Background: Although azacitidine (AZA) has proven effective in myelodysplastic syndromes (MDS), the duration of haematologic response is limited. Introduction: The French Group (Itzykson 2011) identified some clinical and haematologic parameters (poor ECOG performance status, IPSS intermediate and poor risk cytogenetics, circulating blasts, high transfusion need) independently associated with a a poorer outcome, and these 4 criteria were integrated in a 3-group prognostic score. Purpose: These data prompted us to retrospectively analyse our MDS pts treated with AZA who showed a favourable long-lasting response to AZA (i.e: duration of response ≥ 20 months). Materials and Methods: The type of response was defined according to IWG criteria (Cheson 2006): Complete Remission (CR), Marrow CR, Partial Remission (PR), and Hematologic Improvement (HI). Results: Thirty-six pts (M/F: 21/15), from nine Institutions, with a median age of 72 (range 52-84) yrs, showed a response duration ≥ 20 months. At AZA onset, IPSS risk was: low: 3 pts; intermediate- 1: 7 pts; intermediate-2: 21 pts; high: 5 pts. IPSS cytogenetic risk was: low: 23 pts; intermediate: 7 pts; high: 6 pts.Transfusion need was high (≥ 4 RBC units/8 weeks) in 17 pts. Following Itzykson’s AZA prognostic scoring system, the risk was low in 14 pts (38.9%), intermediate in 21 pts (58.3%), and high in 1 pt (2.8%), respectively. The pts received a median of 23.5 cycles of AZA (range: 8-59). The best response achieved was: CR in 21 pts (58.3%); marrow CR: 4 pts (11.1%); PR in 2 pts (5.5%); and HI in 9 pts (25%). Cytogenetic remission was achieved in 7 pts (19.4%). The median duration of response was 24.5 (range: 20-88) months. Twenty-two pts (61.1%) are still maintaining hematologic response, 9 pts (25%) are still alive but discontinued treatment because of disease progression, and 5 pts died (2 for AML). Median OS from the start of AZA was 35.5 (range: 22-120) months. Conclusions: Our data show that a long-lasting hematologic response can be achieved even in a significant fraction of pts presenting one or more poor risk features.
C. Finelli, C. Clissa, M.T. Voso, M.A. Aloe Spiriti, M. Breccia, G. Gaidano, et al. (2013). Long-lasting hematologic response to azacitidine in myelodysplastic syndromes: Multicenter retrospective study of 36 patients. ELSEVIER [10.1016/S0145-2126(13)70343-6].
Long-lasting hematologic response to azacitidine in myelodysplastic syndromes: Multicenter retrospective study of 36 patients
FINELLI, CARLO;FOLLO, MATILDE YUNG;MARTINELLI, GIOVANNI;CAVO, MICHELE
2013
Abstract
Background: Although azacitidine (AZA) has proven effective in myelodysplastic syndromes (MDS), the duration of haematologic response is limited. Introduction: The French Group (Itzykson 2011) identified some clinical and haematologic parameters (poor ECOG performance status, IPSS intermediate and poor risk cytogenetics, circulating blasts, high transfusion need) independently associated with a a poorer outcome, and these 4 criteria were integrated in a 3-group prognostic score. Purpose: These data prompted us to retrospectively analyse our MDS pts treated with AZA who showed a favourable long-lasting response to AZA (i.e: duration of response ≥ 20 months). Materials and Methods: The type of response was defined according to IWG criteria (Cheson 2006): Complete Remission (CR), Marrow CR, Partial Remission (PR), and Hematologic Improvement (HI). Results: Thirty-six pts (M/F: 21/15), from nine Institutions, with a median age of 72 (range 52-84) yrs, showed a response duration ≥ 20 months. At AZA onset, IPSS risk was: low: 3 pts; intermediate- 1: 7 pts; intermediate-2: 21 pts; high: 5 pts. IPSS cytogenetic risk was: low: 23 pts; intermediate: 7 pts; high: 6 pts.Transfusion need was high (≥ 4 RBC units/8 weeks) in 17 pts. Following Itzykson’s AZA prognostic scoring system, the risk was low in 14 pts (38.9%), intermediate in 21 pts (58.3%), and high in 1 pt (2.8%), respectively. The pts received a median of 23.5 cycles of AZA (range: 8-59). The best response achieved was: CR in 21 pts (58.3%); marrow CR: 4 pts (11.1%); PR in 2 pts (5.5%); and HI in 9 pts (25%). Cytogenetic remission was achieved in 7 pts (19.4%). The median duration of response was 24.5 (range: 20-88) months. Twenty-two pts (61.1%) are still maintaining hematologic response, 9 pts (25%) are still alive but discontinued treatment because of disease progression, and 5 pts died (2 for AML). Median OS from the start of AZA was 35.5 (range: 22-120) months. Conclusions: Our data show that a long-lasting hematologic response can be achieved even in a significant fraction of pts presenting one or more poor risk features.I documenti in IRIS sono protetti da copyright e tutti i diritti sono riservati, salvo diversa indicazione.
