The "22nd International Hemophilia Congress" held in Istanbul brought together patients and experts

The 22nd International Hemophilia Congress, organized in collaboration with the Hemophilia Association of Türkiye and the Federation of Hemophilia Societies, brought together patients, their relatives, and experts to discuss the latest developments in hereditary bleeding disorders and treatment methods.

Speaking to an AA correspondent about the congress held at Istanbul University, Istanbul University (IU) Rector and Turkish Hemophilia Association President Prof. Dr. Osman Bülent Zülfikar stated that hereditary bleeding disorders are genetic diseases that begin at birth and continue throughout life.

Zülfikar stated that the symptoms of the disease can manifest as bleeding from some wounds, delayed closure, continued bleeding after surgical interventions, failure to close stitches on time, and external and internal bleeding after any kind of trauma, fall or accident.

Zülfikar noted that hemophilia, which they categorize as hemophilia A and hemophilia B, is at the forefront of these, saying, "While this is only seen in boys, there's another similar disease called Von Willebrand disease, which is equally common. There are also rare factor deficiencies. There are other deficiencies, which we call deficiencies of proteins involved in the clotting system, which stop bleeding. All of these are known as hereditary bleeding disorders, or hemophilia in the current sense."

Zülfikar explained that hemophilia, a disease caused by a genetic defect, has existed since the earliest days of humankind, and continued:

"Therefore, the question, 'Could we fix the disease if we correct this gene?' has been wondered for years. Now, it has become applicable to a certain extent. Furthermore, in this genetic disorder, the body lacks a substance that enables clotting. The question was, 'Could we solve the problems by obtaining that missing substance from healthy people and administering it to them?' And that was also successful. Later, due to the possibility of transmitting the disease to others, they began to be produced in laboratories. This was in the early 1990s, about 30 or 35 years ago. Now, those drugs are on the market. Therefore, they are produced in large quantities."

Prof. Dr. Zülfikar stated that the most significant problem patients face in this regard stems from the fact that these medications must be administered intravenously. He said, "It's not easy to administer them intravenously. And not just once. If we want to avoid bleeding, they need to be administered twice a week. In other words, administering these medications to people from newborns to advanced age isn't as easy. That's why the healthcare industry is working hard to produce new medications."

Prof. Dr. Zülfikar pointed out that the new drugs developed in recent years are administered as injections rather than intravenously, and said that these drugs can be used once a week, once every two weeks, or once every eight weeks.

Zülfikar explained that they are working to keep patients closely informed during this rapid and active process. "We've been sharing this with the public for years," he said. "Because the medications our patients use are covered by public taxes. Unfortunately, all of these medications are imported. We're working on this, and if we can achieve the same in our country and have our own products available to our patients, we'll be very happy."

Underlining that the purpose of these congresses is not just to bring healthcare professionals together, Zülfikar noted:

"Anyone suffering from bleeding disorders and their families are coming to this congress. Therefore, we are holding a mixed congress. We are hosting hematologists, orthopedists, physical therapists, physiotherapists, dentists, and, of course, nurses, who are essential. Tomorrow, we will be holding a special half-day session for nurses. Therefore, our colleagues who have translated this multidisciplinary work into papers and publications are also here."

Zülfikar stated that speakers from various countries, including Thailand, Germany, France, and the United States, attended the congress, adding, "This is a congress that brings together experts in this field, both domestically and internationally, and those who have a real concern."

Professor Dr. Kaan Kavaklı, President of the Federation of Hemophilia Associations, also emphasized the importance of the congress, saying, "Hemophilia is a serious, lifelong blood disease. At the Hemophilia Congress, we bring together all patients, relatives, doctors, and nurses who care for hemophilia patients. We meet in different halls. Sometimes we meet in common rooms. Every year, there's always a two- or three-day meeting. This is very important because doctors, patients, and relatives can closely follow the latest developments and ask questions of the doctors."

Commenting on gene therapy, Kavaklı said, "Regarding gene therapy, hemophilia is a genetic disease, but gene therapy is very new, of course. Research has just been completed. We're also conducting this in Türkiye. It's been 80% successful, but it could take at least five years for it to be reimbursed in the market. It's very costly."

Kavaklı noted that the medications patients have been using for the last 20 years are intravenous drugs that they must use twice a week for life, and said:

"Of course, they start this from infancy. That creates a challenge. That's why our patients are currently interested in the newly developed, subcutaneous drugs that are easier to administer, like the ones diabetics use insulin, and they're asking more questions about them. But of course, it seems it will take a few years for these to be included in the reimbursement system. Currently, at least all our hemophilia patients can get their intravenous medications covered free of charge through the Social Security Institution (SGK). Of course, we hope that in the coming years, more easily administered medications will be reimbursed."

Kavaklı emphasized that hemophilia patients in Türkiye have access to existing treatments, saying, "In Türkiye, there are currently factor medications available to help these patients prevent bleeding. There are also some subcutaneous medications. So, our patients are not helpless. If they use the available medications as prescribed by their doctors, the risk of dying from bleeding is very low. The risk of permanent joint disability is also quite low."

Kavaklı noted that at this congress, they explained to patients how to use both intravenous and subcutaneous factor medications and which medications will be available in the coming years.

It was reported that 185 of the 422 participants attending the congress, held at Istanbul University and ending tomorrow, were patients and their relatives, and it was noted that the information obtained at the congress will also be shared with the Ministry of Health and the Social Security Institution.