Bone Marrow Transplant: Causes, Procedure, and Recovery

Bone marrow transplantation (also called hematopoietic stem cell transplantation) involves harvesting stem cells to rebuild a patient's bone marrow. The new stem cells take over the production of red blood cells.

In some cases, it's possible to harvest the patient's own bone marrow (this procedure is known as autologous transplantation). The bone marrow can be purified of any diseased cells before being re-implanted.

Undergoing a stem cell transplant can be a difficult experience. The medications used to prepare the body can cause unpleasant side effects, and there's a risk of complications, such as the body rejecting the new stem cells, or the new stem cells attacking the body (called "graft-versus-host disease"), as well as a risk of infection. For many people, it takes up to a year to fully recover from the procedure.

What is a Bone Marrow Transplant?

Hematopoietic stem cell transplantation, formerly called "bone marrow transplant", involves transplanting cells that are the origin of all blood corpuscles: red blood cells, white blood cells, and platelets.

The hematopoietic stem cell is an immature cell, the progenitor of all the basic components of the blood. It is capable of multiplying and simultaneously producing daughter cells which, through successive maturation processes, will give rise to the mature elements of the blood (white blood cells, red blood cells, and platelets).

Hematopoietic stem cells are found in the bone marrow and in umbilical cord blood at birth, but they can be mobilized and migrate into the blood of children and adults thanks to appropriate stimulation by special medications called growth factors.

Harvesting and Donor: How Does a Bone Marrow Donation Work?

The classic donation involves harvesting bone marrow blood from the posterior iliac crests (performed under general anesthesia in the operating room), requiring a short hospitalization (no more than 48 hours).

The second method is the donation of hematopoietic stem cells harvested from peripheral blood after stimulation by a granulocyte growth factor (medication). In this case, donation involves administering this medication in the 5 days preceding the harvest.

The growth factor has the ability to stimulate the transfer of hematopoietic stem cells from the bone marrow to the bloodstream, thus increasing the number of hematopoietic stem cells in the blood where they are harvested by apheresis. The blood, harvested from one arm, enters a sterile circuit into a centrifuge where the cellular component useful for transplantation is isolated and collected in a bag, while the rest of the blood is re-injected into the donor through the opposite arm.

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How is a Bone Marrow Transplant Performed?

The transplantation technique involves, as indicated above, harvesting HSCs from a donor (allogeneic transplantation) or from the patient themselves (autologous transplantation). The harvest, which is mainly done in the pelvis, can be easily performed using needles and syringes under general anesthesia.

Complications from this surgery are very rare and never serious. Once harvested and processed, it is re-injected into the recipient who has undergone maximum-dose radio-chemotherapy, potentially myeloablative, i.e., capable of eradicating the patient's bone marrow and, in the case of malignant diseases, also the residual disease cells (conditioning regimen). Conditioning irreversibly suppresses the patient's bone marrow and practically never allows spontaneous hematological recovery. The harvested cells are infused intravenously into the peripheral blood and repopulate the bone marrow, thus giving rise to a new hematopoietic and immune system.

HSC transplantation is therefore not a surgical operation. However, despite this apparent simplicity, it is a complex biological procedure, which can also present certain risks to the patient's life. Transplantation is not just an HSC infusion, but a long journey of weeks and months that will last even after the first discharge.

In the case of autografting, bone marrow or peripheral blood stem cells are harvested from the patient themselves during a remission phase of the leukemic disease and are then subjected to purification (selective removal of any residual malignant contaminating cells by various techniques), cryopreserved in liquid nitrogen, then re-injected after conditioning by radio-chemotherapy. Blood cells from a previously cryopreserved autologous umbilical cord can also be used. In this context, the re-infusion of HSCs aims to allow the administration of chemotherapeutic drugs at doses that would not be possible in the absence of a transplant, in order to restore a bone marrow function that would otherwise be irreversibly impaired.

Why Perform a Bone Marrow Transplant?

Hematopoietic stem cell transplantation is the treatment of choice for a whole series of diseases:

• Neoplastic and non-neoplastic hematological diseases;
• Solid tumors;
• Inborn errors of metabolism;
• Primary immunodeficiencies;

In children with certain genetic diseases, hematopoietic stem cell transplantation makes it possible to replace the child's missing or non-functional cells with healthy cells produced from hematopoietic stem cells harvested from a healthy donor.

Recovery: How to Live After a Bone Marrow Transplant?

The donor's hematopoietic stem cells reach the bone marrow spaces and begin to proliferate. This phase, called engraftment, occurs about 15 days after the transplant and is noticeable by the increase in the number of white blood cells and platelets and hemoglobin values in the blood.

However, the success of a hematopoietic stem cell transplant, especially if it is allogeneic, does not stop at engraftment; it is necessary for the infused cells to learn to live with the recipient's immune system. In general, the most critical phase is the first 100 days after transplantation.

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