Bone Marrow or Stem Cell Transplant

Bone Marrow Transplants have successfully been used for many years to treat individuals with asymptomatic Adrenoleukodystrophy, with a Loes score of less than 8.

There are three types of transplant:

• Allogeneic transplant for genetic diseases uses cells from another person, or a donor, who does not have the disease
• A haploidentical transplant is a type of allogeneic transplant. It uses healthy, blood-forming cells from a half- matched donor to replace the unhealthy ones. The donor is typically a family member.
• An autologous transplant uses a patient’s own cells for the transplant, so no donor is required

A stem cell or bone marrow transplant is a long and complicated process that involves 5 main stages.

1. Tests and examinations – to assess your general level of health.
2. Harvesting – the process of obtaining the stem cells to be used in the transplant, either from you or a donor.
3. Conditioning – treatment to prepare your body for the transplant.
4. Transplanting the stem cells.
5. Recovery – you’ll need to stay in hospital for at least a few weeks until the transplant starts to take effect.

These stages are described in more detail below.

Before a stem cell transplant can be carried out, you’ll need a series of tests and examinations to ensure you’re healthy enough for the procedure to be carried out.

Transplants tend to be more successful in people who are in good general health, despite their underlying condition.

The tests you might have include:

• an electrocardiogram (ECG) – a simple test used to check your heart’s rhythm and electrical activity
• an echocardiogram – a scan used to look at the heart and nearby blood vessels
• an X-ray and/or computerised tomography (CT) scan to check the condition of organs such as the lungs and liver
• blood tests to check the level of blood cells and assess how well the liver and kidneys are working

There are 3 main ways stem cells can be harvested, these are:

• from blood – where the stem cells are removed from the donors blood using a special machine (see below)
• from bone marrow – where a procedure is carried out to remove a sample of bone marrow from the hip bone (see below)
• from cord blood – where donated blood from the placenta and umbilical cord of a new-born baby is used as the source of stem cells (find out more from the NHS Cord Blood Bank)

The most common way to harvest stem cells involves temporarily removing blood from the body, separating out the stem cells, and then returning the blood to the body.

To boost the number of stem cells in the blood, medication that stimulates their production will be given for about 4 days beforehand. On the fifth day, a blood test will be carried out to check there are enough circulating stem cells.

If there are enough cells, veins in each arm will be connected by tubes to a cell-separator machine. Blood is removed from one arm and passed through a filter, before being returned to the body through the other arm.

This procedure isn’t painful and is done while the donor is awake. It takes around 3 hours and may need to be repeated the next day if not enough cells are removed the first time.

An alternative method of collecting stem cells is to remove around a litre of bone marrow from the donors’ hip bone using a needle and syringe.

The needle may need to be inserted into several parts of your hip to ensure enough bone marrow is obtained. This is done under a general anaesthetic, so you’ll be asleep and won’t feel any pain while it’s carried out.

However, the area where the needle is inserted may be painful afterwards and you’ll have marks on your skin where the needles were inserted (usually one on each side).

Treatment with high doses of chemotherapy and sometimes radiotherapy will be needed before the stem cells can be transplanted to:

• destroy existing bone marrow cells – this is to make room for the transplanted tissue
• stop your immune system working – this reduces the risk of the transplant being rejected

As part of the conditioning treatment, you’ll be given a range of medicines, so a tube called a central line will usually be inserted into a large vein near your heart. This means medication can be passed into your body without the need for lots of injections.

The conditioning process usually lasts up to 10 days. You’ll probably need to stay in hospital throughout the treatment.

Conditioning can cause a number of unpleasant side effects, such as sickness, hair loss and tiredness. These are usually temporary. Your treatment team will discuss the risks of treatment with you beforehand.

The transplant will usually be carried out a day or 2 after conditioning has finished.

The stem cells will be passed slowly into your body through the central line. This process often takes around a couple of hours.

The transplant won’t be painful, and you’ll be awake throughout.

Once the transplant is finished, you’ll need to stay in hospital for a few weeks while you wait for the stem cells to settle in your bone marrow and start producing new blood cells.

During this period you may:

• feel weak, and you may experience vomiting, diarrhoea and/or a loss of appetite
• be given fluids by mouth or through a tube running from your nose to your stomach (a nasogastric tube) to prevent malnutrition
• have regular blood transfusions, as you’ll have a low number of red blood cells
• have regular platelet transfusions, as you’ll have a low number of platelets
• stay in a special germ-free room, and visitors may need to wear protective clothing to prevent infections, as you’ll have a low number of infection-fighting white blood cells

Many people are well enough to leave hospital between 1 and 3 months after the transplant. However, if you develop complications such as an infection, you may not be able to leave hospital for longer.

Even after going home, you’ll still be at risk of infections for potentially a year or 2 because it can take a while for your immune system to return to full strength.

If donated stem cells were transplanted, you’ll also usually need to take medicines that stop your immune system from working so strongly, to reduce the risk of your body attacking the transplanted cells (immunosuppressants), or to reduce the risk of the transplanted cells attacking other cells in your body.

Patients will be followed regularly after BMT to ensure that they remain healthy and to carry out routine checks. They will be followed for a number of years post-transplant.