On demand treatment and home therapy of hereditary angioedema in Germany - the Frankfurt experience

Aygören-Pürsün Emel, Martinez-Saguer Inmaculada, Rusicke Eva, Klingebiel Thomas, Kreuz, Kreuz Wolfhart

Description

Manifestation of acute edema in hereditary angioedema (HAE) is characterized by interindividual and intraindividual variability in symptom expression over time. Flexible therapy options are needed. Methods We describe and report on the outcomes of the highly individualized approach to HAE therapy practiced at our HAE center in Frankfurt (Germany). Results The HAE center at the Frankfurt University Hospital currently treats 450 adults with HAE or AAE and 107 pediatric HAE patients with highly individualized therapeutic approaches. 73.9% of the adult patients treat HAE attacks by on-demand therapy with pasteurized pd C1-INH concentrate, 9.8% use additional prophylaxis with attenuated androgens, 1% of the total patient population in Frankfurt has been treated with Icatibant up to now. In addition adult and selected pediatric patients with a high frequency of severe attacks are instructed to apply individual replacement therapy (IRT) with pasteurized pd C1-INH concentrate. Improvement on Quality of Life items was shown for these patients compared to previous long-term danazol prophylaxis. Home treatment of HAE patients was developed in the Frankfurt HAE center in line with experiences in hemophilia therapy and has so far been implemented over a period of 28 years. At present 248 (55%) of the adult patients and 26 (24%) of the pediatric patients are practicing home treatment either as on demand or IRT treatment. Conclusions In conclusion, the individualized home therapies provided by our HAE center, aim to limit the disruption to normal daily activities that occurs for many HAE patients. Furthermore, we seek to optimize the economic burden of the disease while offering a maximum quality of life to our patients.

Informations

• Publié par: biomed
• Publié le: 01 janvier 2010
• Nombre de lectures: 18
• Langue: English

Extrait

AygörenPürsünet al.Allergy, Asthma & Clinical Immunology2010,6:21 http://www.aacijournal.com/content/6/1/21

ALLERGY, ASTHMA & CLINICAL IMMUNOLOGY

R E S E A R C HOpen Access On demand treatment and home therapy of hereditary angioedema in Germany  the Frankfurt experience * Emel AygörenPürsün , Inmaculada MartinezSaguer, Eva Rusicke, Thomas Klingebiel, Wolfhart Kreuz

Abstract Background:Manifestation of acute edema in hereditary angioedema (HAE) is characterized by interindividual and intraindividual variability in symptom expression over time. Flexible therapy options are needed. Methods:We describe and report on the outcomes of the highly individualized approach to HAE therapy practiced at our HAE center in Frankfurt (Germany). Results:The HAE center at the Frankfurt University Hospital currently treats 450 adults with HAE or AAE and 107 pediatric HAE patients with highly individualized therapeutic approaches. 73.9% of the adult patients treat HAE attacks by ondemand therapy with pasteurized pd C1INH concentrate, 9.8% use additional prophylaxis with attenuated androgens, 1% of the total patient population in Frankfurt has been treated with Icatibant up to now. In addition adult and selected pediatric patients with a high frequency of severe attacks are instructed to apply individual replacement therapy (IRT) with pasteurized pd C1INH concentrate. Improvement on Quality of Life items was shown for these patients compared to previous longterm danazol prophylaxis. Home treatment of HAE patients was developed in the Frankfurt HAE center in line with experiences in hemophilia therapy and has so far been implemented over a period of 28 years. At present 248 (55%) of the adult patients and 26 (24%) of the pediatric patients are practicing home treatment either as on demand or IRT treatment. Conclusions:In conclusion, the individualized home therapies provided by our HAE center, aim to limit the disruption to normal daily activities that occurs for many HAE patients. Furthermore, we seek to optimize the economic burden of the disease while offering a maximum quality of life to our patients.

Introduction On demand treatment of acute angioedema in HAE type I and II Hereditary angioedema (HAE) is based on a hereditary, lifelong deficiency of C1esteraseinhibitor (C1INH). Patients with HAE suffer from recurrent, localized, acute edema attacks that can affect any body location. Mainly affected are subcutaneous tissues or mucous membranes, the gastrointestinal tract, and the throat, the latter leading to potentially lifethreatening laryngeal edema. Manifestation of acute edema in hereditary angioedema is characterized by interindividual and intraindividual variability in symptom expression over

* Correspondence: [e-mail]frankfurt.de Centre of Pediatrics III, Department of Hematology, Oncology and Hemostasis, Comprehensive Care Centre for Thrombosis and Hemostasis, Johann Wolfgang Goethe University Hospital, Frankfurt am Main, Germany

time. The onset of the next attack, its location and its severity are unpredictable. Treatment options adapted on the specific needs of the individual patient need to be implemented based on the type and frequency of HAE attacks and should be reevaluated from time to time [1,2]. Worldwide, five different therapy options for on demand therapy of acute attacks based on three distinct pathophysiological approaches are currently under clini cal investigation or already approved in different coun tries. For replacement of lacking or dysfunctional C1INH, three different C1INH concentrates  two plasmaderived (pd) and one expressed in transgenic rabbits  are available or under investigation. Antagon ism of the bradykinin B2receptor, which is supposed to largely convey the increase in vascular permeability lead ing to acute angioedema in HAE, via the B2receptor

© 2010 AygörenPürsün et al; licensee BioMed Central Ltd. This is an Open Access article distributed under the terms of the Creative Commons Attribution License (http://creativecommons.org/licenses/by/2.0), which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.