TY - JOUR
T1 - ITACA
T2 - A new validated international erythropoietic stimulating agent-response score that further refines the predictive power of previous scoring systems
AU - Buckstein, Rena
AU - Balleari, Enrico
AU - Wells, Richard
AU - Santini, Valeria
AU - Sanna, Alessandro
AU - Salvetti, Chiara
AU - Crisà, Elena
AU - Allione, Bernardino
AU - Danise, Paolo
AU - Finelli, Carlo
AU - Clavio, Marino
AU - Poloni, Antonella
AU - Salvi, Flavia
AU - Cilloni, Daniela
AU - Oliva, Esther Natalie
AU - Musto, Pellegrino
AU - Houston, Brett
AU - Zhu, Nancy
AU - Geddes, Michelle
AU - Leitch, Heather
AU - Leber, Brian
AU - Sabloff, Mitchell
AU - Nevill, Thomas J.
AU - Yee, Karen W.
AU - Storring, John M.
AU - Francis, Janika
AU - Maurillo, Luca
AU - Latagliata, Roberto
AU - Spiriti, Maria Antonietta Aloe
AU - Andriani, Alessandro
AU - Piccioni, Anna Lina
AU - Fianchi, Luana
AU - Fenu, Susanna
AU - Gumenyuk, Svitlana
AU - Buccisano, Francesco
AU - ITACA
AU - Emanuele, Angelucci
PY - 2017
Y1 - 2017
N2 - 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.
AB - 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.
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U2 - 10.1002/ajh.24842
DO - 10.1002/ajh.24842
M3 - Article
AN - SCOPUS:85026453194
JO - American Journal of Hematology
JF - American Journal of Hematology
SN - 0361-8609
ER -