Current and Future Therapy of Hereditary Angioedema

Hereditary angioedema (HAE) is an autosomal dominant disorder, mostly due to C1 es-terase inhibitor (C1-INH) deficiency, known by recurring angioedema attacks that are non-pruritic, not accompanying with urticaria, and involve the dermis, intestinal submucosa, and upper respiratory system. The angioedema attacks are not responsive to epinephrine, glucocorticoids, or antihistamine treatments. Whereas HAE patients formerly had a few therapeutic options accessible such as anabolic androgens and antifibrinolytics. Nowadays in many parts of the world there has been remarkable progress in HAE treatment for the last two decades and clinically confirmed medications are presented for prophylactic and attack treatment. Alternatives in attack therapy contain two plasma-derived C1-INH con-centrates, a recombinant C1-INH product, a kallikrein inhibitor, and a bradykinin β2 recep-tor antagonist. Options in prophylactic therapy include other than two plasma-derived C1-INH concentrates, subcutaneous C1-INH replacement and newest subcutaneous plasma kallikrein inhibitor Lanadelumab. In spite of these progresses, HAE patients still run into some challenges of an arduous disorder that can yield to devastating angioedema attacks related with important expenses for patients and the public. Better education of HAE patients and implementation of the self-management policy for “on-demand” therapy will recuperate patients’ life quality and negative effects of the disease. Herein the existing and promising therapeutic options are reviewed in the HAE management.

Günümüz ve Gelecekte Herediter Anjiyoödem Tedavisi

Çoğunlukla C1 esteraz inhibitör (C1-INH) eksikliğine bağlı meydana gelen herediter anjiyoödem (HAÖ) cilt, bağırsak submukozası ve üst solunum yolunu tutan; kaşıntı ve ürtikerle birlikte olmadan tekrarlayan anjiyoödem ataklarıyla seyreden otozomal dominant bir hastalıktır. Herediter anjiyoödem atakları epinefrin, glukokortikoit veya antihistaminik tedavisine cevapsızdır. Herediter anjioödem hastaları önceden anabolik androjen ve antifibrinolitikler gibi birkaç tedavi seçeneğine sahipti. Günümüzde birçok ülkede HAÖ tedavisinde son 20 yıldır belirgin ilerleme olmuş ve klinikte etkinliği kanıtlanmış atak ve profilaktik tedavide kullanılan ilaçlar piyasaya sunulmuştur. Atak tedavisinde alternatif-ler plazmadan üretilen iki C1-INH konsantresini, rekombinan C1-INH ürününü, kallikrein inhibitörü ve bradikinin β2 reseptör antagonistini içerir. Profilaksi tedavi seçenekleri plazmadan üretilen iki plazma kaynaklı C1-INH konsantresi dışında, subkutan C1-INH replasmanını ve en yeni subkutan plazma kallikrein inhibitörü Lanadelumab’ı içerir. Bu gelişmelere rağmen, HAÖ hastaları hasta ve toplum için önemli derecede maliyetle ilişkili ve yıkıcı anjioödem ataklarına yol açan bu zorlu hastalığın hala bazı ciddi sorunlarıyla karşılaşmaktadır. Hastalarının daha iyi eğitimi ve gereğinde tedavi için kendi kendine tedavi stratejisinin yerleşmesi hastaların yaşam kalitesi ve hastalığın negatif etkilerini düzeltir. Bu yazıda, HAÖ tedavisinde mevcut olan ve ümit vaat eden tedavi seçenekleri gözden geçirildi.

Kaynakça

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Kaynak Göster

Southern Clinics of Istanbul Eurasia
  • ISSN: 2587-0998
  • Yayın Aralığı: Yılda 4 Sayı
  • Başlangıç: 2017

832179

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