What to Expect From Hematopoietic Stem Cell Transplantation

A Treatment Intended to Cure Certain Blood and Immune Diseases

Hematopoietic stem cell transplantation is a procedure used to treat certain types of blood cancer and immune disorders. Hematopoietic stem cells produce red blood cells, white blood cells, and platelets, primarily in the bone marrow.

If you are having this procedure, you would need to have a conditioning regimen before the transplant. The conditioning regimen is also described as myeloablation or myeloablative therapy. It includes radiation therapy, which uses beams of intense energy, and/or powerful chemotherapy medications to suppress the immune cells in your bone marrow.

After the conditioning regimen is completed, you would have the transplant, which can be from either your own stem cells collected before the conditioning regimen or from a donor.

Hematopoietic stem cell transplantation is an intravenous infusion

Getty Images / Jim Craigmyle

Reasons for a Hematopoietic Stem Cell Transplant

This procedure is done to provide you with new immune cells or red blood cells. It can be used for treating blood cancer. It is also sometimes used to treat autoimmune disorders, in which the body’s immune system attacks its own tissue. Additionally, it can be used to treat certain blood cell disorders.

Conditions that can be treated with hematopoietic stem cell transplantation include, but are not limited to:

  • Lymphoma: A group of blood cancers
  • Leukemia: A group of blood cancers
  • Multiple myeloma: A type of blood cancer
  • Lupus: A chronic autoimmune disease in which a person's immune system attacks its own tissues
  • Aplastic anemia: A condition in which the bone marrow stops producing blood cells
  • Immune deficiency after treatment with chemotherapy or radiation for cancer
  • Sickle cell disease: An inherited disorder of hemoglobin, the oxygen-carrying molecule in red blood cells
  • Multiple sclerosis: An autoimmune disease in which a person's immune system attacks the sheath covering their nerve cells
  • Stiff person syndrome: A rare nervous system disorder leading to muscle stiffness, which may be autoimmune
  • Chronic inflammatory demyelinating polyneuropathy: A chronic inflammatory condition that damages the sheath covering the nerve cells
  • Thalassemia: A group of inherited conditions that affect hemoglobin production
  • Polycythemia vera: A blood disorder in which too many red blood cells are produced

Sometimes these conditions can be treated effectively with approaches besides hematopoietic stem cell transplantation, and sometimes the transplant is considered a promising option. You and your doctor will have to discuss the potential risks and benefits of the procedure in your specific situation.

Who Is Not a Good Candidate?

Hematopoietic stem cell transplantation can be a curative treatment for many people. However, it isn’t for everyone.

Some factors that can determine whether hematopoietic stem cell transplantation is right for you include:

  • This procedure may have upper age limits, when it is not expected to prolong survival beyond your naturally expected lifetime without the transplant.
  • The disease that you are being treated for should be responsive to the conditioning regimen.
  • The procedure involves substantial medical treatment before and after the transplant. You might not be able to tolerate it if you have severe heart, lung, or kidney disease. 
  • You cannot have an active infection at the time of myeloablation or at the time of the transplant.

You and your doctor will also discuss factors such as the severity of your cancer and whether you are expected to have a better quality of life or improved life expectancy after the transplant.

Types of Hematopoietic Stem Cell Transplant

There are two main categories of hematopoietic stem cell transplantation. These are autologous (using your own stem cells) or allogeneic (using donor cells).

Stem cells are cells in the body that have not yet differentiated. This means that they can still become a different type of cell, such as an immune cell. These cells are collected from blood, bone marrow, or a newborn’s umbilical blood.

Some conditions can be treated with your own stem cells, whole others require donor stem cells, the difference being the following:

  • The advantage of an autologous transplant is that your body will not reject its own stem cells. However, if you have a disease that might be affecting your stem cells, this treatment may not be beneficial as your stem cells would continue the disease.
  • With donor cells, you would have an infusion of cells that should be free of the disease you are being treated for. There is a risk that your body may reject the donor transplant and/or that the transplant cells could reject your cells (graft-vs.-host disease).

Donor-Recipient Selection Process 

If you are having an autologous hematopoietic stem cell transplant, you should not have to wait for a transplant donor to become available because you will receive your own cells.

If you are receiving a transplant from a donor, the limiting factor is finding a good match. Stem cell donors are more available than organ transplant donors (such as heart transplant or liver transplant donors).

Types of Donors

If you are receiving a hematopoietic stem cell transplant from a donor, you will receive the cells from a donor who is alive. Stem cell donation is not harmful to donors' health, and their body will be able to replace their donated stem cells.

Allogeneic donor types include:

  • Related donors (including an identical twin sibling)
  • Unrelated donors
  • Umbilical cord cells

A key component in donor matching is finding a donor who has a matching human leukocyte antigen (HLA) type. These are proteins that you have on the surface of your body’s cells.

Unmatched HLA proteins can cause an immune reaction that leads to problems, such as graft vs. host disease (GvHD) or transplant failure. Generally, people who are related to each other are more likely to have an HLA match.

Before the Transplant

If you are having an autologous transplant, your own stem cells will be harvested. These can be taken from your blood or your bone marrow.

The process of having cells harvested from your bone marrow involves the insertion of a needle into your bone. This can be painful, and you might experience soreness in the area for a few days after the procedure. The cells will be stored until you can receive them during your transplant.

If you have an allogeneic stem cell transplant, your donor will provide the cells either through a blood sample or from their bone marrow.

Before an allogeneic or autologous hematopoietic stem cell transplant, you will undergo a conditioning regimen that includes chemotherapy and/or radiation therapy. You may have blood tests or other tests during this time to monitor your response to the conditioning regimen.

Weakened Immune System

It’s important to be aware that you can have a weakened immune system as a result of myeloablation.

Some conditions treated with hematopoietic stem cell transplantation can also weaken the immune system, and your immune system is likely to be more severely suppressed during the myeloablative phase of your treatment.

This means that your doctor may tell you to avoid certain foods, public places, or contact with anyone who could expose you to an infection.

Transplant Process 

Your transplanted cells are infused into your blood with an intravenous (IV, through a vein) catheter. Usually, a central venous catheter is used. This is a large catheter—larger than an IV that is typically placed in the arm. You may have a central venous catheter surgically placed in your chest.  

Your transplanted cells will be infused into the catheter. The process may take several hours. Your medical team will monitor your vital signs during the infusion, including your blood pressure, heart rate, and breathing rate. 

Complications

During or shortly after your transplant procedure, you might feel weak, dizzy, or short of breath. You might fall asleep, but you can stay awake throughout the infusion. Make sure you tell your medical team about any symptoms you are experiencing.

After Transplant

You will need to have blood tests to assess your health and recovery after your transplant. You can experience tiredness as you recover.

The newly transplanted cells should undergo a process of engraftment, in which they take over cell production in your bone marrow to produce new, healthy cells. This can take weeks or months. You will need to avoid exposure to infections during this time.

If you have any symptoms, such as fever, changes in your urine, pain, discomfort, nausea, vomiting, or feeling sick as you are recovering, tell your medical team.

Prognosis

Generally, a hematopoietic stem cell transplant has a good outcome. You will need to regularly follow up with your doctor, who will assess your condition.

Sometimes the transplant fails, and the new stem cells are rejected by the body. Additionally, acute or chronic graft-vs.-host disease can develop. And some people may have a recurrence of the condition after a period of time.

Support and Coping

If you have had or are going to have hematopoietic stem cell transplantation, you will need to understand all of the issues involved with the procedure, as well as the process before the procedure and after you’ve had it. You can experience substantial improvement in the condition that is being treated.

You might feel a sense of anxiety or depression due to uncertainty or because of how much your condition is affecting your life. It is completely normal for you to have a range of feelings.

Ask your medical team about resources you can use as you cope and adjust. These can include counseling, therapy, or a support group. You can select the type of resources that you feel will work best for you—and you might use different resources throughout your recovery, depending on your personal needs.

Summary

A hematopoietic stem cell transplant is done to treat some types of blood cancers and immune disorders. Donors can be the recipient themselves, or a donor with a matching HLA type may volunteer. The stem cells can be harvested from the peripheral blood, bone marrow, or newborn umbilical blood.

The recipient undergoes a conditioning regimen to clear their bone marrow of hematopoietic stem cells. Then they receive the transplant as an infusion. After the transplant, the cells migrate to the bone marrow and produce new blood cells and immune cells.

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