Acute Promyelocytic Leukemia: Long-Term Outcomes from the HARMONY Project.

Autor:	Voso MT; Tor Vergata, Rome, Italy., Guarnera L; University of Rome 'Tor Vergata', Rome, Italy., Lehmann S; Uppsala University, Uppsala, Sweden., Döhner K; University Hospital of Ulm, Ulm, Germany., Döhner H; University Hospital Ulm, Ulm, Germany., Platzbecker U; University Hospital Leipzig, Department of Hematology and Cell Therapy, Leipzig, Germany., Russell NH; Guy's and St Thomas' NHS Foundation Trust, London, United Kingdom., Dillon RJ; King's College, London, London, United Kingdom., Thomas I; Cardiff University, Cardiff, United Kingdom., Ossenkoppele GJ; Amsterdam UMC, location VUmc, Amsterdam, Netherlands., Haferlach T; MLL Munich Leukemia Laboratory, Munich, Germany., Vignetti M; Fondazione Gimema Onlus, Roma, Italy., La Sala E; Gimema Foundation, Roma, Italy., Piciocchi A; GIMEMA Foundation, Rome, Italy., Fazi P; GIMEMA Foundation, rome, Italy., Villaverde Ramiro Á; Universidad de Salamanca, IBSAL (Instituto de Investigación Biomédica de Salamanca), IBMCC, CSIC, Centro de Investigación del Cáncer, Salamanca, Spain, Salamanca, Spain., Tur Giménez L; GMV Innovating Solutions, Valencia, Spain, Valencia, Spain., Gurnari C; Taussig Cancer Institute, Cleveland Clinic, Cleveland, Ohio, United States., Bullinger L; Charité University Medicine, Berlin, Germany., Hernandez JM; Hospital Universitario de Salamanca, Salamanca, Spain.
Jazyk:	angličtina
Zdroj:	Blood [Blood] 2024 Nov 06. Date of Electronic Publication: 2024 Nov 06.
DOI:	10.1182/blood.2024026186
Abstrakt:	Purpose: Treatment outcomes for acute promyelocytic leukemia (APL) have improved with the widespread use of targeted therapy with all-trans retinoic acid (ATRA) and arsenic trioxide (ATO). Our study aimed to validate these data in a large patient cohort, and to redefine prognostic factors. Patients and Methods: Leveraging the HARMONY Platform, we analyzed 1438 newly-diagnosed APL patients, diagnosed between 1999 to 2022. Patient data derived from the 2 international multicenter GIMEMA-APL0406 and NCRI-AML17 trials, and 4 European registries (HOVON, AMLSG, Swedish AML Registry and SAL). Results: The study cohort included 721 males and 717 females, with a median age of 50.5 years (range 16-94 years). Of 1309 patients starting therapy, 562 received ATRA-ATO, and 747 AIDA-like chemotherapy. Early death (ED) occurred in 85 of 1438 patients (5.9%) at a median of 9 days after APL diagnosis and was independently associated with increasing age and high Sanz risk score (OR:1.06, 95% C.I: 1.04-1.08, and OR:4.65, 95% C.I.:2.55-8.51, respectively).The median follow-up was 5.5 years (IQR=3.2-7.5). ATRA-ATO regimen was associated with the best outcome, reaching 91% 7-year overall survival (vs 81% for AIDA-like, HR:2.14, 95%C.I.:1.51-3.05), 89% event-free survival (vs 71% for AIDA-like, HR:2.72 95%CI: 2.01-3.69) and 3% relapse (vs 13% for AIDA-like, HR:4.19, 95%CI:2.38-7.39, p<0.001 for all outcomes). The survival advantage of ATRA/ATO was independent of patients' age, Sanz-risk score, and treatment scenario. Conclusions: Our study confirms the superiority of ATRA-ATO over ATRA-chemotherapy in APL patients. ED represents an unmet medical need, in particular in older patients and in high-risk APL. (Copyright © 2024 American Society of Hematology.)
Databáze:	MEDLINE
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