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Azacitidine (By injection)

What works?

Learn more about the effects of these drugs. The most reliable research is summed up for you in our featured article.

Azacitidine belongs to the group of medicines known as antimetabolites. It is used to treat some kinds of cancer. Azacitidine interferes with the growth of cancer cells, which are eventually destroyed. Since the growth of normal body cells may also be affected by azacitidine, other effects will also occur. Some of these may be serious and must be reported to your doctor. Some effects may not occur… Read more
Brand names include
Novaplus Azacitidine, PremierPro Rx azaCITIDine, Vidaza
Drug classes About this
Antineoplastic Agent

What works? Research summarized

Evidence reviews

Comparison between decitabine and azacitidine for the treatment of myelodysplastic syndrome: a meta-analysis with 1392 participants

The hypomethylating agents decitabine and azacitidine have been found to improve the outcome of patients with myelodysplastic syndrome (MDS); however, the clinical choice between them is controversial. Therefore, this meta-analysis was performed to compare the efficacy, toxicity, and survival advantage of decitabine and azacitidine in patients with MDS. Eleven trials with a total of 1392 patients with MDS (decitabine, n = 768; azacitidine, n = 624) were included for analysis. The pooled estimates of partial response, hematologic improvement, and overall response rates for azacitidine were significantly higher than for decitabine. There were no differences between these 2 drugs regarding complete response, red blood cell transfusion-independent rates, and grade 3 or 4 hematologic toxicity. When compared with best supportive care, azacitidine significantly improved overall survival (hazard ratio [HR], 0.69; 95% CI, 0.54-0.87) and time to acute myeloid leukemia transformation (HR, 0.51; 95% CI, 0.35-0.74). But these benefits were not found with decitabine. Among patients with higher risk (International Prognostic Scoring System value of 3) or older than 75 years, treatment with azacitidine was a favorable factor, whereas decitabine showed no advantage. Therefore, with higher overall response rates and better survival benefits, azacitidine is recommended as the first-line hypomethylating agent for MDS, especially in elderly patients or those with high risk.

5-azacitidine prolongs overall survival in patients with myelodysplastic syndrome: a systematic review and meta-analysis

This well-conducted review found that hypomethylating agents, particularly 5-azacitidine, improved survival and other outcomes compared with conventional care, but were associated with significant adverse events. These conclusions are likely to be reliable.

Hydroxyurea for the Treatment of Sickle Cell Disease

To synthesize the published literature on the efficacy, effectiveness, and toxicity of hydroxyurea (HU) when used for treatment of sickle cell disease (SCD); and to review the evidence regarding barriers to its use.

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Summaries for consumers

Myelodysplastic Syndromes Treatment (PDQ®): Patient Version

Expert-reviewed information summary about the treatment of myelodysplastic syndromes.

PubMed Health Blog...

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