Optimized EBMT transplant-specific risk score in myelodysplastic syndromes after allogeneic stem-cell transplantation


Por: Gagelmann, N, Eikema, DJ, Stelljes, M, Beelen, D, de Wreede, L, Mufti, G, Knelange, NS, Niederwieser, D, Friis, LS, Ehninger, G, Nagler, A, Yakoub-Agha, I, Meijer, E, Ljungman, P, Maertens, J, Kanz, L, Lopez-Corral, L, Brecht, A, Craddock, C, Finke, J, Cornelissen, JJ, Bernasconi, P, Chevallier, P, Sierra, J, Robin, M, Kroger, N

Publicada: 30 abr 2019
Resumen:
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 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 10(9)/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.

Filiaciones:
Gagelmann, N:
 Univ Med Ctr Hamburg Eppendorf, Hamburg, Germany

Eikema, DJ:
 EBMT Stat, Leiden, Netherlands

Stelljes, M:
 Univ Munster, Munster, Germany

Beelen, D:
 Univ Hosp Essen, West German Canc Ctr, Dept Bone Marrow Transplantat, Essen, Germany

de Wreede, L:
 EBMT Stat, Leiden, Netherlands

Mufti, G:
 GKT Sch Med, London, England

Knelange, NS:
 EBMT Data Off, Leiden, Netherlands

Niederwieser, D:
 Univ Hosp Leipzig, Leipzig, Germany

Friis, LS:
 Rigshosp, Copenhagen, Denmark

Ehninger, G:
 Univ Klinikum Dresden, Dresden, Germany

Nagler, A:
 Chaim Sheba Med Ctr, Tel Hashomer, Israel

Yakoub-Agha, I:
 Univ Lille 2, CHU Lille, INSERM U995, LIRIC, Lille, France

Meijer, E:
 Vrije Univ Amsterdam Med Ctr, Amsterdam, Netherlands

Ljungman, P:
 Karolinska Univ Hosp, Stockholm, Sweden

 Karolinska Inst, Stockholm, Sweden

Maertens, J:
 Univ Hosp Gasthuisberg, Leuven, Belgium

Kanz, L:
 Univ Tubingen, Tubingen, Germany

Lopez-Corral, L:
 Hosp Clin Salamanca, Salamanca, Spain

Brecht, A:
 Deutsch Klin Diagnost, Wiesbaden, Germany

Craddock, C:
 Ctr Clin Haematol, Birmingham, W Midlands, England

Finke, J:
 Univ Freiburg, Freiburg, Germany

Cornelissen, JJ:
 Erasmus Univ, Erasmus MC Canc Inst, Med Ctr, Rotterdam, Netherlands

Bernasconi, P:
 Fdn IRCCS Policlin San Matteo, Pavia, Italy

Chevallier, P:
 CHU Nantes, Nantes, France

Sierra, J:
 Hosp Santa Creu & Sant Pau, Jose Carreras Leukemia Res Inst, Barcelona, Spain

Robin, M:
 Hop St Louis, Paris, France

Kroger, N:
 Univ Med Ctr Hamburg Eppendorf, Hamburg, Germany
ISSN: 07182295





HAEMATOLOGICA
Editorial
FERRATA STORTI FOUNDATION, VIA GIUSEPPE BELLI 4, 27100 PAVIA, ITALY, Italia
Tipo de documento: Article
Volumen: 104 Número: 5
Páginas: 929-936
WOS Id: 000466305600027
ID de PubMed: 30655377
imagen Gold, Green Published

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