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Autologous stem cell transplantation for adults with Philadelphia-negative acute lymphoblastic leukemia in first complete remission. A study by the Acute Leukemia Working Party of the EBMT

R. Swoboda, M. Labopin, S. Giebel, J. Maertens, E. Parovichnikova, J. Versluis, J. Pavlu, A. Kopinska, S. Capria, L. Raida, A. Rambaldi, D. Caillot, F. Folber, D. Nachbaur, M. Ozturk, M. Aljurf, MT. Rubio, NC. Gorin, F. Lanza, A. Nagler, M....

. 2025 ; 25 (1) : 787. [pub] 20250428

Language English Country England, Great Britain

Document type Journal Article

BACKGROUND: The role of autologous hematopoietic stem cell transplantation (AHSCT) in adults with Philadelphia chromosome-negative acute lymphoblastic leukemia (Ph-ALL) remains controversial. The aim of this retrospective study was to analyze results of AHSCT and to identify prognostic factors. METHODS: Overall, 700 patients transplanted in first complete remission between the years 1999-2020 were included. Median patient age was 31.9 years (68% male). B-cell precursor ALL (BCP-ALL) and T-cell precursor ALL (TCP-ALL) was diagnosed in 35% and 65%, respectively. Among 190 patients with available data, negative minimal residual disease (MRD) status was reported in 167 (88%) cases. RESULTS: The probabilities of overall survival (OS) and leukemia-free survival (LFS) at 2 years were 67% and 56%; relapse incidence (RI) and non-relapse mortality (NRM) were 39% and 5%, respectively. TCP-ALL was associated with lower RI (41% vs. 56%, p=0.001), higher LFS (52% vs. 38%, p=0.002) and OS (58% vs 45%, p=0.001) at 5 years when compared to BCP-ALL. In the multivariate analysis, TCP-ALL and longer interval from diagnosis do AHSCT were associated with reduced risk of relapse (HR 0.7, p=0.006 and HR=0.95, p=0.018), better LFS (HR=0.76, p=0.02 and HR=0.95, p=0.01) and OS (HR=0.75, p=0.024 and HR=0.94, p=0.013, respectively). Increasing patient age was associated with higher NRM (HR=1.49, p<0.0001), worse LFS (HR=1.1, p=0.01) and OS (HR=1.17, p=0.0001). CONCLUSIONS: Autologous hematopoietic stem cell transplantation is relatively safe option of late treatment intensification in adults with Ph- ALL. It may be a valuable option especially in patients with TCP-ALL, however it should be proved in prospective clinical trials.

Department of Bone Marrow Transplantation and Onco Hematology Maria Sklodowska Curie National Research Institute of Oncology Gliwice Branch Str Wybrzeze Armii Krajowej 15 Gliwice 44 101 Poland

Department of Haemato Oncology Olomouc University Hospital and Faculty of Medicine and Dentistry Palacký University Olomouc Czech Republic

Department of Haematology Imperial College Hammersmith Hospital London UK

Department of Hematology and Bone Marrow Transplantation Medical University of Silesia Katowice Poland

Department of Hematology and Cellular Therapy National Institute of Health and Medical Research Unit UMR S 938 Sorbonne University and St Anthony Scientific Research Center Public Assistance Hospital of Paris St Anthony Hospital Paris France

Department of Hematology CHU Brabois Nancy France

Department of Hematology Erasmus University Medical Center Rotterdam the Netherlands

Department of Hematology Hôpital Saint Antoine Paris France

Department of Hematology University Hospital Gasthuisberg Leuven Belgium

Department of Internal Medicine 5 Medical University of Innsbruck Anichstrasse 35 Innsbruck A 6020 Austria

Department of Internal Medicine Hematology and Oncology Masaryk University Hospital Brno Brno Czech Republic

Department of Oncology and Hematology University of Milan and Azienda Socio Sanitaria Territoriale Papa Giovanni XXIII Milan Italy

Division of Hematology Sheba Medical Center Tel Hashomer Israel

European Society for Blood and Marrow Transplantation Paris Study Office CEREST TC Paris France

GATA BMT Center Gülhane Military Medical Academy Ankara Turkey

Hematology Unit University Hospital and Romagna Transplant Network Ravenna University of Bologna Bologna Italy

Hopital d'Enfants CHU de Dijon Service Hematologie Adultes Dijon France

King Faisal Specialist Hospital and Research Centre Riyadh Saudi Arabia

National Research Center for Hematology Bone Marrow Transplantation Moscow Russian Federation

Ospedale San Raffaele s r l Haematology and BMT Milano Italy

Univ La Sapienza Dip Biotecnologie Cellulari ed Ematologia Rome Italy

References provided by Crossref.org

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$a Autologous stem cell transplantation for adults with Philadelphia-negative acute lymphoblastic leukemia in first complete remission. A study by the Acute Leukemia Working Party of the EBMT / $c R. Swoboda, M. Labopin, S. Giebel, J. Maertens, E. Parovichnikova, J. Versluis, J. Pavlu, A. Kopinska, S. Capria, L. Raida, A. Rambaldi, D. Caillot, F. Folber, D. Nachbaur, M. Ozturk, M. Aljurf, MT. Rubio, NC. Gorin, F. Lanza, A. Nagler, M. Mohty, F. Ciceri
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$a BACKGROUND: The role of autologous hematopoietic stem cell transplantation (AHSCT) in adults with Philadelphia chromosome-negative acute lymphoblastic leukemia (Ph-ALL) remains controversial. The aim of this retrospective study was to analyze results of AHSCT and to identify prognostic factors. METHODS: Overall, 700 patients transplanted in first complete remission between the years 1999-2020 were included. Median patient age was 31.9 years (68% male). B-cell precursor ALL (BCP-ALL) and T-cell precursor ALL (TCP-ALL) was diagnosed in 35% and 65%, respectively. Among 190 patients with available data, negative minimal residual disease (MRD) status was reported in 167 (88%) cases. RESULTS: The probabilities of overall survival (OS) and leukemia-free survival (LFS) at 2 years were 67% and 56%; relapse incidence (RI) and non-relapse mortality (NRM) were 39% and 5%, respectively. TCP-ALL was associated with lower RI (41% vs. 56%, p=0.001), higher LFS (52% vs. 38%, p=0.002) and OS (58% vs 45%, p=0.001) at 5 years when compared to BCP-ALL. In the multivariate analysis, TCP-ALL and longer interval from diagnosis do AHSCT were associated with reduced risk of relapse (HR 0.7, p=0.006 and HR=0.95, p=0.018), better LFS (HR=0.76, p=0.02 and HR=0.95, p=0.01) and OS (HR=0.75, p=0.024 and HR=0.94, p=0.013, respectively). Increasing patient age was associated with higher NRM (HR=1.49, p<0.0001), worse LFS (HR=1.1, p=0.01) and OS (HR=1.17, p=0.0001). CONCLUSIONS: Autologous hematopoietic stem cell transplantation is relatively safe option of late treatment intensification in adults with Ph- ALL. It may be a valuable option especially in patients with TCP-ALL, however it should be proved in prospective clinical trials.
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