Bone Marrow Transplantation: What All Conditions Can Be Treated With It?

A BMT, or bone marrow/stem cell transplantation, is an advanced type of medical therapy used to treat various cancers and severe blood/immune system disorders. The soft, spongy area found in bones where certain blood cells (red blood cells, white blood cells and platelets) are produced is called bone marrow. When the bone marrow becomes damaged or stops producing healthy blood cells, it may result in a life-threatening condition.

Bone marrow transplantation replaces damaged or diseased bone marrow with a healthy population of stem cells. These stem cells can come from the patient's own body or a compatible donor. Once these healthy stem cells are transplanted into the bone marrow, they migrate there and begin producing new blood cells, restoring normal blood formation and immune function.

Why Do They Need Bone Marrow Transplants?

Bone marrow can no longer grow properly because of an illness or the treatment
Patients diagnosed with specific cancers must have high doses of chemotherapy or radiation therapy as part of their treatment plan (to hinder the creation of healthy blood cells from this area).
To replace diseased bone marrow with healthy/transplanted cells for patients with conditions like leukemia, aplastic anemia, or sickle cell disease.
Can also be used to rebuild the immune system, allowing an individual's body to fight cancer more effectively.
In some cases, chemotherapy and/or radiation treatment do not completely eliminate the cancer, and this may be why a patient is in need of a transplant, as it will help to restore the function of the patientâ€™s bone marrow following aggressive management of their cancer.
To correct certain inherited genetic disorders that impact blood production and/or metabolism.

Different Types of Bone Marrow Transplants

The type of bone marrow transplant a patient receives will depend on their disease, and what type of stem cells they are using (autologous, allogenic, umbilical), therefore, there are three main types of bone marrow transplants:

Autologous: Collection of the patientâ€™s own stem cells is referred to as an autologous transplant. Stem cells can be gathered by the patient in one of two ways - through either bone marrow collection or apheresis. The stem cells are frozen and stored until the patient gets treated with high-dose chemotherapy or total body irradiation.
Allogeneic: This type of transplantation is referred to as an allogeneic transplant. In this case, the patient receives stem cells from a compatible donor (this may be a family member or matched donor) and the stem cells can be obtained through bone marrow collection or peripheral blood stem cell collection. The patient receives cancer treatment and then the donor's stem cells are transplanted into the patient.
Umbilical: When a newborn is delivered, their umbilical cord is often used to aid 'cord blood' transplants, which involve using the blood from the umbilical cord to obtain stem cells (cells that are able to differentiate into almost any type of cell in the body) for transplantation into patients who require stem cells (typically after chemotherapy or radiation treatments).

Diseases Treated with Bone Marrow Transplant

Bone marrow transplant is used to treat several serious diseases that affect the blood, bone marrow, immune system, and metabolism. It helps replace damaged or diseased bone marrow with healthy stem cells so that the body can produce normal blood cells and function properly. Some of the major conditions treated with bone marrow transplant include:

Acute Myeloid Leukemia (AML) is a rapidly growing hematological malignancy that arises from the bone marrow and is characterized by the production of proliferating, dysfunctional white blood cells. Afterward, a bone marrow transplant can effectively restore the bodyâ€™s ability to create normal blood cells through the introduction of healthy stem cells.
Acute Lymphoblastic Leukemia (ALL) is a type of cancer that originates in the blood and bone marrow, and results from the rapid proliferation of abnormal lymphocytes that ultimately suppress the production of healthy blood cells. Following a bone marrow transplant, you can create a healthy ecosystem to produce blood cells.
Chronic Lymphocytic Leukemia (CLL) is a condition that leads to excessive numbers of dysfunctional white blood cells being produced in the bone marrow, thus impinging on normal blood cell production. An individual with CLL may benefit from a bone marrow transplant as a way to rebuild their bone marrow and create an environment conducive to the production of healthy blood cells.
Chronic Myelogenous Leukemia (CML) is considered to be a form of leukemia and is classified as a hematologic malignancy that is created due to the presence of a genetic mutation that leads to the production of an excessive number of white blood cells. Following this type of malignancy, a bone marrow transplant can be utilized to replace diseased bone marrow with healthy cells.
Non-Hodgkin's Lymphoma (NHL) is a group of cancers of the lymphatic system. These cancers arise from uncontrolled growth and multiplication of abnormal lymphocytes, and after chemotherapy, a bone marrow transplant can be performed to restore the number of blood-forming cells to normal levels.
Hodgkin's Lymphoma is considered to be a cancer of the lymphatic system, which causes an abnormal proliferation of lymphocytes and, in the event that the disease recurs following treatment with standard therapies, a bone marrow transplant may be performed to restore the patient to a normal state.
Severe Aplastic Anemia (SAA) is a type of condition where there is an inability for the bone marrow to produce enough of three important components: red blood cells (the type of blood cell that carries oxygen), white blood cells (the type of blood cell responsible for fighting infections), and platelets (the type of blood cell that allows blood to clot).
Severe Combined Immunodeficiency (SCID) is a group of inherited disorders that result in the immune system not working correctly. Bone marrow transplant can assist the patient in developing a healthy immune system.
Wiskott-Aldrich Syndrome (WAS) is also an inherited disorder that affects the immune system and the body's ability to form blood clots. A bone marrow transplant can fix this immune system defect.
Sickle Cell Disease (SCD) is an inherited blood disorder that causes red blood cells to change into a sickle shape and block off blood flow. A bone marrow transplant can replace defective blood-forming cells.
Krabbe Disease is a rare inherited genetic condition that affects the nervous system because of deficient enzyme activity to break down certain fat substances in the body. A bone marrow transplant might slow the progression of the disease.
Hurler Syndrome (MPS-IH) is a metabolic disease caused by the body's inability to break down certain complex sugars. A bone marrow transplant may alleviate some of the symptoms of the condition and stop the progression of the disease.
Adrenoleukodystrophy (ALD) is a genetic disorder of the brain and the adrenal glands because the body breaks down fats incorrectly, called fatty acids. A bone marrow transplant may help slow the neurological deterioration.

Bone Marrow Transplant Procedure

The first step consists of collecting the stem cells either from the donor or the recipient (the recipient will first have stem cells collected prior to treatment). To collect the bone marrow, a thin tube (called a transplant catheter) is placed in large vein (the Transplant Catheter will allow the medical team to collect and administer medications to the recipient while performing the transplant).
After the stem cells have been collected from the donor or the patient (Pre-Transplant), the patient will go through a Stage of Transplant Conditioning (transplant conditioning) (five to seven days of chemotherapy or radiation therapy) to kill off existing cancerous and/or damaged cells in the bone marrow, to make room for new transplant cells.
Once conditioning therapy is finished, healthy stem cells will be given back to the patient via the transplant catheter into the bloodstream. This is done in a similar way to a blood transfusion, and the entire process will take somewhere in the range of 30 minutes to 1 hour.
After surgery, patients will enter the recovery phase. While in recovery, they may receive medication (such as antibiotics) and support care (such as blood transfusions) to help them adjust to their new cells and decrease any risk of infection. The recovery phase typically lasts from 2 to 4 weeks, but it can take longer for patients to completely recover their immune system.

Conclusion

Bone marrow transplantation has been established as an important resource utilized by many patients with various blood-related illnesses, immune system disorders, and/or diseases of the bone marrow. Selecting an appropriate donor and providing the recipient with healthy stem cells will result in restoration of the proper production of normal blood cells; the establishment of an improved functioning immune system; and, ultimately, better survival rates for those individuals living with chronic or complex conditions.

Despite the fact that bone marrow transplants require careful planning and a period of recovery after the procedure, this treatment offers hope to individuals who cannot benefit from other forms of therapy. With appropriate medical management and support from knowledgeable professionals, individuals who undergo bone marrow transplants can expect to achieve significantly improved levels of physical well-being and achieve successful recoveries from serious hematologic and/or genetic disorders.