Oral presentation by Dr. Alan F. List
The present treatment for patients with myelodysplastic syndromes (MDS) who require red blood cell (RBC) transfusions is extremely limited and includes clinical trials or best supportive care. Many agents have been studied over the past few years, but none have been shown to be of benefit. Patients with these diseases are in great need of further treatment options.
Luspatercept is a first-in-class erythroid maturation agent. In this highly selected patient population it demonstrated very promising efficacy. Thirty-eight percent of treated patients achieved transfusion independence for greater than 8 weeks and 28% achieved transfusion independence for greater than 12 weeks. The drug seems to be extremely well tolerated with very little clinical toxicity, consistent with what was reported in the phase II PACE-MDS study by Platzbecker et al. No significant safety concerns were noted in the study; however, it will be very helpful to read the details of the final publication.
As this is an area of great unmet need, once the complete data has been published, this treatment should become standard therapy in my view. Unfortunately, the patient population included in the study is extremely restrictive and I don’t think we can safely extrapolate the benefit of this drug to other categories of MDS without further data. We are hopeful that future studies can show benefit in a wider range of patients. Finally, we are a bit unsure as to what the treatment for patients with MDS will look like in the future, as there have been no significant advances for several years.