Azacitidine Accord: Uses, Dosage & Side Effects
Hypomethylating Agent — Epigenetic therapy for myelodysplastic syndromes and acute myeloid leukaemia
Quick Facts About Azacitidine Accord
Key Takeaways About Azacitidine Accord
- Maintenance therapy for AML: Oral azacitidine is used as maintenance therapy in adults with acute myeloid leukaemia who achieved remission after induction chemotherapy but are not candidates for haematopoietic stem cell transplant
- Epigenetic mechanism: Azacitidine works by inhibiting DNA methyltransferases, reactivating tumour suppressor genes that cancer cells have silenced through abnormal DNA methylation
- Regular blood monitoring essential: Complete blood counts must be checked before each treatment cycle because azacitidine commonly causes myelosuppression (low white blood cells, platelets, and red blood cells)
- Cyclic dosing schedule: Oral azacitidine is taken on days 1 through 14 of each 28-day cycle, followed by a 14-day rest period. The standard dose is 200 mg (one tablet) taken at approximately the same time each day
- Pregnancy contraindicated: Azacitidine can cause serious birth defects. Both male and female patients must use effective contraception during treatment and for a defined period after the last dose
What Is Azacitidine Accord and What Is It Used For?
Azacitidine Accord contains azacitidine, a pyrimidine nucleoside analogue classified as a hypomethylating agent (HMA). It is used for the treatment of myelodysplastic syndromes (MDS), chronic myelomonocytic leukaemia (CMML), and acute myeloid leukaemia (AML). The oral tablet formulation is specifically designed for continued maintenance therapy in AML patients who have achieved remission after intensive induction chemotherapy.
Azacitidine Accord is a prescription-only medication that represents a significant advance in the epigenetic treatment of haematological malignancies. Myelodysplastic syndromes and acute myeloid leukaemia are serious conditions affecting the bone marrow’s ability to produce healthy blood cells. According to the World Health Organization, AML accounts for approximately 80% of adult leukaemias, with an estimated 120,000 new cases diagnosed globally each year. MDS affects approximately 4 per 100,000 people annually, with incidence rising significantly in individuals over 70 years of age.
In healthy bone marrow, haematopoietic stem cells follow a carefully regulated programme of cell division and differentiation to produce mature blood cells — red blood cells for carrying oxygen, white blood cells for fighting infections, and platelets for blood clotting. In MDS and AML, genetic and epigenetic changes disrupt this process, causing the bone marrow to produce abnormal, immature blood cells (blasts) that do not function properly and crowd out healthy cells. This leads to cytopenias — low counts of one or more blood cell types — causing symptoms such as fatigue, increased susceptibility to infections, and easy bruising or bleeding.
One of the critical mechanisms driving these malignancies is aberrant DNA methylation. In cancer cells, the promoter regions of tumour suppressor genes become excessively methylated (hypermethylated), which silences these genes and allows the cancer cells to grow and divide without normal regulatory controls. Azacitidine targets this epigenetic abnormality by inhibiting the enzymes responsible for DNA methylation, effectively “switching back on” the silenced tumour suppressor genes.
Azacitidine is a chemical analogue of cytidine, one of the four nucleoside building blocks of DNA and RNA. Once inside the cell, azacitidine is phosphorylated to its active triphosphate form and incorporated into both DNA and RNA. When incorporated into DNA, azacitidine forms irreversible covalent bonds with DNA methyltransferases (DNMTs), particularly DNMT1, DNMT3a, and DNMT3b. This traps the enzymes and leads to their degradation, resulting in progressive loss of DNA methylation (hypomethylation) with each round of cell division. The resulting reactivation of silenced tumour suppressor genes can restore normal cell differentiation, growth control, and apoptosis. At higher concentrations, direct incorporation of azacitidine into DNA and RNA causes structural damage to nucleic acids, disrupting protein synthesis and triggering direct cytotoxicity.
Approved Indications
Azacitidine is approved for use in the following clinical settings, depending on the formulation:
- Oral azacitidine (tablets) — AML maintenance therapy: For continued treatment of adults with AML who achieved first complete remission (CR) or complete remission with incomplete blood count recovery (CRi) following intensive induction chemotherapy with or without consolidation treatment, and who are not candidates for, or choose not to proceed to, haematopoietic stem cell transplantation (HSCT). The landmark QUAZAR AML-001 trial demonstrated that oral azacitidine maintenance significantly improved overall survival compared to placebo (median 24.7 months vs. 14.8 months).
- Injectable azacitidine — MDS and CMML: For the treatment of adult patients with higher-risk MDS (intermediate-2 or high-risk by IPSS), CMML with 10–29% marrow blasts without myeloproliferative disorder, and AML with 20–30% blasts and multi-lineage dysplasia who are not eligible for haematopoietic stem cell transplantation.
- Injectable azacitidine — AML: For the treatment of adult patients with AML with greater than 30% bone marrow blasts who are not eligible for haematopoietic stem cell transplantation, often in combination with venetoclax as first-line therapy for patients unfit for intensive chemotherapy.
It is important to understand that the oral and injectable formulations of azacitidine have different approved indications, different dosing schedules, and are not interchangeable. The oral formulation (represented by Azacitidine Accord 200 mg film-coated tablets) is specifically designed for the maintenance therapy setting in AML, while the injectable formulation is used as primary treatment for MDS, CMML, and treatment-naïve AML.
The QUAZAR AML-001 Trial
The approval of oral azacitidine for AML maintenance therapy was based on the QUAZAR AML-001 study, a landmark phase 3, randomised, double-blind, placebo-controlled, international trial published in the New England Journal of Medicine in 2020. This trial enrolled 472 patients aged 55 years and older with AML who achieved CR or CRi after intensive induction chemotherapy. Patients were randomised to receive either oral azacitidine 200 mg or placebo on days 1–14 of each 28-day cycle.
The results showed that oral azacitidine significantly improved overall survival (median 24.7 months vs. 14.8 months; hazard ratio 0.69; p = 0.0009) and relapse-free survival (median 10.2 months vs. 4.8 months; hazard ratio 0.65; p < 0.001) compared to placebo. These results established oral azacitidine as the first maintenance therapy to demonstrate an overall survival benefit in AML and changed the standard of care for post-remission management of AML in patients not proceeding to transplantation.
What Should You Know Before Taking Azacitidine Accord?
Before starting azacitidine, your haematologist will evaluate your complete blood counts, liver and kidney function, and overall health status. Azacitidine is contraindicated in patients with advanced malignant hepatic tumours, during pregnancy and breastfeeding, and in patients with known hypersensitivity to azacitidine or mannitol. Regular laboratory monitoring is essential throughout the entire course of treatment.
Treatment with azacitidine should only be initiated and supervised by a physician experienced in the use of chemotherapeutic agents and the management of haematological malignancies. Before your first dose, your medical team will conduct a thorough assessment to ensure that azacitidine is appropriate and safe for you. This assessment typically includes a comprehensive medical history, physical examination, complete blood count with differential, liver function tests (including serum bilirubin, ALT, AST, and alkaline phosphatase), kidney function tests (serum creatinine, estimated GFR), and serum electrolytes.
Contraindications
Azacitidine must not be used in the following circumstances:
- Hypersensitivity: Known allergy to azacitidine, mannitol, or any of the other excipients in the formulation. Severe allergic reactions including anaphylaxis have been reported with azacitidine use.
- Advanced malignant hepatic tumours: Azacitidine is contraindicated in patients with advanced malignant liver tumours due to the risk of fatal hepatic complications. Cases of hepatic failure leading to death have been reported in patients with extensive tumour burden.
- Pregnancy: Azacitidine has demonstrated embryotoxicity and teratogenicity in animal studies. It must not be used during pregnancy. Women of childbearing potential must use effective contraception during treatment and for 6 months after the last dose. A pregnancy test should be performed before starting treatment.
- Breastfeeding: It is not known whether azacitidine or its metabolites are excreted in human milk. Due to the potential for serious adverse reactions in nursing infants, breastfeeding must be discontinued during treatment and for 1 week after the last dose.
Warnings and Precautions
Several important precautions must be considered during treatment with azacitidine:
- Myelosuppression: Azacitidine frequently causes neutropenia, thrombocytopenia, and anaemia, which can be severe. Complete blood counts must be monitored before the start of each treatment cycle. Dose adjustments, treatment delays, or growth factor support may be necessary. Patients should be instructed to report signs of infection (fever, sore throat), bleeding (bruising, blood in urine or stool), or severe fatigue immediately.
- Hepatotoxicity: Liver enzyme elevations and, rarely, hepatic failure have been reported. Liver function should be monitored before initiation and periodically during treatment. Use with caution in patients with pre-existing liver disease.
- Renal toxicity: Renal tubular acidosis, hypokalaemia, and elevated serum creatinine have been reported, particularly with the injectable formulation. Kidney function should be monitored, especially in patients with pre-existing renal impairment.
- Gastrointestinal toxicity: Nausea, vomiting, and diarrhoea are very common and can occasionally be severe. Anti-emetic prophylaxis may be prescribed. Patients should report persistent vomiting or diarrhoea to prevent dehydration and electrolyte imbalances.
- Tumour lysis syndrome (TLS): Although rare with azacitidine, TLS may occur in patients with high tumour burden. Adequate hydration and monitoring of uric acid, potassium, calcium, phosphate, and renal function are recommended in high-risk patients.
Pregnancy and Breastfeeding
Azacitidine is classified as potentially teratogenic based on animal studies and its mechanism of action. In preclinical studies, azacitidine caused embryotoxicity, reduced fetal weight, and developmental abnormalities at doses below those used clinically. The following reproductive precautions are essential:
- Female patients: Must use highly effective contraception during treatment and for at least 6 months after the last dose. A negative pregnancy test is required before starting therapy.
- Male patients: Should use effective contraception during treatment and for at least 3 months after the last dose. Azacitidine may affect sperm quality and male fertility. Patients should be counselled about sperm preservation before starting treatment.
- Breastfeeding: Must be discontinued during treatment and for at least 1 week after the last dose due to the potential risk to the nursing infant.
Do not start, stop, or change the dose of azacitidine without your haematologist’s guidance. Azacitidine is a chemotherapy agent that requires regular blood monitoring. Contact your healthcare team immediately if you develop fever, signs of infection, unusual bleeding or bruising, persistent vomiting or diarrhoea, or any other concerning symptoms.
How Does Azacitidine Accord Interact with Other Drugs?
Azacitidine has the potential to interact with several other medications. Clinically significant interactions may occur with other myelosuppressive agents, hepatotoxic drugs, nephrotoxic agents, and live vaccines. Always inform your haematologist about all medications, supplements, and herbal products you are taking before starting azacitidine therapy.
Drug interactions with azacitidine can occur through several mechanisms, including additive toxicities (particularly bone marrow suppression and organ damage), pharmacokinetic interactions affecting drug metabolism, and pharmacodynamic interactions affecting drug efficacy. Because azacitidine is primarily metabolised by spontaneous hydrolysis and cytidine deaminase rather than cytochrome P450 enzymes, classical CYP-mediated drug interactions are less common. However, clinically important interactions still exist and require careful management.
Your haematologist will review your complete medication list before starting azacitidine and at regular intervals during treatment. This includes prescription medications, over-the-counter drugs, vitamins, supplements, and herbal remedies. Some substances may need to be discontinued, have their dose adjusted, or require more intensive monitoring when used concurrently with azacitidine.
| Drug / Class | Interaction Type | Clinical Significance | Management |
|---|---|---|---|
| Live vaccines (MMR, varicella, yellow fever) | Pharmacodynamic | Risk of disseminated vaccine infection due to immunosuppression | Contraindicated during treatment and for a period after. Use inactivated vaccines instead. |
| Venetoclax | Additive myelosuppression | Increased risk of severe cytopenias, tumour lysis syndrome | Used as approved combination; requires close monitoring, dose adjustments per protocol |
| Cytarabine | Pharmacodynamic antagonism | Both agents compete for intracellular phosphorylation; may reduce efficacy of one or both agents | Avoid concurrent use. Sequential administration may be considered. |
| Methotrexate | Additive toxicity | Increased risk of myelosuppression and hepatotoxicity | Avoid combination if possible. If necessary, intensify monitoring. |
| Hepatotoxic agents (paracetamol high dose, statins, antifungals) | Additive hepatotoxicity | Increased risk of liver enzyme elevations and hepatic injury | Monitor liver function more frequently. Consider alternatives. |
| Nephrotoxic agents (aminoglycosides, NSAIDs, contrast media) | Additive nephrotoxicity | Increased risk of renal impairment | Monitor renal function closely. Ensure adequate hydration. |
| Anticoagulants (warfarin, DOACs) | Increased bleeding risk | Azacitidine-induced thrombocytopenia increases bleeding risk with anticoagulants | Monitor platelet counts and coagulation parameters frequently. Adjust anticoagulant dosing as needed. |
Major Interactions
The most clinically significant interactions involve agents that can exacerbate azacitidine’s bone marrow suppressive effects. Concurrent use with other myelosuppressive chemotherapy agents (such as cytarabine, hydroxyurea, or lenalidomide) requires careful dose coordination and intensified blood count monitoring. The combination of azacitidine with venetoclax has become a standard first-line treatment for AML patients ineligible for intensive chemotherapy, but this combination requires adherence to specific dosing protocols, including dose ramp-up for venetoclax and tumour lysis syndrome prophylaxis.
Live and live-attenuated vaccines must be avoided during treatment with azacitidine because the drug’s immunosuppressive effects could lead to uncontrolled replication of the vaccine organism, potentially causing serious or fatal infections. Inactivated vaccines may be administered, but their efficacy may be reduced due to the immunosuppressed state. Household contacts of patients should also avoid live oral polio vaccine.
Minor Interactions
Azacitidine undergoes hydrolysis and deamination rather than cytochrome P450-mediated metabolism, which means that classical CYP inhibitors and inducers (such as ketoconazole, rifampicin, or grapefruit juice) are unlikely to significantly alter azacitidine levels. However, drugs that affect renal tubular secretion could theoretically alter azacitidine clearance. Proton pump inhibitors (PPIs) and H2 blockers do not appear to significantly affect the absorption of oral azacitidine.
Over-the-counter supplements containing high-dose vitamin C (ascorbic acid) have been theoretically proposed to interfere with hypomethylating agent activity, although clinical evidence for this interaction remains limited and inconclusive. Patients should discuss all supplement use with their haematologist.
What Is the Correct Dosage of Azacitidine Accord?
The recommended dose of oral azacitidine (Azacitidine Accord 200 mg tablets) for AML maintenance therapy is 200 mg (one tablet) taken once daily on days 1 through 14 of each 28-day treatment cycle. Treatment is continued until disease relapse, unacceptable toxicity, or patient decision to discontinue. Dose modifications may be required based on blood count results and tolerability.
Dosing of azacitidine requires precise adherence to the prescribed schedule. The oral formulation uses a cyclic dosing pattern that mirrors the biological rationale of the drug: the 14 days of treatment allow sufficient drug exposure for DNA hypomethylation to occur, while the 14-day rest period allows normal haematopoietic progenitor cells to recover before the next cycle. This approach maximises the therapeutic epigenetic effects while minimising cumulative bone marrow toxicity.
Your haematologist will determine the optimal dosing schedule and make any necessary dose adjustments based on your individual clinical response and tolerance. It is essential that you take the medication exactly as prescribed and do not alter the dose or schedule without medical guidance. The tablets should be swallowed whole with water at approximately the same time each day. They should not be split, crushed, or chewed.
Adults
Standard Maintenance Dosing (Oral Tablets)
Dose: 200 mg (one tablet) once daily
Schedule: Days 1–14 of each 28-day cycle
Duration: Continue until disease relapse, unacceptable toxicity, or patient choice
Administration: Take with or without food (avoid high-fat meals). Swallow whole with water at the same time each day.
Dose Modifications for Haematological Toxicity
If blood counts fall below certain thresholds, your haematologist may:
- Delay the start of the next cycle until counts recover
- Reduce the number of dosing days from 14 to 7 days per cycle
- Prescribe granulocyte colony-stimulating factor (G-CSF) to support neutrophil recovery
- Administer red blood cell or platelet transfusions as needed
| Parameter | Threshold | Action |
|---|---|---|
| Neutrophils (ANC) | < 0.5 × 10⁹/L | Delay cycle until ANC ≥ 0.5 × 10⁹/L. Consider G-CSF support. May reduce dosing days. |
| Platelets | < 25 × 10⁹/L | Delay cycle until platelets ≥ 25 × 10⁹/L. Platelet transfusion if clinically indicated. |
| Haemoglobin | < 80 g/L | Consider red blood cell transfusion. Assess for bleeding or haemolysis. |
| Liver enzymes (ALT/AST) | > 3× upper limit of normal | Interrupt treatment until normalisation. Investigate cause. May restart at same dose. |
| Serum creatinine | ≥ 2× baseline or ≥ 176 μmol/L | Interrupt treatment. Assess renal function. Restart when creatinine returns to baseline. |
Children
The safety and efficacy of oral azacitidine have not been established in children and adolescents under 18 years of age. There are currently no approved paediatric indications for this medication. Any use in paediatric patients would be considered off-label and should only be considered within the context of a clinical trial or under exceptional circumstances with specialist paediatric haematology oversight.
Elderly
No specific dose adjustment is required based on age alone. However, elderly patients (particularly those over 75 years) are more likely to have reduced organ function and may be at higher risk for myelosuppressive complications. The QUAZAR AML-001 trial included patients aged 55 years and older (median age approximately 68 years), and the survival benefit of oral azacitidine was consistent across age subgroups. Nevertheless, careful monitoring and proactive management of cytopenias are particularly important in this population. Renal and hepatic function should be assessed before each cycle, as age-related declines may affect drug clearance.
Missed Dose
If you miss a dose of azacitidine on the same day, take it as soon as you remember, provided it is still the same calendar day. If the entire day has passed and you do not remember until the next day, do not take a double dose to make up for the missed one — simply take the next scheduled dose. If vomiting occurs within 30 minutes of taking a dose, you may retake the same dose on the same day. If vomiting occurs more than 30 minutes after taking a dose, do not retake the dose; take the next dose as scheduled. Record any missed doses and inform your haematologist at your next appointment.
Overdose
There is limited clinical experience with overdose of oral azacitidine. In the event of an overdose, the primary concern would be exaggerated myelosuppression leading to severe neutropenia, thrombocytopenia, and anaemia. There is no specific antidote for azacitidine overdose. Management should be supportive and symptomatic, including close monitoring of blood counts, administration of growth factors (G-CSF) for neutropenia, platelet transfusions for severe thrombocytopenia, and red blood cell transfusions for symptomatic anaemia. Contact your haematologist or emergency services immediately if you suspect an overdose.
What Are the Side Effects of Azacitidine Accord?
Like all chemotherapy agents, azacitidine can cause side effects. The most common are gastrointestinal symptoms (nausea, vomiting, diarrhoea), myelosuppression (low blood counts), and fatigue. Most side effects are manageable with supportive care and dose adjustments. However, some side effects can be serious and require immediate medical attention.
Side effects of azacitidine result from its mechanism of action: as a nucleoside analogue that incorporates into DNA and RNA, it affects not only cancer cells but also rapidly dividing normal cells, particularly those in the bone marrow, gastrointestinal lining, and immune system. The severity and frequency of side effects vary between individuals and may be influenced by factors such as age, organ function, concurrent medications, and the specific disease being treated.
It is important to remember that the benefit of azacitidine therapy in extending survival and maintaining remission generally outweighs the risks of side effects. Your haematology team will monitor you closely and use supportive treatments to minimise the impact of side effects on your quality of life. Always report any new or worsening symptoms to your healthcare team promptly, as early intervention can prevent complications.
The following side effect frequencies are based on data from clinical trials and post-marketing surveillance reports:
Very Common Side Effects
- Nausea (reported in up to 65% of patients)
- Vomiting (up to 60%)
- Diarrhoea (up to 50%)
- Constipation
- Abdominal pain
- Neutropenia (low neutrophil count — increases infection risk)
- Thrombocytopenia (low platelet count — increases bleeding risk)
- Anaemia (low red blood cell count — causes fatigue, shortness of breath)
- Fatigue and weakness
- Decreased appetite
- Febrile neutropenia (fever during period of low white cell count)
- Upper respiratory tract infections
- Urinary tract infections
- Pneumonia
Common Side Effects
- Elevated liver enzymes (ALT, AST)
- Hypokalaemia (low potassium)
- Dizziness
- Headache
- Arthralgia (joint pain)
- Insomnia
- Dyspnoea (shortness of breath)
- Cough
- Petechiae (tiny red spots on skin from minor bleeding)
- Weight loss
- Peripheral oedema (swelling in legs/ankles)
- Back pain
- Sepsis
Uncommon Side Effects
- Renal tubular acidosis
- Tumour lysis syndrome
- Hepatic failure
- Progressive multifocal leukoencephalopathy (PML)
- Interstitial lung disease
- Sweet syndrome (acute febrile neutrophilic dermatosis)
- Severe skin reactions
- Differentiation syndrome
Rare Side Effects
- Anaphylactic reactions
- Necrotising fasciitis
- Pyoderma gangraenosum
- Hepatic coma
- Cardiac failure (in patients with pre-existing cardiac risk factors)
Contact your haematologist or go to your nearest emergency department immediately if you experience: fever above 38°C (especially if your white blood cell count is low), signs of severe infection (chills, rapid breathing, confusion), unusual or uncontrollable bleeding or bruising, blood in urine or stool, persistent vomiting or diarrhoea causing dehydration, sudden shortness of breath, chest pain, yellowing of the skin or eyes (jaundice), or severe skin reactions.
How Should You Store Azacitidine Accord?
Store Azacitidine Accord tablets at room temperature, below 25°C (77°F), in the original packaging to protect from moisture and light. Keep out of reach of children. Do not use after the expiry date printed on the packaging.
Proper storage of azacitidine tablets is essential to maintain the medication’s stability and effectiveness. As a chemotherapy agent, special care should be taken in handling and storing this medication. The following storage guidelines should be followed:
- Temperature: Store below 25°C (77°F). Do not freeze. Brief excursions to temperatures up to 30°C may be tolerated but should be avoided when possible.
- Packaging: Keep the tablets in their original blister packaging until immediately before use. The blister packaging provides protection from moisture and light, which can degrade the active substance.
- Light protection: Avoid prolonged exposure to direct sunlight or strong artificial light.
- Children and pets: Store securely out of reach of children and pets. As a cytotoxic medication, accidental ingestion could be dangerous.
- Expiry date: Do not use the tablets after the expiry date shown on the blister and carton. The expiry date refers to the last day of that month.
- Disposal: Do not dispose of azacitidine tablets in household waste or via wastewater. As a cytotoxic agent, it requires special disposal. Return unused or expired tablets to your pharmacy for safe disposal according to local regulations for hazardous pharmaceutical waste.
If you notice any visible changes in the appearance of the tablets (such as discolouration, crumbling, or an unusual odour), do not take them and consult your pharmacist. Transport the medication in its original packaging, and if you are travelling, keep it in your hand luggage to avoid temperature extremes in aircraft cargo holds.
What Does Azacitidine Accord Contain?
Each Azacitidine Accord film-coated tablet contains 200 mg of azacitidine as the active ingredient, along with pharmaceutical excipients necessary for tablet formulation, stability, and absorption.
Understanding the composition of your medication can be important, particularly if you have known allergies or intolerances to specific pharmaceutical ingredients. The complete qualitative composition of Azacitidine Accord 200 mg film-coated tablets is as follows:
Active Ingredient
- Azacitidine: 200 mg per tablet. Azacitidine (also known as 5-azacytidine) is a pyrimidine nucleoside analogue of cytidine with the molecular formula C₈H₁₂N₄O₅ and a molecular weight of 244.20 g/mol. It is a white to off-white powder that is soluble in water.
Excipients (Inactive Ingredients)
The tablet core and film coating contain pharmaceutical excipients that serve specific functions in the formulation:
- Tablet core: Croscarmellose sodium (disintegrant — helps the tablet break apart for absorption), microcrystalline cellulose (binder/filler), mannitol (filler/diluent), magnesium stearate (lubricant — prevents the tablet from sticking to manufacturing equipment), colloidal anhydrous silica (flow agent)
- Film coating: Hypromellose (coating polymer), titanium dioxide E171 (opacifier/colorant), talc (anti-tack agent), iron oxide red E172 (colourant)
Patients with known hypersensitivity to mannitol or any other excipient should not take this medication and should discuss alternatives with their haematologist. The tablets are lactose-free and gluten-free. Each tablet contains less than 1 mmol sodium (23 mg), meaning the formulation is essentially “sodium-free.”
Frequently Asked Questions About Azacitidine Accord
Medical References & Sources
All information is based on peer-reviewed research, international medical guidelines, and regulatory agency documentation.
- Wei AH, Döhner H, Pocock C, et al. Oral Azacitidine Maintenance Therapy for Acute Myeloid Leukemia in First Remission. N Engl J Med. 2020;383(26):2526-2537. doi:10.1056/NEJMoa2004444
- European Medicines Agency (EMA). Onureg (azacitidine) – Summary of Product Characteristics. Available at: www.ema.europa.eu
- U.S. Food and Drug Administration (FDA). ONUREG (azacitidine tablets) Prescribing Information. Approved September 2020.
- National Comprehensive Cancer Network (NCCN). Clinical Practice Guidelines in Oncology: Acute Myeloid Leukemia, Version 3.2025.
- Döhner H, Wei AH, Appelbaum FR, et al. Diagnosis and management of AML in adults: 2022 recommendations from an international expert panel on behalf of the ELN. Blood. 2022;140(12):1345-1377. doi:10.1182/blood.2022016867
- Fenaux P, Mufti GJ, Hellström-Lindberg E, et al. Efficacy of azacitidine compared with that of conventional care regimens in the treatment of higher-risk myelodysplastic syndromes: a randomised, open-label, phase III study. Lancet Oncol. 2009;10(3):223-232. doi:10.1016/S1470-2045(09)70003-8
- World Health Organization (WHO). Model List of Essential Medicines, 23rd List (2023). Geneva: WHO.
- DiNardo CD, Jonas BA, Pullarkat V, et al. Azacitidine and Venetoclax in Previously Untreated Acute Myeloid Leukemia. N Engl J Med. 2020;383(7):617-629. doi:10.1056/NEJMoa2012971
- British National Formulary (BNF). Azacitidine – Drug Monograph. National Institute for Health and Care Excellence (NICE).
- Savona MR, Odenike O, Giles FJ, et al. A Phase 1 Study of Oral Azacitidine (CC-486) in Patients With Myelodysplastic Syndromes. Blood. 2016;128(22):1761. doi:10.1182/blood.V128.22.1761.1761
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