What are the treatments?

Treatment of aplastic anaemia can be divided into two areas: Supportive therapy and Definitive therapy.

Supportive therapy is directed at treating symptoms such as low red blood cells and platelets with blood and platelet transfusions respectively, and antibiotics and antifungal drugs to prevent and treat infections.

Most patients with aplastic anaemia who need treatment will require transfusions of red blood cells and platelets.

If a patient is being considered for a stem cell transplant it is particularly important that they do not receive transfusions of blood or blood products from any family member. This is because such transfusions may seriously compromise the likelihood of success of a later transplant from a related donor.

Red cell transfusions may rarely produce problems in patients with aplastic anaemia. Some patients produce antibodies to so-called ‘minor’ blood groups. This sometimes means that they need carefully selected blood which may need to be specially ordered from the NHS Blood and Transplant (NHSBT) service (used to be called the National Blood Service). Without these precautions, the presence of antibodies would mean the transfused blood would not last very long.

In rare instances red cell transfusions may cause fever and even rigors (shaking). These can be controlled with injections of hydrocortisone and piriton. Piriton is a strong antihistamine which often makes people sleepy.

One unit of transfused red blood cells typically raises the haemoglobin level by about 1.0. It is usual to need three to four units each month. Repeated red cell transfusions given over many months may lead to accumulation of iron in the body. This can later cause problems with the heart and hormone producing (endocrine) glands unless precautions are taken. Few patients with aplastic anaemia need transfusion programmes which are prolonged enough to cause such problems as there are medicines available to reduce the amount of iron in the body.

Platelets are shorter-lived cells; a single unit transfused should raise the platelet count significantly for about two to three days. Response to platelet transfusions is much more variable from one patient to another. Sometimes patients develop antibodies to platelets and special donors have to be found by the transfusion service.

Definitive therapy is aimed at restoring the bone marrow function so that the patients become free of blood and platelet transfusions, are no longer at risk of bleeding or infections and can hopefully lead a fairly normal life.

Definitive therapy can be divided into two areas: immunosuppressive treatment or stem cell transplant.

Definitive treatment can take many months, and occasionally longer, to achieve its greatest effects; it is very important to continue with supportive therapy during this time when patients are waiting to see if they will respond to definitive treatments as they will still be at risk from anaemia, bleeding/bruising and infections.

Most patients will need some form of definitive therapy except for a small number of patients with non-severe aplastic anaemia who, for example, are not needing any blood or platelet transfusions.

In acquired aplastic anaemia the underlying disease process is the attack on the bone marrow stem cells by the immune system. The principle of treatment, therefore, is to use drugs to ‘quieten’ or ‘dampen down’ the immune system, thus allowing the bone marrow to heal and resume normal production. Curative treatment is aimed at replacing the faulty bone marrow by giving new stem cells and the abnormal immune system by using a stem cell transplant from either a matched sibling or an unrelated donor.