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

H. Farkas; I. Martinez-Saguer; K. Bork; T. Bowen; T. Craig; M. Frank; A. E. Germenis; A. S. Grumach; A. Luczay; L. Varga; A. Zanichelli; W. Aberer; S. Andrejevic; E. Aygoeren-Pürsün; A. Banerji; N. A. Bara; M. Bas; J. Bernstein; S. Betschel; J. Björkander; I. Boccon-Gibod; L. Bouillet; M. Bova; H. H. Boysen; M. Branco-Ferreira; A. Bygum; T. Caballero; M. Cancian; A. Castaldo; S. Christiansen; M. Cicardi; C. Drouet; J. Fabiani; M. Gompels; M. T.. Gonzalez-Quevedo; J. Gooi; R. Gower; N. M. Gökmen; V. Grivcheva-Panovska; M. Guilarte; O. Gülbahar; E. Hack; R. Hakl; G. Harmat; M. Jesenak; S. Jolles; A. Kaplan; Connie Katelaris; M. Kosnik; K. V. Kőhalmi; I. Leibovich; M. Levi; H. Li; H. J. Longhurst; W. Lumry; M. Magerl; A. Malbran; L. Martin; M. Maurer; E. Mihály; D. Moldovan; M. Murdjeva; I. B. Nagy; E. W. Nielsen; S. Nieto; P. Nordenfelt; K. Obtulowitzc; M. Pedrosa; G. Porębski; N. Prior; A. Reshef; M. A. Riedl; B. Rosenkranz; P. Schmid-Grendelmeier; S. Péter; M. Speletas; M. Staevska; M. Stobiecki; M. Triggiani; N. Veszeli; W. Wuillemin; Z. Y. Xiang; B. Yamamoto; B. Zuraw

doi:10.1111/all.13001

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

H. Farkas, I. Martinez-Saguer, K. Bork, T. Bowen, T. Craig, M. Frank, A. E. Germenis, A. S. Grumach, A. Luczay, L. Varga, A. Zanichelli, W. Aberer, S. Andrejevic, E. Aygoeren-Pürsün, A. Banerji, N. A. Bara, M. Bas, J. Bernstein, S. Betschel, J. BjörkanderI. Boccon-Gibod, L. Bouillet, M. Bova, H. H. Boysen, M. Branco-Ferreira, A. Bygum, T. Caballero, M. Cancian, A. Castaldo, S. Christiansen, M. Cicardi, C. Drouet, J. Fabiani, M. Gompels, M. T.. Gonzalez-Quevedo, J. Gooi, R. Gower, N. M. Gökmen, V. Grivcheva-Panovska, M. Guilarte, O. Gülbahar, E. Hack, R. Hakl, G. Harmat, M. Jesenak, S. Jolles, A. Kaplan, Connie Katelaris, M. Kosnik, K. V. Kőhalmi, I. Leibovich, M. Levi, H. Li, H. J. Longhurst, W. Lumry, M. Magerl, A. Malbran, L. Martin, M. Maurer, E. Mihály, D. Moldovan, M. Murdjeva, I. B. Nagy, E. W. Nielsen, S. Nieto, P. Nordenfelt, K. Obtulowitzc, M. Pedrosa, G. Porębski, N. Prior, A. Reshef, M. A. Riedl, B. Rosenkranz, P. Schmid-Grendelmeier, S. Péter, M. Speletas, M. Staevska, M. Stobiecki, M. Triggiani, N. Veszeli, W. Wuillemin, Z. Y. Xiang, B. Yamamoto, B. Zuraw

Western Sydney University

Research output: Contribution to journal › Article › peer-review

171 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 language	English
Pages (from-to)	300-313
Number of pages	14
Journal	Allergy
Volume	72
Issue number	2
DOIs	https://doi.org/10.1111/all.13001
Publication status	Published - 2017

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Farkas, H., Martinez-Saguer, I., Bork, K., Bowen, T., Craig, T., Frank, M., Germenis, A. E., Grumach, A. S., Luczay, A., Varga, L., Zanichelli, A., Aberer, W., Andrejevic, S., Aygoeren-Pürsün, E., Banerji, A., Bara, N. A., Bas, M., Bernstein, J., Betschel, S., ... Zuraw, B. (2017). International consensus on the diagnosis and management of pediatric patients with hereditary angioedema with C1 inhibitor deficiency. Allergy, 72(2), 300-313. https://doi.org/10.1111/all.13001

@article{7c37a01b88f845f0b461a57aa523a1dd,

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 = "H. Farkas and I. Martinez-Saguer and K. Bork and T. Bowen and T. Craig and M. Frank and Germenis, {A. E.} and Grumach, {A. S.} and A. Luczay and L. Varga and A. Zanichelli and W. Aberer and S. Andrejevic and E. Aygoeren-P{\"u}rs{\"u}n and A. Banerji and Bara, {N. A.} and M. Bas and J. Bernstein and S. Betschel and J. Bj{\"o}rkander and I. Boccon-Gibod and L. Bouillet and M. Bova and Boysen, {H. H.} and M. Branco-Ferreira and A. Bygum and T. Caballero and M. Cancian and A. Castaldo and S. Christiansen and M. Cicardi and C. Drouet and J. Fabiani and M. Gompels and Gonzalez-Quevedo, {M. T..} and J. Gooi and R. Gower and G{\"o}kmen, {N. M.} and V. Grivcheva-Panovska and M. Guilarte and O. G{\"u}lbahar and E. Hack and R. Hakl and G. Harmat and M. Jesenak and S. Jolles and A. Kaplan and Connie Katelaris and M. Kosnik and K{\H o}halmi, {K. V.} and I. Leibovich and M. Levi and H. Li and Longhurst, {H. J.} and W. Lumry and M. Magerl and A. Malbran and L. Martin and M. Maurer and E. Mih{\'a}ly and D. Moldovan and M. Murdjeva and Nagy, {I. B.} and Nielsen, {E. W.} and S. Nieto and P. Nordenfelt and K. Obtulowitzc and M. Pedrosa and G. Por{\c e}bski and N. Prior and A. Reshef and Riedl, {M. A.} and B. Rosenkranz and P. Schmid-Grendelmeier and S. P{\'e}ter and M. Speletas and M. Staevska and M. Stobiecki and M. Triggiani and N. Veszeli and W. Wuillemin and Xiang, {Z. Y.} and B. Yamamoto and B. Zuraw",

year = "2017",

doi = "10.1111/all.13001",

language = "English",

volume = "72",

pages = "300--313",

journal = "Allergy",

publisher = "Wiley-Blackwell Publishing",

number = "2",

}

Farkas, H, Martinez-Saguer, I, Bork, K, Bowen, T, Craig, T, Frank, M, Germenis, AE, Grumach, AS, Luczay, A, Varga, L, Zanichelli, A, Aberer, W, Andrejevic, S, Aygoeren-Pürsün, E, Banerji, A, Bara, NA, Bas, M, Bernstein, J, Betschel, S, Björkander, J, Boccon-Gibod, I, Bouillet, L, Bova, M, Boysen, HH, Branco-Ferreira, M, Bygum, A, Caballero, T, Cancian, M, Castaldo, A, Christiansen, S, Cicardi, M, Drouet, C, Fabiani, J, Gompels, M, Gonzalez-Quevedo, MT, Gooi, J, Gower, R, Gökmen, NM, Grivcheva-Panovska, V, Guilarte, M, Gülbahar, O, Hack, E, Hakl, R, Harmat, G, Jesenak, M, Jolles, S, Kaplan, A, Katelaris, C, Kosnik, M, Kőhalmi, KV, Leibovich, I, Levi, M, Li, H, Longhurst, HJ, Lumry, W, Magerl, M, Malbran, A, Martin, L, Maurer, M, Mihály, E, Moldovan, D, Murdjeva, M, Nagy, IB, Nielsen, EW, Nieto, S, Nordenfelt, P, Obtulowitzc, K, Pedrosa, M, Porębski, G, Prior, N, Reshef, A, Riedl, MA, Rosenkranz, B, Schmid-Grendelmeier, P, Péter, S, Speletas, M, Staevska, M, Stobiecki, M, Triggiani, M, Veszeli, N, Wuillemin, W, Xiang, ZY, Yamamoto, B & Zuraw, B 2017, 'International consensus on the diagnosis and management of pediatric patients with hereditary angioedema with C1 inhibitor deficiency', Allergy, vol. 72, no. 2, pp. 300-313. https://doi.org/10.1111/all.13001

TY - JOUR

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

AU - Farkas, H.

AU - Martinez-Saguer, I.

AU - Bork, K.

AU - Bowen, T.

AU - Craig, T.

AU - Frank, M.

AU - Germenis, A. E.

AU - Grumach, A. S.

AU - Luczay, A.

AU - Varga, L.

AU - Zanichelli, A.

AU - Aberer, W.

AU - Andrejevic, S.

AU - Aygoeren-Pürsün, E.

AU - Banerji, A.

AU - Bara, N. A.

AU - Bas, M.

AU - Bernstein, J.

AU - Betschel, S.

AU - Björkander, J.

AU - Boccon-Gibod, I.

AU - Bouillet, L.

AU - Bova, M.

AU - Boysen, H. H.

AU - Branco-Ferreira, M.

AU - Bygum, A.

AU - Caballero, T.

AU - Cancian, M.

AU - Castaldo, A.

AU - Christiansen, S.

AU - Cicardi, M.

AU - Drouet, C.

AU - Fabiani, J.

AU - Gompels, M.

AU - Gonzalez-Quevedo, M. T..

AU - Gooi, J.

AU - Gower, R.

AU - Gökmen, N. M.

AU - Grivcheva-Panovska, V.

AU - Guilarte, M.

AU - Gülbahar, O.

AU - Hack, E.

AU - Hakl, R.

AU - Harmat, G.

AU - Jesenak, M.

AU - Jolles, S.

AU - Kaplan, A.

AU - Katelaris, Connie

AU - Kosnik, M.

AU - Kőhalmi, K. V.

AU - Leibovich, I.

AU - Levi, M.

AU - Li, H.

AU - Longhurst, H. J.

AU - Lumry, W.

AU - Magerl, M.

AU - Malbran, A.

AU - Martin, L.

AU - Maurer, M.

AU - Mihály, E.

AU - Moldovan, D.

AU - Murdjeva, M.

AU - Nagy, I. B.

AU - Nielsen, E. W.

AU - Nieto, S.

AU - Nordenfelt, P.

AU - Obtulowitzc, K.

AU - Pedrosa, M.

AU - Porębski, G.

AU - Prior, N.

AU - Reshef, A.

AU - Riedl, M. A.

AU - Rosenkranz, B.

AU - Schmid-Grendelmeier, P.

AU - Péter, S.

AU - Speletas, M.

AU - Staevska, M.

AU - Stobiecki, M.

AU - Triggiani, M.

AU - Veszeli, N.

AU - Wuillemin, W.

AU - Xiang, Z. Y.

AU - Yamamoto, B.

AU - Zuraw, B.

PY - 2017

Y1 - 2017

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.

UR - https://hdl.handle.net/1959.7/uws:63715

U2 - 10.1111/all.13001

DO - 10.1111/all.13001

M3 - Article

VL - 72

SP - 300

EP - 313

JO - Allergy

JF - Allergy

IS - 2

ER -

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

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