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Treatments with a special molecule (Emicizumab) whose use is approved in phases in Latin America increase options to improve the quality of life of hemophilia patients, agreed several experts at a forum held this week in Cali (
"There have been new studies where new molecules can be used, we have molecules like monoclonal antibodies (type of protein produced in the laboratory that can bind to a substance in the body) and we also have Genetic Therapy where very interesting progress is being made for hemophilia A and B, "explained in an interview with Efe Miguel Antonio Escobar, Medical Director of the University of Texas Research Center
Escobar participated this week in the seventh edition of Roche Press Day, an informative and educational meeting that promotes the continuing education of journalists and where they are The latest news from the health sector in Latin America
There are about 500,000 people in the world suffering from hemophilia, a disease that consists of the absence of a blood protein allowing coagulation.
For haemophilia A it constitutes 85% of cases, there is a lack of factor said VIII and for B (which suffers the remaining 15%), there are weak or nonexistent percentages of factor IX .
To make blood coagulation possible a total of 13 factors must be involved together.
"You have to think about coagulation as if it was a symphony.If a part is missing, it does not work well," says Escobar, who works as a hematologist at the Center. of Hemophilia and Thrombophilia of the Gulf States of the University of Texas (USA).
"One of the molecules (Emicizumab)) has recently been approved, clinical studies have shown that it is very safe and effective from a medical point of view.It avoids bleeding, which is our goal in hemophiliac patients, "says Escobar
Daniel Ciriano, medical director of Roche, l & # 39; pharmaceutical company that developed the molecule, told Efe that it would be administered to patients developing factor VIII inhibitors coagulation.
"Today we are replacing factor VIII with a factor VIII that the body does not produce. Continuous administration means that 30% of patients develop antibodies that block it, inactivate it (with factor VIII), "he added.
The new treatment makes patients work with inhibitors developed and the annual bleeding rate
"In patients treated with the molecule, the annual bleeding rate is almost zero," explains Ciriano.
Patients' quality of life and adherence to treatment. Improve, including children, should not inject two or three times a week to provide the factor they need.
"When injections are done once a week, and studies now show that 39, they can be done every two or four weeks
Escobar also pointed out that some of the so-called "gene therapies" are already in the phase of clinical studies. It is then injected to the individual suffering from hemophilia and the liver is able to produce factor VIII or IX, "says the specialist.
Hemophilia is a genetic disorder that affects the male population (99% of cases) and is pbaded from mother to child.
In Latin America, it is estimated that there are about 58,000 cases, of which 52% have not yet been diagnosed. Reducing this number is the goal of the World Federation of Hemophilia (WFH).
To combat the under-diagnosis, the federation works with the countries of the region where they found disparities in the data. Paraguay has the largest number of subregisters and we have good results in Brazil, Argentina, Venezuela, Costa Rica, Chile and Panama, "said César Garrido, member of the board of directors. of the WFH., formed by a worldwide network of patient organizations in 140 countries.
Up to 1937, the life expectancy of a person with hemophilia was 13 years old.Today, the average is between 65 and 70.
EFE
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