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We want to be treated safely, effectively and in accordance with the current knowledge – call people with hemophilia in Poland
On the occasion of the World Day of Patients with Hemophilia in Warsaw, a conference on the current situation of patients with hemophilia was held, attended by specialists, patients and members of the Polish Association of Patients with Hemophilia. Representatives of the Ministry of Health, the National Health Fund and the National Blood Center were present. On behalf of the specialists, the following spoke: Dr. Ewa Stefańska-Windyga from the Institute of Hematology and Transfusion Medicine in Warsaw and Dr. Andrzej Mital from the Department and Clinic of Hematology and Transplantology at the Medical University of Gdańsk, spoke. During the debate, the most pressing problems faced by patients in the current health care system were considered.
Hemophilia and related bleeding disorders are a problem that affects nearly 4500 patients, their families and relatives in Poland. – We want not only to improve the quality of treatment and access to modern therapy, but also to educate the society so that patients are better understood and accepted – emphasized Bogdan Gajewski, President of the Polish Association of Patients with Hemophilia.
Properly cared for, a person with hemophilia can lead a normal life – work, study, study, pursue his passions, lead a social life, and even play sports. This is the case in European countries richer than Poland. In our country, the majority of adults suffering from severe hemophilia are unfortunately disabled.
What would improve the situation of Polish patients so that they could enjoy the same good quality of life as, for example, our western neighbors? – Organizing complex treatment centers, ensuring prophylaxis for adults and the availability of the safest medicines for children. An important point is to ensure an efficiently operating drug supply system for patients (blood coagulation factors) – lists Bogdan Gajewski. Both patients and doctors are complaining about the electronic ordering system for drugs introduced last year.
The current situation of patients with hemophilia is definitely better than a dozen or even a few years ago. It happened thanks to the National Health Program for People with Hemophilia and the preventive treatment program for children up to 18 years of age – Thanks to the changes that have taken place in recent years, especially new patients have a chance to avoid the consequences of haemophilia – changes in joints leading to irreversible disability – Bogdan Gajewski noted.
Comprehensive hemophilia treatment centers in Poland
The current National Treatment Program for Patients with Hemophilia and Related Hemorrhagic Disorders for the years 2012-18 provides for the establishment of treatment centers where patients would be provided with comprehensive care. Such centers have long existed in Western countries: people with hemophilia in specialized centers can consult not only a hematologist, but also an orthopedist, physiotherapist, hepatologist, surgeon, dentist or psychologist. Comprehensive care brings excellent results: patients are more agile, can work, are not on disability pensions, which at the same time saves the budget. – Treatment centers in Poland should be established as soon as possible so that patients are covered by comprehensive care. We could use six such institutions in our country. You don’t have to create them completely from scratch. It would be enough to better equip the already existing selected hospitals, provide them with the possibility of performing specialist examinations – says Andrzej Mital, MD, PhD, hematologist from the Department and Clinic of Hematology and Transplantology of the Medical University of Gdańsk. The first two comprehensive treatment centers were to be established by the end of last year. Unfortunately, they were not created. The lack of centers also means the lack of proper rehabilitation. Meanwhile, it is essential for people with hemophilia. The right exercise strengthens the muscles. Joint damage worsens in unrehabilitated patients.
Establishment of centers is not possible without proper inpatient treatment of haemophilia, which is now dramatically underestimated. – One day of hospitalization of a patient with hemophilia is estimated at PLN 55. For this amount, at best, food for the sick can be provided. I do not know a center that could offer the treatment in this amount, which patients simply deserve – said Dr. Ewa Stefańska-Windyga. It is simply not profitable for hospitals to treat people with hemophilia. Patients seek help several hundred kilometers from home, and delaying the proper treatment may endanger their health and life.
Prophylactic treatment of haemophilia
In 2008, a program for prophylactic treatment of hemophilia was introduced, which improved the quality of life of patients. Prophylaxis is to prevent bleeding by regular administration of a clotting factor. Its purpose is to prevent or reduce the frequency of strokes. The most important advantage of this type of treatment is that the factor concentrate is administered before bleeding occurs. Children up to 18 years of age in Poland are subject to full prophylaxis. Blood clotting preparations are delivered to the patient’s home. The drug is administered intravenously 2-3 times a week. Thanks to this, the youngest patients can function normally, play with their peers, and go to school. Their joints remain in the best possible condition. Unfortunately, after reaching the age of majority, the patient is excluded from such constant care. From this point on, he can get clotting factors in the event of a stroke. For this reason, the patient’s condition worsens, who often has to stay at home for a long time due to repeated strokes. In such a situation, how to attend university regularly, go to work?
In Sweden, prevention is usually continued throughout life. In many European countries, adults decide for themselves whether they want to continue it. – Prophylaxis is discontinued in 18-year-olds. In the most difficult moment in a young man’s life. He should still take medication to be able to pass his high school diploma, study, find a job – says Bogdan Gajewski. – Lack of prophylaxis limits the activity of patients, hinders the possibility of leading a normal life, it also causes disability and progression of arthropathy, i.e. joint damage – adds Dr. Ewa Stefańska-Windyga. Representatives of the Polish Association of Patients with Haemophilia are striving to ensure that the preventive treatment is continued at least until the age of 25 and that it is introduced as soon as possible in the treatment of adults. – This is the easiest way to avoid disability, and at the same time to save money in the treatment of hemophilia in Poland, because prophylaxis simply pays off: it reduces long-term complications of hemophilia – adds Bogdan Gajewski.
There is good news for adult patients. From next year, the so-called secondary prophylaxis. – It will apply to patients who have more than one haemorrhage per month, or three haemorrhages per three months, or a history of severe bleeding complications such as the central nervous system. I hope that the rest of the patients will also receive such prophylaxis soon. We also hope that full prophylaxis will be extended to the age of 26 – added Dr. Mital.
Electronic drug demand system
Earlier this year, the National Blood Center (NCK) introduced an electronic drug demand system. According to it, a patient with hemophilia was to receive a clotting factor when the doctor wrote an electronic request. Unfortunately, the new system did not work as expected. Instead of improving it, it made it difficult for patients to order medicines electronically. – In many cases, doctors refused to issue electronic requests to patients. The reasons were technical difficulties: no computers, printers in offices, no Internet access, no training for doctors – said Bogdan Gajewski. Fortunately, traditional – paper orders were allowed all the time. Dr. Jolanta Antoniewicz-Papis, the new director of NCK, came to help patients and doctors. The period of validity of electronic requisitions has been postponed until September.
In the world, blood clotting factors – not only children but also adults – are delivered home. This form is not only more convenient for patients, but also safer. In Polish conditions, where the patients are largely disabled, this would be a significant convenience for them: a trip to a distant hospital in many cases costs money, and the budget of disabled patients is limited. They also often require the support of a caregiver – which already involves two people and multiplies the costs in the family. According to Dr. Stefańska-Windyga, delivering home medications to patients is a goal that should be pursued. – However, it is important that the doctor does not lose contact with the patient. Currently, the patient comes to the center for drugs once every 3 months. This is an opportunity to examine it, view the stroke frequency diary, and possibly update the therapy. I can’t imagine losing sight of the patient forever. That he would practically prescribe drugs himself, without any medical control – noted Dr. Mital.
Plasma-derived and recombinant drugs
Prevention and treatment of haemophilia in Poland is carried out by Voivodship Hemophilia Treatment Centers, which implement the assumptions of the National Hemophilia Treatment Program for 2012-2018 and the prophylactic program Prevention of bleeding in children with hemophilia. In 2012, the Institute of Hematology and Transfusion Medicine in Warsaw was selected as the Coordinating Unit for activities aimed at implementing the assumptions of the National Program for the Treatment of Hemophilia. The National Blood Center supervises the provision of blood coagulation factors to patients, which is the responsibility of the Regional Blood Donation and Treatment Centers. In Poland, plasma-derived coagulation factors are mainly used. In developed countries, they have already been largely replaced by modern recombinant drugs produced by genetic engineering. They are considered drugs with the highest safety profile. Plasma factors are obtained from donor plasma. It is enough for one of them to have a viral infection for it to contaminate an entire batch of drugs. Due to such contamination, in the 60s and 70s, many patients were infected, among others, with HIV and HCV viruses (World Haemophilia Federation reports that 22% of haemophiliac patients were then infected with HCV and 5% with HIV as a result of taking plasma-derived clotting factors). Fortunately, many precautions and tests for viruses in plasma have been introduced in the last three decades. There are also many methods of viral inactivation, which means that it is currently impossible to infect a patient with HIV or HCV. – Currently used plasma-derived preparations are safe. The fear of him stems from the experience of 30 years ago. Among the recombinant drugs, the safest are the newest, third-generation drugs – and these are very expensive. In addition to our native capabilities. Due to the high costs in the West, they are used to a limited extent – said Dr. Antoniewicz-Papis.
Recombinant factors are drugs obtained through biotechnology. They were first introduced into treatment in the early 90s. Today it is a technology that has completely eliminated the risk of infection with unknown viruses found in human plasma. However, we do not know what the long-term effects of their use may be. But that’s the way it is in pharmacology. Even aspirin has unwanted side effects. The most important question is whether the benefits of the therapy outweigh the risks. And in the case of both preparations – both plasma-derived and recombinant – there is no doubt that their regular administration dramatically improves the lives of patients.
Treatment of hemophilia in Poland
For many years, the standards of hemophilia treatment in Poland were significantly different from the global recommendations. Until 2007, patients could not even count on providing a sufficient number of doses of clotting factor throughout the year. This resulted in the treatment of complications instead of prophylaxis. Thanks to the National Program for the Treatment of Hemophilia, which was established in 2005 and is being continued, the availability of clotting factors in Poland has significantly improved (from factor VIII consumption per capita of 1 IU in 1.67 to 2004 IU in 4.77).
The first recombinant factors were purchased in Poland only in 2010, mainly to provide modern therapy to children who had not been treated for hemophilia so far. Poland is one of the few European countries with laws that discriminate against children with hemophilia. The prophylactic program stipulates that children who have been treated with plasma-derived coagulation factors cannot receive recombinant factors. Currently, according to the data of the Institute of Mother and Child in Warsaw, 30 children are treated with recombinant drugs in Poland. The rest receive plasma-derived preparations. – There is no medical reason why patients who have so far been treated with plasma-derived factors should not be able to receive recombinant drugs. The patient and the doctor should have the right to choose the drug – emphasizes Dr. Stefańska-Windyga.
Recombinant drugs are widely used not only in Western countries. In Lithuania, they are freely available to all children up to the age of 10, and in the Czech Republic – up to the age of 15. In Lithuania, the doctor, in consultation with the patient, decides on the choice of appropriate drugs: recombinant or plasma-derived. In Poland, patients and doctors still do not have a choice of drugs.
Haemophilia is a genetic disease where one of the clotting factors (factor VIII for haemophilia A or factor IX for haemophilia B) is deficient or absent in the blood. Cases of hemophilia were quite common in European royalty, hence its common name – kings disease. It causes that internal bleeding, mainly into joints and muscles, is dangerous for patients. More serious injuries and cuts can even be life-threatening. Usually it is sick from birth. In 30-50% of cases, no one in the family was sick before. Hemophilia cannot be completely cured. However, thanks to proper, effective therapy, patients can lead a normal, active life – even in the case of severe disease. It is enough to administer the appropriate blood clotting factor in the form of intravenous injections to stop the bleeding. Worldwide, 400 people suffer from hemophilia. patients. In Poland, the incidence of this disease is estimated at 1 case per 12 inhabitants. A patient register has been kept for several years: there are 300 people, 4277 of whom suffer from hemophilia, and the rest of them suffer from other bleeding disorders.
Text: Agnieszka Fedorczyk