Leukeran (chlorambucil) is an alkylating antineoplastic agent specifically formulated for the treatment of chronic lymphocytic leukemia (CLL) and certain types of non-Hodgkin lymphoma. As an oral chemotherapeutic drug, it offers a convenient administration route while delivering cytotoxic effects to malignant lymphocytes through DNA cross-linking. Its established efficacy profile and predictable pharmacokinetics make it a cornerstone in hematologic malignancy protocols, particularly for patients who may not be candidates for more aggressive treatment regimens. Clinical evidence supports its role in both first-line and subsequent therapy lines, providing hematologists with a valuable tool for disease management.

Features

• Active ingredient: Chlorambucil (2mg tablets)
• Mechanism: Bifunctional alkylating agent causing DNA interstrand cross-links
• Administration: Oral formulation with high bioavailability
• Packaging: Available in 2mg scored tablets with 50-tablet bottles
• Stability: Demonstrated chemical stability under recommended storage conditions
• Compatibility: Can be administered with prednisone in combination regimens

Benefits

• Achieves clinically significant reduction in lymphocyte counts and lymph node size
• Provides flexible dosing adjustments based on hematologic parameters
• Enables outpatient treatment management without requiring infusion facilities
• Demonstrates predictable response patterns in treatment-naïve patients
• Offers cost-effective therapy compared to newer biologic agents
• Maintains quality of life through oral administration and manageable side effect profile

Common use

Leukeran is primarily indicated for the palliative treatment of chronic lymphocytic leukemia (CLL), with particular efficacy in patients presenting with advanced-stage disease or symptomatic lymphocytosis. It is also employed in the management of Waldenström’s macroglobulinemia and certain low-grade non-Hodgkin lymphomas. The medication may be used as monotherapy or in combination with prednisone, depending on disease characteristics and patient tolerance. Treatment initiation typically follows confirmation of diagnosis through bone marrow biopsy and flow cytometry, with ongoing monitoring of complete blood counts throughout therapy duration.

Dosage and direction

Initial dosing for Leukeran typically ranges from 0.1 to 0.2 mg/kg body weight administered orally once daily for 3 to 6 weeks. Alternatively, a pulsed dosing regimen of 0.4 mg/kg may be given in divided doses over 4 days, repeated every 2 to 4 weeks. Dosage must be individualized based on hematologic response and toxicity, with regular monitoring of white blood cell and platelet counts. Tablets should be swallowed whole with water, preferably at the same time each day, and may be taken with or without food. Dose adjustments are mandatory for patients with renal impairment or those experiencing myelosuppression.

Precautions

Complete blood counts must be monitored weekly during initiation and at regular intervals throughout therapy. Patients should be advised to report any signs of infection, unusual bleeding, or bruising immediately. Secondary malignancies have been reported with long-term use, requiring careful benefit-risk assessment. Fertility preservation discussions should occur prior to treatment initiation in patients of reproductive potential. Vaccination with live vaccines is contraindicated during treatment. Healthcare providers should exercise caution when handling tablets, using appropriate protective equipment to avoid exposure.

Contraindications

Leukeran is contraindicated in patients with demonstrated hypersensitivity to chlorambucil or any component of the formulation. It should not be administered to patients who have previously exhibited resistance to alkylating agents. Use is prohibited during pregnancy (Category D) and breastfeeding due to potential fetal harm and secretion in human milk. The medication is contraindicated in patients with severe bone marrow suppression prior to treatment initiation. Those with active infections requiring treatment should delay therapy until the infection is controlled.

Possible side effect

• Hematologic: Myelosuppression including leukopenia, thrombocytopenia, anemia (dose-limiting)
• Gastrointestinal: Nausea, vomiting, diarrhea, oral ulceration (usually mild to moderate)
• Dermatologic: Skin rash, urticaria, angioedema
• Neurologic: Seizures (particularly at high doses), peripheral neuropathy
• Pulmonary: Pulmonary fibrosis (rare but serious)
• Reproductive: Amenorrhea, oligospermia, infertility
• Hepatic: Reversible hepatotoxicity with elevated transaminases
• Other: Fever, allergic reactions, secondary malignancies

Drug interaction

• Warfarin: Enhanced anticoagulant effect requiring INR monitoring
• Live vaccines: Diminished immune response and potential vaccine-related complications
• Myelosuppressive agents: Additive bone marrow suppression with other chemotherapeutics
• Allopurinol: May increase incidence of skin reactions
• CYP450 inducers: Potential alteration of chlorambucil metabolism
• Nephrotoxic drugs: Enhanced renal toxicity risk

Missed dose

If a dose is missed, patients should take it as soon as remembered unless it is nearly time for the next scheduled dose. In such cases, the missed dose should be skipped entirely. Patients should never double the dose to make up for a missed administration. Healthcare providers should be notified of missed doses, particularly if multiple doses are omitted, as this may affect treatment efficacy. Documentation of missed doses is essential for accurate toxicity assessment and dose adjustment decisions.

Overdose

Overdose manifests primarily as irreversible bone marrow suppression including pancytopenia, with bleeding complications and overwhelming infection representing the most serious consequences. Gastrointestinal toxicity including nausea, vomiting, and mucositis may occur. There is no specific antidote for Leukeran overdose. Management consists of immediate discontinuation, supportive care including transfusion support, and aggressive treatment of infections. Hematopoietic growth factors may be considered. Hospitalization is required for significant overdoses, with monitoring continuing for several weeks due to the drug’s prolonged effects.

Storage

Store at controlled room temperature (20-25°C or 68-77°F) in the original container with the lid tightly closed. Protect from light and moisture. Keep out of reach of children and pets. Do not transfer tablets to other containers. Proper disposal of unused medication is essential through take-back programs or following specific disposal instructions. Do not use tablets that show signs of discoloration or degradation. The medication should not be stored in bathrooms or other areas with high humidity.

Disclaimer

This information is provided for educational purposes only and does not constitute medical advice. Treatment decisions must be made by qualified healthcare professionals based on individual patient circumstances. The prescribing information provided here may not include all possible uses, directions, precautions, or interactions. Always consult the full prescribing information and clinical guidelines before initiating therapy. Actual patient experiences may vary from clinical trial data.

Reviews

Clinical studies demonstrate overall response rates of 70-80% in treatment-naïve CLL patients, with complete responses observed in approximately 10-15% of cases. Long-term follow-up data show median progression-free survival of 18-24 months in responsive patients. Elderly patients particularly benefit from the manageable toxicity profile compared to fludarabine-based regimens. Combination therapy with anti-CD20 monoclonal antibodies has shown improved response rates in recent studies. Quality of life assessments indicate maintained functional status in most patients during treatment periods.