A New Molecule Helps Hemophilia Patients Live Better – Digital Diary Our Country

"New studies have shown that new molecules can be used, we have molecules like monoclonal antibodies (a type of protein produced in the laboratory that can bind to a substance in the body) and there is also a genetic therapy where very interesting advances are made for hemophilia A and B. "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 given to know the latest developments in the health sector in Latin America.

About 500,000 people worldwide suffer from 39, hemophilia, a disease that consists of the absence of a blood protein that allows coagulation.

For hemophilia A, which constitutes killing 85% of cases, there is a lack of the so-called factor VIII and for B (which suffers the remaining 15%), there is

To make blood clotting possible, it takes a total of 13 factors.

"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 hemorrhages, which is our goal in hemophiliac patients, "says Escobar

Daniel Ciriano, Roche Medical Director, l & rsquo; Pharmaceutical company that developed the molecule, told Efe that it would be administered to patients developing inhibitors of 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 bleeding rate is

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

Patients' quality of life and their adherence to treatment is a serious problem. 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 now 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" 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.

Haemophilia is a genetic disorder that affects the male population (99% of cases) and is transmitted from mother to child.

In Latin America, there are an estimated 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

"Guatemala, Bolivia and Paraguay has the largest number of sub-registers. 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

up to 1937, the life expectancy of a person with hemophilia was 13 years old. The average fluctuates between 65 and 70. Window EFE