Outcome of adult acute myeloid leukemia patients with extramedullary disease and treatment with venetoclax / hypomethylating agents
Sabine Kayser,
Khaled Sanber,
Giovanni Marconi,
Agnese Mattei,
Marlise R. Luskin,
Amar Kelkar,
Marco Cerrano,
Daniel Tuyet Kristensen,
Anne Stidsholt Roug,
Chiara Sartor,
Fabio Giglio,
Marta Riva,
Lorenzo Rizzo,
Francesco Saraceni,
Selene Guerzoni,
Federica Lessi,
Erika Borlenghi,
Mark J. Levis,
Richard F. Schlenk,
Tania Jain,
Cristina Papayannidis
Affiliations
Sabine Kayser
Institute of Transfusion Medicine and Immunology, Medical Faculty Mannheim, Heidelberg University, German Red Cross Blood Service Baden-Württemberg-Hessen, Mannheim, Germany; NCT Trial Center, National Center of Tumor Diseases, Heidelberg University Hospital and German Cancer Research Center (DKFZ), Heidelberg
Khaled Sanber
Sidney Kimmel Comprehensive Cancer Center, Johns Hopkins University, Baltimore, MD
Giovanni Marconi
Hematology Unit, IRCCS Istituto Romagnolo Per Lo Studio Dei Tumori (IRST) "Dino Amadori", Meldola, FC
Agnese Mattei
Hematology Unit, IRCCS Istituto Romagnolo Per Lo Studio Dei Tumori (IRST) "Dino Amadori", Meldola, FC
Marlise R. Luskin
Department of Medical Oncology, Dana-Farber Cancer Institute, Boston, MA
Amar Kelkar
Department of Medical Oncology, Dana-Farber Cancer Institute, Boston, MA
Marco Cerrano
Deptartment of Oncology, Division of Hematology, Presidio Molinette, Torino
Daniel Tuyet Kristensen
Department of Hematology, Aarhus University Hospital, Denmark; Department of Haematology, Clinical Cancer Research Center, Aalborg University Hospital, Aalborg, Denmark; Department of Clinical Medicine, Aalborg University, Aalborg, Denmark
Anne Stidsholt Roug
Department of Hematology, Aarhus University Hospital, Denmark
Chiara Sartor
Istituto di Ematologia “Seràgnoli”, Bologna
Fabio Giglio
Hematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milano, Italy; Onco-Hematology Division, IEO European Institute of Oncology IRCCS, Milano
Marta Riva
S.C. EMATOLOGIA Dipartimento di Ematologia, Oncologia e Medicina Molecolare Niguarda Cancer Center, ASST Grande Ospedale Metropolitano Niguarda, Milano
Lorenzo Rizzo
S.C. EMATOLOGIA Dipartimento di Ematologia, Oncologia e Medicina Molecolare Niguarda Cancer Center, ASST Grande Ospedale Metropolitano Niguarda, Milano
Francesco Saraceni
Department of Hematology and Bone Marrow Transplantation, Ospedali Riuniti, Ancona
Selene Guerzoni
Department of Hematology and Bone Marrow Transplantation, Ospedali Riuniti, Ancona
Federica Lessi
Ematologia Azienda Ospedale Università Padova
Erika Borlenghi
Department of Hematology, ASST Spedali Civili, Brescia
Mark J. Levis
Sidney Kimmel Comprehensive Cancer Center, Johns Hopkins University, Baltimore, MD
Richard F. Schlenk
NCT Trial Center, National Center of Tumor Diseases, Heidelberg University Hospital and German Cancer Research Center (DKFZ), Heidelberg, Germany; Department of Internal Medicine V, Heidelberg University Hospital, Heidelberg, Germany; Department of Medical Oncology, National Center for Tumor Diseases (NCT), Heidelberg University Hospital, Heidelberg
Tania Jain
Sidney Kimmel Comprehensive Cancer Center, Johns Hopkins University, Baltimore, MD
Cristina Papayannidis
IRCCS Azienda Ospedaliero-Universitaria di Bologna, Istituto di Ematologia “Seràgnoli”, Bologna
We evaluated response to VEN/HMA in 46 patients with acute myeloid leukemia (AML) characterized by extramedullary disease (EMD). Median age was 65 (range, 19-81) years. Patients had a median of two EMD sites (range, 1-5) and 35 (76%) patients had concurrent bone marrow involvement. Twenty (43%) patients had highrisk genetic features according to the European Leukemia Net 2022 classification. Twenty-nine (63%) were relapsed or refractory after intensive chemotherapy (CTX) including 13 (28%) with prior allogeneic hematopoietic cell transplantation (allo-HCT). Patients received a median of 2 cycles of VEN/HMA (range, 1-31). Twenty (43%) patients achieved complete remission (CR) or CR with incomplete hematological recovery (CRi) after VEN/HMA and five (11%) achieved a partial remission (PR). Six patients were subsequently consolidated with allo-HCT (CR/CRi, n=4; PR, n=2). Median follow-up was 49.1 months (95%-CI, 26.1 months - not reached) and median overall survival (OS) 6.4 months (95%-CI, 5.1-11 months). One-year and 2-years OS rates were 29.3% (95%-CI, 18.6-46.2%) and 12.3% (95%-CI, 5.5-27.6%), respectively. Age with a cut-off of 60 years had no impact on OS (P=0.90). Relapse occurred in 12 of 20 (60%) patients who achieved CR/CRi after VEN/HMA treatment. Of those, all except one succumbed to their disease. Six (30%) patients were in CR/CRi at last follow-up and 2 (10%) died in CR. In our cohort of patients with AML with EMD with high-risk features, treatment with VEN/HMA resulted in an encouraging ORR of 54% with a CR/CRi rate of 43.5%. However, VEN/HMA alone may not be effective in maintaining disease control.
