@article{3f139ce3cf5f4bc7be24e0ddda08d1bb,
title = "Challenges of phase III trial design for novel treatments in diseases with no standard treatment: The AZA-001 myelodysplasia study model",
abstract = "For cancers lacking standard treatments, comparing new agents with existing treatments is problematic. Here we discuss the study design from the AZA-001 trial, which compared azacitidine with 3 frequently used conventional care regimens (CCR) for higher-risk myelodysplastic syndromes. Before randomization, physicians preselected the most appropriate of 3 CCR for each patient, after thorough examination. Patients were then randomized to azacitidine or CCR. Patients randomized to CCR received their preselected treatment, thus including patients otherwise excluded as poor candidates for a single comparator. This design may serve as a template in other cancers lacking standard therapy.",
keywords = "Azacitidine, CCR, Heterogenous, Novel treatment, Standard, Study design",
author = "Pierre Fenaux and Seymour, {John F.} and Valeria Santini and Lew Silverman and Steven Gore and Alan List and Guillermo Sanz and Mufti, {Ghulam J.} and Eli Estey and Swern, {Arlene S.} and Beach, {C. L.} and Eva Hellstrom-Lindberg",
note = "Funding Information: P.F. has received research funding and honoraria from Celgene Corporation; J.F.S has been a consultant and Speaker's Bureau member for, and has received honoraria and travel support from, Celgene Corporation; V.S. has received honoraria from Celgene Corporation; L.S. has been a consultant for, and has received honoraria from, Celgene Corporation; S.D.G. has been a consultant for, and has received research funding from, Celgene Corporation; A.L. has been a consultant for Celgene Corporation; G.S. has received research funding and honoraria from Celgene Corporation; G.J.M has been a consultant and member of the Speaker's Bureau for, and has received research funding from, Celgene Corporation; E.E. has nothing to disclose; A.S.S. and C.L.B are employed by and have equity ownership in Celgene Corporation; E.H.-L. has been a consultant for, and has received research funding from, Celgene Corporation. Sheila Truten, BS, MC2, Wynnewood, PA provided editorial and writing assistance, which was supported by Celgene Corporation. Celgene Corporation funded the AZA-001 study. ",
year = "2014",
month = feb,
doi = "10.1016/j.leukres.2013.10.014",
language = "English",
volume = "38",
pages = "258--262",
journal = "Leukemia Research",
issn = "0145-2126",
publisher = "Elsevier Ltd.",
number = "2",
}