Lomustine

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Lomustine

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Medically reviewed

Marina Burgos

Last updated on 10/01/2026

This page provides general, reference-level information compiled from official medical sources. It is not a substitute for professional medical advice, diagnosis, or treatment. For decisions about your health, please consult a qualified healthcare professional.

Overview of Lomustine

Quick Facts

Property Description
Active Ingredient Lomustine (CCNU)
Form Hard capsule (Oral administration)
Pharmacological Class Alkylating Agent, Nitrosourea
Common Use Chemotherapy
Origin Synthetic chemical compound

Lomustine (CCNU): Identity and Chemical Classification

Lomustine, often identified by the acronym CCNU (for chloroethyl-cyclohexyl-nitrosourea), is a synthetic therapeutic agent classified as an antineoplastic drug, utilized in specialized chemotherapy protocols. It belongs to the specific class of alkylating agents. Within this classification, it is a member of the nitrosourea sub-class, structurally engineered to modify the genetic material of rapidly dividing cells. The drug is recognized globally as the established INN (International Nonproprietary Name) for this specific compound, simplifying identification across various regions.

Composition, Form, and Origin of the Medicine

The medication is formulated for oral administration and is supplied as a hard capsule, offering a non-intravenous route unique among many potent cytotoxic agents. Lomustine is a single-ingredient product derived from a synthetic chemical process, resulting in a compound that is notably highly lipid-soluble. The capsule contains the active substance Lomustine alongside pharmaceutical fillers and non-therapeutic components (excipients) necessary for proper drug stability and encapsulation.

General Role and Primary Mechanism Feature

The overarching purpose of Lomustine is to function as a cytostatic drug to halt or control the proliferation of abnormal cell populations within the body. Its primary action, achieved through DNA alkylation, is clinically recognized for interfering with cellular replication cycles. This mechanism is key to its role in controlling progressive cellular disorders. Lomustine possesses a highly lipophilic nature, a unique characteristic that enables it to effectively pass through the blood-brain barrier. This intrinsic property links its chemical structure to its general therapeutic utility, particularly when control of cellular disorders within the central nervous system is required.

Regulatory References

  1. NIH MedlinePlus Drug Information on Lomustine

What side effects are possible with Lomustine?

Possible Side Effects and Safety Information

Lomustine's safety profile is defined by its effects on the bone marrow, lungs, and other major organs, requiring careful monitoring as outlined in regulatory documentation.

Key Adverse Reactions and Frequencies

Classification Adverse Reaction Examples
Most Common / Severe Delayed Bone Marrow Suppression (Thrombocytopenia, Leukopenia)
Very Common Nausea, Vomiting, Stomatitis, Alopecia

The most frequent and dose-limiting toxicity is delayed myelosuppression, characterized by a severe drop in platelet and white blood cell counts, which typically peaks 4 to 6 weeks after administration and requires strict weekly monitoring.

Serious and Cumulative Risks

Serious Adverse Reactions documented in official labeling include fatal infections and bleeding (secondary to myelosuppression) and Pulmonary Toxicity (pulmonary fibrosis), which can be fatal. The risk of lung damage is progressive and is strongly associated with the total cumulative dose received over time, leading to mandated lifetime dose limits.

Late-onset toxicities also include progressive renal failure (nephrotoxicity) and the risk of secondary malignancies (acute leukemia and myelodysplasia) after long-term use.

Safety Restrictions and Monitoring

Courses of lomustine must not be repeated more frequently than every 6 weeks to allow for bone marrow recovery, with strict blood count criteria required before subsequent doses. Weekly blood counts and periodic monitoring of liver and renal function are mandated by regulatory agencies.

Population-Specific Safety: Lomustine is classified as Pregnancy Category D / Contraindicated. It is known to cause embryo-fetal toxicity, and strict contraceptive measures are required for patients who are or may become pregnant during and after treatment.

Overdose and Emergency Response

Overdose Manifestations and Regulatory Response

Overdosage of Lomustine is associated with fatal toxicity, establishing any suspected overexposure as a medical emergency. The officially documented clinical manifestations linked to overdose reflect the drug's severe and cumulative toxicity profile, primarily affecting the hematologic system.

Documented signs and symptoms include heightened myelosuppression (bone marrow suppression), abdominal pain, vomiting, anorexia, lethargy, and dizziness. Potential severe outcomes from this toxicity include life-threatening infection and bleeding, as well as the documented risk of pulmonary or renal injury with high cumulative exposure.

When Immediate Medical Help Is Required

Due to the risk of fatal outcomes, immediate medical attention must be sought for suspected overdosage. Management is strictly supportive, as no specific antidote is established. Officially described supportive measures include prompt gastric lavage and appropriate supportive care, such as antibiotic treatment or blood product replacement to manage resulting complications.

To mitigate the risk of accidental toxicity, regulatory constraints mandate that only one dose be prescribed and dispensed at a time. Following any exposure event, continuous monitoring is critical; specifically, blood counts must be monitored weekly for at least six weeks due to the delayed nature of the drug’s primary toxicity. Patients with existing impaired renal function may experience a greater risk of toxic reactions.

Therapeutic Uses of Lomustine

What Lomustine Treats: Main Uses and Benefits

Lomustine is commonly used across two primary therapeutic domains, specifically malignant primary and metastatic brain tumors and Hodgkin's lymphoma. This medication plays a role in managing conditions presenting with disruptive symptom manifestations in these specific types of cancer.

It is generally applied in contexts where additional symptomatic support is needed, often relevant when supportive symptom management is appropriate. The medication helps address groups of symptoms that may become intense or disruptive over time, which contributes to easing the overall symptom burden.

“This medication is commonly used when short-term symptomatic assistance is needed and helps maintain a sense of stability when symptoms are more noticeable.”

Quick Fact: Relief for Neurological Discomfort

Lomustine is relevant for managing symptoms that interfere with daily comfort, such as the neurological or physical discomfort associated with CNS tumors. It supports the patient during difficult episodes by easing distress and assists with maintaining functional stability during treatment for recurrent or progressive disease.

Eligibility and Restrictions for Use

Eligibility Profile: Who Can and Cannot Use Lomustine

Official regulatory information defines specific populations that are ineligible for Lomustine or require stringent conditional use.

Absolute Exclusions (Contraindications)

The medicine must not be used in individuals with:

  • A known hypersensitivity to lomustine, other nitrosoureas, or any of the product’s excipients.
  • Severe bone marrow depression (severe leukopenia and/or thrombocytopenia).
  • Pregnancy or breastfeeding, due to the potential for fetal harm and risk to the nursing infant. Lomustine is strictly contraindicated during these states.
  • Concomitant use with live vaccines, including yellow fever vaccine, while immunosuppressed.

Conditional Use and Restrictions

Use of Lomustine requires special caution and monitoring in the following groups:

  • Impaired Organ Function: Patients with existing renal or hepatic impairment are at greater risk of toxic effects, requiring careful dose selection and monitoring.
  • Compromised Hematologic Status: Treatment is restricted; a repeat course is only allowed once blood cell counts (leukocytes and platelets) have returned to acceptable levels.
  • Reproductive Potential: Both male and female patients of reproductive potential must use effective contraception during treatment and for a specified period after the final dose.
  • Elderly Patients: Use requires caution, as decreased renal function is more common in this population.

What should I know about interactions with other medicines?

Interactions with other medicines and products

The regulatory profile for Lomustine establishes specific constraints concerning co-administration with other substances, classifying interactions based on their documented effect on toxicity or drug exposure.

Formal Contraindicated Combinations

The co-administration of Yellow Fever Vaccine and other Live Vaccines is formally contraindicated in immunosuppressed patients. This restriction is documented in the prescribing information due to the increased risk of severe adverse effects or systemic infection from the vaccine.

Timing-Based Separation Requirement

Official labeling advises that patients should not receive live vaccines until at least 3 months after the end of treatment with Lomustine.

Pharmacodynamic and Pharmacokinetic Interactions

Specific interactions are documented where co-administration results in either the potentiation of toxicity or altered clearance:

  • Potentiation of Bone Marrow Toxicity: Concomitant use with agents such as Theophylline or the H2-receptor antagonist Cimetidine may potentiate bone marrow toxicity. Furthermore, co-treatment with other cytostatic drugs or radiation therapy can increase bone marrow depression (additive myelosuppression).

  • Exposure Modification: Pre-treatment with Phenobarbital (an enzyme inducer) results in microsomal liver enzyme induction, which accelerates Lomustine's elimination. This specific pharmacokinetic interaction is documented to cause a reduced antitumour effect.

Mechanism of Action

The action of Lomustine is defined by its function as a lipophilic alkylating agent, which works by chemically damaging critical components of the cell. The mechanism operates through two core domains: introducing chemical lesions that challenge repair mechanisms and accessing anatomically protected sites.

Irreversible Chemical Modification of DNA and Proteins

Lomustine decomposes in vivo to yield highly reactive intermediates that perform alkylation on DNA (specifically guanine bases) and carbamoylation on functional proteins. This permanent molecular interference leads directly to the formation of interstrand cross-links (ICLs) in the DNA. The formation of these irreversible ICLs acts as a physical block, saturating the cell's repair mechanisms and preventing the separation of DNA strands required for replication and transcription. This cascade ultimately causes the targeted cells to halt the cell cycle and initiate apoptosis (programmed cell death), which results in the antiproliferative physiological consequence.

Enhanced Systemic Access and Mechanistic Limitations

Lomustine's high lipophilicity allows it to readily pass through biological lipid barriers, including the blood-brain barrier (BBB), enabling the active intermediates to access targets in the central nervous system. Conversely, the mechanism faces constraints from certain cellular defense systems, notably the O^6-methylguanine DNA methyltransferase (MGMT) enzyme. High activity of MGMT can rapidly repair the initial alkylation damage, thereby preventing the formation of cytotoxic ICLs and subsequently limiting the intended physiological effect.

Dosage and Administration Information

Administration Map: Official Usage Guidelines

Lomustine is an oral chemotherapy agent, supplied exclusively as hard capsules in strengths of 5 mg, 10 mg, 40 mg, and 100 mg. The official usage protocol is structured around a highly precise, infrequent, and cyclic schedule, distinct from daily medication regimens.

Instruction Details
Route of Administration Oral route (swallowed by mouth).
Standard Dosing Regimen The standard single dose is 130 mg/ m^2 of body surface area (BSA). For patients with compromised bone marrow function, the starting dose is 100 mg/ m^2.
Mandatory Frequency A single course must not be repeated for at least 6 weeks. The bone marrow response must be monitored weekly for this period.
Capsule Handling The capsules must be swallowed whole and must not be opened or crushed. The total required dose may be comprised of a combination of the different capsule strengths.
Dosing Adjustment Rule Subsequent doses are governed by the patient's hematological recovery. A new course may only be given once blood counts return to acceptable levels (e.g., platelets 100,000/ mm^3 and leukocytes 4,000/ mm^3).
Cumulative Dose Limit The maximum cumulative dose should generally not exceed 1,000 mg/ m^2.

This protocol defines Lomustine's use as a highly regulated, intermittent cycle of therapy. Administration is a single event, followed by a minimum six-week recovery period, where the decision to proceed with the next course is based strictly on laboratory results, not a fixed calendar date. Standard guidelines emphasize dispensing only the capsules required for a single dose to prevent over-administration, which reinforces the critical nature of the 6-week interval.

Recent Clinical Evidence

Overview of Studies and Research for Lomustine (CCNU)

This section summarizes the structure of available clinical research and key study findings for Lomustine across its approved uses. It focuses on the types of studies conducted and the outcomes that were measured, without offering medical advice or making claims about individual results. Findings describe group patterns observed in the studies, not personal outcomes.


Evidence Framework for Malignant Primary and Metastatic Brain Tumors

Research into the use of Lomustine for brain tumors, such as Glioblastoma and Astrocytoma, primarily involves Randomized Controlled Trials (RCTs) and various other clinical trials. These studies generally focus on adult patients and often evaluate Lomustine as part of a combination regimen alongside other drugs or radiation therapy. The main outcomes researchers have evaluated focus on patient survival time. These include Overall Survival (OS) and Progression-Free Survival (PFS). Studies also monitor the rate of radiographic response and assess Quality of Life and neurocognitive function.

Evidence Framework for Progressive Hodgkin's Lymphoma

Studies for Lomustine in Hodgkin's lymphoma are mainly designed as Phase II/III clinical trials and retrospective analyses. This research typically focuses on adult patients whose disease has relapsed or has been refractory. In these research scenarios, Lomustine is commonly evaluated as a component of complex, multi-agent "salvage" chemotherapy regimens. Researchers in these settings primarily evaluate outcomes related to achieving disease status changes and long-term survival rates. Studies report the percentage of patients achieving certain measured endpoints and describe patterns related to survival, but comparative evidence isolating the findings of Lomustine as a single agent in this complex setting is often lacking.


Limitations and Areas of Uncertainty in Existing Evidence

A significant limitation is that the research has often examined Lomustine as part of a combination treatment. Because of this, subgroup findings are uncertain regarding Lomustine's singular contribution compared to the overall drug regimen. Follow-up durations were limited in some trials, meaning that data on long-term consequences are not fully characterized. For recurrent brain tumors, evidence comparing Lomustine to all other existing strategies is limited, and sample sizes were modest in some studies. Final, certainty remains low in areas where findings were mixed or where trials were conducted before modern genetic testing was widely applied, meaning research provides limited insight into application for newly defined tumor types.

Frequently Asked Questions (FAQ)

Common questions about Lomustine (FAQ)


Q: Why is Lomustine often given for brain tumors?

According to official product information, Lomustine is a highly fat-soluble drug. This chemical property allows it to effectively cross the blood-brain barrier, which is a protective layer in the brain. This unique ability relates to its documented use in treating both primary and metastatic brain tumors.


Q: How does Lomustine compare to Temozolomide (TMZ) at a high level?

Lomustine is an alkylating nitrosourea compound, which is a specific class of chemotherapy. Regulatory guidelines and clinical practice sometimes refer to its use alongside or in sequence with other treatments, such as Temozolomide (TMZ), in certain brain tumor protocols, as part of a combination approach.


Q: What kind of blood tests are needed when taking Lomustine?

Official guidelines state that blood counts, including white blood cell counts (leukocytes) and platelet counts, must be monitored weekly for at least six weeks after each dose. Periodic monitoring of liver and kidney function is also required by official guidelines.


Q: How often do the low blood cell counts (myelosuppression) usually recover?

The most significant drop in blood counts, known as delayed myelosuppression, is expected to peak about 4 to 6 weeks after the dose is taken. Platelet counts generally recover within 5 to 6 weeks, and white blood cell counts usually recover within 6 to 8 weeks, which reinforces the minimum six-week waiting period between doses.


Q: What are the possible late side effects of Lomustine, like lung or kidney issues?

Documented serious and cumulative risks include Pulmonary Toxicity (lung damage) and progressive renal failure (kidney damage). For this reason, official guidance mandates limits on the total lifetime dosage received, as the risk is associated with the cumulative dose.


Q: For how long after treatment must contraception be used by men and women?

Regulatory documents state that females of reproductive potential should use effective contraception during treatment and for 2 weeks after the final dose. Males with female partners should use effective contraception during treatment and for 3.5 months after the final dose, due to documented concerns regarding fetal development and reproductive function.


Q: Why is Lomustine typically given as an oral capsule instead of an infusion?

Lomustine is approved as a highly fat-soluble molecule, which facilitates its absorption after oral administration. This property enables the drug to be administered as an oral capsule rather than requiring an intravenous (IV) infusion, a route that is documented to be effective for this agent.


Q: Does Lomustine cause hair loss for most people?

Official documentation lists hair loss, or Alopecia, as a common or very common adverse reaction. This indicates that it is a frequent side effect expected to occur in a significant number of patients undergoing treatment.


Q: Is it normal to feel nausea and vomiting after taking Lomustine?

Yes, nausea and vomiting are listed in regulatory documents as very common side effects associated with Lomustine. These gastrointestinal effects typically start a few hours after taking the dose.


Q: How long do the common stomach side effects from Lomustine typically last?

According to official product information, the nausea and vomiting usually last for less than 24 hours after the single dose is administered. Official labeling notes that anti-nausea medication may be administered before or with the dose to manage these effects.


Q: Are vision changes or confusion considered common side effects of Lomustine?

Official labeling lists neurological reactions such as disorientation and lethargy (sluggishness), and reports of visual disturbances. While these are documented, they are reported infrequently and are not listed among the most common adverse reactions.


Q: Why is Lomustine sometimes used alongside other drugs like Avastin or as part of the PCV regimen?

Official guidelines and clinical protocols describe Lomustine being used as part of a combination regimen with other drugs or radiation therapy. The documented use of combination treatment is designed to target abnormal cells using multiple agents. Clinical documentation describes this as a common therapeutic strategy.


Q: Why do official guidelines suggest taking Lomustine on an empty stomach?

Regulatory information notes that administering the drug to fasting patients can help reduce the frequency and severity of common gastrointestinal side effects, such as nausea and vomiting.


Q: Is the brand name Gleostine the same as the generic drug Lomustine?

Yes, Lomustine is the international nonproprietary name (generic name) for the active chemical compound. Gleostine is one of the brand names under which the active ingredient Lomustine is available and marketed.


Q: Can Lomustine treatment cause fatigue or extreme tiredness?

Unusual tiredness or weakness is listed as a potential side effect in patient information summaries. The drug's systemic effects, especially the impact on blood cell counts, may contribute to general disorders like fatigue.


Q: Why do some people experience mouth sores or ulcers during Lomustine treatment?

Mouth sores, known medically as Stomatitis, are listed as a very common side effect. This occurs because the drug acts on rapidly dividing cells, including those that line the mouth and digestive tract, which is a common pattern for chemotherapy.


Q: Does Lomustine have any specific restrictions for elderly patients?

Caution is advised for elderly patients. This is because decreased kidney function is more common in older adults, and since Lomustine and its byproducts are substantially excreted by the kidneys, monitoring of renal function is necessary.


Q: Are there any special handling or safety precautions for the capsule itself?

Yes, since Lomustine is a cytotoxic drug, special precautions apply. The capsules must be swallowed whole and never opened or crushed. Caregivers are advised in official documentation to use gloves when handling the capsules to minimize the potential for accidental skin exposure.


Q: Are there different strengths of capsules that make up a single dose?

Yes, Lomustine is available in several capsule strengths, such as 5 mg, 10 mg, 40 mg, and 100 mg. Official documents confirm that the total dose prescribed for a patient may consist of a combination of these different capsule strengths.


Q: Do official documents describe any risk of secondary cancers with Lomustine use?

Yes, official documentation includes a warning that secondary malignancies can occur with long-term use. Specifically, there is a documented risk of acute leukemia and myelodysplasia (disorders of the blood and bone marrow).


Q: What are the general research themes regarding Lomustine and oligodendroglioma?

While regulatory indications cover brain tumors generally, clinical research and guidelines frequently discuss Lomustine’s use for specific tumor types, including oligodendroglioma. It is often evaluated as part of combination treatment protocols for these tumor types.


Q: Can Lomustine cause jaundice or signs of liver damage?

Yes, official labeling includes a warning about Hepatotoxicity (liver damage). A reversible form of liver toxicity, indicated by elevated liver enzyme and bilirubin levels, has been reported in a small percentage of patients.


Q: Why are patients sometimes advised to check their temperature regularly during treatment?

Lomustine causes delayed myelosuppression, which can lead to a severe drop in white blood cell counts. Fever may be a sign of infection due to low white blood cell levels, as documented in the safety profile. Patients are cautioned in official documents to report signs of infection immediately.


Q: Is it common for a patient's appetite to be reduced for a few days after taking the dose?

Loss of appetite, or anorexia, is listed as a potential side effect in patient information summaries. This general reduction in appetite is documented separately from direct stomach side effects like nausea and vomiting.


Q: What is the maximum number of cycles a patient might receive Lomustine?

Regulatory guidance sets a maximum cumulative lifetime dose limit of approximately 1,000 mg/m^2 to reduce the risk of serious lung toxicity. The number of cycles a patient receives is limited by this total cumulative dose and their individual blood count recovery.


Q: Are there special disposal instructions for unused Lomustine capsules?

Regulatory documents state that unused, partially used, or expired capsules must be handled as cytotoxic waste. Disposal protocols typically involve returning the product to the dispensing pharmacy or clinic, in accordance with local cytotoxic waste regulations, rather than disposing of it in general trash or drains.


Q: Does Lomustine affect a person's ability to drive or operate machinery?

Lomustine may cause side effects that affect the central nervous system, such as disorientation or visual disturbances. Regulatory documents advise caution regarding driving or operating heavy machinery if these types of side effects are experienced.


Q: Does Lomustine contain any lactose or gluten based on official documents?

Official European product documentation lists anhydrous lactose and wheat starch among the inactive ingredients (excipients) in the capsules. It is noted that the presence of wheat starch may be a consideration for patients with celiac disease or a wheat allergy.


Q: Can Lomustine treatment cause anemia (low red blood cells)?

Yes, official documentation lists anemia, which is a decrease in red blood cell count, as a very common side effect. It is categorized as a blood and lymphatic system disorder, similar to the documented drop in white blood cells and platelets.

How should Lomustine be stored and disposed of?

Storage and Disposal of Lomustine Capsules

Lomustine capsules must be stored in the original container, which must be kept tightly closed, to ensure protection from light and moisture.

Required Storage Conditions

Condition Requirement
Temperature Store at room temperature; do not exceed 25 C and do not freeze.
Child Safety Must be kept out of the reach and sight of children.
Container Integrity The capsules must not be opened.

Handling and Disposal

As a cytotoxic drug, special handling procedures apply. Individuals should wear impervious gloves when handling the bottles or capsules. Any unused or expired product and contaminated materials must be treated as cytotoxic waste and disposed of in accordance with local regulations and specialized waste management protocols, avoiding release into drains or waterways.

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

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