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Myelodysplastic Syndromes
Optimized EBMT transplant-specific risk score in myelodysplastic syndromes after allogeneic stem-cell transplantation
Ferrata Storti Foundation
Haematologica 2019 Volume 104(5):929-936
Nico Gagelmann,1 Diderik-Jan Eikema,2 Matthias Stelljes,3 Dietrich Beelen,4 Liesbeth de Wreede,2 Ghulam Mufti5, Nina Simone Knelange,6 Dietger Niederwieser,7 Lone S. Friis,8 Gerhard Ehninger,9 Arnon Nagler,10 Ibrahim Yakoub-Agha,11 Ellen Meijer,12 Per Ljungman,13 Johan Maertens,14 Lothar Kanz,15 Lucia Lopez-Corral,16 Arne Brecht,17 Charles Craddock,18 Jürgen Finke,19 Jan J. Cornelissen,20 Paolo Bernasconi,21 Patrice Chevallier,22 Jorge Sierra,23 Marie Robin24 and Nicolaus Kröger1
1University Medical Center Hamburg-Eppendorf, Hamburg, Germany; 2EBMT Statistics, Leiden, the Netherlands; 3University of Münster, Germany; 4Department of Bone Marrow Transplantation, West German Cancer Center, University Hospital of Essen, Germany; 5GKT School of Medicine, London, UK; 6EBMT Data Office, Leiden, the Netherlands; 7University Hospital Leipzig, Germany; 8Rigshospitalet, Copenhagen, Denmark; 9Universitätsklinikum Dresden, Germany; 10Chaim Sheba Medical Center, Tel-Hashomer, Israel; 11CHU de Lille, LIRIC, INSERM U995, Université Lille2, France; 12VU University Medical Center, Amsterdam, the Netherlands; 13Karolinska University Hospital and Karolinska Institutet, Stockholm, Sweden; 14University Hospital Gasthuisberg, Leuven, Belgium; 15Universität Tübingen, Germany; 16Hospital Clínico, Salamanca, Spain; 17Deutsche Klinik für Diagnostik, Wiesbaden, Germany; 18Centre for Clinical Haematology, Birmingham, UK; 19University of Freiburg, Germany; 20Erasmus MC Cancer Institute, Erasmus University Medical Center, Rotterdam, the Netherlands; 21Fondazione IRCCS Policlinico San Matteo, Pavia, Italy; 22CHU Nantes, France; 23Hospital Santa Creu i Sant Pau, Jose Carreras Leukemia Research Institute, Barcelona, Spain and 24Hopital St. Louis, Paris, France
ABSTRACT
The aim of this study was to develop and validate a clinical and trans- plant-specific prognostic score using data from a large cohort of patients with myelodysplastic syndromes reported to the European Society for Blood and Marrow Transplantation registry. A Cox model was fitted to detect clinical and transplant-related variables prognostic of out- come. Then, cross-validation was performed to evaluate the validity and consistency of the model. Seven independent risk factors for survival were identified: age ≥50 years, matched unrelated donor, Karnofsky Performance Status <90%, very poor cytogenetics or monosomal karyotype, positive cytomegalovirus status of the recipient, blood blasts >1%, and platelet count ≤50 x 109/L prior to transplantation. Incorporating these factors into a four-level risk score yielded hazard ratios for death, with low-risk (score of 0-1) as reference, of 2.02 (95% CI: 1.41-2.90) for the intermediate-risk group (score of 2-3), 3.49 (95% CI: 2.45-4.97) for the high-risk group (score of 4-5), and 5.90 (95% CI: 4.01-8.67) for the very high-risk group (score of >5). The score was predictive of survival, relapse-free survival, relapse, and non-relapse mortality (P<0.001, respectively). Cross-validation yielded sig- nificant and reproducible improvement in prognostic ability with C-statis- tics being 0.609 (95% CI: 0.588-0.629) versus 0.555 for the Gruppo Italiano Trapianto di Midollo Osseo registry and 0.579 for the Center for Blood and Marrow Transplant Research registry. Prediction was even further aug- mented after applying a nomogram using age and platelets as continuous variables showing C-statistics of 0.628 (95% CI: 0.616-0.637). In conclu- sion, compared to existing prognostic systems, this proposed transplant- specific risk score offers improved performance with respect to post-trans- plant risk stratification in myelodysplastic syndromes.
Correspondence:
NICOLAUS KRÖGER
nkroeger@uke.uni-hamburg.de
Received: July 2, 2018. Accepted: January 9, 2019. Pre-published: January 17, 2019.
doi:10.3324/haematol.2018.200808
Check the online version for the most updated information on this article, online supplements, and information on authorship & disclosures: www.haematologica.org/content/104/5/929
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