{"doi":"10.3390/ijms24054790","title":"Targeting Measurable Residual Disease (MRD) in Acute Myeloid Leukemia (AML): Moving beyond Prognostication","abstract":"<jats:p>Measurable residual disease (MRD) assessment in acute myeloid leukemia (AML) has an established role in disease prognostication, particularly in guiding decisions for hematopoietic cell transplantation in first remission. Serial MRD assessment is now routinely recommended in the evaluation of treatment response and monitoring in AML by the European LeukemiaNet. The key question remains, however, if MRD in AML is clinically actionable or “does MRD merely portend fate”? With a series of new drug approvals since 2017, we now have more targeted and less toxic therapeutic options for the potential application of MRD-directed therapy. Recent approval of NPM1 MRD as a regulatory endpoint is also foreseen to drastically transform the clinical trial landscape such as biomarker-driven adaptive design. In this article, we will review (1) the emerging molecular MRD markers (such as non-DTA mutations, IDH1/2, and FLT3-ITD); (2) the impact of novel therapeutics on MRD endpoints; and (3) how MRD might be used as a predictive biomarker to guide therapy in AML beyond its prognostic role, which is the focus of two large collaborative trials: AMLM26 INTERCEPT (ACTRN12621000439842) and MyeloMATCH (NCT05564390).</jats:p>","journal":"International Journal of Molecular Sciences","year":2023,"id":636971,"datarank":0.5101796072493234,"base_score":3.4011973816621555,"endowment":3.4011973816621555,"self_citation_contribution":0.5101796072493234,"citation_network_contribution":0.0,"self_endowment_contribution":0.5101796072493234,"citer_contribution":0.0,"corpus_percentile":null,"corpus_rank":null,"citation_count":29,"citer_count":0,"citers_with_citation_signal":0,"citers_with_endowment":0,"datacite_reuse_total":0,"is_dataset":false,"is_dataset_confidence":null,"is_data_producer":false,"deposit_databanks":null,"is_oa":false,"file_count":0,"downloads":0,"has_version_chain":false,"published_date":null,"fair_score":null,"fair_percentile":null,"algorithm_id":"datarank_citation_only_1hop_v6","ranking_scope":"data_only","authors":[{"id":1031903,"name":"Sun Loo","orcid":"0000-0001-6294-2691","position":1,"is_corresponding":false},{"id":109578,"name":"Ing Soo Tiong","orcid":"0000-0001-7417-4343","position":0,"is_corresponding":false}],"reference_count":0,"raw_metadata":{"has_enrichment":true,"resolved":true,"title":"Targeting Measurable Residual Disease (MRD) in Acute Myeloid Leukemia (AML): Moving beyond Prognostication","abstract":"<jats:p>Measurable residual disease (MRD) assessment in acute myeloid leukemia (AML) has an established role in disease prognostication, particularly in guiding decisions for hematopoietic cell transplantation in first remission. Serial MRD assessment is now routinely recommended in the evaluation of treatment response and monitoring in AML by the European LeukemiaNet. The key question remains, however, if MRD in AML is clinically actionable or “does MRD merely portend fate”? With a series of new drug approvals since 2017, we now have more targeted and less toxic therapeutic options for the potential application of MRD-directed therapy. Recent approval of NPM1 MRD as a regulatory endpoint is also foreseen to drastically transform the clinical trial landscape such as biomarker-driven adaptive design. In this article, we will review (1) the emerging molecular MRD markers (such as non-DTA mutations, IDH1/2, and FLT3-ITD); (2) the impact of novel therapeutics on MRD endpoints; and (3) how MRD might be used as a predictive biomarker to guide therapy in AML beyond its prognostic role, which is the focus of two large collaborative trials: AMLM26 INTERCEPT (ACTRN12621000439842) and MyeloMATCH (NCT05564390).</jats:p>","is_dataset_classified":null,"base_score":3.367295829986474,"endowment":3.367295829986474,"datacite_reuse_total":0,"file_count":0,"downloads":0,"views":0,"has_version_chain":false,"is_dataset":false,"is_oa":false,"pmid":"36902217","pmcid":"PMC10003715","openalex_id":"https://openalex.org/W4322770890","authors":[],"funders":[],"total_grants":0,"fwci":5.1418,"citation_percentile":0.96245525,"influential_citations":0,"citation_trend":[{"year":2023,"count":3},{"year":2024,"count":8},{"year":2025,"count":11},{"year":2026,"count":6}],"oa_status":"gold","license":"cc-by","oa_locations":[{"url":"https://www.mdpi.com/1422-0067/24/5/4790/pdf?version=1678006834","host_type":"journal"},{"url":"https://www.mdpi.com/1422-0067/24/5/4790/pdf?version=1678006834","host_type":"publisher"},{"url":"https://www.mdpi.com/1422-0067/24/5/4790/pdf","host_type":"publisher"},{"url":"https://doi.org/10.3390/ijms24054790","host_type":"journal"},{"url":"https://pubmed.ncbi.nlm.nih.gov/36902217","host_type":"repository"},{"url":"https://www.ncbi.nlm.nih.gov/pmc/articles/10003715","host_type":"repository"},{"url":"https://dx.doi.org/10.3390/ijms24054790","host_type":"repository"},{"url":"https://pmc.ncbi.nlm.nih.gov/articles/PMC10003715/pdf/ijms-24-04790.pdf","host_type":"repository"},{"url":"https://europepmc.org/articles/PMC10003715","host_type":"Europe_PMC"},{"url":"https://europepmc.org/articles/PMC10003715?pdf=render","host_type":"Europe_PMC"}],"fields_of_study":["Acute Myeloid Leukemia Research","Sarcoma Diagnosis and Treatment","Retinoids in leukemia and cellular processes"],"mesh_terms":["Nucleophosmin","Humans","Nuclear Proteins","Prognosis","Remission Induction","Biomarkers, Tumor","Leukemia, Myeloid, Acute","Neoplasm, Residual","Hematopoietic Stem Cell Transplantation"],"keywords":["Myeloid leukemia","Minimal residual disease","Medicine","Residual","Disease","Oncology","Leukemia","Myeloid","Internal medicine","Cancer research","Intensive care medicine","Computer science","AMl","Relapse","Novel Therapy","Npm1","Mrd"],"sdg_mappings":[{"sdg_number":0,"sdg_label":"Good health and well-being"}],"linked_datasets":[],"clinical_trials":[],"software_tools":[],"database_accessions":[{"name":"nct"}],"source":"live","citation_network_status":"fetched"},"created_at":"2026-08-06T18:03:39.493296Z","pmid":null,"pmcid":null,"fwci":null,"citation_percentile":null,"influential_citations":0,"oa_status":null,"license":null,"views":0,"total_file_size_bytes":0,"version_count":0,"fair_f":null,"fair_a":null,"fair_i":null,"fair_r":null,"fair_zscore":null,"fair_rationale":null,"fair_model":null,"fair_agent_version":null,"fair_fulltext_source":null,"fair_has_llm":null,"fair_computed_at":null,"clinical_trials":[],"software_tools":[],"db_accessions":[],"linked_datasets":[],"topics":[]}