Patients with hemophilia, a serious blood clotting disorder, are being treated better and better in Poland, but many changes are still needed to implement the recommendations of the European Parliament in this regard, experts and patients said at a press conference in Warsaw on Tuesday.
It was organized on the initiative of the Polish Association of Patients with Hemophilia in cooperation with the European Haemophilia Consortium – EHC.
The most important changes for the better in the care of patients with haemophilia include the introduction of the National Haemophilia Treatment Program in 2005 and the commencement – in August 2008 – of a program for the prevention of muscle and joint bleeding in children with this disease. We are very grateful to Minister Ewa Kopacz for introducing this program. It brought a huge change in the lives of sick children, who thanks to it can avoid painful haemorrhages and disabilities – emphasized Bogdan Gajewski, President of the Polish Association of Patients with Hemophilia.
Hemophilia is a rare genetic disease. It consists in the deficiency or absence of one of the blood clotting factors, which makes bleeding last much longer. Mainly men suffer from it, but women are carriers of the defective hemophilia gene. In Poland, there are currently over 2,5 thousand registered. patients with hemophilia.
The disease manifests itself as painful bleeding into joints, muscles and internal organs. They lead to irreversible damage to the joints, causing disability, but can also be deadly, such as a stroke.
In 2009, the EP clarified the rules of care for people with hemophilia in the European Union. They talk about the need to provide patients with safe and effective coagulation factors, access to bleeding prophylaxis, comprehensive care and treatment of a complication of therapy, such as the appearance of a clotting factor inhibitor.
As assessed by the President of the Irish Haemophiliac Association, Brian O’Mahony, for Poland to fully implement the EP recommendations for the care of hemophilia patients, it is necessary to extend the prevention program to adolescents over 18 years of age and to all adults who need it. Prophylaxis consists in administering the missing clotting factor several times a week, not just when needed. The data presented by O’Mahony shows that it is very profitable, because it reduces by over 80 percent. absenteeism from work and school, as well as the number of tissue bleeding which is very expensive to treat. The fact that it is worth using is best evidenced by the fact that in Western countries patients with hemophilia work normally, while in Poland most of them are on disability pensions.
O’Mahony emphasized that Poland is one of the few European countries where only blood coagulation factors are used and there is no access to the safest recombinant factors obtained by biotechnology.
As Gajewski reminded, in the 90s blood-derived factors caused massive infections with HIV and hepatitis B and C viruses (HCV and HBV) in children and adults with haemophilia. In July 2010, the District Court in Warsaw received a lawsuit against the State Treasury on behalf of 378 patients from all over Poland who had been infected with HCV in the past as a result of receiving blood coagulation factors.
According to the director of the Center for the Treatment of Hemophilia and Thrombosis at the University of Oxford, Dr. Paula Giangrande, while currently no plasma-derived clotting factor carries a risk of HIV or HCV infection, no one can predict the risk of transmitting infectious prions causing a new variant of Creutzfeldt-Jakob disease.
For many years, recombinant factors have been very expensive. But recently the situation has changed a lot. According to Dr. Giangrande, in the UK, where only recombinant agents have been used in haemophiliac patients for more than five years, prices have caught up with blood medicines and, in some cases, have fallen lower.
The hematologist also noted that – contrary to previous concerns – these drugs do not increase the incidence of a serious complication of hemophilia, which is the appearance of a clotting factor inhibitor.
In January 2010, the Minister of Health signed a new version of the prevention program for children with hemophilia. It ensures that the youngest children with a newly diagnosed disease who have not previously been treated with plasma products will receive recombinant factors.
This aroused great controversy among parents of children who have already started therapy with blood derivatives. I am very grateful to everyone who contributed to the preventive treatment of children. However, like every parent of a sick child, I would also like my son to receive the safest drugs, in this case recombinant clotting factors – said Agnieszka Cybulska, mother of seven-year-old Jaś with hemophilia, who was present at the conference.
For the time being, for organizational reasons, the new version of the program has not been launched, so children diagnosed with haemophilia in January 2010 will not qualify for it, because they have started using plasma factors. (PAP)