International consensus on the diagnosis and management of pediatric patients with hereditary angioedema with C1 inhibitor deficiency

HAWK

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46 Citations (Scopus)

Abstract

Background: The consensus documents published to date on hereditary angioedema with C1 inhibitor deficiency (C1-INH-HAE) have focused on adult patients. Many of the previous recommendations have not been adapted to pediatric patients. We intended to produce consensus recommendations for the diagnosis and management of pediatric patients with C1-INH-HAE. Methods: During an expert panel meeting that took place during the 9th C1 Inhibitor Deficiency Workshop in Budapest, 2015 (www.haenet.hu), pediatric data were presented and discussed and a consensus was developed by voting. Results: The symptoms of C1-INH-HAE often present in childhood. Differential diagnosis can be difficult as abdominal pain is common in pediatric C1-INH-HAE, but also commonly occurs in the general pediatric population. The early onset of symptoms may predict a more severe subsequent course of the disease. Before the age of 1 year, C1-INH levels may be lower than in adults; therefore, it is advisable to confirm the diagnosis after the age of one year. All neonates/infants with an affected C1-INH-HAE family member should be screened for C1-INH deficiency. Pediatric patients should always carry a C1-INH-HAE information card and medicine for emergency use. The regulatory approval status of the drugs for prophylaxis and for acute treatment is different in each country. Plasma-derived C1-INH, recombinant C1-INH, and ecallantide are the only agents licensed for the acute treatment of pediatric patients. Clinical trials are underway with additional drugs. It is recommended to follow up patients in an HAE comprehensive care center. Conclusions: The pediatric-focused international consensus for the diagnosis and management of C1-INH-HAE patients was created.

Original languageEnglish (US)
Pages (from-to)300-313
Number of pages14
JournalAllergy: European Journal of Allergy and Clinical Immunology
Volume72
Issue number2
DOIs
StatePublished - Feb 1 2017

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Hereditary Angioedemas
Consensus
Pediatrics
Drug Approval
Emergency Medicine
Politics
Abdominal Pain
Differential Diagnosis
Clinical Trials
Newborn Infant
Education

All Science Journal Classification (ASJC) codes

  • Immunology and Allergy
  • Immunology

Cite this

@article{b9112006b5c5402397608337ef98839e,
title = "International consensus on the diagnosis and management of pediatric patients with hereditary angioedema with C1 inhibitor deficiency",
abstract = "Background: The consensus documents published to date on hereditary angioedema with C1 inhibitor deficiency (C1-INH-HAE) have focused on adult patients. Many of the previous recommendations have not been adapted to pediatric patients. We intended to produce consensus recommendations for the diagnosis and management of pediatric patients with C1-INH-HAE. Methods: During an expert panel meeting that took place during the 9th C1 Inhibitor Deficiency Workshop in Budapest, 2015 (www.haenet.hu), pediatric data were presented and discussed and a consensus was developed by voting. Results: The symptoms of C1-INH-HAE often present in childhood. Differential diagnosis can be difficult as abdominal pain is common in pediatric C1-INH-HAE, but also commonly occurs in the general pediatric population. The early onset of symptoms may predict a more severe subsequent course of the disease. Before the age of 1 year, C1-INH levels may be lower than in adults; therefore, it is advisable to confirm the diagnosis after the age of one year. All neonates/infants with an affected C1-INH-HAE family member should be screened for C1-INH deficiency. Pediatric patients should always carry a C1-INH-HAE information card and medicine for emergency use. The regulatory approval status of the drugs for prophylaxis and for acute treatment is different in each country. Plasma-derived C1-INH, recombinant C1-INH, and ecallantide are the only agents licensed for the acute treatment of pediatric patients. Clinical trials are underway with additional drugs. It is recommended to follow up patients in an HAE comprehensive care center. Conclusions: The pediatric-focused international consensus for the diagnosis and management of C1-INH-HAE patients was created.",
author = "HAWK and H. Farkas and I. Martinez-Saguer and K. Bork and T. Bowen and Timothy Craig and M. Frank and Germenis, {A. E.} and Grumach, {A. S.} and A. Luczay and L. Varga and A. Zanichelli and Werner Aberer and Sladjana Andrejevic and Emel Aygoeren-P{\"u}rs{\"u}n and Alena Banerji and Bara, {Noemi Anna} and Murat Bas and Jonathan Bernstein and Stephen Betschel and Janne Bj{\"o}rkander and Isabelle Boccon-Gibod and Laurence Bouillet and Maria Bova and Boysen, {Henrik Halle} and Manuel Branco-Ferreira and Anette Bygum and Teresa Caballero and Mauro Cancian and Anthony Castaldo and Sandra Christiansen and Marco Cicardi and Christian Drouet and Jose Fabiani and Mark Gompels and Gonzalez-Quevedo, {Maria Teresa} and Jimmy Gooi and Richard Gower and G{\"o}kmen, {Nihal Mete} and Vesna Grivcheva-Panovska and Mar Guilarte and Okan G{\"u}lbahar and Erik Hack and Roman Hakl and Gy{\"o}rgy Harmat and Miloš Jeseň{\'a}k and Stephen Jolles and Allen Kaplan and Connie Katelaris and Mitja Kosnik and Kőhalmi, {Kinga Vikt{\'o}ria}",
year = "2017",
month = "2",
day = "1",
doi = "10.1111/all.13001",
language = "English (US)",
volume = "72",
pages = "300--313",
journal = "Allergy: European Journal of Allergy and Clinical Immunology",
issn = "0105-4538",
publisher = "Wiley-Blackwell",
number = "2",

}

TY - JOUR

T1 - International consensus on the diagnosis and management of pediatric patients with hereditary angioedema with C1 inhibitor deficiency

AU - HAWK

AU - Farkas, H.

AU - Martinez-Saguer, I.

AU - Bork, K.

AU - Bowen, T.

AU - Craig, Timothy

AU - Frank, M.

AU - Germenis, A. E.

AU - Grumach, A. S.

AU - Luczay, A.

AU - Varga, L.

AU - Zanichelli, A.

AU - Aberer, Werner

AU - Andrejevic, Sladjana

AU - Aygoeren-Pürsün, Emel

AU - Banerji, Alena

AU - Bara, Noemi Anna

AU - Bas, Murat

AU - Bernstein, Jonathan

AU - Betschel, Stephen

AU - Björkander, Janne

AU - Boccon-Gibod, Isabelle

AU - Bouillet, Laurence

AU - Bova, Maria

AU - Boysen, Henrik Halle

AU - Branco-Ferreira, Manuel

AU - Bygum, Anette

AU - Caballero, Teresa

AU - Cancian, Mauro

AU - Castaldo, Anthony

AU - Christiansen, Sandra

AU - Cicardi, Marco

AU - Drouet, Christian

AU - Fabiani, Jose

AU - Gompels, Mark

AU - Gonzalez-Quevedo, Maria Teresa

AU - Gooi, Jimmy

AU - Gower, Richard

AU - Gökmen, Nihal Mete

AU - Grivcheva-Panovska, Vesna

AU - Guilarte, Mar

AU - Gülbahar, Okan

AU - Hack, Erik

AU - Hakl, Roman

AU - Harmat, György

AU - Jeseňák, Miloš

AU - Jolles, Stephen

AU - Kaplan, Allen

AU - Katelaris, Connie

AU - Kosnik, Mitja

AU - Kőhalmi, Kinga Viktória

PY - 2017/2/1

Y1 - 2017/2/1

N2 - Background: The consensus documents published to date on hereditary angioedema with C1 inhibitor deficiency (C1-INH-HAE) have focused on adult patients. Many of the previous recommendations have not been adapted to pediatric patients. We intended to produce consensus recommendations for the diagnosis and management of pediatric patients with C1-INH-HAE. Methods: During an expert panel meeting that took place during the 9th C1 Inhibitor Deficiency Workshop in Budapest, 2015 (www.haenet.hu), pediatric data were presented and discussed and a consensus was developed by voting. Results: The symptoms of C1-INH-HAE often present in childhood. Differential diagnosis can be difficult as abdominal pain is common in pediatric C1-INH-HAE, but also commonly occurs in the general pediatric population. The early onset of symptoms may predict a more severe subsequent course of the disease. Before the age of 1 year, C1-INH levels may be lower than in adults; therefore, it is advisable to confirm the diagnosis after the age of one year. All neonates/infants with an affected C1-INH-HAE family member should be screened for C1-INH deficiency. Pediatric patients should always carry a C1-INH-HAE information card and medicine for emergency use. The regulatory approval status of the drugs for prophylaxis and for acute treatment is different in each country. Plasma-derived C1-INH, recombinant C1-INH, and ecallantide are the only agents licensed for the acute treatment of pediatric patients. Clinical trials are underway with additional drugs. It is recommended to follow up patients in an HAE comprehensive care center. Conclusions: The pediatric-focused international consensus for the diagnosis and management of C1-INH-HAE patients was created.

AB - Background: The consensus documents published to date on hereditary angioedema with C1 inhibitor deficiency (C1-INH-HAE) have focused on adult patients. Many of the previous recommendations have not been adapted to pediatric patients. We intended to produce consensus recommendations for the diagnosis and management of pediatric patients with C1-INH-HAE. Methods: During an expert panel meeting that took place during the 9th C1 Inhibitor Deficiency Workshop in Budapest, 2015 (www.haenet.hu), pediatric data were presented and discussed and a consensus was developed by voting. Results: The symptoms of C1-INH-HAE often present in childhood. Differential diagnosis can be difficult as abdominal pain is common in pediatric C1-INH-HAE, but also commonly occurs in the general pediatric population. The early onset of symptoms may predict a more severe subsequent course of the disease. Before the age of 1 year, C1-INH levels may be lower than in adults; therefore, it is advisable to confirm the diagnosis after the age of one year. All neonates/infants with an affected C1-INH-HAE family member should be screened for C1-INH deficiency. Pediatric patients should always carry a C1-INH-HAE information card and medicine for emergency use. The regulatory approval status of the drugs for prophylaxis and for acute treatment is different in each country. Plasma-derived C1-INH, recombinant C1-INH, and ecallantide are the only agents licensed for the acute treatment of pediatric patients. Clinical trials are underway with additional drugs. It is recommended to follow up patients in an HAE comprehensive care center. Conclusions: The pediatric-focused international consensus for the diagnosis and management of C1-INH-HAE patients was created.

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U2 - 10.1111/all.13001

DO - 10.1111/all.13001

M3 - Article

C2 - 27503784

AN - SCOPUS:84987949061

VL - 72

SP - 300

EP - 313

JO - Allergy: European Journal of Allergy and Clinical Immunology

JF - Allergy: European Journal of Allergy and Clinical Immunology

SN - 0105-4538

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ER -