Azacitidine for the treatment of patients with acute myeloid leukemia: Report of 82 patients enrolled in an Italian compassionate program

Background: The efficacy of azacitidine for the treatment of high-risk myelodysplastic syndromes has prompted the issue of its potential role even in the treatment of acute myeloid leukemia (AML). Methods: The authors analyzed 82 patients with AML who were diagnosed according to World Health Organization criteria. The median patient age was 72 years (range, 29-87 years), and 27 patients (33%) had secondary AML. Of 62 patients with evaluable cytogenetics, 18 patients (29%) had a poor-risk karyotype, and 44 patients (71%) had an intermediate karyotype. Thirty-five patients (43%) received azacitidine as front-line treatment, and 47 patients (57%) had previously received 1 or more line of chemotherapy. Results: The overall response rate was 32% (26 of 82 patients) and included 12 (15%) complete remissions (CRs), 4 (5%) CRs with incomplete blood count recovery (CRi), and 10 (12%) partial responses (PRs). Responses were observed more frequently among untreated patients compared with pretreated patients; in fact, 17 of 35 untreated patients (48%) responded, including 11 responses (31%) classified as CR/CRi. Conversely, only 9 of 47 pretreated patients (19%) responded, including 5 responses (11%) that were classified as CR/Cri. The response rate was significantly higher for untreated patients (P =.006) and those who had white blood cell counts <10 × 10 ⁹/L (P =.006). For untreated patients who achieved a response, the median overall response duration was 13 months, and the 1-year and 2-years overall survival rates were 58% and 24%, respectively. Conclusions: The current results indicated that azacitidine promises to be an effective therapy for elderly patients with untreated AML and with white blood cell counts <10 × 10 ⁹/L.