Is PFS the Right Endpoint to Assess Outcome of Maintenance Studies in Multiple Myeloma? Results of a Patient Survey Highlight Quality-of-Life As an Equally Important Outcome Measure

Background: So far, planning of clinical trials in multiple myeloma (MM) has been based on traditional outcome measures such as progression free (PFS) and overall survival (OS). However, treatment with drugs, which prolong survival, usually impacts quality of life (QoL). A good example is lenalidomide (LEN) maintenance therapy in transplant eligible newly diagnosed MM. While LEN is the only approved drug for maintenance therapy, clearly and consistently prolonging PFS in a number of trials, treatment comes with an increased risk for infections, secondary malignancies, fatigue and diarrhea. One consequence could be to define QoL as an additional study endpoint but the MM patient's perspective on this topic has not been systematically investigated yet.