Histiocytosis is a disease which causes excessive multiplication of histiocytes, the cells belonging to the immune system. Histiocytes can build up in any organ and cause damage. The clinical manifestations vary greatly, depending on the patient's age or affected organs. These manifestations can present as cranial vault bone lesions, protrusion of the eyeballs, diabetes insipidus, bone marrow, liver and other organ failure, impaired appetite and weight loss. It is a very rare disease, it is estimated 2 in a million babies are born with it.
“Histiocytosis is a standard indication for stem cell transplantation. Today, transplant doctors shouldn't ask themselves the question if the given histiocytosis qualifies for a transplant but should ask themselves what they should do to obtain even better results if the transplantation is performed at the right time,” says Dr Dariusz Boruczkowski, specialist in paediatric diseases and clinical transplantation.
Miłosz lives with his parents and brother Piotruś (Peter) in a small town near Poznań. He was the first, and therefore very much awaited, baby of Grażyna and her husband Przemek. There were no problems during her pregnancy and there were no indications that the baby may be ill. However, several months after Miłosz's birth, his routine test results turned out to be alarming. Doctors started a series of specific examinations and after a long time of uncertainty, the parents were given the diagnosis – Miłosz suffered from histiocytosis. It wasn't the only shocking news they learnt. Another, completely unexpected announcement, was that Grażyna was pregnant. The parents’ feelings were a mixture of both joy and sadness but as Miłosz's mother recalls, sadness prevailed.
A diagnosis of histiocytosis meant that Miłosz had to spend the following months in hospital. His parents had to share their duties. Dad visited his son at weekends, while mum, even when she was in the late stages of her pregnancy, would spend every weekday in the clinic in Poznań. The therapy and the search for a bone marrow donor started. The first step was to test the parents but the lack of genetic compliance excluded them as donors. There was no other option but to wait until doctors found a matching bone marrow donor. During his battle with the disease, Miłosz would bravely undergo treatment and never once did he complain about the number of injections he would get. He often had to stay in a sterilized isolation room where only his mum and doctors could accompany him. The need to protect him against any infection meant restricting visits from other family members. Miłosz spent his second birthday in hospital and couldn't even try his birthday cake because he was on a special diet. And this wasn't even the worst of what he had to go through. Thanks to his incredible energy and cheerfulness he became a favourite of all the staff and for his bravery and good behaviour he was awarded with a diploma for courage, which his parents are very proud of.
Less than a year later, doctors informed the family that a bone marrow donor had been found but the bone marrow wasn't a full HLA match. At the same time Grażyna learned about the possibility to collect stem cells from the umbilical cord blood at the delivery of her second child. Umbilical cord blood stem cells are primitive, unspecialised and multipotent cells. Their extremely valuable property is their ability to self-renew and transform into other cells of which human tissue is built of. They are an alternative to bone marrow stem cells and can be transplanted even when they are not fully compliant. Grażyna decided to wait until the delivery of her newborn baby and collect the umbilical cord blood from her second child, Piotruś. Her intuition told her that it was worth taking the risk and the doctors supported her decision. The umbilical cord blood was deposited at the Polish Stem Cell Bank, the only family bank in Poland with experience in stem cell transplantation.
Everybody felt relieved when the tests showed that Miłosz could be the recipient of his brother's stem cells. The transplantation took place at the Clinic of Paediatric Oncology, Haematology and Transplantation of Poznan Medical University a month after Piotruś’ umbilical cord blood was collected. The boys’ parents had to wait another 30 days for the results of the leukocytes examination. To the great joy of everyone involved, the number was normal and Miłosz slowly started to recover.
Miłosz saw his younger brother soon after his birth, before the transplantation. They didn't have any contact for the first half year. The boys' parents worried how their sons would react when they saw each other the second time. However, the brotherly bond turned out to be stronger than separation. They hugged each other for some five minutes, as Grażyna recalls.
“My oldest son is a hero because he won his battle with the disease. He was very brave, and fought all the way. And my younger son gave his umbilical cord blood and saved the life of his brother,' says Grażyna.
Now, almost a year after transplantation, Miłosz can enjoy his childhood like any other child his age. He only has to visit hospital for control tests and maintain a special diet. However, it seems that the worst is behind him. His parents look to the future with hope.
The first umbilical cord blood stem cells transplantation in a child took place in Poland in 2000. Since then, some 20 procedures alike have been performed. Blood from the family bank, the Polish Stem Cell Bank (PBKM), was used for 8 of them, while the blood for the remaining procedures was acquired from public banks abroad. Umbilical cord blood stem cells have been used for transplantation in the world since 1988.