Myelodysplasia

Question 1

What is meant by the term ‘myelodysplastic syndromes’?

Answer 1

These are a group of clonal disorders of the bone marrow where there is bone marrow failure as a result of ineffective haematopoiesis

Question 2

In what age group is myelodysplasia most common?

Answer 2

> 60 years

Question 3

At what stage can myelodysplasia progress to acute leukaemia?

Answer 3

When there are greater than 20% of blast cells in the bone marrow

Question 4

Describe the pathophysiology of myelodysplasia?

Answer 4

In myelodyspalsia, haematopoietic stem cells are damaged so they give rise to faulty blood cells which don’t mature but instead persist as immature cells called blasts. In early disease. these blast cells undergo apoptosis which causes peripheral blood cytopenias. In later disease, there is decreased apoptosis with characteristic gene mutations, enhanced survival of myeloblasts and potential expansion of a leukaemic clone

Question 5

Myelodysplasia can occur de novo or may be secondary to…?

Answer 5

Chemotherapy or radiotherapy for another malignancy

Question 6

What are the symptoms of myelodyspalsia?

Answer 6

Patients may be asymptomatic and when symptoms do occur they can range from mild anaemia to the consequence of severe marrow failure (profound anaemia, recurrent infections, bleeding). pronounced symptoms are more common in individuals with large number of blasts

Question 7

What diagnostic tests can be used to investigate potential myelodysplasia?

Answer 7

FBC
Peripheral blood smear
Bone marrow biopsy
Chromosome analysis

Question 8

What factors determine a myelodysplasia patient’s risk status?

Answer 8

FBC
Proportion of blast cells in the marrow
Type of chromosome abnormalities present

Question 9

What is involved ins supportive treatment for myelodysplasia?

Answer 9

Regular blood transfusion to control symptoms of anaemia - may also need iron chelators
Platelet transfusion to manage haemorrhage
Antibiotics to manage infection

Question 10

Describe the treatment of low risk patients with myelodysplasia?

Answer 10

Anaemia treated with EPO +/- G-CSF

Lenalidomide used for patients with a del 5q chromosome abnormalities to improve anaemia

Question 11

What is the aim of treatment in low risk patients with myelodysplasia?

Answer 11

To improve cytopenias

Question 12

What is the aim of treatment in high risk patients with myelodysplasia?

Answer 12

To prolong survival and delay transformation to acute myeloid leukaemia

Question 13

Describe the treatment of high risk patients with myelodysplasia?

Answer 13

Chemotherapy

Allogeneic stem cell therapy