Hereditary angioedema in children and adolescents
Main Article Content
Keywords
Hereditary angioedema, C1 inhibitor, child, attack, treatment, pediatric, prophylaxis
Abstract
Hereditary angioedema is a genetic disease with autosomal dominant inheritance and, in most cases, caused by C1 inhibitor deficiency. Patients present with recurrent edema affecting sub-cutaneous and mucus membranes with variable onset and severity. More than 50% of patients may become symptomatic before 10 years of age. Family history can help with the diagnosis; however, approximately 25% of the cases are de novo mutations. Biochemical diagnosis should be delayed until after 1 year of age. Children were often excluded from advances in therapy for hereditary angioedema since most of the new medicines were tested in adults and thus excluded by the Food and Drug Administration (FDA) and other agencies for approval to be used in children. Treatment of attacks is available for the pediatric patient; however, barriers still exist for the use of long-term prophylaxis in young children.
References
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7. Bygum A, Aygören-Pürsün E, Beusterien K, Hautamaki E, Sisic Z, Wait S, et al. Burden of illness in hereditary angioedema: a conceptual model. Acta Derm Venereol. 2015 Jul;95(6):706–710. 10.2340/00015555-2014
8. Freda MF, Savarese L, Dolce P, Picione RL. Caregivers’ sensemaking of children’s hereditary angioedema: a semiotic narrative analysis of the sense of grip on the disease. Front Psychol. 2019 Nov 27;10:2609. 10.3389/fpsyg.2019.02609
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14. Bork K, Wulff K, Witzke G, Hardt J. Hereditary angioedema with normal C1-INH with versus without specific F12 gene mutations. Allergy. 2015 Aug;70(8):1004–1012. 10.1111/all.12648
15. Deroux A, Boccon-Gibod I, Fain O, Pralong P, Ollivier Y, Pagnier A, et al. Hereditary angioedema with normal C1 inhibitor and factor XII mutation: a series of 57 patients from the French National Center of Reference for Angioedema. Clin Exp Immunol. 2016 Sep;185(3):332–337. 10.1111/cei.12820
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17. Bork K, Machnig T, Wulff K, Witzke G, Prusty S, Hardt J. Clinical features of genetically characterized types of hereditary angioedema with normal C1 inhibitor: a systematic review of qualitative evidence. Orphanet J Rare Dis. 2020 Oct 15;15(1):289. 10.1186/s13023-020-01570-x
18. Bafunno V, Firinu D, D’Apolito M, Cordisco G, Loffredo S, Leccese A, et al. Mutation of the angiopoietin-1 gene (ANGPT1) associates with a new type of hereditary angioedema. J Allergy Clin Immunol. 2018;141(3):1009–1017. 10.1016/j.jaci.2017.05.020
19. Bork K, Wulff K, Steinmüller-Magin L, Braenne I, StaubachRenz P, Witzke G, et al. Hereditary angioedema with a mutation in the plasminogen gene. Allergy. 2018 Feb;73(2):442–450. 10.1111/all.13270
20. Bork K, Wulff K, Rossmann H, Steinmuller-Magin L, Braenne I, Witzke G, et al. Hereditary angioedema cosegregating with a novel kininogen 1 gene mutation changing the N-terminal cleavage site of bradykinin. Allergy. 2019;74(12):2479–2481. 10.1111/all.13869
21. Ariano A, D’Apolito M, Bova M, Bellanti F, Loffredo S, D’Andrea G, et al. A myoferlin gain-of-function variant associates with a new type of hereditary angioedema. Allergy. 2020 Nov;75(11):2989–2992. 10.1111/all.14454
22. Bork K, Wulff K, Möhl BS, Steinmüller-Magin L, Witzke G, Hardt J, et al. Novel hereditary angioedema linked with a heparan sulfate 3-O-sulfotransferase 6 gene mutation. J Allergy Clin Immunol. 2021 Oct;148(4):1041–1048. 10.1016/j.jaci.2021.01.011
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24. Araújo-Simões J, Boanova AGP, Constantino-Silva RN, Fragnan NTML, Pinto JA, Minafra FG, et al. The challenges in the follow-up and treatment of Brazilian children with hereditary angioedema. Int Arch Allergy Immunol, 2021;182(7):585–591. 10.1159/000512944
25. Piotrowicz-Wójcik K, Porebski G. Life-threatening laryngeal attacks in hereditary angioedema patients. Otolaryngol Pol. 2020 Mar 31;74(2):1–5. 10.5604/01.3001.0014.0619
26. Bernstein JA, Cremonesi P, Hoffmann TK, Hollingsworth J. Angioedema in the emergency department: a practical guide to differential diagnosis and management. Int J Emerg Med. 2017 Dec;10(1):15. 10.1186/s12245-017-0141-z
27. Otani IM, Lumry WR, Hurwitz S, Li HH, Craig TJ, Holtzman NS, et al. Subcutaneous icatibant for the treatment of hereditary angioedema attacks: Comparison of home self-administration with administration at a medical facility. J Allergy Clin Immunol Pract. 2017 Mar–Apr;5(2):442–447.e1. 10.1016/j.jaip.2016.09.023
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31. García Sánchez P, Plata Gallardo M, Pedrosa Delgado M, Caballero Molina MT, de Ceano-Vivas la Calle M. Pediatric emergency department management of C1 inhibitor deficiency. Pediatr Emerg Care. 2022;38(2):e844–e848. 10.1097/PEC.0000000000002443
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33. Baker JW, Bernstein JA, Harper JR, Relan A, Riedl MA. Efficacy of recombinant human C1 esterase inhibitor across anatomic locations in acute hereditary angioedema attacks. Allergy Asthma Proc. 2018 Sep 28;39(5):359–364. 10.2500/aap.2018.39.4151
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36. Farkas H, Reshef A, Aberer W, Caballero T, McCarthy L, Hao J, et al. Treatment effect and safety of icatibant in pediatric patients with hereditary angioedema. J Allergy Clin Immunol Pract. 2017 Nov–Dec;5(6):1671–1678.e2. 10.1016/j.jaip.2017.04.010
37. Wentzel N, Panieri A, Ayazi M, Ntshalintshali SD, Pourpak Z, Hawarden D, et al. Fresh frozen plasma for on-demand hereditary angioedema treatment in South Africa and Iran. World Allergy Organ J. 2019 Oct 12;12(9):100049. 10.1016/j.waojou.2019.100049
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39. Zanichelli A, Ghezzi M, Santicchia I, Vacchini R, Cicardi M, Sparaco A, et al. Short-term prophylaxis in patients with angioedema due to C1-inhibitor deficiency undergoing dental procedures: an observational study. PLoS One. 2020 Mar 12;15(3):e0230128. 10.1371/journal.pone.0230128
40. Bork K, Hardt J, Staubach-Renz P, Witzke G. 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 Endod. 2011 Jul;112(1):58–64. 10.1016/j.tripleo.2011.02.034
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2. Farkas H, Martinez-Saguer I, Bork K, Bowen T, Craig T, Frank M, et al. International consensus on the diagnosis and management of pediatric patients with hereditary angioedema with C1 inhibitor deficiency. Allergy. 2017 Feb;72(2):300–313. 10.1111/all.13001
3. Grivcheva-Panovska V, Giannetti B. Hereditary angioedema attack in utero and treatment of the mother and fetus. Mayo Clin Proc Innov Qual Outcomes. 2020 Aug 22;4(5):595–600. 10.1016/j.mayocpiqo.2020.06.004
4. Abdon Barbosa A, de Oliveira Martins R, Martins R, Grumach AS. Assessment on hereditary angioedema burden of illness in Brazil: a patient perspective. Allergy Asthma Proc. 2019 May 1;40(3):193–197. 10.2500/aap.2019.40.4207
5. Banerji A, Li Y, Busse P, Riedl MA, Holtzman NS, Li HH, et al. Hereditary angioedema from the patient’s perspective: a follow-up patient survey. Allergy Asthma Proc. 2018 May 1;39(3):212–223. 10.2500/aap.2018.39.4123
6. Savarese L, Freda MF, De Luca Picione R, Dolce P, De Falco R, Alessio M, et al. The experience of living with a chronic disease in pediatrics from the mothers’ narratives: The clinical interview on parental Sense of Grip on the Disease. Health Psychol Open. 2020 Dec 8;7(2):2055102920971496. 10.1177/2055102920971496
7. Bygum A, Aygören-Pürsün E, Beusterien K, Hautamaki E, Sisic Z, Wait S, et al. Burden of illness in hereditary angioedema: a conceptual model. Acta Derm Venereol. 2015 Jul;95(6):706–710. 10.2340/00015555-2014
8. Freda MF, Savarese L, Dolce P, Picione RL. Caregivers’ sensemaking of children’s hereditary angioedema: a semiotic narrative analysis of the sense of grip on the disease. Front Psychol. 2019 Nov 27;10:2609. 10.3389/fpsyg.2019.02609
9. Ponard D, Gaboriaud C, Charignon D, Ghannam A, WagenaarBos IGA, Roem D, et al. SERPING1 mutation update: mutation spectrum and C1 inhibitor phenotypes. Hum Mutat. 2020 Jan;41(1):38–57. 10.1002/humu.23917
10. Veronez CL, Mendes AR, Leite CS, Gomes CP, Grumach AS, Pesquero JB, et al. The panorama of primary angioedema in the Brazilian population. J Allergy Clin Immunol Pract. 2021 Jun;9(6):2293–2304.e5.
11. Cicardi M, Aberer W, Banerji A, Bas M, Bernstein JA, Bork K, et al. Classification, diagnosis, and approach to treatment for angioedema: consensus report from the Hereditary Angioedema International Working Group. Allergy. 2014 May;69(5):602–616. 10.1111/all.12380
12. Maurer M, Magerl M, Ansotegui I, Aygören-Pürsün E, Betschel S, Bork K, et al. The international WAO/EAACI guideline for the management of hereditary angioedema–the 2017 revision and update. Allergy. 2018 Aug;73(8):1575–1596. 10.1111/all.13384
13. Germenis AE, Margaglione M, Pesquero JB, Farkas H, Cichon S, Csuka D, et al. International consensus on the use of genetics in the management of hereditary angioedema. J Allergy Clin Immunol Pract. 2020 Mar;8(3):901–911. 10.1016/j.jaip.2019.10.004
14. Bork K, Wulff K, Witzke G, Hardt J. Hereditary angioedema with normal C1-INH with versus without specific F12 gene mutations. Allergy. 2015 Aug;70(8):1004–1012. 10.1111/all.12648
15. Deroux A, Boccon-Gibod I, Fain O, Pralong P, Ollivier Y, Pagnier A, et al. Hereditary angioedema with normal C1 inhibitor and factor XII mutation: a series of 57 patients from the French National Center of Reference for Angioedema. Clin Exp Immunol. 2016 Sep;185(3):332–337. 10.1111/cei.12820
16. Veronez CL, Moreno AS, Constantino-Silva RN, Maia LSM, Ferriani MPL, Castro FFM, et al. Hereditary angioedema with normal C1 inhibitor and F12 mutations in 42 Brazilian families. J Allergy Clin Immunol Pract. 2018 Jul–Aug;6(4):1209–1216.e8. 10.1016/j.jaip.2017.09.025
17. Bork K, Machnig T, Wulff K, Witzke G, Prusty S, Hardt J. Clinical features of genetically characterized types of hereditary angioedema with normal C1 inhibitor: a systematic review of qualitative evidence. Orphanet J Rare Dis. 2020 Oct 15;15(1):289. 10.1186/s13023-020-01570-x
18. Bafunno V, Firinu D, D’Apolito M, Cordisco G, Loffredo S, Leccese A, et al. Mutation of the angiopoietin-1 gene (ANGPT1) associates with a new type of hereditary angioedema. J Allergy Clin Immunol. 2018;141(3):1009–1017. 10.1016/j.jaci.2017.05.020
19. Bork K, Wulff K, Steinmüller-Magin L, Braenne I, StaubachRenz P, Witzke G, et al. Hereditary angioedema with a mutation in the plasminogen gene. Allergy. 2018 Feb;73(2):442–450. 10.1111/all.13270
20. Bork K, Wulff K, Rossmann H, Steinmuller-Magin L, Braenne I, Witzke G, et al. Hereditary angioedema cosegregating with a novel kininogen 1 gene mutation changing the N-terminal cleavage site of bradykinin. Allergy. 2019;74(12):2479–2481. 10.1111/all.13869
21. Ariano A, D’Apolito M, Bova M, Bellanti F, Loffredo S, D’Andrea G, et al. A myoferlin gain-of-function variant associates with a new type of hereditary angioedema. Allergy. 2020 Nov;75(11):2989–2992. 10.1111/all.14454
22. Bork K, Wulff K, Möhl BS, Steinmüller-Magin L, Witzke G, Hardt J, et al. Novel hereditary angioedema linked with a heparan sulfate 3-O-sulfotransferase 6 gene mutation. J Allergy Clin Immunol. 2021 Oct;148(4):1041–1048. 10.1016/j.jaci.2021.01.011
23. Frank MM, Zuraw B, Banerji A, Bernstein JA, Craig T, Busse P, et al. Management of children with hereditary angioedema due to C1 inhibitor deficiency. Pediatrics. 2016 Nov;138(5):e20160575. 10.1542/peds.2016-0575
24. Araújo-Simões J, Boanova AGP, Constantino-Silva RN, Fragnan NTML, Pinto JA, Minafra FG, et al. The challenges in the follow-up and treatment of Brazilian children with hereditary angioedema. Int Arch Allergy Immunol, 2021;182(7):585–591. 10.1159/000512944
25. Piotrowicz-Wójcik K, Porebski G. Life-threatening laryngeal attacks in hereditary angioedema patients. Otolaryngol Pol. 2020 Mar 31;74(2):1–5. 10.5604/01.3001.0014.0619
26. Bernstein JA, Cremonesi P, Hoffmann TK, Hollingsworth J. Angioedema in the emergency department: a practical guide to differential diagnosis and management. Int J Emerg Med. 2017 Dec;10(1):15. 10.1186/s12245-017-0141-z
27. Otani IM, Lumry WR, Hurwitz S, Li HH, Craig TJ, Holtzman NS, et al. Subcutaneous icatibant for the treatment of hereditary angioedema attacks: Comparison of home self-administration with administration at a medical facility. J Allergy Clin Immunol Pract. 2017 Mar–Apr;5(2):442–447.e1. 10.1016/j.jaip.2016.09.023
28. Busse PJ, Christiansen SC, Riedl MA, Banerji A, Bernstein JA, Castaldo AJ, et al. US HAEA medical advisory board 2020 guidelines for the management of hereditary angioedema. J Allergy Clin Immunol Pract. 2021 Jan;9(1):132–150.e3. 10.1016/j.jaip.2020.08.046
29. Bork K, Bernstein JA, Machnig T, Craig TJ. Efficacy of different medical therapies for the treatment of acute laryngeal attacks of hereditary angioedema due to C1-esterase inhibitor deficiency. J Emerg Med. 2016 Apr;50(4):567–580.e1. 10.1016/j.jemermed.2015.11.008
30. Craig T. Triggers and short-term prophylaxis in patients with hereditary angioedema. Allergy Asthma Proc. 2020 Nov 1;41(Suppl 1):S30–S34. 10.2500/aap.2020.41.200058
31. García Sánchez P, Plata Gallardo M, Pedrosa Delgado M, Caballero Molina MT, de Ceano-Vivas la Calle M. Pediatric emergency department management of C1 inhibitor deficiency. Pediatr Emerg Care. 2022;38(2):e844–e848. 10.1097/PEC.0000000000002443
32. Serpa FS, Mansour E, Aun MV, Giavina-Bianchi P, Chong Neto HJ, Arruda LK, et al. Hereditary angioedema: how to approach it at the emergency department? Einstein (Sao Paulo). 2021 Apr 9;19:eRW5498. 10.31744/einstein_journal/2021RW5498
33. Baker JW, Bernstein JA, Harper JR, Relan A, Riedl MA. Efficacy of recombinant human C1 esterase inhibitor across anatomic locations in acute hereditary angioedema attacks. Allergy Asthma Proc. 2018 Sep 28;39(5):359–364. 10.2500/aap.2018.39.4151
34. Longhurst HJ. Management of acute attacks of hereditary angioedema: potential role of icatibant. Vasc Health Risk Manag. 2010 Sep 7;6:795–802. 10.2147/VHRM.S4332
35. Maurer M, Bork K, Martinez-Saguer I, Aygören-Pürsün E, Botha J, Andresen I, et al. Management of patients with hereditary angioedema in Germany: comparison with other countries in the Icatibant Outcome Survey. J Eur Acad Dermatol Venereol. 2019 Jan;33(1):163–169. 10.1111/jdv.15232
36. Farkas H, Reshef A, Aberer W, Caballero T, McCarthy L, Hao J, et al. Treatment effect and safety of icatibant in pediatric patients with hereditary angioedema. J Allergy Clin Immunol Pract. 2017 Nov–Dec;5(6):1671–1678.e2. 10.1016/j.jaip.2017.04.010
37. Wentzel N, Panieri A, Ayazi M, Ntshalintshali SD, Pourpak Z, Hawarden D, et al. Fresh frozen plasma for on-demand hereditary angioedema treatment in South Africa and Iran. World Allergy Organ J. 2019 Oct 12;12(9):100049. 10.1016/j.waojou.2019.100049
38. Grumach AS, Staubach-Renz P, Villa RC, Diez-Zuluaga S, Reese I, Lumry WR. Triggers of exacerbation in chronic urticaria and recurrent angioedema–prevalence and relevance. J Allergy Clin Immunol Pract. 2021 Jun;9(6):2160–2168. 10.1016/j.jaip.2021.04.023
39. Zanichelli A, Ghezzi M, Santicchia I, Vacchini R, Cicardi M, Sparaco A, et al. Short-term prophylaxis in patients with angioedema due to C1-inhibitor deficiency undergoing dental procedures: an observational study. PLoS One. 2020 Mar 12;15(3):e0230128. 10.1371/journal.pone.0230128
40. Bork K, Hardt J, Staubach-Renz P, Witzke G. 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 Endod. 2011 Jul;112(1):58–64. 10.1016/j.tripleo.2011.02.034
41. Ajewole O, Lanlokun M, Dimanche S, Craig T. Short-term prophylaxis for children and adolescents with hereditary angioedema. Allergy Asthma Proc. 2021 May 1;42(3):205–213. 10.2500/aap.2021.42.210006
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Apr 7, 2022
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Mansour, E., Veronez, C. L., Craig, T., & Grumach, A. S. (2022). Hereditary angioedema in children and adolescents. Allergologia Et Immunopathologia, 50(SP1), 1-6. https://doi.org/10.15586/aei.v50iSP1.535
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Special Issue : Approach to immune-allergic diseases in childhood and adolescence
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How to Cite
Mansour, E., Veronez, C. L., Craig, T., & Grumach, A. S. (2022). Hereditary angioedema in children and adolescents. Allergologia Et Immunopathologia, 50(SP1), 1-6. https://doi.org/10.15586/aei.v50iSP1.535