Efficacy of Azacitidine In the Treatment of Chronic Myelomonocytic Leukemia

Abstract

4017 Background: Chronic myelomonocytic leukemia (CMML) is a heterogeneous disease sharing features of myelodysplastic syndromes (MDS) and myeloproliferative neoplasms (MPN). FDA approved indications for azacitidine and decitabine include CMML as subset of MDS. Fewer than 10 patients with CMML, however, were treated in each of the original studies. In this study we report our institutional experience of azacitidine treatment of CMML patients. Methods: This was a retrospective review of CMML patients who received azacitidine at Moffitt Cancer Center. The primary endpoint was determining response rate to azacitidine utilizing International Working Group 2006 criteria (IWG 2006). Secondary objectives were to assess treatment tolerance and overall survival. Descriptive statistics were used for baseline characteristics and response rates. Kaplan-Meier estimates were used for evaluation of overall survival. Results: Between July 2004 and December 2009, 35 CMML patients treated with azacitidine were identified. Table-1 summarizes baseline characteristics of those patients. Based on Dusseldorf CMML risk criteria one patient (2.9%) was low risk, 17 (48.7%), intermediate, 7 (20%) high risk and 10 (28.6%) were unknown. According to MD Anderson CMML risk model, 11 (31.4%) were low risk, 12 (34.3%) int-1, 2 (5.7%) int-2, 1 (2.9%) high risk and 9(25.7%) unknown. The median number of azacitidine cycles was 6.0 (1-34) The best response rates by IWG 2006 criteria were complete response (CR) 5 (14.3%), marrow CR 4 (11.4%), partial response (PR) 1 (2.9%), and hematological improvement (HI) 7 (20%). The overall response rate was 48.6%. The median OS was 25 month (95%CI 13.8–36.1 mo). Conclusions: In this retrospective analysis, response to azacitidine in CMML was similar to response rates reported in other MDS patients on azacitidine studies. The median overall survival is comparable to AZA-001 randomized clinical study. Disclosures: Lancet: Celgene: Research Funding. List:Celgene: Research Funding. Komrokji:Celgene: Research Funding, Speakers Bureau.

Azacitidine has limited activity in ‘real life’ patients with MDS and AML: a single centre experience

“…Although our CMML group included a small number of patients, our overall response rates were comparable with those of Teichman et al . 17 (48.5% vs. 60% in our study). In this group, we observed drug‐related fever, increase in BUN, creatinine and amylase, nausea and dizziness as complications of Aza‐C.…”

“…One patient was receiving Aza-C maintenance every 3 months and doing well at 14þ months and the other two patients were alive under supportive care at 21þ and 43þ months at the time of study entry. Although our CMML group included a small number of patients, our overall response rates were comparable with those of Teichman et al [17] (48.5% vs. 60% in our study). In this group, we observed drug-related fever, increase in BUN, creatinine and amylase, nausea and dizziness as complications of Aza-C.…”