Shannon Bradshaw Trust


What is aplastic anaemia?

Aplastic anaemia is a rare blood disorder, which causes blood cell production to break down so that not enough important blood cells are made.


Anaemia is a condition where there are too few red blood cells, which are needed to carry oxygen around the body. Although anaemia can occur on its own, it usually develops as a symptom of many other disease, including blood cancers.

Aplastic anaemia, which is being discussed here, is a much more serious condition that develops, independent of other blood disorders, during a patient’s life.

Developing aplastic anaemia

Aplastic anaemia develops in the bone marrow – the soft tissue in the middle of our bones where all our blood cells are made. Mother cells, called stem cells reside here and make every type of blood cell that we need.

Stem cells constantly produce new blood cells to replace old and damaged ones. New blood cells only leave the bone marrow once they have fully matured. This process is well controlled so that we have just the right amount of each blood cell in our body.

When someone has aplastic anaemia blood cell production breaks down. The body is therefore nolonger able to make sufficient numbers of blood cells. This usually happens because cells in the immune system begin to attack and destroy stem cells in the bone marrow. The spaces this leaves in the bone marrow are then filled up with fat cells.

Aplastic anaemia is not a blood cancer, which is the over production of abnormal, or cancerous, blood cells. Rather, it is a lack of healthy blood cells, but this in itself leads to serious problems

There are no specific symptoms of aplastic anaemia and it can often be confused with other common illnesses.

The onset of aplastic anaemia is gradual, and many patients experience mild or even no symptoms at first. In fact most patients are diagnosed while having blood tests done for other illnesses.

The common symptoms of aplastic anaemia include:

  • Tiredness and breathlessness
  • Paleness
  • Frequent and persistent infections
  • Unusual bleeding and bruising
  • These are caused by a lack of healthy red and white cells and platelets in the blood. People with aplastic anaemia may experience all, or just some, of these symptoms.

Some forms of aplastic anaemia are inherited, but these are very rare. This information relates to aplastic anaemia that develops during a patient’s life – called acquired aplastic anaemia.

It is thought that most cases of aplastic anaemia are caused by an auto-immune reaction. This is when the cells of the immune system become confused and begin to attack the person’s healthy tissue, particularly the stem cells in the bone marrow . There does not appear to be any underlying trigger behind the onset of this auto-immunity, in the majority of cases.

Factors that are thought to contribute to the development of aplastic anaemia are:

  • Some medicinal drugs
  • Exposure to some chemicals
  • Viruses
  • However, there is no proven link in the majority of cases and developing aplastic anaemia from any of the above is very rare.

Treatment for Aplastic Anaemia:

Blood transfusions, which boost the number of blood cells, are the most important part of treatment for some patients with aplastic anaemia, who have a very low red blood cell count.

Supportive care, which helps with the prevention and relief of infections and treatment complications, is major part of treatment for nearly all patients with aplastic anaemia. These complications include:

Anaemia, for which patients are given blood transfusions and growth factors to boost the number of healthy red cells in the blood.

Thrombocytopenia, which causes excessive bleeding, is treated with transfusion of platelets.
Infections, which are caused by too few white blood cells are a dangerous complication for patients with aplastic anaemia. Immediate courses of antibiotics and occasionally short stays in hospital are used to control these.

These patients are monitored closely in process called ‘watch and wait’ where they are given regular check-ups to look for any progress in their illness.

Definitive therapy is used to describe the main, or first line, treatment, which aims to cure patients rather than just controlling their symptoms. When possible, this includes a stem cell transplant. If patients are not able to have a transplant, drugs that stop the immune system from damaging the stem cells, are used instead. This is called immunosuppressive therapy. Most patients will need some form of definite therapy, which should be started as soon as possible after diagnosis.

Stem cell transplant is an intensive form of treatment that is only feasible for patients who are in relatively good health. This is therefore only recommended to patients who have severe aplastic anaemia, and to children who tend to suffer less severe side effects and generally do better after treatment. Patients have transplants using stem cells from a donor, called an allogeneic transplant. This also means that the immune cells responsible for destroying the stem cells in the first places, are replaced with harmless immune cells.

Specific Treatments:

Drugs called antilymphocyte or antithymocyte globulins (ALG or ATG) are used to treat patients receiving immunosuppressive therapy. These work by attacking and destroying the immune cells responsible for damaging the bone marrow.

This treatment is given to patients in hospital because it suppresses normal defences and patients need careful nursing to protect against infection. Alongside this, most patients will also receive another immunosuppressant drug called cyclosporine for up to six months

Side effects:

It is very common for patients to experience side effects as a direct result of cancer treatments.

Blood transfusions can cause fever and shaking. But these side effects are short-term and easily managed with injections of hydrocortisone and Piriton™.

Repeated transfusions over many years may also cause iron to build up in the body, which can affect the heart and hormone levels. This can be managed using drugs that help the body to excrete iron.

Immunosuppression drugs can also cause fever and shaking, as well as skin rashes. These side effects are immediate and recurrent. But they are easily managed with steroids and analgesic drugs.

Possible side effects associated with stem cell transplants include graft versus host disease (GvHD), in which the new donor cells attack the patient’s healthy tissues. This can be very serious, but generally the side effects are manageable.

Prognosis of Aplastic Anaemia:

Aplastic anaemia is much more successfully treated now than ever before.

Here you will find information about the likely outcome for patients with aplastic anaemia. If you are a patient, you may prefer not to read this now, so do not continue. However, you may like a family member or friend to read it instead.

Nearly three quarters of all patients with aplastic anaemia who have a stem cell transplant as part of their treatment are cured. This treatment is particularly successful in younger patients who have had transplants using stem cells from a sibling, as this tends to avoid serious side effects.

It is possible to recover from aplastic anaemia at any stage of the disease. Therefore it is important to try and stay in the best possible health using antibiotics and blood transfusions, even if this does impose some lifestyle difficulties.

With immunosuppressive treatment more than half of patients with aplastic anaemia will achieve remission, meaning that the disease seems to go away and they no longer need treatment. But there is a risk that these patients will go on to develop other blood disorders.

However around one in ten of these may relapse later on. For relapsed patients, or those who don’t respond to treatment, a stem cell transplant is a good alternative.

Aplastic Anaemia – After Treatment

Even when patients have finished definitive therapy and achieved remission they still need to visit their doctor regularly for many years after their treatment has finished.

This is because there is a chance that this blood disorder may come back. One in ten patients who have achieved remission will relapse. This is more common in women who are pregnant and in people who have received multiple vaccinations.

Many patients may have a low blood count for up to ten years after following treatment, and so regular check ups are recommended to make sure they stay in good health.

A minority of patients will develop a myelodysplastic syndrome or acute myeloid leukaemia following aplastic anaemia. Regular check-ups will make sure that this is caught early and other treatments are offered.

Patients who experience no complications will not need check-ups so often.