Azacitidine for the treatment of lower risk myelodysplastic syndromes : a retrospective study of 74 patients enrolled in an Italian named patient program.
BACKGROUND
Azacitidine induces responses and prolongs overall survival compared with conventional care regimens in patients who have high-risk myelodysplastic syndromes (MDS). However, limited data are available concerning the efficacy and safety of azacitidine in patients who have lower risk MDS.


METHODS
The authors retrospectively evaluated 74 patients with International Prognostic Scoring System low-risk or intermediate 1-risk MDS, who received azacitidine on a national named patient program. At baseline, 84% of patients were transfusion-dependent, 57% had received erythropoietin, and 51% were aged >70 years. Azacitidine was administered subcutaneously for 5 days (n = 29 patients), 7 days (n = 43 patients), or 10 days (n = 2 patients) every month at a dose of 75 mg/m(2) daily (n = 45 patients) or at a fixed dose of 100 mg daily (n = 29 patients) and for a median of 7 cycles (range, 1-30 cycles).


RESULTS
According to the 2006 International Working Group criteria, overall response rate (ORR) was 45.9%, including complete responses (10.8%), partial responses (9.5%), hematologic improvements (20.3%), and bone marrow complete responses (5.4%). The ORR was 51.6% in 64 patients who completed > or = 4 cycles of treatment. The median duration of response was 6 months (range, 1-30 months). After a median follow-up of 15 months, 71% of patients remained alive. A survival benefit was observed in responders versus nonresponders (94% vs 54% of patients projected to be alive at 2.5 years, respectively; P < .0014). The most common grade 3 or 4 adverse events were myelosuppression (21.6%) and infection (6.8%).


CONCLUSIONS
The current results indicated that azacitidine may be a feasible and effective treatment for patients with lower risk MDS.
Unit of Hematology and Stem Cell Transplantation, IRCCS, Centro di Riferimento Oncologico della Basilicata, Via Padre Pio 1, 85028 Rionero in Vulture, Potenza, Italy. p.musto@crob.it
Pellegrino
P
Musto
Luca
L
Maurillo
Alessandra
A
Spagnoli
Antonella
A
Gozzini
Flavia
F
Rivellini
Monia
M
Lunghi
Oreste
O
Villani
Maria Antonietta
MA
Aloe-Spiriti
Adriano
A
Venditti
Valeria
V
Santini
D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D000964:Antimetabolites, Antineoplastic / Q000627:therapeutic use*; D001374:Azacitidine / Q000009:adverse effects / Q000627:therapeutic use*; D004341:Drug Evaluation; D005260:Female; D006801:Humans; D007558:Italy; D008297:Male; D008875:Middle Aged; D009190:Myelodysplastic Syndromes / Q000188:drug therapy*; D012189:Retrospective Studies; D012306:Risk; D016896:Treatment Outcome
D000964:Antimetabolites, Antineoplastic; D001374:Azacitidine