TY - JOUR
T1 - Hereditary Angioedema Caused by C1-Esterase Inhibitor Deficiency
T2 - A Literature-Based Analysis and Clinical Commentary on Prophylaxis Treatment Strategies
AU - Gower, Richard G.
AU - Busse, Paula J.
AU - Aygören-Pürsün, Emel
AU - Barakat, Amin J.
AU - Caballero, Teresa
AU - Davis-Lorton, Mark
AU - Farkas, Henriette
AU - Hurewitz, David S.
AU - Jacobs, Joshua S.
AU - Johnston, Douglas T.
AU - Lumry, William
AU - Maurer, Marcus
N1 - Publisher Copyright:
© 2011 World Allergy Organization; licensee BioMed Central Ltd.
PY - 2011/2/15
Y1 - 2011/2/15
N2 - Hereditary angioedema (HAE) caused by C1-esterase inhibitor deficiency is an autosomal-dominant disease resulting from a mutation in the C1-inhibitor gene. HAE is characterized by recurrent attacks of intense, massive, localized subcutaneous edema involving the extremities, genitalia, face, or trunk, or submucosal edema of upper airway or bowels. These symptoms may be disabling, have a dramatic impact on quality of life, and can be life-threatening when affecting the upper airways. Because the manifestations and severity of HAE are highly variable and unpredictable, patients need individualized care to reduce the burden of HAE on daily life. Although effective therapy for the treatment of HAE attacks has been available in many countries for more than 30 years, until recently, there were no agents approved in the United States to treat HAE acutely. Therefore, prophylactic therapy is an integral part of HAE treatment in the United States and for selected patients worldwide. Routine long-term prophylaxis with either attenuated androgens or C1-esterase inhibitor has been shown to reduce the frequency and severity of HAE attacks. Therapy with attenuated androgens, a mainstay of treatment in the past, has been marked by concern about potential adverse effects. C1-esterase inhibitor works directly on the complement and contact plasma cascades to reduce bradykinin release, which is the primary pathologic mechanism in HAE. Different approaches to long-term prophylactic therapy can be used to successfully manage HAE when tailored to meet the needs of the individual patient.
AB - Hereditary angioedema (HAE) caused by C1-esterase inhibitor deficiency is an autosomal-dominant disease resulting from a mutation in the C1-inhibitor gene. HAE is characterized by recurrent attacks of intense, massive, localized subcutaneous edema involving the extremities, genitalia, face, or trunk, or submucosal edema of upper airway or bowels. These symptoms may be disabling, have a dramatic impact on quality of life, and can be life-threatening when affecting the upper airways. Because the manifestations and severity of HAE are highly variable and unpredictable, patients need individualized care to reduce the burden of HAE on daily life. Although effective therapy for the treatment of HAE attacks has been available in many countries for more than 30 years, until recently, there were no agents approved in the United States to treat HAE acutely. Therefore, prophylactic therapy is an integral part of HAE treatment in the United States and for selected patients worldwide. Routine long-term prophylaxis with either attenuated androgens or C1-esterase inhibitor has been shown to reduce the frequency and severity of HAE attacks. Therapy with attenuated androgens, a mainstay of treatment in the past, has been marked by concern about potential adverse effects. C1-esterase inhibitor works directly on the complement and contact plasma cascades to reduce bradykinin release, which is the primary pathologic mechanism in HAE. Different approaches to long-term prophylactic therapy can be used to successfully manage HAE when tailored to meet the needs of the individual patient.
KW - C1-esterase inhibitor
KW - HAE
KW - angioedema
KW - attenuated androgen
KW - prophylaxis
UR - http://www.scopus.com/inward/record.url?scp=85014207459&partnerID=8YFLogxK
U2 - 10.1186/1939-4551-4-S2-S9
DO - 10.1186/1939-4551-4-S2-S9
M3 - Article
AN - SCOPUS:85014207459
SN - 1939-4551
VL - 4
SP - S9-S21
JO - World Allergy Organization Journal
JF - World Allergy Organization Journal
ER -