BACKGROUND: In 'real-life', the Nordic score guides Erythropoietic stimulating agent (ESA) use in lower-risk myelodysplastic syndrome (MDS) with predicted response rates of 25% or 74%. As new treatments emerge, a more discriminating score is needed. OBJECTIVES: To validate existing ESA predictive scores and develop a new score that identifies non-responders. METHODS: ESA-treated patients were identified in 3 MDS registries in Italy and Canada (FISM 555, GROM 233, and MDS-CAN 208). Clinical and disease-related variables were captured. Nordic, MDS-CAN, and IPSS-R-based ESA scores were calculated and documented ESA responses compared. RESULTS: 996 ESA-treated patients were identified. Overall response rate (ORR) was 59%. The database was randomly divided into balanced derivation (n = 463) and validation (n = 462) cohorts. By multivariate analysis, transfusion independence, erythropoietin (EPO) level <100 IU/L, and IPSS low-risk were independently predictive of response. Assigning a score of 1 to each resulted in a scoring system of 0-3 with response rates of 23%, 43%, 67%, and 85%. ORR was concordant in the validation cohort. The 'ITACA' score had the highest discriminating power of response. CONCLUSION: ITACA is an internally-validated predictive SS of ESA response in real-life 'good risk' MDS patients derived from a large international dataset that surpasses others. The incorporation of biologic markers to better identify non-responders is still needed
ITACA: A new validated international erythropoietic stimulating agent-response score that further refines the predictive power of previous scoring systems / Buckstein, R; Balleari, E; Wells, R; Santini, V; Sanna, A; Salvetti, C; Crisà, E; Allione, B; Danise, P; Finelli, C; Clavio, M; Poloni, A; Salvi, F; Cilloni, D; Oliva, En; Musto, P; Houston, B; Zhu, N; Geddes, M; Leitch, H; Leber, B; Sabloff, M; Nevill, Tj; Yee, Kw; Storring, Jm; Francis, J; Maurillo, L; Latagliata, R; Spiriti, Maa; Andriani, A; Piccioni, Al; Fianchi, L; Fenu, S; Gumenyuk, S; Buccisano, F.. - In: AMERICAN JOURNAL OF HEMATOLOGY. - ISSN 0361-8609. - 92(10):(2017), pp. 1037-1046. [10.1002/ajh.24842]
ITACA: A new validated international erythropoietic stimulating agent-response score that further refines the predictive power of previous scoring systems
Poloni AMembro del Collaboration Group
;
2017-01-01
Abstract
BACKGROUND: In 'real-life', the Nordic score guides Erythropoietic stimulating agent (ESA) use in lower-risk myelodysplastic syndrome (MDS) with predicted response rates of 25% or 74%. As new treatments emerge, a more discriminating score is needed. OBJECTIVES: To validate existing ESA predictive scores and develop a new score that identifies non-responders. METHODS: ESA-treated patients were identified in 3 MDS registries in Italy and Canada (FISM 555, GROM 233, and MDS-CAN 208). Clinical and disease-related variables were captured. Nordic, MDS-CAN, and IPSS-R-based ESA scores were calculated and documented ESA responses compared. RESULTS: 996 ESA-treated patients were identified. Overall response rate (ORR) was 59%. The database was randomly divided into balanced derivation (n = 463) and validation (n = 462) cohorts. By multivariate analysis, transfusion independence, erythropoietin (EPO) level <100 IU/L, and IPSS low-risk were independently predictive of response. Assigning a score of 1 to each resulted in a scoring system of 0-3 with response rates of 23%, 43%, 67%, and 85%. ORR was concordant in the validation cohort. The 'ITACA' score had the highest discriminating power of response. CONCLUSION: ITACA is an internally-validated predictive SS of ESA response in real-life 'good risk' MDS patients derived from a large international dataset that surpasses others. The incorporation of biologic markers to better identify non-responders is still neededI documenti in IRIS sono protetti da copyright e tutti i diritti sono riservati, salvo diversa indicazione.
