Measurable Residual Disease in High-Risk Acute Myeloid Leukemia
Open accessAbstract
Mounting evidence suggests measurable residual disease (MRD) assessments are prognostic in acute myeloid leukemia (AML). High-risk AML encompasses a subset of AML with poor response to therapy and prognosis, with features such as therapy-related AML, an antecedent hematologic disorder, extramedullary disease (in adults), and selected mutations and cytogenetic abnormalities. Historically, few patients with high-risk AML achieved deep and durable remission with conventional chemotherapy; however, newer agents might be more effective in achieving MRD-negative remission. CPX-351 (dual-drug liposomal encapsulation of daunorubicin/cytarabine at a synergistic ratio) demonstrated MRD-negativity rates of 36–64% across retrospective studies in adults with newly diagnosed high-risk AML and 84% in pediatric patients with first-relapse AML. Venetoclax (BCL2 inhibitor) demonstrated MRD-negativity rates of 33–53% in combination with hypomethylating agents for high-risk subgroups in studies of older adults with newly diagnosed AML who were ineligible for intensive therapy and 65% in combination with chemotherapy in pediatric patients with relapsed/refractory AML. However, there is no consensus on optimal MRD methodology in AML, and the use of different techniques, sample sources, sensitivity thresholds, and the timing of assessments limit comparisons across studies. Robust MRD analyses are needed in future clinical studies, and MRD monitoring should become a routine aspect of AML management.
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MRD is a good surrogate marker for OS in a prospective frontline HR-MDS study.
“MRD is a good surrogate marker for OS as recently shown in a prospective frontline HR‐MDS study.”
From Long‐term follow‐up and combined Phase 2 results of eprenetapopt and azacitidine in patients with <i>TP53</i> mutant MDS/AML (2025) · cites this paper as [46]
Section 3, “Prognostic Value of MRD in AML” (page unavailable), reports an association between MRD negativity and better overall survival in AML. It does not describe a prospective frontline high-risk MDS study or establish MRD as a surrogate marker for overall survival in that setting. (LLM, conf=0.85)
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Sallman, D. A., Komrokji, R. S., Dezern, A. E., Sebert, M., Garcia‐Manero, G., Rahmé, R., Winer, E. S., Lehmann‐Che, J., Roboz, G. J., Madelaine, I., et al. (2025) · HemaSphere · doi:10.1002/hem3.70164 · Cited by 20 · see citing works
“39,46 In our study, a potentially curative approach was possible for m TP53 patients obtaining a response with NGS negativity followed by allo ‐ HCT.”
Self-citation
- Authors
- Cluzeau, T., Lemoli, R. M., McCloskey, J., & Cooper, T.
- Affiliations
- Université Côte d'Azur (FR)
- Ospedale Policlinico San Martino (IT)
- Hackensack University Medical Center (US)
- Seattle Children's Hospital (US)
- Corresponding author
- Cluzeau, Thomas
- Venue
- Cancers
- Publisher
- MDPI AG
- Published
- 2022-03-01
- Volume
- 14
- Issue
- 5
- Pages
- 1278
- ISSN
- 2072-6694 (ISSN-L)
- PMC
- PMC8909238
- OpenAlex
- W4214924516
- Cited by
- 13
- References
- 54
- Citations by year
- 2026: 1 · 2025: 5 · 2024: 3 · 2023: 3 · 2022: 1
- FWCI
- 0.53
- Type
- journal article
- Language
- en
- Open access
- gold
- doi.org
- Licence
- Locations
- Cancers (publishedVersion, cc-by)
- PubMed Central (publishedVersion, cc-by)
- PubMed (publishedVersion)
- DOAJ (DOAJ: Directory of Open Access Journals) (submittedVersion, cc-by-sa)
- CINECA IRIS Institutial Research Information System (University of Genoa) (submittedVersion, other-oa)
- Topics
- Health Sciences › Medicine › Hematology › Acute Myeloid Leukemia Research
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › Retinoids in leukemia and cellular processes
- Health Sciences › Medicine › Public Health, Environmental and Occupational Health › Acute Lymphoblastic Leukemia research
- Keywords
- minimal residual disease, high-risk acute myeloid leukemia, MRD negativity, CPX-351, daunorubicin, cytarabine, liposomal chemotherapy, venetoclax, BCL-2 inhibitor, hypomethylating agents, relapsed/refractory AML, therapy-related myeloid neoplasms, prognostic factors, MRD monitoring
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