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

Salvetti, Chiara;Crisà, Elena;Cilloni, Daniela;
2017

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.
AMERICAN JOURNAL OF HEMATOLOGY
92
10
1037
1046
http://onlinelibrary.wiley.com/journal/10.1002/(ISSN)1096-8652
Aged; Aged, 80 and over; Canada; Databases, Factual; Female; Hematinics; Humans; International Cooperation; Italy; Logistic Models; Male; Predictive Value of Tests; Prognosis; Prospective Studies; Registries; Survival Rate; Myelodysplastic Syndromes; Hematology
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
File in questo prodotto:
File Dimensione Formato  
Erythropoietic stimulating agents.docx

non disponibili

Tipo di file: PREPRINT (PRIMA BOZZA)
Dimensione 195.81 kB
Formato Adobe PDF
195.81 kB Adobe PDF   Visualizza/Apri   Richiedi una copia
Cilloni_R1_ITACA.pdf

non disponibili

Tipo di file: PDF EDITORIALE
Dimensione 891.57 kB
Formato Adobe PDF
891.57 kB Adobe PDF   Visualizza/Apri   Richiedi una copia

I documenti in IRIS sono protetti da copyright e tutti i diritti sono riservati, salvo diversa indicazione.

Utilizza questo identificativo per citare o creare un link a questo documento: http://hdl.handle.net/2318/1704592
Citazioni
  • ???jsp.display-item.citation.pmc??? 3
  • Scopus 14
  • ???jsp.display-item.citation.isi??? 13
social impact