PhD defence J. (Hanne) Rozema

Real-world studies of patients with myelodysplastic syndromes

Survival, treatment and outcomes in a population-based setting

Myelodysplastic syndromes (MDS) are a type of blood cancer that derive from ineffective hematopoiesis. This leads to cytopenias and dysplasias in erythrocytes, neutrophils and thrombocytes, which can present itself as fatigue, frequent infections or bruises and bleedings. MDS is a relatively rare disease and is predominantly found in the elderly (>65 years). The prognosis can vary from months to years.

The research of Hanne Rozema showed that 50% of the Frisian MDS patients were alive after 25 months. Two thirds of her study population had a relevant comorbidity. Most of these patients would most likely not be included in clinical trials. The treatment duration was limited to several months and once initial treatment was abrogated, median overall survival was half a year. Patients mainly discontinued treatment due to treatment failure (44%).

The majority of MDS patients received regular blood transfusions. Transfusions require regular hospital visits. Additionally, they can lead to the development of allo-antibodies (in 9% of our study population) and iron overload. Iron overload was monitored in 47% and monitoring appeared to be primarily based on patients’ clinical performance.

Moreover, the research of Rozema showed that MDS patients had a 2.5 times higher chance of receiving anti-infective agents as compared to before diagnosis. This equals three weeks of treatment per year. The MINDSET study then showed a diversity of reasons and considerations whether or not to prescribe infection prophylaxis in MDS patients.

With her thesis, Rozema hopes to contribute to a better understanding of MDS and treatment of MDS patients in a real-world setting.

Promotores Prof.dr. E.N. van Roon, dr. M. Hoogendoorn, dr. R.E. Kibbelaar