The subcutaneous drug is administered in seconds once every two weeks. Lives change, parents return to work, children forget about the disease, they say they have regained their lives anew," says Ewelina Matuszak, president of the Sanguis Foundation - Hemophilia and Related Hemorrhagic Diathesis, which appeals to policymakers to make hemophilia treatment in Poland humane, which subcutaneous therapy provides.
Hemophilia: Reclaiming Life
Published May 27, 2025 07:15
What are the Sanguiz Foundation's key demands regarding access to subcutaneous treatment for children with severe hemophilia?
Our children are in the B15 drug program, albeit access to intravenous therapy. Imagine that it has never been possible to purchase prolonged-acting factors for children, despite the fact that these factors are included in the drug program. This is absurd, for the reason that hemophilia treatment involves intravenous administration of clotting factor even every two days. So for a young patient, in whom prophylaxis starts around the age of one, it's really a traumatic experience, but also for the families, because the whole family gets sick together with this child. Unfortunately, as I said: access to treatment is there, but this access is truncated. In addition, this treatment is very taxing. Hemophilia patients receive medication at home and families carry out this treatment at home themselves.
So are they performing a medical procedure?
Yes. This is a medical procedure that involves administering fairly large volumes intravenously. Imagine a child of a year, two years, three years even five years old, who has to have a clotting factor administered in this way every two days. And then there's this absurdity: we don't have prolonged-acting factors, they have never been purchased despite the fact that they are reimbursed. In addition, there is already subcutaneous therapy, which is used all over the world, because medicine is moving forward. We know that there are cutting-edge therapies and that the world is treated very differently. Poland since 2008 (which was a breakthrough, because the drug program for hemophilia was created then), has been left behind, nothing has changed. Thanks to the demands of the Foundation and the patients, an extension to the drug program for children was implemented in July, where some of the patients most in need according to specially defined criteria have access to subcutaneous drugs.
These parents tell me that the subcutaneous drug is administered in seconds once every two weeks. Life changes, the parents go back to work, the children forget about the disease, they say they have regained their lives all over again. And now we have this mirror image, where some children ask: "Mom why don't I get this treatment? Mom can I deserve it?". The intravenous agent is a limitation for such a child. When we go on a trip, we have to know to give this factor, either the parent goes with the child or has to supply a nurse there to give the IV.
This is a limitation for the whole family....
Yes, even more so when the child is already older. It has to learn how to administer the medicine. He doesn't run around with children, he doesn't think of a carefree childhood just has to learn how to tame this disease. Despite the fact that it's a preventative, these children still bleed.
Some children have already received subcutaneous treatment. About 70 patients have had their lives transformed by 180 degrees. Parents often say that they have been given a new life, while the rest, unfortunately, have not. The public has finally seen what hemophilia treatment looks like, because until now it was hidden behind four walls in homes. It was the parents who were responsible for this prevention, for giving the drug to their children. As they showed on their social media what the supply of clotting factor looks like, the media, the public reacted and an appeal was created, which was signed by 100,000 people in two weeks.
The public expects a change in treatment, that in the 21st century we cannot allow such omissions and such treatment for primarily the pediatric part. These are children who do not have psychological care, where such treatment also takes a toll on their overall health, due to the fact that it is intravenous administration of clotting factor every other day.
You are calling for access to this subcutaneous treatment for children, but you are also bringing together adult patients. What about them?
Yes. First of all, as I said, 100,000 signatures cannot pass in front of decision-makers. We call for extending these criteria to the entire pediatric part. Of course, we have stocked treatments for adults. These are also factors administered intravenously. But here the issue is reversed, because adults, these are usually disabled people in whom the disease has already made great progress. They haven't had prophylaxis before, they have destroyed veins, they can't move, they have destroyed joints and often can't administer the drug to themselves.
They are like second-class patients because they don't have access to subcutaneous treatment, despite the fact that this therapy is already available in other countries. In the second quarter of 2023, a reimbursement application has been submitted for some adult patients, so that the most needy also have access to the subcutaneous drug. These people often cannot give themselves intravenous prophylaxis. They also need rest, they are really in a very severe condition, so why are we turning our eyes away from them?
What would you appeal to policy makers for?
I ask you very much that the treatment of hemophilia in Poland should change, because it is no longer something that is innovative, it is something that should be. We need to catch up with the treatment as it is in European countries.
It's not our phenaboys, it's something is just humanitarian. So I appeal that finally someone should hear us and hear the suffering of these people and that subcutaneous treatment should be made available.
