Blood Disorders

There are many illnesses treated by bone marrow transplants. This section focuses on 2 main areas: blood cancers and disorders. Your doctor will also be able to tell you more about the conditions, and advise you accordingly. Every patient is different, and treatments for each condition vary. For certain conditions such as Acute Myelogeneous Leukaemia (AML), Acute Lymphoblastic Leukaemia (ALL), the need for a transplant is mostly determined by th tte likelihood of the disease returning – which is affected by the patient’s general health, some of the leukaemia’s features, and the risk factors. Whereas for Non-Hodgkin’s Lymphoma and Multiple Myeloma, factors including age, overall health, stage and growth speed of the disease will determine the risks and benefits of having a transplant. The doctor will thus determine whether a transplant is an option.

BLOOD DISORDERS

There are more than 70 blood disorders treatable by a bone marrow transplant, for more information about your condition, do check with your doctor. This section includes 3 blood disorders.

Severe combined immunodeficiency (SCID, all types)

  • SCID is a group of inherited immune system disorders that are present at birth.
  • They are potentially life threatening within the first year of life if left untreated.
  • Usually results in the onset of one or more serious infections within the first few months of life.
  • These infections are usually serious, and may even be life threatening, they may include pneumonia, meningitis or bloodstream infections.
  • Allogeneic transplants are the only known treatment, and are recommended as soon as possible. Patients with SCID are at risk for life-threatening infections thus increasing the urgency for transplants – those with severe infections might even be too weak to get a transplant.

Severe Aplastic Anaemia

  • People with severe aplastic anaemia have bone marrow that is unable to produce sufficient blood cells for the body.
  • They have low amounts of all three types of blood cells.
  • Can affect people of all ages, but is prevalent amongst young adults.
  • Infections or bleeding caused by the lack of blood cells can be life threatening and require immediate medical attention.
  • A stem cell or bone marrow transplant can be the first treatment recommended, or used after drug therapy has failed.
  • In the early stages of the disease, referral to a transplant doctor may offer the best route to a cure.

Thalassemia (Major)

  • Genetic blood disease
  • Thalassemics’ bodies are unable to produce normal haemoglobin needed to produce healthy red blood cells.
  • Not infectious – personal contact will not pass the condition from one individual to another.
  • A special blood test, haemoglobin electrophoresis, is used to determine if one has the Thalassemia trait.
  • The combination of one gene from the mother and one from the father leads to three different types of Thalassemia: Major, Intermediate, and Minor.
  • As those with Minor and Intermediate Thalassemia are not hugely impacted by the condition, they do not require a transplant.

Major

  • Occurs when a child inherits two mutated genes, one from each parent.
  • Symptoms of severe anaemia within the first year of life will be developed.
  • They are unable to produce normal, adult haemoglobin, and experience chronic fatigue.
  • Poor growth, impaired physical activities, facial and other bone deformities, fragile bones and enlargement of the spleen are effects of the severe anaemia and expansion of the bone marrow in the body’s effort to produce more red blood cells.
  • Potentially fatal within the first decade of life is left untreated.
  • The only treatment to combat severe anaemia is regular blood transfusions and iron chelation therapy – when that fails, a stem cell or bone marrow transplant will be recommended.