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Question 6
Myelodysplastic syndromes (MDS) are a group of malignant cancers. In MDS, the bone marrow does not produce healthy blood cells. Haematopoietic stem cell transplanta... show full transcript
Step 1
Answer
Haematopoietic stem cell transplantation (HSCT) effectively treats MDS by replacing faulty bone marrow with healthy stem cells. This procedure consists of the following steps:
Production of Healthy Cells: HSCT involves the generation of healthy blood cells by introducing stem cells that are not affected by MDS.
Elimination of Faulty Cells: Before transplantation, the patient's own unhealthy bone marrow is destroyed, eliminating the source of defective blood cells.
Stem Cell Replication: The transplanted stem cells replicate and differentiate, leading to the formation of functional blood cells, thereby restoring the hematopoietic function.
Step 2
Answer
Reduction of Methylation: AZA reduces methylation of DNA, specifically targeting cytosine bases in the promoter region of tumour suppressor genes. This demethylation promotes the expression of these genes.
Gene Activation: By allowing the transcription of tumour suppressor genes, AZA controls aberrant cell division, thus preventing the uncontrolled proliferation typical of MDS. This mechanism helps restore normal cell cycle regulation and reduces the severity of MDS.
Step 3
Answer
Effectiveness Comparison: Conventional drugs are established treatments for MDS, providing a baseline for evaluating the effectiveness of AZA.
Ethical Considerations: It is important to use a control group for ethical reasons, ensuring that patients in the study receive a recognized standard of care.
Step 4
Answer
At 10 months, the proportion of patients surviving with AZA is 0.58 while the conventional drugs have 0.74.
To find the difference in the number of patients:
Calculate Surviving Patients:
Difference in Proportions:
This indicates that there is a difference of 16% in survival rates after 10 months.
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