Phase II Study of the ALK5 Inhibitor Galunisertib in Very Low-, Low-, and Intermediate-Risk Myelodysplastic Syndromes.
Valeria SantiniDavid ValcarcelUwe PlatzbeckerRami S KomrokjiAnn L CleverlyMichael M LahnJan JanssenYumin ZhaoAlan ChiangAristoteles GiagounidisSusan C GubaIvelina GueorguievaAllicia C GirvanMariana da Silva FerreiraTushar D BhagatKith PradhanUlrich SteidlAshwin SridharanBritta WillAmit VermaPublished in: Clinical cancer research : an official journal of the American Association for Cancer Research (2019)
In summary, galunisertib treatment has an acceptable safety profile and was associated with hematologic improvements in lower- and intermediate-risk MDS, with responses in heavily transfusion-dependent patients and in those with signs of an early stem cell differentiation block.