For young patients with high-risk CLL, BTK-/PI3K-inhibitors or allogeneic stem cell transplantation (alloHCT) are considered. Patients with a low risk of non-relapse mortality (NRM) but a high risk of failure of targeted therapy may benefit most from alloHCT. We performed Cox regression analyses to identify risk factors for 2-year NRM and 5-year event-free survival (using EFS as a surrogate for long-term disease control) in a large, updated EBMT registry cohort (n= 694). For the whole cohort, 2-year NRM was 28% and 5-year EFS 37%. Higher age, lower performance status, unrelated donor type and unfavorable sex-mismatch had a significant adverse impact on 2-year NRM. Two-year NRM was calculated for good- and poor-risk reference patients. Predicted 2-year-NRM was 11 and 12% for male and female good-risk patients compared with 42 and 33% for male and female poor-risk patients. For 5-year EFS, age, performance status, prior autologous HCT, remission status and sex-mismatch had a significant impact, whereas del(17p) did not. The model-based prediction of 5-year EFS was 55% and 64%, respectively, for male and female good-risk patients. Good-risk transplant candidates with high-risk CLL and limited prognosis either on or after failure of targeted therapy should still be considered for alloHCT.

BMT Unit Department of Hematology Rigshospitalet Copenhagen Denmark

Bone Marrow Transplantation Center University Hospital Eppendorf Hamburg Germany

Clinical Trials Unit DKMS gemeinnützige GmbH Tübingen Germany

Department for Hematology University Hospital Basel Switzerland

Department Medical Statistics and Bioinformatics Leiden University Medical Center Leiden The Netherlands

Department of Bone Marrow Transplantation University Hospital Essen Germany

Department of Hematology Hospital Clinic Institute of Hematology and Oncology Barcelona Spain

Department of Hematology Institute of Hematology and Blood Transfusion Prague Czech Republic

Department of Hematology Oncology Charles University Hospital Pilsen Czech Republic

Department of Internal Medicine Division of Hematology University Medical Center Maastricht The Netherlands

Department of Medicine at Huddinge Hematology Center Karolinska and Karolinska Institutet Stockholm Sweden

Department of Medicine Hematology University of Freiburg Oncology Freiburg Germany

Division of Stem Cell Transplantation and Immunotherapy University Hospital Schleswig Holstein Kiel Germany

Hematologia Hospital de la Santa Creu i Sant Pau Barcelona Spain

Hématologie Center Hospitalier Lyon Sud Lyon France

Medical Department 1 University Hospital Technische Universität Dresden Dresden Germany

Medizinische Klinik und Poliklinik 5 Ruprecht Karls Universität Heidelberg Heidelberg Germany

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

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