– This is a bum! A disgrace for a nation that murders its own children – Zbigniew Wojkowski, father of 11-year-old Ludwik from Krakow, does not hide his indignation. The boy’s mother, Lidia Kiersztyn, wrote a letter to the president on this matter. Mr. President, I am begging you on my knees for help. They take the drug. The children are dying.
Ludwik, son of Zbigniew and Lidia, suffers from an ultra-rare genetic disease: mucopolysaccharidosis, also known as Hunter’s syndrome. A child with MPS does not produce the enzyme that causes the sugar to build up. The mucopolysaccharides accumulated in the body damage the cells and organs of the body, leading to the degradation of the entire body and death. A chance to extend life and reduce suffering is to administer an intravenous injection of an enzyme called elaprazole once a week. Annual therapy with this drug costs about PLN 800. zloty. If not for the treatment with the drug elaprase (Ludwik takes it from the age of six), he probably would not be in the world anymore.
– Our son’s life hangs in the balance because the National Health Fund wants to withdraw elaprase from the list of reimbursed drugs – says Zbigniew. – For children with Hunter syndrome, it means death in agony. It is like stopping insulin for diabetics or stopping chemotherapy in people with cancer. Better that they do euthanasia.
— How is Ludwik?
– Our son is still running, watching movies and cuddling up to us – replies the desperate father. – He lives carefree in his world. There is no awareness of what is happening at all. That he is standing on a precipice, like the remaining handful of boys in Poland suffering from the most severe form of mucopolysaccharidosis. When we received information that the National Health Fund was consistently deleting subsequent patients from the list of people covered by elaprase therapy, we do not have a day of peace. Every six months Ludwiczek is consulted by the Coordination Team for Ultra-rare Diseases at the Children’s Health Center in Warsaw, which manages further therapy. Based on medical records, without a direct examination of the child, as everything is done in absentia. Every six months we die of anxiety, so experts will order it. When my wife and I learned that our friend Ludwiczek from Częstochowa, eight-year-old Bartek Walaszczyk, had been refused further therapy, our hands literally dropped. This is a death sentence for a helpless child! And it is death in excruciating pain!
— Na how long is your son guaranteed treatment reimbursement?
Two more months, says Lydia. – What will happen next, fear to think. When our child was diagnosed with the disease, he was over four years old. We had to move heaven and earth in order for the son to be properly diagnosed. Until the age of four, there were no symptoms. Ludwik was developing wonderfully. He already wrote some letters on the blackboard, kicked a ball with his dad. But it started to bother us that the little one stops talking. He, usually so cheerful, becomes withdrawn. There were problems with the digestive system, frequent diarrhea, but also recurrent throat infections. During the evening baths, I noticed that my son had an enlarged liver. We did most of the tests on our own, because the doctors did not see the problem. We heard that the baby still has time to talk. That if he doesn’t want to eat, it’s our problem that we can’t feed him. Only when we got to the hospital of St. Ludwik in Krakow, things turned right. The son fell into the hands of a wonderful doctor, Dr. Zbigniew Żubr, and it was he who first noticed that they had to be examined for Hunter. At the Children’s Memorial Health Institute, appropriate tests were performed on Ludwicz. I was at a training session in Warsaw when I got a call from my husband. Listen, I have two pieces of news, good and bad. Ludwiczek has an inherited genetic disease called mucopolysaccharidosis. The prognosis is not interesting. But there is already a drug in the world that compensates for the deficiency of the enzyme. Our child has a chance to live and function normally. Only the drug needs to be started as soon as possible.
– My wife was waiting for a child for 13 years – says Zbigniew. – Ludwik is a long-awaited child. Sure there was a temptation to break down. I know many parents who are very depressed about their child’s terminal illness. When they diagnosed Ludwiczek, our daughter, Matylda, was already at the candle. We didn’t have time to despair. We had to act to help our son and not to neglect Matilda.
– It took one and a half years for Ludwik to receive elaprase therapy. Reason? Objection of the then Minister of Health, Bolesław Piecha, who argued that the money for the therapy of children with Hunter syndrome is wasted amounts. The therapy of our children is unprofitable! During that year and a half, the disease seriously progressed and Ludwik ended up in a wheelchair. He cried a lot. His joints ached, he had trouble breathing, and his heart defect was getting worse. There were nights when he howled in pain. A child of several years. It is simply impossible to describe. When we finally managed to get therapy for my son at the end of the fifth year, we were amazed at its effects. Already after three months, there was an improvement in the child’s motor skills. Ludwik started to get to his feet. He overcame his fear of people, began to smile and talk. Someone who expresses the opinion that elaprase does not help children with Hunter, that it is only the parents’ whim, should live with us for at least a few weeks and see with their own eyes how the child functions with and without the drug. The information that elarpase therapy is not being used in the West is sucked from the finger. In highly developed European countries, to the rank of which we aspire, they cannot even imagine depriving a seriously ill child of a drug that helps him! It is possible in our country. Elimination of non-prognostic units. But what does it actually mean? Not shocking? Are we building a civilization of life or a civilization of death?
— On September 20, in Warsaw, you spoke personally with representatives of the Medical Technology Agency, which was to decide about the legitimacy of treatment with the drug elaprase in children with Hunter syndrome. How was the meeting?
– We got there in just a few people. Me with my wife Lidia, Agnieszka Walaszczyk, Bartek’s mother, MP Lidia Gądek and a few friends. We managed to meet the President of AOTM, Wojciech Matusewicz, the AOTM press spokesman, Ms Katarzyna Jagodzińska-Kalinowska and a few other employees. The meeting lasted about 2 hours. We handed over to the president our appeal regarding not collecting the medicine. In the conversation, we also managed to convey a lot of arguments in favor of treating our children, but as Wojciech Matusewicz told us, neither he, nor any of the people present at the meeting, was a decision-making person in the matter of which we were interested. The opinion on the treatment of elaprase was to be issued by the Transparency Council established especially for this purpose.
— And a few days later, the decision of the Transparency Council about the unprofitability of treating children with MPSII with the drug elaprase was made. The Minister of Health, Bartosz Arłukowicz, is to comment on this decision. And nerves again.
– I must emphasize it strongly – says Zbigniew – it is not only the matter of our son! Little is said about the fact that there is a sizable fraction of the population that are also deficient in the enzyme, but at a much lower percentage. Please imagine two bathtubs. Above one there is a tap turned on to the maximum, water is dripping a drop from the tap at the other bathtub. The worsening of our son’s disease is like running a full stream of water. But there are people who also have this condition, but don’t know it! The effects of the deposition of mucopolysaccharides in the body will become known only after many years. In the form of premature osteoporosis, cardiovascular diseases, rheumatism. We are not single parents who are crazy about our own child and make a buzz about him. But in the fight against the bureaucratic machine, we feel very lonely, unfortunately also powerless.
– Coming back to the Minister of Health Arłukowicz. Yes, the final decision is up to him. However, will it dare to stand against the unfair opinion of the Transparency Council, which, it is worth noting, was not unanimous? I feel a bit like I’m actually deja vu. A repeat from the times of Minister Piecha. Instead of focusing on work and raising children, I have to spend time and energy fighting windmills. But it is about the life of my son and children like him, such as Bartek Walaszczyk. That’s why I do it.