The benefit of Azacitidine treatment in survival of high-risk myelodysplastic syndromes ( MDS ) patients compared with conventional care treatment ( CCT ) has not been established outside clinical trials. To assess its effectiveness, researchers have compared overall survival between Azacitidine and conventional treatment in high-risk MDS patients, excluding those undergoing stem cell transplantation, submitted to the Spanish MDS registry from 2000 to 2013.
Several Cox regression and competing risk models, considering Azacitidine as a time-dependent covariate, were used to assess survival and acute myeloblastic leukemia ( AML ) progression.
Among 821 patients included, 251 received Azacitidine.
Median survival was 13.4 ( 11.8–16 ) months for Azacitidine-treated patients and 12.2 ( 11–14.1 ) for patients under conventional care treatment ( P=0.41 ).
In a multivariate model, age, International prognostic scoring system and lactate dehydrogenase were predictors of overall survival whereas Azacitidine was not ( adjusted odds ratio 1.08, 95% confidence interval 0.86–1.35, P=0.49 ).
However, in patients with chromosome 7 abnormalities, a trend toward a better survival was observed in Azacitidine-treated patients ( median survival 13.3 [ 11–18 ] months ) compared with conventional care treatment ( median survival 8.6 [ 5–10.4 ] months, P=0.08 ).
In conclusion, the data have shown that, in spite of a widespread use of Azacitidine, there is a lack of improvement in survival over the years.
Identification of predicting factors of response and survival is mandatory. ( Xagena )
Bernal T et al, Leukemia 2015; 29: 1875–1881