Bone marrow transplantation (hematopoietic stem cell transplantation, HSCT) is a medical procedure in which diseased or damaged bone marrow cells are replaced with healthy stem cells. Transplantation is used to treat children with various conditions, including leukemia, aplastic anemia, immunodeficiency disorders, certain inherited metabolic diseases, and some forms of lymphoma.
This procedure can be life-saving, especially for children with severe blood disorders that do not respond to standard therapies.
Types of bone marrow transplantation
- Autologous transplantation. Stem cells are collected from the patient, frozen, and later returned after chemotherapy or radiation therapy. This type of transplantation is used less frequently in children, as it is generally less effective for malignant diseases.
- Allogeneic transplantation. Stem cells are obtained from a donor, who may be a related donor (such as a sibling) or an unrelated donor identified through a donor registry. Donor selection is based on HLA (human leukocyte antigen) compatibility. Allogeneic transplantation is more commonly used for oncologic and inherited diseases.
Indications for bone marrow transplantation
Oncologic diseases — acute lymphoblastic leukemia (ALL) and acute myeloid leukemia (AML), particularly in cases of relapse or resistance to standard therapy.
Lymphomas (in complex or refractory cases).
Inherited disorders — aplastic anemia, sickle cell disease, and severe forms of thalassemia.
Immunodeficiency disorders — severe combined immunodeficiency (SCID), in which the child’s immune system cannot effectively fight infections.
Metabolic diseases — inherited disorders of metabolism that cause severe systemic dysfunction.
Preparation for bone marrow transplantation
- Donor search. In allogeneic transplantation, compatibility testing is performed with potential donors, including siblings. If no suitable related donor is found, a search is conducted in international donor registries.
- Pre-transplant conditioning therapy. Before transplantation, the child receives intensive chemotherapy and/or radiation therapy to destroy malignant cells, suppress the immune system, and create space in the bone marrow for new stem cells. This also helps prevent rejection of donor cells.
- Medical evaluation. A comprehensive medical assessment is performed to ensure that the child is fit for transplantation. Heart, lung, kidney, and other organ functions are carefully evaluated.
Bone marrow transplantation procedure
- Stem cell infusion. Stem cells are administered intravenously, similar to a blood transfusion. The procedure is painless and lasts several hours. After infusion, the stem cells migrate to the bone marrow and begin producing new blood cells.
- Recovery period. After transplantation, the child remains hospitalized for several weeks or months. During this time, doctors monitor engraftment and manage potential complications. The immune system is severely weakened, so strict infection control and protective isolation are required.
Recovery after transplantation
- Immune system recovery. Full immune reconstitution may take several months to a year. During this period, the child must avoid exposure to infections and follow strict hygiene measures.
- Diet and nutrition. A special diet may be required to reduce infection risk and support recovery. A dietitian helps develop an individualized nutrition plan.
- Physical activity. Rehabilitation may include physiotherapy to maintain physical strength and restore mobility.
Long-term follow-up
- Regular medical check-ups. The child will need ongoing follow-up visits to monitor overall health, organ function, and potential late complications.
- Vaccination. After transplantation, revaccination is required because the immune system has been “reset” and no longer retains immunity from previous vaccinations.
Bone marrow transplantation in children is a complex and potentially life-saving procedure that offers a chance for cure in severe diseases. Treatment requires coordinated work by a multidisciplinary medical team, including oncologists, hematologists, immunologists, surgeons, and psychologists. Recovery after transplantation can be prolonged and demanding for both the child and the family. However, with appropriate medical care, support, and follow-up, many children are able to regain their health and return to a full and active life.
