Topoisomerase I inhibitor Pregnancy: Topotecan has been shown to cause embryo-foetal lethality and malformations in preclinical studies and may cause foetal harm; women of childbearing potential should avoid becoming pregnant and use effective contraception during treatment and for 6 months after completion; men should use effective contraception and not father a child during treatment and for 3 months after completion. If used during pregnancy, or if the patient becomes pregnant during therapy, the patient must be warned of the potential hazards to the foetus. Breast-feeding is contraindicated.

Topotecan (Specialist drug)

Brand names: Hycamtin

Topotecan is a topoisomerase I inhibitor cytotoxic agent used in the treatment of relapsed ovarian cancer, small-cell lung cancer and cervical cancer. It is a specialist drug given under oncology supervision.

Auto-extracted from the source labelling — not yet independently clinician-verified. These values were distilled from the UK SPC (or the US label where noted) but have not had a clinician sign-off. Confirm against the current SmPC before prescribing.

Adult dose

Dose: Ovarian carcinoma and small cell lung carcinoma (monotherapy): 1.5 mg/m2 body surface area per day

Route: Intravenous infusion over 30 minutes (must be reconstituted and further diluted before use)

Frequency: Daily for five consecutive days, with a three-week interval between the start of each course; if well tolerated, treatment may continue until disease progression

Use should be confined to units specialised in the administration of cytotoxic chemotherapy and given only under the supervision of a physician experienced in the use of chemotherapy. Before the first course, patients must have a baseline neutrophil count of at least 1.5 x 10^9/l, a platelet count of at least 100 x 10^9/l and a haemoglobin level of at least 9 g/dl (after transfusion if necessary). Cervical carcinoma (combination with cisplatin): topotecan 0.75 mg/m2/day as a 30-minute intravenous infusion on days 1, 2 and 3, with cisplatin given as an intravenous infusion on day 1 at 50 mg/m2/day following the topotecan dose; this schedule is repeated every 21 days for six courses or until progressive disease. Subsequent doses (monotherapy): do not re-administer unless neutrophils are at least 1 x 10^9/l, platelets at least 100 x 10^9/l and haemoglobin at least 9 g/dl; for severe neutropenia (neutrophils below 0.5 x 10^9/l) for seven days or more, neutropenia with fever or infection, or treatment delay due to neutropenia, reduce the dose by 0.25 mg/m2/day to 1.25 mg/m2/day (and subsequently to 1.0 mg/m2/day if necessary); reduce similarly if platelets fall below 25 x 10^9/l; in clinical studies topotecan was discontinued if a further reduction below 1.0 mg/m2/day was required. Subsequent doses (cervical carcinoma): re-administer only if neutrophils are at least 1.5 x 10^9/l, platelets at least 100 x 10^9/l and haemoglobin at least 9 g/dl; for the same triggers reduce the dose by 20% to 0.60 mg/m2/day for subsequent courses (and subsequently to 0.45 mg/m2/day if necessary). Hepatic impairment: a small number of patients with serum bilirubin between 1.5 and 10 mg/dl received 1.5 mg/m2/day for five days every three weeks and a reduction in topotecan clearance was observed, but there are insufficient data to make a dose recommendation; not recommended in severe hepatic impairment (serum bilirubin 10 mg/dl or above) due to cirrhosis. Paediatric population: currently available data are described in sections 5.1 and 5.2 but no recommendation on a posology can be made. When topotecan is used in combination with cisplatin, the full prescribing information for cisplatin should be consulted.

Dose adjustments

Renal

Monotherapy (ovarian and small cell lung carcinoma): the recommended dose in patients with a creatinine clearance between 20 and 39 ml/min is 0.75 mg/m2/day for five consecutive days; there is insufficient experience in severe renal impairment (creatinine clearance below 20 ml/min) and use is not recommended in this group. Combination therapy (cervical carcinoma): in clinical studies therapy was only initiated in patients with serum creatinine 1.5 mg/dl or less; if serum creatinine exceeds 1.5 mg/dl during topotecan/cisplatin therapy, consult the cisplatin prescribing information for advice on dose reduction/continuation.

Dose auto-extracted from UK Summary of Product Characteristics (SPC) via the eMC; US FDA prescribing information (openFDA / DailyMed) — cross-check; US labelling may differ from UK — not yet clinician-verified. Always confirm against the product SmPC and your local formulary before prescribing.

Contraindications

Severe hypersensitivity to the active substance or to any of the excipients
Breast-feeding
Severe bone marrow depression prior to starting the first course, as evidenced by baseline neutrophils below 1.5 x 10^9/l and/or a platelet count below 100 x 10^9/l

Side effects

Very common: febrile neutropenia, neutropenia, thrombocytopenia, anaemia, leucopenia; common pancytopenia; myelosuppression leading to sepsis and fatalities due to sepsis have been reported
Very common: nausea, vomiting and diarrhoea (all of which may be severe), constipation, abdominal pain, mucositis; neutropenic colitis (including fatal cases) has been reported as a complication of topotecan-induced neutropenia; gastrointestinal perforation (frequency not known)
Very common: infection; very common anorexia (which may be severe)
Very common: alopecia, pyrexia, asthenia, fatigue; common pruritus and malaise
Rare: interstitial lung disease (some cases have been fatal); rare anaphylactic reaction, angioedema, urticaria; common hypersensitivity reaction including rash; common hyperbilirubinaemia

Interactions

No in vivo human pharmacokinetic interaction studies have been performed — the SPC §4.5 text was truncated at the source-fetch limit in this bundle, so the full interaction section must be checked in the SPC by the reviewing clinician

Clinical monograph

How it works

It inhibits topoisomerase I, stabilising the enzyme-DNA complex and causing DNA strand breaks that are lethal to dividing cells.

Prescribing in practice

It causes severe, dose-limiting myelosuppression, particularly neutropenia, so blood counts must be checked before each course and treatment withheld until adequate recovery.
Diarrhoea, fatigue and mucositis are common, and the dose requires reduction in renal impairment.
It is teratogenic and must be handled using cytotoxic precautions, with effective contraception advised.

Monitoring

Monitor full blood count before and during each cycle, together with renal function.

Counselling the patient

Report fever, sore throat, bruising or bleeding straight away as your white cell count may drop.
Tell your team about diarrhoea, mouth ulcers or extreme tiredness.
Use reliable contraception during treatment.

Evidence & guidelines

Use is guided by the manufacturer's SPC and NICE guidance on topotecan in relevant gynaecological and lung cancers.

Reference: NICE TA183; SmPC; Drug verified in RxNorm (NLM); confirm dosing against the manufacturer SPC (eMC). Verify against your local formulary and current prescribing references before prescribing. The structured dose values shown have been reviewed by a clinician. Monograph status: clinician-reviewed (2026-07-04).

Curated clinical cross-links plus same-class fallbacks.

Calculators

Drugs

Irinotecan (Specialist drug) · Topoisomerase I inhibitor

Pathways