ITACA: A new validated international erythropoietic stimulating agent-response score that further refines the predictive power of previous scoring systems Academic Article uri icon

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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 needed.

authors

  • Buckstein, Rena
  • Balleari, Enrico
  • Wells, Richard
  • Santini, Valeria
  • Sanna, Alessandro
  • Salvetti, Chiara
  • Crisà, Elena
  • Allione, Bernardino
  • Danise, Paolo
  • Finelli, Carlo
  • Clavio, Marino
  • Poloni, Antonella
  • Salvi, Flavia
  • Cilloni, Daniela
  • Oliva, Esther Natalie
  • Musto, Pellegrino
  • Houston, Brett
  • Zhu, Nancy
  • Geddes, Michelle
  • Leitch, Heather
  • Leber, Brian
  • Sabloff, Mitchell
  • Nevill, Thomas J
  • Yee, Karen W
  • Storring, John M
  • Francis, Janika
  • Maurillo, Luca
  • Latagliata, Roberto
  • Spiriti, Maria Antonietta Aloe
  • Andriani, Alessandro
  • Piccioni, Anna Lina
  • Fianchi, Luana
  • Fenu, Susanna
  • Gumenyuk, Svitlana
  • Buccisano, Francesco

publication date

  • October 2017