Decitabine is a cytosine analogue and an intravenously administered antineoplastic agent used in the therapy of myelodysplastic syndromes. Decitabine is associated with a low rate of transient serum enzyme elevations during therapy, but has not been implicated in causing clinically apparent liver injury with jaundice.


Decitabine (dee sye' ta been) is a pyrimidine analogue (5-aza-deoxy-cytidine) which is the deoxyribose form of 5-azacitidine. Decitabine is converted intracellularly to a triphosphate which becomes incorporated into DNA and appears to inhibit DNA methylation, resulting in increased expression of silenced genes including tumor suppressor genes. Studies done in vitro and in vivo have shown that decitabine induces differentiation of bone marrow cells and results in normalization of bone marrow in a proportion of patients with myelodysplasia. Decitabine was approved for use in the United States in 2006 and the current single indication is for therapy of myelodysplastic syndromes (MDS). Decitabine is available as a powder or solution for injection in 50 mg vials generically and under the trade name of Dacogen. The usual dosage regimen in adults is 15 to 20 mg/m2 of body surface area given intravenously (iv) in several day regimens, with repeat cycles every 4 or 6 weeks. A minimum of 4 courses is recommended. Common side effects include bone marrow suppression, nausea, vomiting, diarrhea, stomatitis, bruising, abdominal pain, myalgias, headache, dizziness, fatigue, fever, rash and pruritus.

Decitabine is poorly absorbed orally due to its metabolism by cytidine deaminase found in the intestine and liver. In 2020, cedazuridine, an oral inhibitor of cytidine deaminase, was approved for use with a fixed dose of oral decitabine as treatment of adults with MDS. This oral combination therapy yielded similar plasma concentrations as iv administered decitabine and had similar effects on DNA methylation and clinical responses. The adverse event rates to the oral combination therapy were similar to those of iv decitabine, although direct comparisons were limited to single initial cycles of therapy. The oral form of decitabine is available only as a fixed combination with cedazuridine as tablets of 35 mg of decitabine and 100 mg of cedazuridine under the brand name Inqovi. The oral administration of decitabine avoids the inconvenience and difficulties of its iv administration on multiple days of each course of therapy.


In early clinical trials using high doses of decitabine, serum enzyme elevations occurred in up to 16% of patients with underlying liver disease or liver metastases, but rarely in persons without hepatic illness. In subsequent studies, serum ALT elevations were reported in 5% to 15% of treated patients, but all were self-limited and no clinically apparent liver injury was reported. Recent studies have reported elevations in serum bilirubin levels in 7% to 12% of treated patients, but the elevations resolved rapidly and were not associated with other clinical or laboratory evidence of liver injury. Monitoring of serum enzyme levels during treatment is recommended only in patients with concurrent liver disease.

The oral combination therapy of decitabine with cedazuridine appears to have a similar frequency and pattern of adverse events as iv decitabine alone. In several prospective clinical trials, single cycles of oral vs iv decitabine had similar rates of aminotransferase elevations and long term, multi-course regimens of the oral fixed dose combination therapy resulted in serum aminotransferase elevations in 20% to 37% of patients, which were above 5 times the upper limit of normal (ULN) in 2% to 3% with no cases of clinically apparent liver injury attributable to the chemotherapeutic agent.

Thus, despite widescale use as therapy of MDS, decitabine has not been convincingly linked to cases of clinically apparent liver injury. Nevertheless, the frequency of serum enzyme elevations with therapy make it difficult to say that decitabine is totally without potential for causing drug induced liver injury.

Likelihood score: E* (unproven but suspected, rare cause of clinically apparent liver injury).

Mechanism of Injury

Hepatotoxicity from decitabine appears to be rare and confined mostly to asymptomatic elevations in serum enzymes in patients receiving the highest doses or with underlying liver disease. Thus, the liver injury is likely due to direct toxicity which is generally minimal or mild except in susceptible patients.

Outcome and Management

The severity of the liver injury linked to decitabine therapy is usually mild in severity and without accompanying symptoms or jaundice. Decitabine has not been linked to cases of severe acute hepatitis, acute liver failure, chronic hepatitis or vanishing bile duct syndrome. There is no information on cross sensitivity to hepatic injury between decitabine and other cytidine analogues including azacitidine.

Drug Class: Antineoplastic Agents

Other drugs for myelodysplastic syndromes: Azacitidine, Cedazuridine, Luspatercept



Decitabine – Generic, Dacogen®

Decitabine and Cedazuridine – Inqovi®


Antineoplastic Agents


COMPLETE LABELING (Decitabine and Cedazuridine)

Product labeling at DailyMed, National Library of Medicine, NIH



References updated: 27 July 2023

Abbreviations: AML, acute myelogenous leukemia; iv, intravenous; MDS, myelodysplastic syndrome; sc, subcutaneously.

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