A New Molecule Helps Hemophiliacs Live Better | Cuyo Newspaper

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, several experts agreed in a forum this week in Cali ( Colombia)

"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 there are also has genetic therapy where very interesting advances are made for hemophilia A and B, "explained Miguel Antonio Escobar, medical director of the University of Texas Research Center, in an interview with Efe.

About 500,000 people suffer from hemophilia, a condition that consists of the absence of a protein in the blood that allows clotting.

For the hemophilia A, which accounts for 85% of cases, there is a lack of factor VIII and for B (who suffers from the remaining 15%), there are low or nonexistent percentages of factor IX.

To make blood clotting possible, a total of 13 factors must be combined.

"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, United States.

"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 prevents bleeding, which is our goal in patients with hemophilia, "says Escobar

Daniel Ciriano, Roche Medical Director, The Company drug that developed the molecule, told Efe that it would be administered to patients developing inhibitors of coagulation factor VIII.

"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 rate of bleeding.

"In patients treated with the molecule, the annual bleeding rate is almost nil," said Ciriano.

Quality of life of patients 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 made once a week, and nowadays Studies show that they can be done every two or four weeks These are benefits appreciated by patients, "he said.

Escobar also noted that some of the so-called "gene therapies" have already been studied.

"Then injected to the individual suffering from hemophilia and the liver is able to produce factor VIII or IX," says the specialist.

It is a genetic disease that affects the male population (99% of cases) and is pbaded from mother to child. Until 1937, the life expectancy of a hemophiliac was 13 years old. Today, the average is between 65 and 70 years old.

> 58,000 in the region

In Latin America, it is estimated that there are about 58,000 cases, of which 52% have not yet been diagnosed. To combat the under-diagnosis, the federation works with countries in the region where they found disparities in terms of data. "Guatemala, Bolivia and Paraguay have 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 World Federation of Hemophilia. international non-profit organization established in 1963.

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