Aplastic anemia-what is the disease?
Aplastic anemia, or aplastic anemia, is a disease of the bone marrow. In its course, the production of blood cells is impaired, leading to so-called “pancytopenia”. The number of platelets, erythrocytes and white blood cells (leukocytes) is reduced. Aplastic anemia can be congenital or acquired. It can also be the result of various other diseases, such as viral diseases.
Symptoms of aplastic anemia include all symptoms that result from a deficiency of blood morphotic elements. Symptoms of an insufficient number of erythrocytes include shortness of breath, impaired concentration, weakness, dizziness or pale skin. Reduced white blood cell count carries immune disorders, susceptibility to infections or fever. Thrombocytopenia, on the other hand, can manifest as skin and mucosal bleeding, petechiae or bleeding gums, for example.
Treatment of aplastic anemia depends on the course and severity of the disease. In the case of severe symptoms, it is necessary to implement specialized hematological treatment. The treatments used include immunosuppressive treatment, as well as bone marrow transplantation.
One method is to treat aplastic anemia using the patient’s own stem cells, with the goal of stimulating the bone marrow to produce blood cells. Without the implementation of appropriate treatment, aplastic anemia is a fatal disease. Patients usually die from severe bacterial or fungal infections.
What is the story of Antek?
Antek was born as a healthy boy. Until the age of three, he developed normally.
The first symptoms of Antek’s illness that his parents noticed were immune disorders, prolonged fevers, and numerous infections. Every infection, even trivial ones for others, caused him high fever and severe symptoms. In addition, he developed weakness and a complete lack of energy.
During one of these infections, Antek was hospitalized. Laboratory tests performed there revealed leukopenia (a too-low number of white blood cells), thrombocytopenia, and anemia. Specialists diagnosed Antek with acquired aplastic anemia.
Given the severity of his disease, efforts began to find the most optimal treatment. One possibility was a bone marrow transplant from a related donor (allogeneic transplant), but Antek’s sister, a tissue match for him, was too young to be a donor. Another option was severe immunosuppression, but the effectiveness of this therapy is limited to only one-third of children.
The best option for Antek was to use his own stem cells, allowing for less aggressive immunosuppressive treatment, which would reduce potential complications. Doctors suggested to Antek’s parents that they use his own stem cells from umbilical cord blood, which had been collected at his birth, as part of the treatment.
After the initial phase of treatment, autologous stem cells were transplanted into Antek using lighter immunosuppression.
Effects of Antek’s treatment
The boy was three years old at the time of his diagnosis with aplastic anemia. Upon admission to the hospital, he had virtually no immunity and was highly susceptible to infections. He easily caught all sorts of illnesses that were harmless to other children, such as ear infections.
When doctors suggested a treatment method using his own stem cells, Antek’s parents did not hesitate, determined to help their child regain full health. Just 39 days after the transplantation of his own stem cells, the number of leukocytes in his blood began to increase. “The very fact that the bone marrow started to work, that the stem cells, that this transplant helped Antoś, was an incredible joy for us,” said Mrs. Małgosia, Antek’s mother.
Bone marrow biopsies taken after the transplant showed normal function of Antek’s bone marrow and production of blood cells. Twenty months after the transplant, no abnormalities were found in his blood count results. Antek is now developing normally; he is an active child, full of energy and strength.
The stem cells from the umbilical cord blood taken at his birth saved his life. Without the implementation of this treatment, even a trivial infection could have been fatal for him.