Therapy for aplastic anemia in children is customized to the individual and might include some or all of the following options:
- Hospitalization for severe symptoms
- Blood transfusions
- Immune suppressive therapies
- Growth factors to stimulate blood cell production
- Antibiotics to prevent or treat infections
- Hematopoietic Cell Transplant (also called bone marrow transplant or stem cell transplant or cord blood transplant)
Which of these options are considered and their timing depends on:
- Severity of the bone marrow failure symptoms
- Cause of the aplastic anemia:
- unknown or “idiopathic” (the most common type)
- underlying inherited condition (needs to be ruled out by specialized testing)
- viral illness
- exposure to toxins, radiation, or certain medications
- Availability of a suitable bone marrow donor
- Consultation between your child’s hematologist and one of our Hutchinson Center Transplant doctors.
In severe aplastic anemia, extremely low blood cell counts may be life-threatening and requires immediate hospitalization for supportive treatment, which usually includes blood transfusions and antibiotics. In certain situations, your hematologist may recommend that your child proceed as quickly as possible towards a bone marrow transplant because replacing diseased bone marrow with healthy bone marrow from a donor often successfully cures aplastic anemia.
However, the option of proceeding quickly to bone marrow transplant relies on there being a brother or sister who is a match and is able to donate bone marrow. If a matched brother or sister is not available the alternative is to treat the child with immune suppressive therapies and growth factors.
Having a blood transfusion refers to a patient receiving blood cells from a donor. Transfusions help insure that your anemic child’s blood cell count stays at a safe level. Platelet transfusions reduce the risk of bleeding problems caused when platelet counts are too low. Red blood cell transfusions reduce symptoms of fatigues.
However, having too many blood transfusions means that iron from red blood cell transfusions can build up in the body and cause organ damage, or, the immune system may develop antibodies that attack the transfused cells. Therefore, blood transfusions are used as sparingly as possible. This is particularly the case if the child is to receive a bone marrow transplant.
Because aplastic anemia is most often the result of the patient’s own immune system working against itself, immunosuppressive drugs are often given to try to induce the failing marrow to revert to normal cell production. If a child must wait for a stem cell transplant they will be maintained in the meantime on immunosuppressive medication. If no suitable donor can be identified, then the child may stay on these medications for a longer course, and sometimes indefinitely.
The decision to continue immunosuppressive medication that is not working very well can be complicated when the alternative involves a bone marrow transplant from a donor other than a matched brother or sister. In this more complex situation, detailed counsel will be encouraged with your child’s hematologist and a pediatric transplant doctor at Hutchinson Center.
Growth factors are medications that help the body make more blood cells, which may be given to reduce the number or need for blood transfusions. Given with immunosuppressive therapy or after a bone marrow transplant, growth factors can help blood cell production to relieve the symptoms of aplastic anemia.
Having aplastic anemia leaves a person without enough white blood cells which makes them susceptible to all kinds of infections. White blood cells cannot be effectively replaced with a transfusion and their numbers cannot always be boosted with growth factors, so antibiotics may be administered to prevent certain types of infection, or when fever or infection appear.
Bone marrow transplantation
Fred Hutchinson Cancer Research Center physicians pioneered bone marrow transplants for aplastic anemia treatment. If bone-marrow transplantation is indicated, children will be treated by Hutchinson Center doctors who work at Seattle Children's and are world leaders in bone-marrow transplantation. When patients have the opportunity to use a matched sibling’s stem cells or bone marrow, there is a 90 percent success rate when circumstances are optimized. When the first choice of a matched brother or sister is not available Hutchinson Center has been able to achieve a 75 to 85 percent success rate when a matched unrelated donor is available and a modified preparation for transplant is used.
Marrow transplants for children with aplastic anemia are most successful when:
- Matched siblings are the donors
- Patients are under 20 years of age and early after diagnosis
- Patients have had minimal prior (irradiated) transfusions
- Marrow is used instead of stem cells or cord blood
- Preparation for a transplant from a sibling is with cyclophosphamide and antithymocyte globulin (ATG)
- Preparation for a transplant from a matched unrelated donor is with a reduced intensity approach that uses a very low dose of total body irradiation, chemotherapy and ATG.
A consultation with a transplant doctor at the Hutchinson Center doctor will be able to address in further detail the process and risks involved in bone marrow transplantation for aplastic anemia versus the alternative approaches of continued immunosuppressive therapy and supportive care.