Short-term prophylaxis in hereditary angioedema due to deficiency of the C1-inhibitor - A long-term survey

H. Farkas, Z. Zotter, D. Csuka, E. Szabó, Z. Nébenfåhrer, G. Temesszentandrási, L. Jakab, L. Varga, G. Harmat, I. Karádi

Research output: Contribution to journalArticle

38 Citations (Scopus)

Abstract

Background Hereditary angioedema is a potentially life-threatening disorder, because edema occurring in the mucosa of the upper airways can lead to suffocation. The management of HAE consists of avoiding the triggering factors, prophylaxis, and the acute treatment of edematous episodes. Medical procedures can also provoke edematous attacks, and therefore, short-term prophylaxis (STP) is recommended before such interventions. Our aim was to evaluate the efficacy and safety of STP administered before medical procedures. Methods We conducted a retrospective analysis before and a prospective survey after establishing the diagnosis in a group of 137 (60 males, 77 females; 20 pediatric and 117 adult) patients with HAE. Both were implemented using questionnaires, patient diaries and hospital charts focusing on medical interventions provoking edematous attack, and the medicinal products (C1-INH concentrate, tranexamic acid, and danazol) administered for STP. Results Comparing surgical interventions performed without pre-event STP (in 39/89 patients before HAE was diagnosed), or after STP (in 3/55 cases after diagnosis), we found a significant (P <0.0001, Fisher's exact test) reduction in the number of edematous episodes. Evaluating the efficacy of the drugs administered for STP revealed that C1-INH concentrate (Berinert®, CSL Behring, Marburg, Germany) was significantly (P = 0.0096, Fisher's exact test) superior to orally administered drugs in reducing the instances of postprocedural edema. None of the medicinal products caused adverse events potentially related to STP. Conclusions STP reduces the number of postprocedural edematous episodes. C1-INH concentrate is safe and effective for prophylaxis. When this agent is not available, danazol is a potential alternative for prophylaxis before elective medical interventions.

Original languageEnglish
Pages (from-to)1586-1593
Number of pages8
JournalAllergy: European Journal of Allergy and Clinical Immunology
Volume67
Issue number12
DOIs
Publication statusPublished - Dec 2012

Fingerprint

Hereditary Angioedemas
Danazol
Edema
Tranexamic Acid
Asphyxia
Pharmaceutical Preparations
Germany
Mucous Membrane
Pediatrics
Safety
Surveys and Questionnaires
Therapeutics

Keywords

  • C1-inhibitor deficiency
  • hereditary angioedema
  • short-term prophylaxis

ASJC Scopus subject areas

  • Immunology
  • Immunology and Allergy

Cite this

Short-term prophylaxis in hereditary angioedema due to deficiency of the C1-inhibitor - A long-term survey. / Farkas, H.; Zotter, Z.; Csuka, D.; Szabó, E.; Nébenfåhrer, Z.; Temesszentandrási, G.; Jakab, L.; Varga, L.; Harmat, G.; Karádi, I.

In: Allergy: European Journal of Allergy and Clinical Immunology, Vol. 67, No. 12, 12.2012, p. 1586-1593.

Research output: Contribution to journalArticle

Farkas, H. ; Zotter, Z. ; Csuka, D. ; Szabó, E. ; Nébenfåhrer, Z. ; Temesszentandrási, G. ; Jakab, L. ; Varga, L. ; Harmat, G. ; Karádi, I. / Short-term prophylaxis in hereditary angioedema due to deficiency of the C1-inhibitor - A long-term survey. In: Allergy: European Journal of Allergy and Clinical Immunology. 2012 ; Vol. 67, No. 12. pp. 1586-1593.
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AU - Csuka, D.

AU - Szabó, E.

AU - Nébenfåhrer, Z.

AU - Temesszentandrási, G.

AU - Jakab, L.

AU - Varga, L.

AU - Harmat, G.

AU - Karádi, I.

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AB - Background Hereditary angioedema is a potentially life-threatening disorder, because edema occurring in the mucosa of the upper airways can lead to suffocation. The management of HAE consists of avoiding the triggering factors, prophylaxis, and the acute treatment of edematous episodes. Medical procedures can also provoke edematous attacks, and therefore, short-term prophylaxis (STP) is recommended before such interventions. Our aim was to evaluate the efficacy and safety of STP administered before medical procedures. Methods We conducted a retrospective analysis before and a prospective survey after establishing the diagnosis in a group of 137 (60 males, 77 females; 20 pediatric and 117 adult) patients with HAE. Both were implemented using questionnaires, patient diaries and hospital charts focusing on medical interventions provoking edematous attack, and the medicinal products (C1-INH concentrate, tranexamic acid, and danazol) administered for STP. Results Comparing surgical interventions performed without pre-event STP (in 39/89 patients before HAE was diagnosed), or after STP (in 3/55 cases after diagnosis), we found a significant (P <0.0001, Fisher's exact test) reduction in the number of edematous episodes. Evaluating the efficacy of the drugs administered for STP revealed that C1-INH concentrate (Berinert®, CSL Behring, Marburg, Germany) was significantly (P = 0.0096, Fisher's exact test) superior to orally administered drugs in reducing the instances of postprocedural edema. None of the medicinal products caused adverse events potentially related to STP. Conclusions STP reduces the number of postprocedural edematous episodes. C1-INH concentrate is safe and effective for prophylaxis. When this agent is not available, danazol is a potential alternative for prophylaxis before elective medical interventions.

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