Real Life Data on Efficacy and Safety of Azacitidine Therapy for Myelodysplastic Syndrome, Chronic Myelomonocytic Leukemia and Acute Myeloid Leukemia

Abstract

The administration of azacitidine (AZA) was found to be more effective than conventional care regimen (CCR) in patients with higher-risk myelodysplastic syndromes (MDS), chronic myelomonocytic leukemia (CMML) and acute myeloid leukemia (AML) with lower blast count. We designed a study to determine efficacy and safety of AZA therapy in “real life” patients with MDS, CMML and AML. The study included 83 patients (65% male) with a median age at diagnosis of 68 years. 43 patients were diagnosed with higher-risk MDS, 30 had AML and 10-CMML. Median AZA dose was comparable between treated groups. AZA dose reduction was required for 44% of MDS, 17% of AML and 25% of CMML patients. Complete remission (CR) was achieved in 14% of MDS, 7% of AML and 10% of CMML patients. Overall response rate was following: 27% for MDS, 20% for AML and 20% for CMML. Estimated OS at 12 months was 75% for MDS, 60% for AML and 75% for CMML. Median follow-up for MDS/AML/CMML from AZA initiation to last follow-up was 9.0, 9.4 and 9.4 months, respectively. The most common toxicity of AZA therapy was myelosuppression and infections. AZA treatment was effective in a limited number of patients with acceptable safety profile.

Successful Long-Term Treatment with Azacitidine in Patient with Chronic Myelomonocytic Leukemia

“…Duration of the response varied between 3 and 24 months. 13 In our case, although the patient had proliferative features of CMML, OS was 11 years and 5 months, which is far longer than OS mentioned in previous publications. Patient’s quality of life on azacitidine was seemingly unimpaired, putting aside some minor respiratory infections and low grades azacitidine related adverse reactions.…”

“… 17 Myelosuppression and infections were mostly observed and are responsible for treatment delay. 13 We reduced azacitidine dose due to neutropenia several times. And postponed some of the applications because of minor respiratory infections which normally occurred in 2.5% of azacitidine treated patients.…”

Hypomethylating Chemotherapeutic Agents as Therapy for Myelodysplastic Syndromes and Prevention of Acute Myeloid Leukemia

“…However, febrile neutropenia, anemia, neutropenic sepsis, and pneumonia were noticed in a minimal percentage of patients in the AZA PH GL 2003 CL 001 clinical study. Myelosuppression and infections are common forms of toxicity experienced by patients with subcutaneous azacitidine treatment [ 103 ]. Moreover, subcutaneous administration of Vidaza leads to post-injection site erythema, ecchymosis, and inflammation, potentially causing swelling, itching, pain, redness, warmth, rash, or hives [ 91 ].…”

High-dose regimens of hypomethylating agents promote transfusion independence in IPSS lower-risk myelodysplastic syndromes: a meta-analysis of prospective studies

“…The AEs of higher-dose regiments warrant consideration because the myelosuppressive effects of HMAs can accumulate with increasing doses. A lack of data hindered our analysis of AEs in patients with LR-MDS treated with AZA 75×7, a regimen that is widely used for AML [ 36 – 38 ] and higher-risk MDS [ 39 , 40 ] that is associated with a higher rate of hematologic AEs than what was shown by our pooled data. Thus, although higher-dose regimens can achieve better response or TI, AEs would potentially occur more frequently than with lower doses.…”