title: Risk factors for treatment failure after allogeneic transplantation of patients with CLL: a report from the European Society for Blood and Marrow Transplantation.
abstract: 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.
authors.affiliation: Medical Department I, University Hospital, Technische Universität Dresden, Dresden, Germany.|Clinical Trials Unit, DKMS, gemeinnützige GmbH, Tübingen, Germany.
authors.forename: J
authors.initials: J
authors.lastname: Schetelig
authors.affiliation: Clinical Trials Unit, DKMS, gemeinnützige GmbH, Tübingen, Germany.|Department Medical Statistics & Bioinformatics, Leiden University Medical Center, Leiden, The Netherlands.
authors.forename: L C
authors.initials: LC
authors.lastname: de Wreede
authors.affiliation: Department of Internal Medicine, Division of Hematology, University Medical Center Maastricht, The Netherlands.
authors.forename: M
authors.initials: M
authors.lastname: van Gelder
authors.affiliation: BMT Unit, Department of Hematology, Rigshospitalet, Copenhagen, Denmark.
authors.forename: N S
authors.initials: NS
authors.lastname: Andersen
authors.affiliation: Hematologia, Hospital de la Santa Creu i Sant Pau, Barcelona, Spain.
authors.forename: C
authors.initials: C
authors.lastname: Moreno
authors.affiliation: Department of Hematology, Institute of Hematology and Blood Transfusion, Prague, Czech Republic.
authors.forename: A
authors.initials: A
authors.lastname: Vitek
authors.affiliation: Department of Hematology/Oncology, Charles University Hospital, Pilsen, Czech Republic.
authors.forename: M
authors.initials: M
authors.lastname: Karas
authors.affiliation: Hématologie, Center Hospitalier Lyon-Sud, Lyon, France.
authors.forename: M
authors.initials: M
authors.lastname: Michallet
authors.affiliation: Department of Medicine at Huddinge, Hematology Center Karolinska and Karolinska Institutet, Stockholm, Sweden.
authors.forename: M
authors.initials: M
authors.lastname: Machaczka
authors.affiliation: Division of Stem Cell Transplantation and Immunotherapy, University Hospital Schleswig-Holstein, Kiel, Germany.
authors.forename: M
authors.initials: M
authors.lastname: Gramatzki
authors.affiliation: Department of Bone Marrow Transplantation, University Hospital, Essen, Germany.
authors.forename: D
authors.initials: D
authors.lastname: Beelen
authors.affiliation: Department of Medicine-Hematology, University of Freiburg, Oncology, Freiburg, Germany.
authors.forename: J
authors.initials: J
authors.lastname: Finke
authors.affiliation: Department of Hematology, Hospital Clinic, Institute of Hematology & Oncology, Barcelona, Spain.
authors.forename: J
authors.initials: J
authors.lastname: Delgado
authors.affiliation: Stem Cell Transplantation Unit, Helsinki University Hospital Comprehensive Cancer Center, Helsinki, Finland.
authors.forename: L
authors.initials: L
authors.lastname: Volin
authors.affiliation: Department for Hematology, University Hospital, Basel, Switzerland.
authors.forename: J
authors.initials: J
authors.lastname: Passweg
authors.affiliation: Medizinische Klinik und Poliklinik V, Ruprecht-Karls-Universität Heidelberg, Heidelberg, Germany.
authors.forename: P
authors.initials: P
authors.lastname: Dreger
authors.affiliation: Department Medical Statistics & Bioinformatics, Leiden University Medical Center, Leiden, The Netherlands.
authors.forename: A
authors.initials: A
authors.lastname: Henseler
authors.affiliation: Department Medical Statistics & Bioinformatics, Leiden University Medical Center, Leiden, The Netherlands.
authors.forename: A
authors.initials: A
authors.lastname: van Biezen
authors.affiliation: Medical Department I, University Hospital, Technische Universität Dresden, Dresden, Germany.
authors.forename: M
authors.initials: M
authors.lastname: Bornhäuser
authors.affiliation: Medizinische Klinik und Poliklinik V, Ruprecht-Karls-Universität Heidelberg, Heidelberg, Germany.
authors.forename: S O
authors.initials: SO
authors.lastname: Schönland
authors.affiliation: Bone Marrow Transplantation Center, University Hospital Eppendorf, Hamburg, Germany.
authors.forename: N
authors.initials: N
authors.lastname: Kröger
mesh_terms: D000328:Adult; D000367:Age Factors; D000368:Aged; D001782:Blood Donors; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods*; D006801:Humans; D015451:Leukemia, Lymphocytic, Chronic, B-Cell / Q000175:diagnosis / Q000401:mortality / Q000628:therapy*; D008297:Male; D008875:Middle Aged; D011379:Prognosis; D018570:Risk Assessment; D012307:Risk Factors; D012737:Sex Factors; D016019:Survival Analysis; D014184:Transplantation, Homologous; D017211:Treatment Failure; D055815:Young Adult