Overall survival of myelodysplastic syndrome patients after azacitidine discontinuation and applicability of the North American MDS Consortium scoring system in clinical practice

Marino Clavio, Elena Crisà, Maurizio Miglino, Fabio Guolo, Manuela Ceccarelli, Flavia Salvi, Bernardino Allione, Dario Ferrero, Enrico Balleari, Carlo Finelli, Antonella Poloni, Carmine Selleri, Paolo Danise, Daniela Cilloni, Anna Angela Di Tucci, Gianni Cametti, Roberto Freilone, Renato Fanin, Catia Bigazzi, Renato ZambelloMonica Crugnola, Esther N. Oliva, Riccardo Centurioni, Francesco Alesiani, Massimo Catarini, Andrea Castelli, Antonio Abbadessa, Silvana F. Capalbo, Pellegrino Musto, Emanuele Angelucci, Valeria Santini

Research output: Contribution to journalArticlepeer-review

Abstract

Background: Azacitidine (AZA) is the standard treatment for myelodysplastic syndromes (MDS); however, many patients prematurely stop therapy and have a dismal outcome. Methods: The authors analyzed outcomes after AZA treatment for 402 MDS patients consecutively enrolled in the Italian MDS Registry of the Fondazione Italiana Sindromi Mielodisplastiche, and they evaluated the North American MDS Consortium scoring system in a clinical practice setting. Results: At treatment discontinuation, 20.3% of the patients were still responding to AZA, 35.4% of the cases had primary resistance, and 44.3% developed adaptive resistance. Overall survival (OS) was better for patients who discontinued treatment while in response because of planned allogeneic hematopoietic stem cell transplantation (HSCT; median OS, not reached) in comparison with patients with primary resistance (median OS, 4 months) or adaptive resistance (median OS, 5 months) or patients responsive but noncompliant/intolerant to AZA (median OS, 4 months; P =.004). After AZA discontinuation, 309 patients (77%) received best supportive care (BSC), 60 (15%) received active treatments, and 33 (8%) received HSCT. HSCT was associated with a significant survival advantage, regardless of the response to AZA. The North American MDS Consortium scoring system was evaluable in 278 of the 402 cases: patients at high risk had worse OS than patients at low risk (3 and 7 months, respectively; P <.001). The score was predictive of survival both in patients receiving BSC (median OS, 2 months for high-risk patients vs 5 months for low-risk patients) and in patients being actively treated (median OS, 8 months for high-risk patients vs 16 months for low-risk patients; P <.001), including transplant patients. Conclusions: Real-life data confirm that this prognostic scoring system for MDS patients failing a hypomethylating agent seems to be a useful tool for optimal prognostic stratification and for choosing a second-line treatment after AZA discontinuation.

Original languageEnglish
Pages (from-to)2015-2024
JournalCancer
Volume127
Issue number12
DOIs
Publication statusPublished - 2021

Keywords

  • azacitidine
  • myelodysplastic syndromes (MDS)
  • prognostic scoring system

ASJC Scopus subject areas

  • Oncology
  • Cancer Research

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