Hereditary angioedema (HAE) is a rare genetic condition characterised by recurring attacks of local swelling, caused by low levels of functional C1-inhibitor (C1INH) protein in the plasma. HAE attacks are often disabling and those obstructing the airway can be fatal. Treatment options for HAE attacks include plasma-derived C1INH, recombinant human C1INH (rhC1INH), icatibant and ecallantide. A rhC1INH (Ruconest™) has been approved in the EU for the treatment of acute HAE attacks. The 50U/kg approved dose effectively reduces the time-to-symptom relief and to resolution of the attack relative to placebo. rhC1INH is an alternative treatment option to plasma-derived C1INH, with no risk of human virus transmission and a high therapeutic success rate. In clinical trials, 95% of attacks treated with 50U/kg of rhC1INH showed symptom relief within four hours, relative to 69.8% of attacks treated with 20U/kg of plasma-derived C1INH. rhC1INH is a new treatment option for acute HAE attacks with high safety and efficacy benefits. © 2011 Touch Briefings.
A review of hereditary angioedema and recombinant human C1-inhibitor treatment
TRIGGIANI, MASSIMO;
2011
Abstract
Hereditary angioedema (HAE) is a rare genetic condition characterised by recurring attacks of local swelling, caused by low levels of functional C1-inhibitor (C1INH) protein in the plasma. HAE attacks are often disabling and those obstructing the airway can be fatal. Treatment options for HAE attacks include plasma-derived C1INH, recombinant human C1INH (rhC1INH), icatibant and ecallantide. A rhC1INH (Ruconest™) has been approved in the EU for the treatment of acute HAE attacks. The 50U/kg approved dose effectively reduces the time-to-symptom relief and to resolution of the attack relative to placebo. rhC1INH is an alternative treatment option to plasma-derived C1INH, with no risk of human virus transmission and a high therapeutic success rate. In clinical trials, 95% of attacks treated with 50U/kg of rhC1INH showed symptom relief within four hours, relative to 69.8% of attacks treated with 20U/kg of plasma-derived C1INH. rhC1INH is a new treatment option for acute HAE attacks with high safety and efficacy benefits. © 2011 Touch Briefings.I documenti in IRIS sono protetti da copyright e tutti i diritti sono riservati, salvo diversa indicazione.