Myelodysplastic syndromes and aplastic anaemia Flashcards
Which one result of the follow falls within the normal reference range?
Hb 29.0 g/L
Total White Cell Count 109.3 x 10^9/L
Platelet count 273 x 10^9/L
Neutrophil count 0.34 x 10^9/L
Blood urea 29.5 mmol/l
Platelet count 273 x 10^9/L
What is MDS?
Biologically heterogeneous group of acquired haemopoietic stem cell disorders (~ 4 per 100,000 persons)
What is MDS characterised by?
The development of a clone of marrow stem cells with abnormal maturation resulting in -
functionally defective blood cells AND
a numerical reduction
This results in:
i. Cytopenia(s).
ii. Qualitative (i.e. functional) abnormalities of erythroid, myeloid and megakaryocyte maturation.
iii. Increased risk of transformation to leukaemia
What is the general clinical picture of myelodysplasia?
Typically a disorder of the elderly.
Symptoms/signs are those of general marrow failure
Develops over weeks & months
What are Some Blood & Bone Marrow Morphological Features of MDS?
Pelger-Huet anomaly (bilobed neutrophils)
Dysganulopoieses of neutrophils
Dyserythropoiesis of red cells
Dysplastic megakaryocytes – e.g. micro-megakaryocytes
Increased proportion of blast cells in marrow (normal < 5%)
What is this?
Refractory anaemia dysgranulopoiesis
What is myelokathexis?
Myelokathexis is a congenital disorder of the white blood cells that causes severe, chronic leukopenia (a reduction of circulating white blood cells) and neutropenia (a reduction of neutrophil granulocytes). The disorder is believed to be inherited in an autosomal dominant manner.
What is this?
Ringed sideroblasts
What is this?
Myeloblast with Auer Rods
What is the WHO classification of MDS?
Refractory anaemia (RA)
with ringed sideroblasts (RARS)
without ringed sideroblasts
Refractory cytopenia with multilineage dysplasia (RCMD)
Refractory anaemia with excess of blasts (RAEB)
RAEB-I (BM blasts 5-9%)
RAEB-II (BM blasts 10-19%)
5q- syndrome
Unclassified MDS: MDS with fibrosis, childhood MDS, others
What is the Revised International Prognostic Scoring System (IPSS-R) in MDS (2012)?
How do you use IPSS R for prognosis?
What is the evolution of myelodysplasia?
- Deterioration of blood counts
• Worsening consequences of marrow failure
- Development of acute myeloid leukaemia
– Develops in 5-50%< 1 year (depends on subtype)
– Some cases of MDS are much slower to evolve
– AML from MDS has an extremely poor prognosis and is usually not curable
- As a rule of thumb
- 1/3 die from infection
- 1/3 die from bleeding
- 1/3 die from acute leukaemia
What is the treatment of MDS?
At present, the only two treatments that can prolong survival are:
allogeneic stem cell transplantation (SCT)
intensive chemotherapy
but only a minority of MDS patients can really benefit from them
What are the specific treatments of MDS?
1 Supportive care
Blood product support
Antimicrobial therapy
Growth factors (Epo, G-CSF)
2 Biological Modifiers
Immunosuppressive therapy
Azacytidine ) Hypomethylating
Decitabine ) agents
Lenalidomide
3 Oral chemotherapy
Hydroxyurea
4 Low dose chemotherapy
Subcutaneous low dose cytarabine
5 Intensive Chemotherapy/SCT (for high risk MDS)
AML type regimens
Allo/VUD standard/ reduced intensity
Which one of the following about MDS is true?:
Myelodysplasia has a bi-modal age distribution
The primary modality of treatment of MDS is by intensive chemotherapy
One third of MDS patients can be expected to die from leukaemic transformation
There is no good correlation between the severity of the cytopenias and the overall life expectancy
White cell function is frequently well preserved in MDS
One third of MDS patients can be expected to die from leukaemic transformation
Draw out the haematopoietic cell lines