Molecular relapse after first-line intensive therapy in patients with CBF or NPM1-mutated acute myeloid leukemia - a FILO study.
Corentin OrvainSarah BertoliPierre PeterlinYohann DesbrossesPierre-Yves DumasAlexandre IatMarie-Anne HospitalMartin CarreEmmanuelle TavernierJérémie RiouAnne BouvierAudrey BidetSylvie TondeurFlorian RenosiMarie-Joelle MozziconacciPascale Flandrin-GrestaBérengère Dadone-MontaudiéEric DelabesseArnaud PigneuxMathilde M Hunault-BergerChristian RecherPublished in: Leukemia (2024)
Patients with Core-Binding Factor (CBF) and NPM1-mutated acute myeloid leukemia (AML) can be monitored by quantitative PCR after having achieved first complete remission (CR) to detect morphologic relapse and drive preemptive therapy. How to best manage these patients is unknown. We retrospectively analyzed 303 patients with CBF and NPM1-mutated AML, aged 18-60 years, without allogeneic hematopoietic cell transplantation (HCT) in first CR, with molecular monitoring after first-line intensive therapy. Among these patients, 153 (51%) never relapsed, 95 (31%) had molecular relapse (53 received preemptive therapy and 42 progressed to morphologic relapse at salvage therapy), and 55 (18%) had upfront morphologic relapse. Patients who received preemptive therapy had higher OS than those who received salvage therapy after having progressed from molecular to morphologic relapse and those with upfront morphologic relapse (three-year OS: 78% vs. 51% vs. 51%, respectively, P = 0.01). Preemptive therapy included upfront allogeneic HCT (n = 19), intensive chemotherapy (n = 21), and non-intensive therapy (n = 13; three-year OS: 92% vs. 79% vs. 58%, respectively, P = 0.09). Although not definitive due to the non-randomized allocation of patients to different treatment strategies at relapse, our study suggests that molecular monitoring should be considered during follow-up to start preemptive therapy before overt morphologic relapse.
Keyphrases
- acute myeloid leukemia
- end stage renal disease
- chronic kidney disease
- free survival
- ejection fraction
- newly diagnosed
- stem cell transplantation
- randomized controlled trial
- peritoneal dialysis
- bone marrow
- prognostic factors
- squamous cell carcinoma
- allogeneic hematopoietic stem cell transplantation
- acute lymphoblastic leukemia
- mesenchymal stem cells
- cell proliferation
- cell death
- high dose
- locally advanced
- replacement therapy
- double blind
- ulcerative colitis
- phase ii