My son is waiting for a bone marrow transplant. Will he win the race against time?
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– What to wish me in the New Year? Good news: we have a donor, Hubert’s future is secure. We talk with Natalia, the mother of a six-year-old who has been diagnosed with a rare mutation in the PIK3CD gene, about what everyday life looks like in self-isolation and how one lives with hope …

Mrs. Natalia, how did you find out that Hubert had a PIK3CD mutation, which causes severe immunodeficiency?

It’s a long story … In fact, for the last six years we’ve been trying to find out what our son is suffering from. His health problems started when he was three months old. Cough, runny nose, RSV pneumonia. After doctors at the hospital in Lębork diagnosed Hubert’s anemia, we were transferred to the University Clinical Center, Department of Paediatrics, Hematology and Oncology in Gdańsk. The first diagnosis was: infantile myelomonocytic leukemia (JMML).

Type of chronic myeloid leukemia?

Yes, fortunately the diagnosis was not confirmed, but we still didn’t know what was wrong with him … In the meantime, our six-year-old went through a lot: blood transfusions, lymph node biopsy, three bone marrow biopsies looking for neoplastic changes. Even a clipping of his scalp was taken. Later, for several years, we were convinced that our son had a caspase 10 gene mutation, and finally there was a breakthrough in September 2021. After conducting genetic tests again, Hubert was diagnosed with a pathogenic mutation in the PIK3CD gene, which determines severe immunodeficiency.

That’s when you learned that a son’s “to be or not to be” depends on an unrelated bone marrow donor?

Doctors immediately explained to us that the only salvation was a bone marrow transplant. There is no other medicine than hematopoietic stem cells. The worst thing, however, was that for several years we lived in the unconscious and Hubert did not receive any treatment. And as we found out later, undiagnosed and untreated immunodeficiencies can lead to serious complications, including cancer. On the other hand, there was some relief: we finally know… and we can help him.

How has the “new diagnosis” changed your life?

Diametrically … We had to learn not only Hubert’s disease, but also how to live with it, which was not easy. I quit my job, and Hubert quit school and play with other children. All this to reduce exposure to infections. Our daily routine includes drugs, namely steroids, whose task is to stop immune cells from attacking their own body. Due to the fact that steroid therapy affects the carbohydrate metabolism, we measure the sugar level every day, or actually three times a day. We have also changed the diet – we keep an eye on the glycemic index of the products. Instead of kajzerek, wholemeal bread or graham, instead of wheat pasta, coarse groats.

And no sweets?

Which is not easy. Hubert has always been a gourmand. Even before the diagnosis, he could eat a whole bar of chocolate. Today, as long as his sugar is good, we allow him one lump. Even recently, when we baked Christmas gingerbread together, he only got one.

Self-isolation in the place of childhood – must it be hard for him?

In the beginning, it was the hardest – because of his illness, he had to give up many activities that he liked and that were important to him. He often sat in the room sad and thoughtful. We tried to console him, explaining that our isolation was a temporary situation. Now it is also different, sometimes she has worse days and sometimes better. Although he is only six years old, he is very mature about his own disease. He even said to me: Mom, don’t be afraid, I’ll be fine. He knows we’ll be with him, whatever happens. The fact that we have each other is the most important thing. We also do not hide anything from him and try to explain everything to him, so that he knows what still awaits us and he understands the seriousness of the situation. He certainly misses school, friends and girlfriends the most. Sometimes we connect with his class at Zoom so they can see and talk for a while. This gives him the feeling that his peers have not forgotten him and miss him as much as he does for them.

How does the boy spend his free time today?

Unlike before, he was replaced by playing with himself as a place to play with other children. Sometimes he follows me or his dad step by step and asks: “play with me.” He draws a lot, builds houses and garages from blocks, everything with a ruler. He’s a little fagot. We go for walks practically every day, in his case exercise is very advisable.

I know that the search for Hubert’s “genetic twin” is still ongoing …

The DKMS Foundation helps us a lot from the very beginning. Honestly, it never occurred to me at first that we might not find a compatible unrelated bone marrow donor. I was more afraid of the risks associated with the transplant itself and possible complications. Now I know that every fifth patient in Poland does not find his “genetic twin” and has no chance of a transplant. That is why we bend over backwards to publicize our son’s story – in January, a concert for Hubert will take place at the Municipal Cultural Center in Potęgowo. His dad, who is a soldier, will also try to organize a registration campaign in his unit. Each new person registered in the donor database is an opportunity for us.

You can also join the database of potential bone marrow donors via the Internet, by ordering the registration package on the DKMS Foundation’s website – for Hubert and others looking for a compatible donor. It is just a few clicks, taking a smear and sending it to the foundation, and thanks to this our son can find his “genetic twin” and get a chance for a healthy life.

What do you wish for in the New Year?

Good news: we have a donor, Hubert’s future is secure. Our greatest dream is to give our son a normal life, not question marks.

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