title: Allogeneic stem cell transplantation in patients with atypical chronic myeloid leukaemia: a retrospective study from the Chronic Malignancies Working Party of the European Society for Blood and Marrow Transplantation.
abstract: Atypical chronic myeloid leukaemia (aCML) is an aggressive malignancy for which allogeneic haematopoietic stem cell transplantation (allo-HSCT) represents the only curative option. We describe transplant outcomes in 42 patients reported to the European Society for Blood and Marrow Transplantation (EBMT) registry who underwent allo-HSCT for aCML between 1997 and 2006. Median age was 46 years. Median time from diagnosis to transplant was 7 months. Disease status was first chronic phase in 69%. Donors were human leucocyte antigen (HLA)-identical siblings in 64% and matched unrelated (MUD) in 36%. A reduced intensity conditioning was employed in 24% of patients. T-cell depletion was applied in 87% and 26% of transplants from MUD and HLA-identical siblings, respectively. According to the EBMT risk-score, 45% of patients were 'low-risk', 31% 'intermediate-risk' and 24% 'high-risk'. Following allo-HSCT, 87% of patients achieved complete remission. At 5 years, relapse-free survival was 36% and non-relapse mortality (NRM) was 24%, while relapse occurred in 40%. Patient age and the EBMT score had an impact on overall survival. Relapse-free survival was higher in MUD than in HLA-identical sibling HSCT, with no difference in NRM. In conclusion, this study confirmed that allo-HSCT represents a valid strategy to achieve cure in a reasonable proportion of patients with aCML, with young patients with low EBMT risk score being the best candidates.
authors.affiliation: Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, University of Milan, Milan, Italy.
authors.forename: Francesco
authors.identifier: 0000-0002-2991-3474
authors.initials: F
authors.lastname: Onida
authors.affiliation: Department of Medical Statistics & Bioinformatics, Leiden University Medical Centre, Leiden, The Netherlands.|DKMS Clinical Trials Unit, Dresden, Germany.
authors.forename: Liesbeth C
authors.initials: LC
authors.lastname: de Wreede
authors.affiliation: Department of Medical Statistics & Bioinformatics, Leiden University Medical Centre, Leiden, The Netherlands.
authors.forename: Anja
authors.initials: A
authors.lastname: van Biezen
authors.affiliation: Department of Medical Statistics & Bioinformatics, Leiden University Medical Centre, Leiden, The Netherlands.
authors.forename: Diderik-Jan
authors.initials: DJ
authors.lastname: Eikema
authors.affiliation: Nottingham University Hospitals Trust, Nottingham, UK.
authors.forename: Jenny L
authors.initials: JL
authors.lastname: Byrne
authors.affiliation: Azienda Policlinico Umberto I, 'La Sapienza' University, Rome, Italy.
authors.forename: Anna P
authors.initials: AP
authors.lastname: Iori
authors.affiliation: Universitair Ziekenhuis Brussel, Brussels, Belgium.
authors.forename: Rik
authors.initials: R
authors.lastname: Schots
authors.affiliation: Charles University Hospital, Pilsen, Czech Republic.
authors.forename: Alexandra
authors.initials: A
authors.lastname: Jungova
authors.affiliation: Universitaetsklinikum Dresden, Dresden, Germany.
authors.forename: Johannes
authors.initials: J
authors.lastname: Schetelig
authors.affiliation: University of Freiburg, Freiburg, Germany.
authors.forename: Jürgen
authors.initials: J
authors.lastname: Finke
authors.affiliation: Department of Haematology, Leiden University Medical Centre, Leiden, The Netherlands.
authors.forename: Hendrik
authors.initials: H
authors.lastname: Veelken
authors.affiliation: Sahlgrenska University Hospital, Göteborg, Sweden.
authors.forename: Jan-Erik
authors.initials: JE
authors.lastname: Johansson
authors.affiliation: Queen Elisabeth Hospital, Birmingham, UK.
authors.forename: Charles
authors.initials: C
authors.lastname: Craddock
authors.affiliation: University of Münster, Münster, Germany.
authors.forename: Matthias
authors.initials: M
authors.lastname: Stelljes
authors.affiliation: University Medical Centre Mainz, Mainz, Germany.
authors.forename: Matthias
authors.initials: M
authors.lastname: Theobald
authors.affiliation: University Regensburg, Regensburg, Germany.
authors.forename: Ernst
authors.initials: E
authors.lastname: Holler
authors.affiliation: University Hospital, Zürich, Switzerland.
authors.forename: Urs
authors.initials: U
authors.lastname: Schanz
authors.affiliation: Radboud University - Nijmegen Medical Centre, Nijmegen, The Netherlands.
authors.forename: Nicolaas
authors.initials: N
authors.lastname: Schaap
authors.affiliation: University Hospital, Homburg, Germany.
authors.forename: Jörg
authors.initials: J
authors.lastname: Bittenbring
authors.affiliation: Hammersmith Hospital, London, UK.
authors.forename: Eduardo
authors.initials: E
authors.lastname: Olavarria
authors.affiliation: Hematology Division, Hôpitaux Universitaires de Genève and Faculty of Medicine, University of Geneva, Geneva, Switzerland.
authors.forename: Yves
authors.initials: Y
authors.lastname: Chalandon
authors.affiliation: University Hospital Eppendorf, Hamburg, Germany.
authors.forename: Nicolaus
authors.initials: N
authors.lastname: Kröger
mesh_terms: D000328:Adult; D000368:Aged; D018572:Disease-Free Survival; D005260:Female; D006085:Graft Survival; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods* / Q000401:mortality; D006801:Humans; D054438:Leukemia, Myeloid, Chronic, Atypical, BCR-ABL Negative / Q000401:mortality / Q000628:therapy*; D008297:Male; D008875:Middle Aged; D012008:Recurrence; D012189:Retrospective Studies; D012307:Risk Factors; D014184:Transplantation, Homologous
keywords: Myelodyslastic/Myeloproliferative Neoplasms (MDS/MPN); Ph-negative CML: BCR-ABL1-negative; allogeneic transplantation; atypical chronic myeloid leukaemia