Biological therapy in hereditary angioedema: transformation of a rare disease.
Expert Opin Biol Ther
; 20(5): 493-501, 2020 05.
Article
en En
| MEDLINE
| ID: mdl-31994957
ABSTRACT
Introduction:
Hereditary angioedema, a disabling condition, with high mortality when untreated, is caused by C1 inhibitor deficiency and other regulatory disorders of bradykinin production or metabolism. This review covers the remarkable progress made in biological therapies for this rare disorder.Areas covered Over the past 10 years, several evidence-based parenteral treatments have been licensed, including two plasma-derived C1 inhibitor replacement therapies and one recombinant C1 inhibitor replacement for acute treatment of angioedema attacks and synthetic peptides for inhibition of kallikrein or bradykinin B2 receptors, with oral small molecule treatments currently in clinical trial. Moreover, recent advances in prophylaxis by subcutaneous C1 inhibitor to restore near-normal plasma function or by humanized antibody inhibition of kallikrein have resulted in freedom from symptoms for a high proportion of those treated.Expert opinion This plethora of treatment possibilities has come about as a result of recent scientific advances. Collaboration between patient groups, basic and clinical scientists, physicians, nurses, and the pharmaceutical industry has underpinned this translation of basic science into treatments and protocols. These in their turn have brought huge improvements in prognosis, quality of life and economic productivity to patients, their families, and the societies in which they live.Palabras clave
Texto completo:
1
Colección:
01-internacional
Base de datos:
MEDLINE
Asunto principal:
Terapia Biológica
/
Angioedemas Hereditarios
Tipo de estudio:
Guideline
Aspecto:
Patient_preference
Límite:
Humans
Idioma:
En
Revista:
Expert Opin Biol Ther
Asunto de la revista:
BIOLOGIA
/
TERAPEUTICA
Año:
2020
Tipo del documento:
Article
País de afiliación:
Reino Unido