A Plain Language Summary of the ASCERTAIN Trial: Oral Decitabine and Cedazuridine Versus Intravenous Decitabine for MDS or CMML

Authors

Guillermo Garcia-Manero
James McCloskey
Elizabeth A Griffiths
Karen W L Yee
Amer M Zeidan
Aref Al-Kali
H Joachim Deeg
Mitchell Sabloff
Mary-Margaret Keating
Nancy Zhu
Nashat Y Gabrail
Salman Fazal
Joseph Maly
Olatoyosi Odenike
Amy E DeZern
Casey L O'Connell
Gail J Roboz
Lambert Busque
Rena Buckstein
Harshad Amin
Brian Leber
Aditi Shastri
Aram Oganesian
Harold N Keer
Mohammad Azab
Michael R Savona

Publication Date

4-1-2025

Journal

Future Oncology

DOI

10.1080/14796694.2025.2468578

PMID

40051275

PMCID

PMC11938952

PubMedCentral® Posted Date

3-6-2025

PubMedCentral® Full Text Version

Post-print

Abstract

What is this summary about?This summary describes results from a phase 3 clinical trial called ASCERTAIN.Researchers compared two treatments in people with myelodysplastic syndromes (MDS) or chronic myelomonocytic leukemia (CMML), either of which can develop into acute myeloid leukemia (AML). One group was treated with oral decitabine and cedazuridine (oral DEC-C) and the other with intravenous decitabine.Treatment was given in a series of cycles. In the first cycle, people were randomly assigned to receive oral DEC-C or intravenous decitabine and then switched to the other treatment in the second cycle. After the first two cycles, everyone received oral DEC-C. The aim was to compare whether the same amount of decitabine reached people’s bloodstream in a certain time period after receiving oral DEC-C or intravenous decitabine (known as pharmacokinetics). Researchers also looked at how long people lived while taking treatment, how long people lived without developing AML, and the side effects from receiving treatment.

What were the results?The amount of decitabine in the bloodstream during the first 5 days of treatment was almost the same (99% similar) between people who received oral DEC-C and those who received intravenous decitabine. Among people treated with oral DEC-C, the median overall survival was 31.8 months. Overall, people lived without developing AML for a median of 31.7 months. Among people who required red blood cell transfusion at the start of the trial, 52% achieved transfusion independence during the trial with oral DEC-C. Side effects of oral DEC-C were similar to those of intravenous decitabine,most commonly thrombocytopenia and neutropenia. There were five treatment-related deaths during the trial; two in the oral DEC-C group and three in the intravenous decitabine group.

What do the results mean?Oral DEC-C was as effective, and had similar side effects, as treatment with intravenous decitabine. Although the trial did not focus on quality of life, oral medicines require fewer visits to a clinic compared with intravenous medicines, which could make it easier for people to stay on treatment. This trial supports using oral DEC-C for treating people with MDS or CMML.[Box: see text].

Published Open-Access

yes

Recommended Citation

Garcia-Manero, Guillermo; McCloskey, James; Griffiths, Elizabeth A; et al., "A Plain Language Summary of the ASCERTAIN Trial: Oral Decitabine and Cedazuridine Versus Intravenous Decitabine for MDS or CMML" (2025). Faculty, Staff and Student Publications. 4412.
https://digitalcommons.library.tmc.edu/uthgsbs_docs/4412