TY - JOUR
T1 - Ascertaining QUAZARs
T2 - slow-motion and light-speed development of oral azacitidine and decitabine
AU - Feld, Jonathan
AU - Tremblay, Douglas
AU - Navada, Shyamala C.
AU - Silverman, Lewis R.
N1 - Publisher Copyright:
© 2022 Informa UK Limited, trading as Taylor & Francis Group.
PY - 2023
Y1 - 2023
N2 - Myelodysplastic syndromes (MDS) and acute myeloid leukemia (AML) are devastating diseases that frequently rely on the use of parenteral hypomethylating agents (HMAs), either as monotherapy or in combination, as first-line treatment for many patients. Two new oral HMAs, decitabine/cedazuridine (DC) for use in place of azacitidine or decitabine in MDS, and azacitidine (CC-486) for use as maintenance treatment in AML, were recently approved by the FDA. We will discuss the development of these oral HMAs, including the advantages/disadvantages in transitioning to oral HMAs and an in depth look at the pivotal phase III trials that led to their FDA approval–ASCERTAIN for DC and QUAZAR-AML-001 for CC-486. We also review how these agents have been and are being studied in other malignancies, and examine the future role that these exciting novel agents will play in both MDS and AML.
AB - Myelodysplastic syndromes (MDS) and acute myeloid leukemia (AML) are devastating diseases that frequently rely on the use of parenteral hypomethylating agents (HMAs), either as monotherapy or in combination, as first-line treatment for many patients. Two new oral HMAs, decitabine/cedazuridine (DC) for use in place of azacitidine or decitabine in MDS, and azacitidine (CC-486) for use as maintenance treatment in AML, were recently approved by the FDA. We will discuss the development of these oral HMAs, including the advantages/disadvantages in transitioning to oral HMAs and an in depth look at the pivotal phase III trials that led to their FDA approval–ASCERTAIN for DC and QUAZAR-AML-001 for CC-486. We also review how these agents have been and are being studied in other malignancies, and examine the future role that these exciting novel agents will play in both MDS and AML.
KW - Hypomethylating agent
KW - acute myeloid leukemia
KW - azacitidine
KW - decitabine
KW - myelodysplastic syndromes
KW - oral chemotherapy
UR - http://www.scopus.com/inward/record.url?scp=85142173468&partnerID=8YFLogxK
U2 - 10.1080/10428194.2022.2142051
DO - 10.1080/10428194.2022.2142051
M3 - Review article
C2 - 36370098
AN - SCOPUS:85142173468
SN - 1042-8194
VL - 64
SP - 525
EP - 539
JO - Leukemia and Lymphoma
JF - Leukemia and Lymphoma
IS - 3
ER -