Adult B-cell precursor acute lymphoblastic leukemia (BCP-ALL) with t(4;11)(q21;q23);KMT2A/AFF1 is a poor-prognosis entity. This registry-based study was aimed to analyze outcome of patients with t(4;11) BCP-ALL treated with allogeneic hematopoietic stem cell transplantation (alloHSCT) in first complete remission (CR1) between 2000 and 2017, focusing on the impact of measurable residual disease (MRD) at the time of transplant. Among 151 patients (median age, 38) allotransplanted from either HLA-matched siblings or unrelated donors, leukemia-free survival (LFS) and overall survival (OS) at 2 years were 51% and 60%, whereas relapse incidence (RI) and non-relapse mortality (NRM) were 30% and 20%, respectively. These results were comparable to a cohort of contemporary patients with diploid normal karyotype (NK) BCP-ALL with equivalent inclusion criteria (n = 567). Among patients with evaluable MRD pre-alloHSCT, a negative status was the strongest beneficial factor influencing LFS (hazard ratio [HR] = 0.2, p < 0.001), OS (HR = 0.14, p < 0.001), RI (HR = 0.23, p = 0.001), and NRM (HR = 0.16, p = 0.002), with a similar outcome to MRD-negative NK BCP-ALL patients. In contrast, among patients with detectable pretransplant MRD, outcome in t(4;11) BCP-ALL was inferior to NK BCP-ALL (LFS: 27% vs. 50%, p = 0.02). These results support indication of alloHSCT in CR1 for t(4;11) BCP-ALL patients, provided a negative MRD status is achieved. Conversely, pre-alloHSCT additional therapy is warranted in MRD-positive patients.

Center for Hematopoietic Cell Transplantation Deutsche Klinik für Diagnostik Helios Klinik Wiesbaden Germany

Charles University Hospital Pilsen Czech Republic

CHU Bordeaux Hôpital Haut Lévêque Pessac France

Department d'Hematologie CHU Nantes Nantes France

Department of Bone Marrow Transplantation and Onco Hematology Maria Sklodowska Curie Institute Oncology Center Gliwice Branch Gliwice Poland

Department of Hematology and Oncology Augsburg University Hospital Augsburg Germany

Department of Hematology Hospital Saint Antoine Paris France

Department of Hematology Nijmegen Medical Centre Radboud University Nijmegen The Netherlands

Department of Hematology The 1st Hospital Affiliated to Soochow University Suzhou China

Department of Translational and Precision Medicine Sapienza University Rome Italy

Division of Hematology Hospital St Louis and University Paris Paris France

EBMT Paris Study Office Department of Hematology and Cell Therapy Hôpital Saint Antoine Paris France

Erasmus MC Daniel den Hoed Cancer Centre Rotterdam The Netherlands

Hematology Department Hospital Clínic of Barcelona IDIBAPS University of Barcelona Barcelona Spain

Hematology Division Chaim Sheba Medical Center Tel Hashomer Israel

Hôpital Claude Huriez Lille France

Hôpital Edouard Herriot Lyon France

Nottingham University Hospitals and University of Nottingham Nottingham UK

Stem Cell Transplantation Unit Comprehensive Cancer Center Helsinki University Hospital Helsinki Finland

University Hospital Center Zagreb School of Medicine University of Zagreb Zagreb Croatia

University Hospital Gasthuisberg Leuven Belgium

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