Population based studies in myeloid disorders

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The study authors conclude that the life expectancy of CML patients improved over time, particularly among younger patients between the 1990s and mid-late-2000s. The most reassuring finding was that the life expectancy of CML patients across all age groups ultimately approached that of the general population. This finding among contemporary diagnosed patients can essentially be attributed to the introduction of several TKIs across various therapy lines. Collectively, CML patients can look forward to a near-normal life expectancy in an era with modern approaches to manage CML.


The study authors reported for the first-time real-life data about ELT in LR-MDS and CMML patients with thrombocytopenia. They confirmed that ELT is effective with a sustained effect on platelet counts reduction in the platelet transfusion burden and in severe bleeding. The effect of ELT was often prolonged after drug discontinuation. The tolerance profile was favorable in this population with no excess of blast, but previous history of thrombosis remains a major risk factor for thrombotic events on treatment.


The study authors evaluated the impact of ELT use in real-life in AA. Two-thirds of the patients were responders to ELT. Two years after starting ELT, 42% had a complete response (cumulative incidence). More than half of the patients achieved transfusion independence. Clonal progression was identified in 11% of patients, which is comparable to the data in the literature. The safety profile was similar to the one observed in ELT clinical studies. According to the authors the results of this real-life study confirm the positive benefit-risk ratio of ELT in these patients.


The study authors conclude that within the limitations of an observational study, these real-life data support an overall survival benefit of ruxolitinib over hydroxyurea alone in patients with primary (PMF) and secondary myelofibrosis (sMF).


Presentation ID p411-5


Presenter: Morten Kranker Larsen, Frederikssund, Denmark

The study authors conclude that their study shows that CHIP CALR-mutations in a general population study associate with a significant reduction in estimated glomerular filtration rate (eGFR) as a proxy for renal function. Furthermore, cytosis is associated with a significant decrease in eGFR. Chronic inflammation and MPN-associated mutations may contribute to a decrease in eGFR even in the very early stage of MPN-development.