Little Wiktoria saves her older brother's life, her umbilical cord blood is the alternative to bone marrowMoreFor more than 2 years, seven year old Kacper struggled with aplastic anaemia – severe bone marrow dysfunction. The only chance to save him was by a transplantation but unfortunately, the donor retracted at the last moment. However, rescue came from his younger sister. Her umbilical cord blood had been collected and then transplanted to Kacper. This procedure was the 9th family umbilical cord blood stem cells transplantation in Poland.
Bone marrow aplasia is a disease caused by a disorder of blood stem cells, where the rate of division and differentiation is decreased. This results in bone marrow suppression and consequently, in pancytopenia. Pancytopenia is a decrease in the number of all types of blood cells (red blood cells, white blood cells and platelets) and results in bone marrow atrophy. This is a relatively rare disease and can occur in people of all age groups. Within every population size, comparable to Poland’s (ca. 38.5 million), several dozen cases of this disease are reported every year.
Kacper's case history started in November 2011, when he was 5 years old. He started to wake up at night with strong knee pain. Initially, there were no clues that this can be a symptom of the disease. However, when these incidents started to occur more often, the boy's parents decided to consult their GP. The blood count test results surprised not only Kacper's parents but also his doctors. It turned out that Kacper was suffering from platelet deficiency (thrombocytopenia). The doctors suspected the worst - leukaemia. 'Our whole life fell to pieces at once like a house of cards,' recalls Kacper's dad.
Kacper was admitted to hospital in Częstochowa and was transferred later that very same day to Katowice, where they performed a biopsy. The results of the first examination were promising, doctors excluded cancer and leukaemia. Kacper's parents were relieved, but unfortunately their joy didn't last long. Unexpectedly, the results of each subsequent test dramatically worsened. The doctors decided to perform a bone biopsy and it turned out that his bone marrow was empty. After a long period of uncertainty, the doctors made the final diagnosis. “Kacper suffered from severe aplastic anaemia and needed numerous blood and platelet transfusions. He also had a low level of granulocytes making him more at risk to bacteria and fungi infections.” stated Prof. Krzysztof Kałwak from the Clinic of Paediatric Oncology, Haematology and Bone Marrow Transplantation in Wrocław.
Only a bone marrow transplant could save him. The countdown to save his life from the disease had started. Kacper's parents and brother were examined to see if they qualified as bone marrow donors. Sadly, his parents were only a fifty percent genetic match and his younger brother, Mikołaj, had only 7 out of 10 matching antigens. Kacper’s family members were excluded as bone marrow donors as the required compliance for transplantation is at
least 9/10 matching antigens. Unfortunately, Mikołaj's parents hadn’t decided on the collection and storage of umbilical cord blood at delivery, which could have been used to save Kacper’s life. Now they could only wait until a quality donor was found. The months went by, until finally in November 2012, the phone rang.
'We were informed that a donor had been found,' says Kacper's dad. 'They started to prepare my son for the transplantation. They pulled out his bad teeth and inserted a special catheter that allows the collection and infusion of liquids, medicines and blood without the need of inserting additional cannulas,' he adds.
The transplantation was scheduled for 27th November 2012 at the Clinic of Paediatric Oncology, Haematology and Bone Marrow Transplantation in Wrocław. However, ten days before the transplantation was to go ahead, a doctor from the hospital phoned and Kacper's parents heard the news they would never have expected, the donor had retracted at the last moment. They would never learn why. 'We were so happy that all this would soon be over, that everything would be good at last. Instead we were only disappointed, both ourselves and Kacper. He had hoped everything would soon be over, that he wouldn't have to have transfusions and injections any more, that his suffering would end soon. Suddenly, the situation had changed.' describes Kacper's mum . The struggle for Kacper's life had started again. 'We had to fight very hard to remain positive, because it wouldn't have helped anything,' says Kacper's dad.
The prognosis wasn't good – both the number of platelets and haemoglobin levels kept falling. But there was a glimmer of hope. Kacper's parents were expecting the birth of their new baby. 'We learned from the head of the ward at the clinic, that there was a possibility to collect umbilical cord blood and transplant it to Kacper instead of the bone marrow of an unrelated donor,' says Kacper's dad. Umbilical cord blood stem cells are more valuable than stem cells extracted from other sources (e.g. bone marrow) as they are primitive, unspecialised and multipotent. They are extremely valuable due to their ability to self-renew and transform into other cells which the human body is made up of. 'Umbilical cord blood stem cells ensure a significantly better survival rate in children with metabolic diseases than e.g. stem cells from peripheral blood or bone marrow,' says Prof. Kałwak.
In January 2013 Wiktoria was born. Stem cells extracted from her umbilical cord blood were delivered to the laboratory of the Polish Stem Cell Bank (PBKM). The parents were looking forward to learn the results of the specialised tests. Everybody was relieved when it turned out that the blood collected at their daughter's delivery was compliant with Kacper's. Three weeks later the transplantation was performed. Everything went well, the boy was conscious for most of the time. The results of the examination carried out 30 days later, confirmed that the transplant had been accepted and the number of leucocytes was normal. 'We called our daughter Wiktoria (Victory), to honour the fact, that there was a full compliance of stem cells. We thought of it as a gift from God,' recalls Kacper's mum . After the transplantation, Kacper started to recover and could be discharged from hospital. Now he is at home, with his family. In January he went back to school and saw his friends, whom he had missed so much.
'We are glad that Kacper's parents entrusted PBKM with Wiktoria's precious blood. There is nothing more satisfying in our work than helping to save a child's life,' says Dr Tomasz Baran of The Polish Stem Cell Bank. Over 35,000 umbilical cord blood transplantations in children and adults have been carried out in the world. In Poland, so far dozens of transplantations, mainly in children, have been performed. As much as 10 of them used blood collected and stored by the Polish Stem Cell Bank; the rest were used with blood from foreign public banks. In September 2012, the European Parliament adopted a resolution on voluntary and unpaid donation of tissues and cells (among others, from umbilical cord blood). Therefore, emphasising the need to establish both public and family umbilical cord blood banks in every member state.
A story of 12-year-old Dominika cured thanks to her brother’s umbilical cord bloodMoreFrom the day of her birth Dominika had problems with her health. The day after she was born she had the first operation connected with the incorrect structure of her alimentary tract. Then a cardiac effect was diagnosed. When she was three she suffered from continuous vomiting and diarrhoea. After some tests, the doctors diagnosed her with thrombocytopenia – a disease which causes a lack of blood platelets. For a few years she had to have transfusions, to begin with, just once a month, then later once every two weeks. In the summer her condition usually got worse, her organ resistance decreased and the number of blood platelets reduced. Then doctors made punctures, which luckily did not show any abnormality that would be a sign of a tumour.
Dominika developed normally, she could play with other children, go to school and devote time to her hobbies, such as horse riding. Nevertheless, the older she got, the longer her absences at school were. Doctors were afraid that she might get seriously ill. The danger increased when Dominika’s medical test results got worse. Mrs Dorota, the girl’s mother, decided to look for help with other specialists. The analysis of medical tests in Warsaw showed that Dominika was suffering from Franconi anaemia. If she were to be diagnosed with this disease, it would mean that she would be at high risk of tumour development.
The diagnosis was confirmed by more tests and soon a search for a bone marrow donor began. Over the course of a year and a half a few potential donors were found, but each time a tissue compatibility test was done, it turned out that the donors were not suitable. During that time, Dominika’s parents decided to have another child. One day, as a heavily pregnant Mrs Dorota was visiting her sick child in hospital, she came across a leaflet from the Polish Stem Cell Bank (PBKM) which collects and stores umbilical cord blood. Mrs Dorota had never heard about such a possibility before. She made an appointment with the Bank’s consultant, who explained the whole process and clarified any doubts she had. After her meeting with the consultant, Mrs Dorota decided to have her child’s umbilical cord blood collected.
Although a suitable donor was found before the birth of Dominika’s sibling, the doctors decided to wait before making the decision for the transplant. They wanted to check if the umbilical cord blood collected during the birth would be compatible with Dominika’s blood. The specialists claim that a sibling’s blood is better for a transplant than a non-relative’s blood because the risk of the transplant rejection, as well as complications, is much smaller.
In January 2010 Kacper was born. Everybody was relieved when it turned out that the stem cells collected from his umbilical cord blood could be transplanted to Dominika. The doctors decided to wait one more year before transplanting her brother’s umbilical cord blood and bone marrow, as Dominika was just 11 years old at the time. With admirable patience and resistance, Dominika waited for the transplantation. Mrs Dorota recalls her daughter’s brave decision to shave her head and get rid of her precious hair. Up to then, Dominika only allowed a few centimetres of her hair to be cut but just before the transplant she did not protest at all. She knew it was necessary and she accepted shaving her head with dignity.
In accordance with the procedures connected with the transplant, Dominika was treated with intensive chemotherapy. The aim of this was to destroy diseased bone marrow cells and consequently, destroy her immune system. She had to stay in a sterile isolation ward, where only doctors and her closest family members could enter, having maintained the highest safety standards. They had to wear special masks, aprons and protectors as any kind of infection could put her life in danger.
One year after Kacper was born, the transplant was performed in the Clinic of Paediatric Bone Marrow Transplantation, Oncology and Haematology at Wrocław Medical University. It was a big event for the hospital staff. A lot of employees were interested in Dominika’s case because transplants of umbilical cord blood stem cells are not a method of treatment often used in Poland, although in other Western countries it is a routine medical procedure. The reason for this is due to the small number of stored umbilical cord blood units in Poland.
One and a half months after the transplant, Dominika’s medical test results were so good that she could leave hospital. Doctors often affirm that going back home is a factor that often increases the motivation to fight a disease and can speed up the recovery process. This was exactly what happened in Dominika’s case. Day after day, the effects of the treatment were more and more satisfactory and she could get back to her normal life, she could visit a friend or go out. At the moment Dominika feels excellent and she can enjoy activities like riding a bike, roller-skating and going to the swimming pool. Her time of illness has come to an end and the time of making her wishes come true has begun.
Real brotherhood of blood - the story of the seventh successful family stem cells transplantation in polandMoreIn this picture, Miłosz is a 3 year old, vigorous, smiling boy, always willing to help the others. When you look at him, it's hard to believe that not so long ago he had to overcome the severe oncological disease known as histiocytosis. In his struggle he was helped by his younger brother whose umbilical cord blood stem cells were transplanted to Miłosz. This was the seventh family transplantation of umbilical cord blood stem cells performed in Poland.
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.