Optimized EBMT transplant-specific risk score in myelodysplastic syndromes after allogeneic stem-cell transplantation
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Optimized EBMT transplant-specific risk score in myelodysplastic syndromes after allogeneic stem-cell transplantation. / Gagelmann, Nico; Eikema, Diderik-Jan; Stelljes, Matthias; Beelen, Dietrich; de Wreede, Liesbeth; Mufti, Ghulam; Knelange, Nina Simone; Niederwieser, Dietger; Friis, Lone S; Ehnninger, Gerhard; Nagler, Arnon; Yakoub-Agha, Ibrahim; Meijer, Ellen; Ljungman, Per; Maertens, Johan; Kanz, Lothar; Lopez-Corral, Lucia; Brecht, Arne; Craddock, Charles; Finke, Jürgen; Cornelissen, Jan J; Bernasconi, Paolo; Chevallier, Patrice; Sierra, Jorge; Robin, Marie; Kröger, Nicolaus.
In: HAEMATOLOGICA, Vol. 104, No. 5, 05.2019, p. 929-936.Research output: SCORING: Contribution to journal › SCORING: Journal article › Research › peer-review
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TY - JOUR
T1 - Optimized EBMT transplant-specific risk score in myelodysplastic syndromes after allogeneic stem-cell transplantation
AU - Gagelmann, Nico
AU - Eikema, Diderik-Jan
AU - Stelljes, Matthias
AU - Beelen, Dietrich
AU - de Wreede, Liesbeth
AU - Mufti, Ghulam
AU - Knelange, Nina Simone
AU - Niederwieser, Dietger
AU - Friis, Lone S
AU - Ehnninger, Gerhard
AU - Nagler, Arnon
AU - Yakoub-Agha, Ibrahim
AU - Meijer, Ellen
AU - Ljungman, Per
AU - Maertens, Johan
AU - Kanz, Lothar
AU - Lopez-Corral, Lucia
AU - Brecht, Arne
AU - Craddock, Charles
AU - Finke, Jürgen
AU - Cornelissen, Jan J
AU - Bernasconi, Paolo
AU - Chevallier, Patrice
AU - Sierra, Jorge
AU - Robin, Marie
AU - Kröger, Nicolaus
N1 - Copyright © 2019, Ferrata Storti Foundation.
PY - 2019/5
Y1 - 2019/5
N2 - The aim of this study was to develop and validate a clinical and transplant-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 outcome. 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 × 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 significant and reproducible improvement in prognostic ability with C-statistics 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 augmented after applying a nomogram using age and platelets as continuous variables showing C-statistics of 0.628 (95% CI: 0.616-0.637). In conclusion, compared to existing prognostic systems, this proposed transplant-specific risk score offers improved performance with respect to post-transplant risk stratification in myelodysplastic syndromes.
AB - The aim of this study was to develop and validate a clinical and transplant-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 outcome. 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 × 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 significant and reproducible improvement in prognostic ability with C-statistics 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 augmented after applying a nomogram using age and platelets as continuous variables showing C-statistics of 0.628 (95% CI: 0.616-0.637). In conclusion, compared to existing prognostic systems, this proposed transplant-specific risk score offers improved performance with respect to post-transplant risk stratification in myelodysplastic syndromes.
KW - Journal Article
U2 - 10.3324/haematol.2018.200808
DO - 10.3324/haematol.2018.200808
M3 - SCORING: Journal article
C2 - 30655377
VL - 104
SP - 929
EP - 936
JO - HAEMATOLOGICA
JF - HAEMATOLOGICA
SN - 0390-6078
IS - 5
ER -
