Within a year we will have definitive answers regarding the MDS study to the following questions (among others):
8 wk TI—the primary end point, asked and answered. This will include data from part I of the MDS study which we already know as well as part II.
24 wk TI—asked and answered
Evaluations of CR, PR, HI, PFS, OS and disease modification. Asked and answered.
Effects on specific mutations: asked and answered
Potential for front line use (especially in RS- MDS), asked and answered
Comparison with population similar to Luspatercept patients (RS+) including duration of drug effect: asked and answered
Effect on QT interval: asked and answered
Effect on symptom control/QOL: asked and answered
Definitive side effect profile: asked and answered
Assessment of burden relief on the health care system asked and answered
European/British Commonwealth approval/partnership/ strategy asked and answered
Effect on progression to AML: asked and answered
Number of blood transfusions that can be saved world wide: asked and answered
There are other questions some of which may require waiting for bottom line data less than a year after top line
Outlook for the results of the MF study: asked
Asked and Answered
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