Phase III, Randomized, Placebo-Controlled Trial of CC-486 (Oral Azacitidine) in Patients With Lower-Risk Myelodysplastic Syndromes.
Guillermo Garcia ManeroValeria SantiniAntonio AlmeidaUwe PlatzbeckerAnna JonasovaLewis R SilvermanJose FalantesGianluigi RedaFrancesco BuccisanoPierre FenauxRena BucksteinMaria Diez CampeloStephen LarsenDavid ValcarcelParesh VyasValentina GiaiEsther Natalie OlivaJake ShorttDietger NiederwieserMoshe MittelmanLuana FianchiIgnazia La TorreJianhua ZhongEric LailleDaniel Lopes de MenezesBarry SkikneC L BeachAristoteles GiagounidisPublished in: Journal of clinical oncology : official journal of the American Society of Clinical Oncology (2021)
CC-486 significantly improved RBC-TI rate and induced durable bilineage improvements in patients with LR-MDS and high-risk disease features. More early deaths occurred in the CC-486 arm, most related to infections in patients with significant pretreatment neutropenia. Further evaluation of CC-486 in MDS is needed.