Stem Cells in Treatment of Pediatric Diseases Homework Help
What is a stem cell?
Stem cells can be described as the special cells that are made in the bone marrow. A stem cell transplant is done to replace damaged stem cells with those that are healthy. Stem cell transplants become white blood cells, red blood cells, more stem cells, and platelets. A stem cell transplant can also be called a bone marrow transplant.
Children suffering from diseases like lymphoma and leukemia or those with bone marrow and blood diseases require stem cell transplants. Deciding to have a stem cell transplant is a serious decision that requires both the parent and the treatment team to contemplate and decide on.
Stem cell transplant procedures can be risky. The process is long and painful. Whether a stem cell transplant will be successful or not will depend on the general health of the child, the disease they are suffering from, and the age of the child.
Stem cell transplant is not always successful. However, the good thing is that when the procedure is successful, it increases the chances of curing the disease and the chances of remission.
A stem cell transplant can be described as the process of infusing healthy stem cells into the body to trigger the growth of new bone marrow. Stem cells increase a person’s chance of fighting an infection.
In children, stem cell transplants are done in those whose stem cells have not been destroyed by invasive treatments such as radiation therapy and chemotherapy or damaged by diseases.
Types of stem cell transplants can be divided into two:
- Autologous transplants – in this type of stem cell transplant, the doctors collect the patient’s stem cells, store them in a special laboratory, and will intravenously introduce them back into the system of the patient.
- Allogeneic transplants – in this type of stem cells transplant, doctors collect stem cells from a tissue-matched donor. This could be a sibling of the patient, umbilical cord blood, or an unrelated donor. The stem cell is then delivered into the patient’s system intravenously.
Allogeneic transplants are used to treat children diagnosed with the following illnesses:
- Acute leukemia
- Wiskot-Aldrich syndrome
- Amegakaryocytosis or congenital thrombocytopenia
- Paroxysmal nocturnal hemoglobinuria
- Aplastic anemia or refractory anemia
- Familial erythrophagocytosis lymphohistiocytosis
- Myelodysplastic syndrome of another myelodysplastic disorder
- Chronic lymphocytic leukemia
Autologous and allogeneic stem cell transplants are done to treat several cancerous and non-cancerous conditions. The process of stem cell transplant is complicated and can take several months to complete. The transplant coordinator, nurses, and doctors need to carefully review the information about the transplant at the consent meeting.
Are stem cell transplants safe for children?
Stem cell transplants can be done for patients who are as young as a few months old. However, several risks come with stem cell transplants. In a case where a child is suffering from a non-cancerous disease such as severe combined immunodeficiency, it is important to do the transplant as soon as possible. Children with this disease have a weaker immune system which increases their risk for infection.
For a child with inherited disorders, the doctors will need to watch the child for a few years to allow organs to mature before doing a stem cell transplant.
What are the allogenic stem cell donor options for pediatric patients?
Donated stem cells should match the cells of the child for them to be suitable. In some cases, siblings are the perfect donors although this is not the case for everybody. In cases where a sibling is not a suitable match, the doctor will look for an unrelated donor who matches the child’s cells.
In case the doctor or the transplant coordinator is not able to find a matched donor, an alternate donor is used. An alternate donor can be a haploidentical donation that is half-matched with a family member, a mismatched unrelated donor, or an umbilical cord blood donation.
Parents with children with conditions that require stem cell transplant and are in the process of expecting a baby should preserve the umbilical cord of the baby. This preservation of the umbilical cord is known as umbilical cord blood banking. The cord should be preserved so that if the cells of the siblings match, the cord can be used for transplant later.
Stem cells are extracted in 2 ways. One is peripheral withdrawal and the other is bone marrow harvesting. During peripheral withdrawal, the donor is given shots of growth factors to stimulate the production of stem cells. The cells are removed intravenously using apheresis. This process is similar to that of blood donation.
With bone marrow harvesting, surgery is done to remove stem cells from the pelvic bone. This process requires general anesthesia. Doctors decide on the best method of extraction depending on the overall health of the child and the physical size of the donor.
Special precautions should be taken if the donor is a child. It is important to be sure that the donor enters the process willingly. To help the donor child to understand the procedure, he or she should meet with a doctor, a child life specialist, an ethicist, and a psychologist.
There are numerous preliminary appointments that a child should go through once they are eligible for a stem cell transplantation. In addition to the appointments, there are several tests and blood work that has to be done. All these are scheduled by the stem cell transplant patient coordinator.
The stem cell transplant patient coordinator acts as the child’s liaison to the medical team. Parents will also be required to meet with a senior member of the transplant team to deeply discuss the child’s plan as well as to give consent by signing the consent forms.
If the child is to receive an autologous peripheral blood stem cell transplant, the doctor will collect the child’s stem cells before admitting them for transplant. The process of stem cell collection is coordinated by the stem cell transplant coordinator and the care provider.
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Stem Cell Transplant Process Timeline
There are several stages of stem cell transplant. Stem cell transplant is a complex process that may take several months. A great part of the treatment is done at the hospital while the rest is done, outpatient.
The duration of the treatment depends on how the child responds to treatment, the disease the child is suffering from, and any problems the child may have from the treatment.
During preparation, the child will require several appointments with the care and transplant team for several weeks before the day of the transplantation. During these appointments, a member of the transplant team or the primary transplant doctor will discuss with the parent the procedure including its benefits and risks.
During preparation, the doctor will:
- do blood tests, X-rays, and Other imaging scans
- stem cells from the child or the child’s donor will be collected and stored
- a central iv line may be inserted to give the child medications and chemotherapy. The child should be sedated during this procedure.
There are resources available that can help the child and their family to manage stress. Parents should talk to the nurses or doctors or social workers for more information on how they can get this help.
Several tests will be done to ensure the child is in good health to be able to handle radiation and chemotherapy because they are part of the process. This step may take days to several weeks.
Deciding on treatment
Both the doctor and the parent will go through the test results of the child. They will also discuss the emotional and physical challenges the child is likely to encounter. By the end of this step, the parent may opt to go on with the treatment or choose a different method of treatment.
Another type of stem cell transplant besides the two discussed above is the stem cells that come from the blood in the umbilical cord. In normal circumstances, the umbilical cord is thrown away after birth.
However, for this type of stem cell transplant, the blood is banked or saved to be used later. The blood in the cord may be from one or more donors. It may also come from the child if the blood of the cord was banked when the child was born.
Admission to the hospital
The child needs to go to the hospital 1-3 weeks before day 0 (the day of the transplant) to start their conditioning therapy. Conditioning helps to prepare the child’s immune system for the transplant. Conditioning includes radiation, chemotherapy, and immunotherapy.
Conditioning will depend on the medical needs of the child and the type of transplant they will be having. Conditioning therapy has numerous goals. Conditioning therapy involves high-dose radiation or chemotherapy or both that children go through before stem cell infusion.
The day the child receives the stem cells or the bone marrow is called day 0. Conditioning therapy is done before day 0. The goals of conditioning therapy include killing cancer cells in children with the malignant disorder and killing normal cells in the bone marrow cavities to make room for new bone marrow cells to grow.
In children who are receiving cells from another person, the goal of conditioning therapy is to suppress the immune system of the child so that the immune system does not reject the cells of the donor.
Radiation and chemotherapy are done to kill any bone marrow or cancer cells and to help prepare the body of the child for the new stem cells. This process takes 1 to 2 weeks. Possible side effects are poor appetite, mouth sores, nausea, and loss of hair. Side effects can last for weeks or months. Medication can be given to ease side effects.
The day of the transplant is known as day 0. On day 0, the child is given medication to assist the body to accept the new stem cells. Medications will be given through IV. The process is done while the child is awake and it is not painful.
The process is done in a hospital room in the presence of the transplant team members and care teams.
Once the damaged cells have been destroyed, the process of putting the healthy stem cells into the bloodstream of the child starts, a process known as an infusion. This process takes several hours. The infused cells travel through the blood to the child’s bone marrow where they start making healthy blood cells in 1-4 weeks.
After the transplant, the child may remain in the hospital for 4 weeks or longer. Radiation therapy and chemotherapy may destroy white blood cells in the child. Without white blood cells, the body is unable to fight infections.
If this happens, the doctor will give medication to protect the child from infection s as their body makes new white blood cells. It is necessary for visitors, caregivers, and family members to be extra cautious to protect the child from infection.
What to expect after a stem cell transplant?
A common side effect of stem cell transplant is a decrease in white blood cells, platelets, and red blood cells. Mucositis is common after radiation therapy and conditioning chemotherapy. Mucositis causes ulcers and irritation throughout the mouth and the digestive tract. Some patients may also have diarrhea, painful sores, and stomach discomfort. There are medications to help with such situations.
Another risk after a stem cell transplant is infection. During and after a stem cell transplant, a person is with no immune system to help their infections. Patients should be closely monitored to identify side effects before they escalate to become life-threatening. This is the reason why patients remain in the hospital for up to 6 weeks or more.
The goal of the caregivers before the child leaves the hospital is for them to start growing new stem cells before they leave the hospital. They would also want to ensure that the child is comfortable, that they can take sips of water, walk and sleep comfortably until their blood count is up. Once ready for discharge, they should go to the hospital for checkups 3-5 days a week.
After the stem cell transplant, the child will remain in the hospital for a month as the doctors look for any signs of engraftment. Engraftment is when the stem cells that were transplanted begin to make white blood cells enough to protect the child against infections.
The doctors will look for signs of engraftment by looking at the number of neutrophils and white blood cells. Neutrophils are a type of white blood cell that fight bacterial and fungi germs.
Going home from the hospital
After the transplant, the child may remain in the hospital for 4 weeks or more. At home, the child will need care throughout. The parent or the caregiver and the child may need to hang around the transplant center for the first 3 months after the transplant.
The year after the transplant, the child will need to see the doctor often for blood tests. The doctor may restrict activities such as being in public spaces or going to school until their concerns of the child developing an infection go away.
There may be a need for blood transfusions until the body of the child can make enough blood cells on its own. Children who receive stem cells from a donor will be checked for signs of the body rejecting the cells or signs of new cells attacking cells in the body.
If there is any sign of infection, it is good to talk to the doctor. After engraftment, children can be discharged after one month if there are no complications or infections.
After a stem cell transplant, follow-up care is very important for the treatment and safety of the child. It is important to ensure the child attends all their appointments. If the child develops any problems or is having signs of an infection, it is important to call their doctor immediately.
In addition, parents should know the results of tests done on their children and keep a list of all the medicine the child is taking. Parents who are considering a stem cell transplant for their child should ask for help from their child’s doctor or the care team to help them understand what it entails. They may also them with the right resources that can help them with the process at their own pace. This is because there is a lot to learn about stem cell transplants that can be overwhelming.
Scientists and doctors are currently researching kids with blood diseases. There is a new type of immunotherapy known as CAR T that is given like a stem cell transplant. CAR T is used to treat certain lymphomas and B-cell acute lymphoblastic leukemia.
However, the side effects of CAR T can be very severe. Guidelines for the management of side effects associated with CAR T are still being developed to enable more patients to receive CAR T cell therapy safely.
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