Hemophilia: The basics are safety, good access to drugs and development of treatment centers

April 17 - World Hemophilia Day - a day that reminds us of the needs of those living with this rare disease and an opportunity to reflect on the path that patients, doctors and the health care system have gone through to talk about it not as a sentence, but as a challenge that can be lived with. Poland has made tremendous progress in treatment in recent years. However, there are still challenges that hemophilia patients face on a daily basis.

- For us, the most important thing is safety, ensuring access to treatment anywhere in the country, both in scheduled and emergency situations. The National Hemophilia Treatment Program provides very good access to medication. In an emergency situation, clotting factors are provided free of charge to any place where it is necessary, to any hospital where a hemophilia patient finds himself due to an emergency or elective surgery. This is the basis of our safety," noted Bogdan Gajewski, president of the Polish Association of Hemophilia Patients.

Hemophilia doesn't have to shorten life. Patients fight for its good quality

In Poland, about 6,000 people suffer from hemophilia and related hemorrhagic diathesis. - It's a genetic disease, meaning it's most often inherited, but in about 30% of cases genetic mutations occur de novo, meaning hemophilia appears in a family where it didn't occur before. The biggest problem is strokes (bleeding), most often in the muscles and joints. The hemorrhages can occur not only after an injury, but also for no apparent reason: for example, the patient goes to sleep and gets up in the morning with a hemorrhage. Repeated bleeding leads to joint destruction and progressive disability. Some bleeding can be very dangerous, even life-threatening, e.g. to the central nervous system, gastrointestinal tract, so it is necessary to act quickly," said Prof. Pawel Laguna, head of the Department of Pediatric Oncology, Hematology, Clinical Transplantation and Pediatrics at UCK WUM, consultant in pediatric oncology and hematology for the Mazovian province

Improving treatment means that today the life expectancy of a hemophilia patient is not necessarily different from that of a healthy person. - The primary form of treatment is prophylactic treatment, which protects against bleeding. As a result, children with hemophilia who receive prophylactic treatment from the beginning can live and function normally. The new World Federation of Hemophilia (WFH) definition of prophylaxis says that we are to treat children so that their quality of life is comparable to that of healthy people. That is: if a boy wants to play ball, we're not supposed to forbid him from doing so, we're supposed to adjust the dosage of preventive treatment so that he can play ball. We individualize preventive treatment; I have patients who compete in swimming competitions and are just preparing for the Olympics," Prof. Laguna noted.

In a more difficult situation are adult hemophilia patients, who have serious joint problems, often disabilities, resulting from inadequate treatment lasting many years. In their case, the basic need today is not only good access to drugs, but also comprehensive care in treatment centers. - Today the amount of factor is adequate to my needs. This was not the case when I was a child, which is why I have joint problems, recently a knee synovectomy was necessary. It would certainly simplify my life to have home deliveries of medicines, but even so, at present I rate the treatment very well. I can work, run a business, and have a family, " said Radoslaw Kamycki, a hemophilia patient.

The program that changed the standards of hemophilia treatment in Poland

Treatment of hemophilia in Poland is currently based on the operation of two programs. The first is "Prevention of bleeding in children with hemophilia A and B," under which children up to the age of 18 receive preventive treatment. The second is the National Program for the Treatment of Patients with Hemophilia and Related Hemorrhagic Diathesis, which began 20 years ago (it is now in its fourth edition). Prior to its inception, access to drugs (clotting factors) in Poland was at a level that the World Health Organization (WHO) defined as the minimum necessary to save lives. Today, the situation is dramatically different: -We areat the forefront of European countries in terms of the use of factor VIII in hemophilia A and factor IX for hemophilia B," Prof. Laguna noted.

The Polish system for treating patients with hemophilia and related hemorrhagic diathesis and the central distribution of medicines is very well organized and is also praised abroad. - Medications are available at Regional Blood Donation and Hemotherapy Centers and hospital depositories (there are almost 70 of them), distributed throughout Poland. Patients have continuous access to drugs through the central distribution of cooperating Regional Blood Donation and Hematology Centers. This allows transfers to be made, if necessary, to other regions of Poland, which also translates into rational management of purchased drugs and their use within the expiration date ," noted Dr. Sebastian Twaróg, director of the National Blood Center (NCK).

NCK purchases medicines - as part of unrestricted tender proceedings conducted through the Department of Public Procurement under the Minister of Health. - Last year, we organized more than 40 proceedings for the purchase of various drugs: clotting factors, desmopressin, medicinal products that mimic factor VIII. We monitor drug stocks and consumption on an ongoing basis, and centrally manage inventories ," Director Twaróg stressed.

Many adult patients are disabled, so a system of home delivery of medicines is being developed. - Currently, nearly 300 hemophilia patients are receiving home deliveries. We are trying to extend home deliveries to a growing group of patients. The health care system provides patients with hemophilia and related hemorrhagic diathesis with security ," said Director Twaróg.

Hemophilia treatment centers: development and comprehensive care needed

Poland currently has 27 hemophilia treatment centers for adult patients and 15 centers for children. - The role of the center is to provide comprehensive care for patients. Let's remember that hemophilia is a rare disease; there are doctors who will never come across such a patient. Therefore, the patient must know where he can get help," Prof. Pawel Laguna noted.

At the center, the patient should - if necessary - get help not only from a hematologist, but also from other specialists, including an orthopedist, physiotherapist, dentist, psychologist, geneticist. This is important in both pediatric and adult treatment centers. - Hemophilia patients today are living longer and longer, which means they also have other diseases of civilization, such as hypertension, diabetes, heart disease, inflammatory bowel disease. At our center, we had a hemophilia patient who developed plasmocytic myeloma. Good care of the congenital diathesis made it possible for him to have a bone marrow transplant. Multispecialty care is the basis ," said Prof. Maria Podolak-Dawidziak of the Department of Hematology, Blood Tumors and Bone Marrow Transplantation at the Wrocław Medical University, chair of the Hemostasis Group of the Polish Society of Hematologists and Transfusionists.

There should be 24-hour telephone contact with the hemophilia treatment center: when an emergency situation (such as an accident) occurs, a paramedic or ED doctor will be able to contact the doctor on duty and consult the course of action. - In our center, such contact with the clinic, with the doctor on duty is provided 24 hours a day. Unfortunately, sometimes if a patient with an injury ends up in another hospital, there can be problems with proper management, despite the fact that every hemophilia patient carries a patient's chart, which states: "First give the factor, then diagnose." We are trying to spread this knowledge," noted Andrzej Mital, MD, from the Department of Hematology and Transfusiology at the University Clinical Center in Gdansk.

Although the situation is improving, there is still a problem in many centers with access to rehabilitation, which should be part of hemophilia treatment. Every hemophilia patient should be under the care of a physiotherapist. - Thanks to modern treatment, the musculoskeletal status of children is no different from that of their peers; these patients require sports counseling, possibly supervision and an annual fitness evaluation. In contrast, essentially 100 percent of adult patients who have not had the benefit of modern treatment have developed hemophilic arthropathy. They basically require ongoing rehabilitation treatment, as well as physiotherapy in preparation for orthopedic surgery and after orthopedic surgery. More and more physiotherapists are training in this field, but such good access to physiotherapy is still lacking in many centers , said Janusz Zawilski, MD, of the INTERLAB Medical Center in Poznań.

Access to medicines - the basis for patient safety

Both the Children's Prevention Program and the National Hemophilia Treatment Program are currently funded through the budget of the National Health Fund. - The budget is secure, predictable and stable. The preventive program is well organized, there are home deliveries for all children, there is individualization of treatment, most children receive recombinant factors. Also for the National Program, funding is stable, we do not foresee problems. We can assure patients that the situation will be as favorable to them as possible," noted Iwona Kasprzak, director of the Drug Management Department of the National Health Fund.

The portfolio of drugs used in hemophilia is growing: today these include clotting factors (plasma-derived, recombinant, extended-release), antibodies (administered subcutaneously), and the first gene therapies are also appearing. - We evaluate access to drugs and their distribution very positively. As for new drugs, we are monitoring their effectiveness; we are very interested in introducing new drugs, but we want this to be done within the framework of the National Program for the Treatment of Patients with Hemophilia and Related Hemorrhagic Diathesis, as it guarantees access to treatment throughout Poland. We do not want separate drug programs, as drug programs are implemented by specific hospitals and will not guarantee the safety of treatment for the patient throughout Poland," Bogdan Gajewski noted.

- As part of the National Program, we can already include patients in treatment with products other than clotting factors, the Program Council can make such a decision if the patient requires it. However, it has happened many times that a doctor sends a requirement for a patient for a particular drug, we find that there are other options for treatment, we establish contact with the doctor and in fact he often agrees that the treatment we proposed is possible," stressed Prof. Laguna.

Also according to the Ministry of Health, access to medicines today is very well organized and at a high level. - There are few patients with another disease entity that has such well-organized treatment, both in terms of access to therapy, logistics and the entire system. Patients with hemophilia have a very well-organized system and treatment options ," assessed Matthew Oczkowski, deputy director of the Health Ministry's Drug Policy and Pharmacy Department.

He also pointed out that there was a point in operating two programs (the preventive program and the National Program) when the former was managed by the National Health Service and the latter by the Ministry of Health. - I don't know if today there is a point in keeping the preventive program for children and the National Hemophilia Treatment Program separate. It would be better if the preventive program was attached to the national program. In general, I think we lack opportunities for price negotiations in central purchasing. If such opportunities arose, we could further reduce drug prices. In general, we should strive for patients to be able to use drugs that are administered infrequently, provided that they actually provide great protection against bleeding. This is something we must always pay attention to," Director Oczkowski noted.

Source: press mat.