3 € Pharming schreef op 22 augustus 2019 07:40:
via @pharmingforum (twitter)
accesalud.femexer.org/farmaco-para-an...Drug for hereditary angioedema in pediatric patients
BY YTZAMEN PEÑA · AUGUST 1, 2019
hereditary angioedema sisters
A drug for hereditary angioedema shows safety and efficacy in pediatric patients, a study shows.
History of Angioedema News, a recent clinical trial showed that the drug conestat alfa (marketed as Ruconest) was able to successfully treat 96 percent of episodes of acute inflammation in children with the rare genetic inherited angioedema disorder.
The drug was approved as a treatment for the disease in Europe in 2010 and in the USA. UU. In 2014. However, its effects on childhood patients were not supported by solid data.
About hereditary angioedema
Hereditary angioedema is a genetic disorder characterized by chronic episodes of swelling that can affect multiple areas of the body. The condition is caused by mutations that affect the HAE gene. Swelling attacks usually occur every two weeks or so; They can usually last several days. Swelling can affect the extremities, the digestive tract, the face and the respiratory tract, with the blockage of the respiratory tract being the most dangerous complication. Vomiting and abdominal pain may accompany the attack also if the digestive tract is involved. Treatment is to reduce the likelihood of attacks and prevent them from getting worse when they do.
Hereditary angioedema is usually life threatening if left untreated.
The prevalence of the condition is estimated at around one in 10,000 to one in 50,000, at least in the United States and Canada.
About Conestat Alfa
Conestat alfa works by replacing a critical C1 inhibitor protein that, in patients, is deficient or absent as a result of the genetic mutation. The symptoms of inflammation are triggered by the lack of this protein in the blood; therefore, supplementing that protein with conestat alfa can relieve swelling.
Results of the study
The trial included a total of 20 patients with hereditary childhood angioedema who were between five and 13 years old. Patients should receive an intravenous dose of conestat alfa in response to episodes of acute inflammation as they appeared. The dose level was 50 U / kg. The medication was used to treat a total of 73 attacks during the study. The primary endpoint was the duration from the moment the attack appeared until the onset of symptom relief. A secondary endpoint was the time for minimal symptoms.
The drug was able to stop 95.9 percent of the attacks. More than a third of the participants required treatment for four or more episodes. The average time for symptom relief was 60 minutes and the average time for minimum symptoms was 122.5 minutes.
This study clearly demonstrated that this medication was effective in this patient population without serious safety problems.
JAMES MOORE
Mooi resultaat, fijn voor de patiënten.