Topotecan
Topotecan is a chemotherapy medication classified as a topoisomerase I inhibitor. It works by binding to the topoisomerase I-DNA complex, preventing the repair of single-strand breaks in DNA, which leads to double-strand DNA damage and cell death. This medication is primarily used to treat ovarian cancer, small cell lung cancer, and certain types of cervical cancer.
Topotecan is an injectable chemotherapy drug that inhibits topoisomerase I to treat various cancers by causing DNA damage that kills cancer cells.
This medication carries a boxed (black box) warning — the strongest type of FDA warning. Review the full prescribing information before use.
Key facts at a glance
- ATC
- L01CE01
- RxNorm
- 57308
- Generic
- Topotecan
- Class
- atc-l01
- Brands
- Hycamtin
- Availability
- rx
- Lactation
- unknown
- Routes
- oral
- Forms
- injection, capsule, solution
- Regions
- US, EU, CN, JP
- Monitoring
- —
This page is for informational purposes only.
Do not use this information to diagnose yourself or to replace a physician's advice. Always consult a qualified healthcare provider before starting, stopping, or changing any medication. If you believe you are experiencing a medical emergency, call your local emergency number immediately.
Dosage and administration
- Usual dose
- 2.3 mg/m² once daily
- Maximum
- consult physician
- Frequency
- daily for 5 days every 21 days
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
- Usual dose
- 2 mg/m² once daily
- Maximum
- consult physician
- Frequency
- daily for 5 days every 21 days
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
- Usual dose
- 1.8 mg/m² once daily
- Maximum
- consult physician
- Frequency
- daily for 5 days every 21 days
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
Overdose information
Special Populations
9 groupspregnancy
first trimesteravoid if possible
avoid use during pregnancy unless benefits outweigh risks; discuss potential fetal harm
regular fetal monitoring including ultrasound
Use of this medication in this population requires careful consideration of risks and benefits.
pregnancy
second third trimesteravoid if possible
avoid use during pregnancy unless benefits outweigh risks
regular fetal monitoring
Use of this medication in this population requires careful consideration of risks and benefits.
lactation
breastfeedingdiscontinue breastfeeding
do not breastfeed during treatment and for at least 1 week after last dose
monitor infant for signs of toxicity
Use of this medication in this population requires careful consideration of risks and benefits.
geriatric
elderly 65consider starting at lower end of dosing range
use with caution due to increased risk of myelosuppression; monitor renal function closely
more frequent CBC monitoring; assess renal function before each cycle
Use with caution and monitor for adverse effects.
pediatric_age
children adolescentsdose based on body surface area and specific indication
use in pediatric patients with neuroblastoma, rhabdomyosarcoma, or other approved pediatric cancers under specialist supervision
close hematologic monitoring; monitor for signs of infection
Use with caution and monitor for adverse effects.
renal_impairment
mild renal impairmentreduce starting dose if CrCl 40-60 mL/min
monitor renal function; adjust dose based on tolerability
monitor creatinine clearance before each cycle
Use with caution and monitor for adverse effects.
renal_impairment
moderate severe renal impairmentsignificant dose reduction required
use with extreme caution; consider alternative therapy if CrCl less than 30 mL/min
frequent renal function monitoring; monitor for signs of toxicity
Use of this medication in this population requires careful consideration of risks and benefits.
hepatic_impairment
mild hepatic impairmentno major initial dose adjustment needed
use with caution; monitor for increased toxicity
monitor hepatic function and blood counts
Use with caution and monitor for adverse effects.
hepatic_impairment
moderate severe hepatic impairmentreduce dose by 50% or more
use with extreme caution; not recommended in severe hepatic impairment
frequent monitoring of liver function and hematologic parameters
Use of this medication in this population requires careful consideration of risks and benefits.
Statistics and clinical data
FDA approval year
Initial FDA approval for Topotecan (Hycamtin)
Drug class
Antineoplastic agent classification
Mechanism of action
Primary pharmacological mechanism
FDA-approved indications
Therapeutic indications
Topotecan is FDA-approved for the treatment of ovarian cancer (after platinum-based chemotherapy failure), small cell lung cancer (after first-line chemotherapy failure), and cervical cancer (in combination with other agents). It may also be used for other solid tumors in clinical settings.
Topotecan binds to and stabilizes the topoisomerase I-DNA cleavage complex, preventing the re-ligation of single-strand DNA breaks. This creates persistent DNA damage during replication, leading to double-strand breaks, cell cycle arrest, and apoptotic cell death in rapidly dividing cancer cells.
Topotecan is administered intravenously and requires regular blood count monitoring due to significant myelosuppression risk (neutropenia, thrombocytopenia, anemia). It should not be used during pregnancy or breastfeeding. Caution is needed in patients with renal impairment. The drug is typically handled as a hazardous agent in healthcare settings.
Contraindications
Conditions and patient states where this medication should be avoided or used with caution.
Do not use (1)
Known allergy to this medication or its components
Use caution (1)
Consult healthcare provider for benefits vs risks assessment
Official drug label
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This information is for educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment.
This content is for general informational and educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment. Always seek the guidance of a qualified healthcare provider with any questions you have about a medical condition, and never disregard professional medical advice or delay seeking it because of something you read here.
If you think you may have a medical emergency, call your local emergency number or go to the nearest emergency department immediately.
Medically reviewed by WellAlly Editorial Team Last reviewed July 26, 2026. · Read our editorial policy