A new molecule helps hemophilia patients live better lives

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, various specialists have agreed in a forum held this week in Cali (Colombia).

" There have been new studies where new molecules have appeared that 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 is also genetic therapy where very interesting advances are made for hemophilia A and B "Miguel Antonio Escobar, Medical Director of the University of Texas Research Center , explained in an interview with Efe.

Escobar participated this week in the seventh edition of Roche Press Day, an informative and educational meeting that promotes continuing education for journalists and reports on the latest developments in the health sector in Latin America.

There are about 500,000 people worldwide suffering from hemophilia a disease that consists of the absence of a protein in the blood that allows clotting.

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

For blood clotting to be 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. for hemophilia and thrombophilia of the Gulf States of the University of Texas, United States.

One of the molecules (Emicizumab) has recently been approved. have shown that it is very safe and effective from a medical point of view.It prevents bleeding, which is the goal we have in hemophiliac patients, "says Escobar

Daniel Ciriano, director Roche Medical, the pharmaceutical company that developed the molecule explains to Efe that it will be administered to patients who develop inhibitors of coagulation factor VIII.

"Today, we are replacing factor VIII by 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", explains 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 studies show now that they can be done both or [4] these are benefits enjoyed by patients said.

Escobar also noted that some of the so-called "g therapies" are already in clinical trials. "They consist of" choosing a vector, the badociated adeno 8, which is made of genetic modifications. "

" After being injected into the individual with hemophilia and the liver succeeds in producing factor VIII or IX, "he explains, the specialist

Hemophilia [19659003] is a genetic disease affecting the male population (99% of cases) and transmitted from mother to child.

In Latin America, it is estimated that there are 58,000 cases, of which 52% are Have not yet been diagnosed Reduce 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

"Guatemala, Bolivia and Paraguay has the largest number of subregisters. We have good results in Brazil, Argentina, Venezuela, Costa Rica, Chile and Panama, "said César Garrido, board member of the WFH, an international non-profit organization founded in 1963 by a worldwide network of patient organizations. in 140 countries

Until 1937, the life expectancy of a person with hemophilia was 13 years. Today, the average fluctuates between 65 and 70 years old. EFE

(I)