Вайдаза

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Вайдаза

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Overview of Вайдаза

Quick Facts

Property Description
Active ingredient Azacitidine
Form Lyophilized powder for suspension/solution
Pharmacological class Hypomethylating agent, Antimetabolite
Common use Specialized treatment for blood and bone marrow disorders
Origin Synthetic compound, Pyrimidine analog

What is Вайдаза and its Active Component Azacitidine?

The drug Вайдаза (a brand name for Azacitidine) is a synthetic, single-component medicinal preparation primarily classified as an antineoplastic agent. Its sole active ingredient is Azacitidine (C₈H₁₂N₄O₅), which is chemically characterized as a pyrimidine analog of the nucleoside cytidine. The substance is supplied as a sterile lyophilized powder intended for reconstitution into a suspension or solution, making it an injectable preparation for parenteral administration, typically via subcutaneous or intravenous injection. The preparation is designed exclusively for use under the direction of a physician experienced in specialized cancer therapies.

Classification of Вайдаза: Form, Origin, and Purpose

Azacitidine belongs to the specialized pharmacological class of hypomethylating agents, also broadly categorized as antimetabolites. This classification highlights its primary function as a DNA methyltransferase inhibitor. This unique mechanism promotes the hypomethylation of DNA in abnormal blood-forming cells. The general purpose of this therapy is to utilize targeted cellular modification to help inhibit the growth of pathological cells and support their differentiation, aiming to correct underlying cellular abnormalities in the bone marrow.

Regulatory References

  1. EMA
  2. antineoplastic agent
  3. Azacitidine
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What side effects are possible with Вайдаза?

Possible Side Effects and Safety Information

The following safety information for Vidaza (Azacitidine) is based strictly on authoritative government regulatory documents, detailing officially documented adverse reactions and safety restrictions. No dosing instructions, mechanism of action, or therapeutic benefits are included.

Adverse Reactions and Frequency

The most frequent and clinically significant adverse reactions are related to suppression of blood cell production (myelosuppression). The official classification uses the following frequency framework:

Classification Examples of Reactions
Very Common (ge 1/10) Low white blood cells (Neutropenia, Leukopenia), low platelets (Thrombocytopenia), low red cells (Anemia), Infections, Fever (Pyrexia), Nausea, Vomiting, Fatigue, Injection site reactions, Constipation.
Common (ge 1/100 to <1/10) Sepsis, Pneumonia, Pancytopenia, Hypersensitivity reactions, Renal failure, Bleeding in the digestive tract.
Uncommon (ge 1/1,000 to <1/100) Pericarditis, Interstitial lung disease, Progressive hepatic coma, Sweet’s syndrome.

Serious Safety Considerations

Serious adverse reactions documented in regulatory sources include severe myelosuppression (potentially leading to Bone Marrow Failure), life-threatening infections (Sepsis, Pneumonia), Intracranial Hemorrhage, Pericardial Effusion, and Progressive Hepatic Coma.

Safety-Related Restrictions and Limitations

  • Contraindications: The medicine is officially contraindicated (must not be used) in individuals with known hypersensitivity to azacitidine or mannitol, or in patients with advanced malignant hepatic tumors.
  • Mandatory Monitoring: Government labels require mandatory, continuous monitoring of Complete Blood Counts (CBC), Liver Function Tests, and Renal Function Tests before and during each cycle to manage toxicity and assess the need for dose modification.
  • Population Notes: Specific dose adjustments are required for patients with moderate to severe renal impairment.

This structure reflects the regulatory emphasis on managing hematologic risk and the mandatory requirements for patient surveillance.

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Overdose and Emergency Response

The official regulatory profile for Azacitidine (Вайдаза) overdose defines the specific signs and mandated emergency actions required in the event of over-exposure. Due to the drug’s intended effect, the primary consequence of an overdose is an intensification of adverse reactions, particularly severe bone marrow suppression.

Overdose Scope

Field Official Regulatory Information
Documented overdose presentations Intensification of adverse effects, severe gastrointestinal symptoms (diarrhea, nausea, vomiting), and severe hematologic toxicity
Physiological systems affected Hematologic system (severe myelosuppression, peripheral cytopenias), Gastrointestinal system
Population-specific overdose notes Patients with severe preexisting hepatic impairment or renal impairment are identified as requiring careful monitoring for increased toxicity.

Emergency Actions and Management

Field Official Regulatory Information
Antidote availability No specific antidote is known for Azacitidine overdose.
Mandated immediate help Patients must seek immediate medical attention for a suspected overdose. Emergency services (911) should be called immediately if the person has collapsed, had a seizure, has trouble breathing, or cannot be awakened.
Management measures Management is strictly symptomatic and supportive, requiring close monitoring of the patient’s hematologic parameters and overall condition.

This regulatory framework mandates urgent clinical intervention to manage the potential for severe, life-threatening outcomes, focusing on supportive care and the close observation of hematologic and renal function.

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Therapeutic Uses of Вайдаза

What Вайдаза treats: Main Uses and Benefits

The medication is used to address adult patients who are not eligible for haematopoietic stem cell transplantation (HSCT) and have higher-risk Myelodysplastic Syndromes (MDS), certain subtypes of Chronic Myelomonocytic Leukemia (CMML), and Acute Myeloid Leukemia (AML) in specific patient contexts. This therapeutic approach is relevant for supporting the management of the disease course and may assist with achieving long-term disease control.

The approach is commonly used for managing the symptom cluster of peripheral cytopenias—low counts of red blood cells, white blood cells, and platelets. The treatment may assist in supporting the improvement of blood cell counts, which generally helps lessen the patient's reliance on regular blood transfusions (transfusion dependence). This improvement may help address the burden of chronic fatigue and can support general well-being and daily function. The approach is relevant in supporting the management of the risk of the condition progressing to a more aggressive state, such as AML.


Quick Fact: Relief for Cytopenia-Related Symptoms

Property Description
Primary Indication Areas Higher-risk MDS, CMML, specific AML cases
Symptom Cluster Managed Peripheral cytopenias (low counts of blood cells)
Key Patient Benefit Supports the lessening of transfusion dependence
Clinical Goal Supporting the management of disease progression risk
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Eligibility and Restrictions for Use

The eligibility for Azacitidine (Vaidaza) is defined by official regulatory documentation, outlining specific populations for whom use is allowed, restricted, or strictly prohibited.

Contraindicated Populations

Use of the medicine is contraindicated in patients with a known hypersensitivity to the active substance Azacitidine or the excipient Mannitol. Use is also strictly prohibited in patients diagnosed with advanced malignant hepatic tumors. Due to the high risk of fetal harm, the medicine is contraindicated during pregnancy, and breast-feeding must be discontinued during treatment.

Age and Health Restrictions

The medicine is primarily indicated for adult patients. While the US label permits use in pediatric patients one month and older for Juvenile Myelomonocytic Leukemia (JMML), for the main indications, use is not recommended in children and adolescents (0-17 years) as safety and efficacy are not fully established.

Patients with renal impairment or severe preexisting hepatic impairment require close monitoring and caution. Additionally, caution is advised for patients with a history of severe congestive heart failure or unstable cardiac disease, as the safety profile in these specific populations has not been established by pivotal regulatory studies.

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What should I know about interactions with other medicines?

Metabolic and Pharmacokinetic Profile

The official regulatory documents state that formal clinical drug-drug interaction studies with Azacitidine for injection have not been conducted. Consequently, specific interaction patterns with co-administered medicinal products are not fully established. Regarding metabolism, data indicates that an interaction with common Cytochrome P450 (CYP) isoenzymes such as 1A2, 2C19, or 3A4/5 is unlikely, as the drug is not metabolized by these enzymes.

Population and Formulation Constraints

A significant timing and formulation restriction exists: the injectable Azacitidine product must not be substituted for oral products as they are not bioequivalent and have documented, non-equivalent pharmacokinetic properties. Furthermore, patients with severe pre-existing hepatic impairment or renal impairment require close monitoring. This caution reflects the increased risk of toxic reactions in these populations because Azacitidine and its metabolites are primarily eliminated by the kidneys, which can modify overall drug clearance.

Contraindications and Restrictions

The drug is formally contraindicated in patients with a documented hypersensitivity to Azacitidine or the excipient mannitol. Use is also restricted by a condition-specific constraint: it is contraindicated in patients with advanced malignant hepatic tumors.

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Mechanism of Action

Molecular Action on DNA Methyltransferases (DNMTs)

The drug's active metabolites are incorporated into the DNA of dividing cells, resulting in the covalent trapping and subsequent degradation of DNMT1 enzymes. This inhibition halts the enzymatic process responsible for maintaining methylation patterns on newly synthesized DNA. This molecular action triggers progressive DNA hypomethylation, which facilitates the reactivation of genes previously silenced by aberrant methylation. This cascade is linked to the increased expression of pathways involved in cellular differentiation.

Nucleoside Analog Incorporation and Anti-proliferative Effects

As a nucleoside analog, the drug is also significantly incorporated into the cell's RNA, causing functional errors that disrupt protein synthesis. This cascade introduces a component of direct cellular toxicity, existing alongside the epigenetic reprogramming. This dual mechanistic action results in the death of rapidly proliferating abnormal cells via apoptosis, affecting the cellular composition of the hematopoietic system.

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Dosage and Administration Information

The administration of Вайдаза (Azacitidine for injection) follows defined routes, dosing, and schedules. The medicine is administered through either subcutaneous injection or intravenous infusion.

Standard Dosing Schedule

Treatment follows a 28-day cycle, consisting of a starting dose of 75 mg/m² of body surface area given once daily for 7 consecutive days, followed by a 21-day treatment-free period. Subsequent cycles are repeated every 4 weeks. The dose may be increased to 100 mg/m² only if no beneficial effect is observed after the first two cycles and no toxicity other than nausea or vomiting has occurred. Treatment is continued for a minimum of 4 to 6 cycles.

Preparation and Administration

For administration, the 100 mg lyophilized powder vial requires reconstitution with sterile water. The reconstituted suspension for subcutaneous use must not be filtered and is incompatible with 5% Dextrose and solutions containing bicarbonate. Subcutaneous injection sites, which include the upper arm, thigh, and abdomen, must be rotated. Doses requiring a volume greater than 4 mL must be divided and injected into two separate sites.

Population-Specific Use

While no initial dose adjustment is required for older adults or patients with renal impairment, dose modification rules apply based on monitored laboratory values. A dose reduction by 50% for the next cycle is required if unexplained reductions in serum bicarbonate levels to less than 20 mEq/L or specific elevations in BUN/creatinine occur. For pediatric patients, the dosing for Juvenile Myelomonocytic Leukemia (JMML) is based on body surface area or weight.

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Recent Clinical Evidence

Research Evidence Overview and Studies for Вайдаза (Azacitidine)


Evidence Overview for Higher-Risk Myelodysplastic Syndromes (MDS)

Research exploring Вайдаза use in higher-risk Myelodysplastic Syndromes (MDS) includes key Randomized Controlled Trials (RCTs), as well as systematic reviews and observational studies. These studies were conducted to examine adults, often including patients around the median age of 67–70 years, who were diagnosed with higher-risk MDS and were generally ineligible for haematopoietic stem cell transplantation (HSCT). Research examined outcomes related to systemic or functional imbalance by monitoring Overall Survival (OS) and the measured rate of disease progression, including transformation to Acute Myeloid Leukemia (AML).

Studies explored measurements of disease progression, specifically monitoring time until the condition progressed to AML or death. Research describes patterns observed in the studies related to overall survival measurements. Research also described the changes measured during the study period for outcomes like the observed rate of transfusion dependence.


Evidence Overview for Chronic Myelomonocytic Leukemia (CMML)

Research exploring Вайдаза use in Chronic Myelomonocytic Leukemia (CMML) was evaluated in observational studies, retrospective analyses, and data derived from subgroup analyses within larger clinical trials focused on related myeloid disorders. These studies monitored outcomes related to systemic or functional imbalance, such as overall remission rates, progression-free survival, and Overall Survival (OS). Evidence quality varies across studies, as there is a lack of standalone, large-scale RCTs specifically dedicated to the CMML patient population.


️ Evidence Overview for Acute Myeloid Leukemia (AML) in Specific Older Patients

Вайдаза was studied for newly diagnosed Acute Myeloid Leukemia (AML) primarily in older adult patients (aged 65 years) who were ineligible for intensive chemotherapy or HSCT due to existing health conditions. Researchers conducted pivotal Phase 3 RCTs. The studies reported measurements of overall survival when comparing treatment groups to conventional care regimens. However, initial analysis of the key trial did not record a clear pattern for OS measurements until specific adjustments (censoring) were made for patients who later received alternative AML treatments. Results apply only to the populations studied—older adults who are unfit for intensive therapy.


What Is Still Uncertain About Вайдаза Research

Certainty remains low for certain findings, particularly for CMML, where reliance on observational data is common. Furthermore, comparative evidence is lacking against the full range of newer treatments now approved. Long-term effects are not fully established across all studied subgroups and indications, and research is ongoing to address these gaps.

Key Studies & References

  1. Azacitidine for the treatment of myelodysplastic syndromes, chronic myelomonocytic leukaemia and acute myeloid leukaemia | Guidance | NICE (TA218)
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Frequently Asked Questions (FAQ)

Common questions about Вайдаза (FAQ)


Q: Can I take Vaidaza with other medicines?

Official documents state that formal drug-drug interaction studies with Vaidaza have not been conducted. However, based on how the body processes the drug, interaction with common liver enzymes is considered unlikely. Patients are generally advised to discuss all other medicines with their healthcare team.


Q: How long must I take Vaidaza?

Official documentation describes treatment schedules involving a minimum of 4 to 6 cycles. Treatment may continue as long as the patient is observing clinical benefit, or until unacceptable side effects occur or the disease progresses. The duration of treatment is determined by the prescribing physician based on monitoring the patient’s condition.


Q: What is the difference between Vaidaza and other MDS medicines?

Vaidaza belongs to a specialized pharmacological class called hypomethylating agents, which means its mechanism involves affecting DNA methylation in abnormal cells. Official research documents indicate that comparative studies against the full range of newer approved treatments are currently lacking. Information on how the drug’s mechanism differs from other available treatments is typically discussed by the prescribing physician.


Q: How is injectable Vaidaza different from oral azacitidine medicines?

The injectable form of Vaidaza and oral forms of the same active ingredient are not considered bioequivalent, meaning they are not interchangeable in the body. They have different approved uses and dosing regimens, and the injectable form must not be substituted for the oral tablet form, according to official product information.


Q: Why is Vaidaza treatment given in cycles?

The official dosing schedule prescribes treatment cycles that include treatment days followed by a long rest period (21 days). This cyclical schedule is established to help manage potential side effects, particularly those affecting the blood cell production (myelosuppression). This approach allows the body time to recover between treatments.


Q: What is known about the safety of Vaidaza with other chronic diseases?

Official safety documents specify that close monitoring is required for patients who have pre-existing moderate to severe kidney or severe liver impairment, as these conditions can affect how the drug is cleared from the body. Caution is also advised for patients with a history of severe congestive heart failure or unstable cardiac disease.


Q: When can one expect Vaidaza to start working?

Full or partial response to therapy may require more than 4 to 6 cycles of treatment. The body's response is generally tracked by your healthcare provider using regular blood tests and monitoring of your bone marrow. Some patients may not notice immediate physical changes.


Q: What should be done if a dose of Vaidaza is missed?

Vaidaza is administered by a healthcare professional according to a fixed schedule. If a scheduled dose is missed, it is necessary to contact the doctor or medical team immediately for guidance on adjusting the treatment plan.


Q: What is known about the long-term use of Vaidaza?

Treatment is intended to continue as long as the patient is observed to be receiving benefit. Published studies, including randomized controlled trials, describe the outcomes of long-term use, including effects on overall survival. Long-term safety and benefit are continuously assessed by the treating physician.


Q: What are the signs that Vaidaza is working?

Signs of improvement are typically monitored by the physician using clinical data. These may include measurable changes in blood counts, such as a reduced need for blood transfusions or the documented stabilization of the underlying disease, as observed in clinical trials.


Q: What should be done about bruising or bleeding while taking Vaidaza?

Bruising and bleeding can be linked to thrombocytopenia (low platelet counts), a very common side effect of the medicine. If signs of unusual bleeding or extensive bruising are observed, it is necessary to promptly inform the prescribing physician, as this may indicate the need for medical assessment and potential dose review.


Q: What will happen if I suddenly stop taking Vaidaza?

The decision to stop treatment is typically made in consultation with the prescribing physician. Treatment is generally continued until the disease progresses. Abruptly discontinuing treatment without medical guidance could be associated with the return or progression of the underlying disease.


Q: Can Vaidaza cause sleep problems?

Yes, sleep problems such as insomnia (trouble sleeping) are documented in official safety information as a very common side effect associated with the medicine.


Q: How often do I need to visit the doctor while on Vaidaza treatment?

Official requirements mandate that blood counts (CBC), liver function tests, and kidney function tests must be monitored before the start of each treatment cycle. The overall frequency of doctor visits is determined by the healthcare team based on these required monitoring tests and the patient's clinical condition.


Q: How does Vaidaza affect appetite?

A decrease in appetite (loss of appetite) is listed in official safety documents as a very common side effect associated with Vaidaza treatment.


Q: Does Vaidaza affect driving or operating machinery?

Vaidaza may cause fatigue (tiredness). Official instructions advise caution regarding driving or operating machinery until the individual's reaction to the medicine is known, due to this potential side effect.


Q: What symptoms should I look out for during Vaidaza treatment?

Patients are advised to be aware of symptoms that may indicate serious adverse reactions. These include signs of infection, such as fever, as well as signs of unusual bleeding or unexplained bruising, which are associated with the very common blood-related side effects of the drug.


Q: Can Vaidaza cause skin problems?

Yes, very common localized reactions at the injection site, including redness, pain, and rash, are documented. Rare, serious skin reactions, such as Sweet’s syndrome, have also been documented in official product information.


Q: How does Vaidaza interact with anticoagulants (blood thinners)?

While formal drug interaction studies with anticoagulants have not been conducted, Vaidaza very often causes thrombocytopenia (low platelet counts). The risk of bleeding requires that the prescribing physician exercise caution and perform close monitoring if the drug is used concurrently with medicines that affect blood clotting.


Q: What is known about the use of Vaidaza during pregnancy?

Vaidaza is formally contraindicated (must not be used) during pregnancy because, based on its mechanism and animal data, it carries a high risk of harming the fetus. Patients who are able to become pregnant are required to use effective contraception during the treatment period, as specified in regulatory documents.


Q: Are there restrictions on vaccinations (vaccines) while taking Vaidaza?

Vaidaza can cause low white blood cell counts, which increases the risk of serious infections. Because of the potential for immunosuppression, the use of live vaccines warrants careful consideration and discussion with a healthcare specialist.


Q: Why is Vaidaza given subcutaneously or intravenously?

Vaidaza is formulated as a lyophilized powder that is reconstituted for injection, either subcutaneously (under the skin) or intravenously (into a vein). It is administered parenterally because the injectable product has different properties from any oral forms and must be given by injection.


Q: Is Vaidaza associated with hair loss?

Yes, hair loss (alopecia) is listed as a frequent side effect associated with treatment in the official safety data.


Q: Can I drink alcohol while on Vaidaza treatment?

Official documents do not list a direct contraindication for alcohol. Alcohol consumption is a topic typically discussed between the patient and their prescribing physician, particularly since the drug may affect liver function and cause fatigue.


Q: Does Vaidaza affect the ability to get pregnant or have children?

Studies in animals have documented adverse effects on male fertility. Patients who wish to have children in the future are advised to seek information on fertility preservation (such as sperm or egg cryopreservation) before starting treatment with Vaidaza.


Q: What should I do if I get a fever while on Vaidaza treatment?

Fever (pyrexia) is a very common side effect and may be a sign of a serious infection, potentially due to low white blood cell counts (febrile neutropenia). If a fever occurs, it is required that the patient contact their doctor or medical team immediately, as this symptom may be a sign of a serious infection.


Q: Is Vaidaza a cure or a symptom reliever?

Vaidaza is classified as an antineoplastic agent (a type of therapy that inhibits tumor growth). Its goal is to alter the course of the disease by targeting and modifying pathological cells. Official documents do not use the term 'cure' to describe the expected outcome of the therapy.


Q: Is there pain or discomfort when Vaidaza is administered?

Pain or discomfort at the injection site is officially documented as a very common side effect. These localized reactions are classified as 'injection site reactions' and can include pain, redness, and bruising at the site where the medicine is administered.


Q: What are the key differences in Vaidaza use in older adults?

Official documentation notes that initial dose modification is typically not required for older adult patients. However, the pivotal clinical trials supporting its use were primarily conducted on older patients who were not considered suitable candidates for intensive chemotherapy.

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How should Вайдаза be stored and disposed of?

Storage and Disposal of Azacitidine for Injection

The un-reconstituted lyophilized powder for injection must be stored at controlled room temperature (25 C or 77 F), with excursions permitted between 15 C and 30 C. Storage must protect the powder from direct sunlight.

After reconstitution, the stability of the product is time-limited. For immediate administration, administration must be completed within 1 hour. For delayed subcutaneous use, the refrigerated suspension (2 C to 8 C) is stable for up to 22 hours under specific preparation conditions. Before use, the product must be visually inspected for particulate matter or discoloration.

As a cytotoxic drug, Vaidaza is for single-use only, and any unused portions of the vial must be discarded. The product must be stored locked up and kept out of the sight and reach of children. Disposal must follow applicable local and institutional regulations for hazardous pharmaceutical waste; it must not be emptied into drains or released into the environment.

Attention! Always consult to a doctor or pharmacist before using pills or medicines.

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