-019-0376-8
  • European Medicines Agency. Cinryze - Assessment report. EMA [Internet]. Available from: https://www.ema.europa.eu/en/medicines/human/EPAR/cinryze.
  • Gavigan G, Yang WH, Santucci S, et al. The prophylactic use of C1 inhibitor in hereditary angioedema patients undergoing invasive surgical procedures: a retrospective study. Allergy, Asthma Clin Immunol. 2014;10(1):1–5. doi: 10.1186/1710-1492-10-17
  • Lumry W, Manning ME, Hurewitz DS, et al. Nanofiltered C1-esterase inhibitor for the acute management and prevention of hereditary angioedema attacks due to c1-inhibitor deficiency in children. J Pediatr. 2013;162:1017–1022.e2. InternetAvailable from. doi: 10.1016/j.jpeds.2012.11.030
  • Balla Z, Ignácz B, Varga L, et al. How angioedema quality of life questionnaire can help physicians in treating C1-inhibitor deficiency patients? Clin Rev Allergy Immunol. 2021;61:50–59. InternetAvailable from. doi: 10.1007/s12016-021-08850-9
  • 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(20):2108–2121. doi: 10.1001/jama.2018.16773
  • Busse P, Tachdjian R, Longhurst H, et al. Long-term efficacy and safety of lanadelumab for prophylactic treatment in adolescent patients with hereditary angioedema (HAE). J Allergy Clin Immunol. 2020;145(2):AB103. doi: 10.1016/j.jaci.2019.12.623
  • Banerji A, Bernstein JA, Johnston DT, et al. Long-term prevention of hereditary angioedema attacks with lanadelumab: the HELP OLE Study. Allergy: Eur J Allergy Clin Immunol. 2022;77(3):979–990. doi: 10.1111/all.15011
  • BioCryst. BioCryst announces approval of ORLADEYO TM (berotralstat) in Japan for the prophylactic treatment of hereditary angioedema. 2021.
  • FDA. ORLADEYO® (berotralstat) Label. 2020. 1–13.
  • Zuraw B, Lumry WR, Johnston DT, et al. Oral once-daily berotralstat for the prevention of hereditary angioedema attacks: a randomized, double-blind, placebo-controlled phase 3 trial. J Allergy Clin Immunol. 2021;148(1):164–172.e9. doi: 10.1016/j.jaci.2020.10.015
  • Wedner HJ, Aygören-Pürsün E, Bernstein J, et al. Randomized trial of the efficacy and safety of berotralstat (bcx7353) as an Oral Prophylactic Therapy for Hereditary Angioedema: results of APeX-2 through 48 weeks (Part 2). J Allergy Clin Immunol Pract. 2021;9:2305–2314.e4. InternetAvailable from. doi: 10.1016/j.jaip.2021.03.057
  • Farkas H, Stobiecki M, Peter J, et al. Long-term safety and effectiveness of berotralstat for hereditary angioedema: the open-label APeX-S study. Clin Transl Allergy. 2021;11(4):11. doi: 10.1002/clt2.12035
  • Ohsawa I, Honda D, Suzuki Y, et al. Oral berotralstat for the prophylaxis of hereditary angioedema attacks in patients in Japan: a phase 3 randomized trial. Allergy: Eur J Allergy Clin Immunol. 2021;76(6):1789–1799. doi: 10.1111/all.14670
  • Zuraw BL, Busse PJ, White M, et al. Nanofiltered C1 Inhibitor concentrate for treatment of hereditary angioedema. N Engl J Med. 2010;363:513–522. doi: 10.1056/NEJMoa0805538
  • Zuraw BL, Kalfus I. Safety and efficacy of prophylactic nanofiltered C1-inhibitor in hereditary angioedema. Am j med. 2012;125:.e938.1–.e938.7. InternetAvailable from. doi: 10.1016/j.amjmed.2012.02.020
  • Aygören-Pürsün E, Soteres D, Moldovan D, et al. Preventing HEREDITARY ANGIOEDEMA ATTACKS IN CHILDREN USing Cinryze®: interim efficacy and safety phase 3 findings. Int Arch Allergy Immunol. 2017;173(2):114–119. doi: 10.1159/000477541
  • Aygören-Pürsün E, Soteres DF, Nieto-Martinez SA, et al. A randomized trial of human C1 inhibitor prophylaxis in children with hereditary angioedema. Pediatr Allergy Immunol. 2019;30(5):553–561. doi: 10.1111/pai.13060
  • Craig T, Shapiro R, Vegh A, et al. Efficacy and safety of an intravenous C1-inhibitor concentrate for long-term prophylaxis in hereditary angioedema. Allergy Rhinol. 2017;8:13–19. doi: 10.2500/ar.2017.8.0192
  • Behring C. HAEGARDA® (C1 Esterase Inhibitor Subcutaneous [Human]). 2020;1–26.
  • Longhurst H, Cicardi M, Craig T, et al. Prevention of hereditary angioedema attacks with a subcutaneous C1 inhibitor. N Engl J Med. 2017;376(12):1131–1140. doi: 10.1056/NEJMoa1613627
  • 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(6):1793–1802.e2. doi: 10.1016/j.jaip.2019.01.054
  • Levy D, Caballero T, Hussain I, et al. Long-term efficacy of subcutaneous C1 inhibitor in pediatric patients with hereditary angioedema. Pediatr Allergy Immunol Pulmonol. 2020;33(3):136–141. doi: 10.1089/ped.2020.1143
  • Bork K, Schneiders V. Danazol-induced hepatocellular adenoma in patients with hereditary angio-oedema [3]. J Hepatol. 2002;36(5):707–709. doi: 10.1016/S0168-8278(02)00035-1
  • Berkel AEM, Bouman DE, Schaafsma MR, et al. Hepatocellular carcinoma after danazol treatment for hereditary angio-oedema. Neth J Med. 2014;72:380–382.
  • Rahal S, Gilabert M, Ries P, et al. Hepatocellular carcinoma in a noncirrhotic liver after long-term use of danazol for hereditary angioedema. Case Rep Oncol. 2014;7(3):825–827. doi: 10.1159/000370106
  • Crampon D, Barnoud R, Durand M, et al. Danazol therapy: an unusual aetiology of hepatocellular carcinoma. J Hepatol Netherlands. 1998;29:1035–1036. doi: 10.1016/S0168-8278(98)80140-2
  • Thoufeeq MH, Ishtiaq J, Abuzakouk M. Danazol-induced hepatocellular carcinoma in a patient with hereditary angioedema. J Gastrointest Cancer. 2012;43(S1):280–282. doi: 10.1007/s12029-012-9402-5
  • Craig TJ. Appraisal of danazol prophylaxis for hereditary angioedema. Allergy Asthma Proc. 2008;29(3):225–231. doi: 10.2500/aap.2008.29.3107
  • Bork K, Bygum A, Hardt J. Benefits and risks of danazol in hereditary angioedema: a long-term survey of 118 patients. Ann Allergy Asthma Immunol. 2008;100:153–161. InternetAvailable from. doi: 10.1016/S1081-1206(10)60424-3
  • Farkas H, Czaller I, Csuka D, et al. The effect of long-term danazol prophylaxis on liver function in hereditary angioedema-a longitudinal study. Eur J Clin Pharmacol. 2010;66:419–426. doi: 10.1007/s00228-009-0771-z
  • Farkas H, Csuka D, Zotter Z, et al. Prophylactic therapy in children with hereditary angioedema. J Allergy Clin Immunol. 2013;131:579–582.e2. doi: 10.1016/j.jaci.2012.08.001
  • Kohalmi KV, Veszeli N, Zotter Z, et al. The effect of long-term danazol treatment on haematological parameters in hereditary angioedema Rare immune deficiencies. Orphanet J Rare Dis. 2016;11(1):1–7. doi: 10.1186/s13023-016-0386-2
  • Kőhalmi KV, Veszeli N, Luczay A, et al. Effect of danazol treatment on growth in pediatric patients with hereditary angioedema due to C1-inhibitor deficiency. Orv Hetil. 2017;158:1269–1276. doi: 10.1556/650.2017.30806
  • FDA. DANOCRINE® (Brand of DANAZOL) Label. 2013. 1–9. Available from: http://www.accessdata.fda.gov/scripts/cder/iig/index.cfm
  • Horiuchi T, Hide M, Yamashita K, et al. The use of tranexamic acid for on-demand and prophylactic treatment of hereditary angioedema—A systematic review. J Cutan Immunol Allergy. 2018;1:126–138. doi: 10.1002/cia2.12029
  • Sheffer AL, Austen KF, Rosen FS. Tranexamic acid therapy in hereditary angioneurotic edema. N Engl J Med. 1972;287(9):452–454. doi: 10.1056/NEJM197208312870907
  • Agostoni A, Marasini B, Cicardi M, et al. Hepatic function and fibrinolysis in patients with hereditary angioedema undergoing long‐term treatment with tranexamic acid. Allergy. 1978;33(4):216–221. doi: 10.1111/j.1398-9995.1978.tb01537.x
  • Zanichelli A, Vacchini R, Badini M, et al. Standard care impact on angioedema because of hereditary C1 inhibitor deficiency: a 21-month prospective study in a cohort of 103 patients. Allergy. 2011;66(2):192–196. doi: 10.1111/j.1398-9995.2010.02433.x
  • Bygum A. Hereditary angioedema - Consequences of a new treatment paradigm in Denmark. Acta Derm Venereol. 2014;94:436–441. doi: 10.2340/00015555-1743
  • Wintenberger C, Boccon-Gibod I, Launay D, et al. Tranexamic acid as maintenance treatment for non-histaminergic angioedema: analysis of efficacy and safety in 37 patients. Clin Exp Immunol. 2014;178(1):112–117. doi: 10.1111/cei.12379

Reprints and Corporate Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

To request a reprint or corporate permissions for this article, please click on the relevant link below:

Academic Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

Obtain permissions instantly via Rightslink by clicking on the button below:

If you are unable to obtain permissions via Rightslink, please complete and submit this Permissions form. For more information, please visit our Permissions help page.