Topotecan
Pronunciation: (toe-poe-TEE-kan).
- Generic name
- topotecan
- Brand names
- Hycamtin
- Drug class
- Topoisomerase I inhibitor (chemotherapy)
- Legal status
- Prescription-only; administered in healthcare settings
Availability and supply rules vary by country
Trade names by country: United States · Canada
Australia: · Full directory
What topotecan is and how it works
Topotecan is an intravenous chemotherapy medicine (a topoisomerase I inhibitor) given by healthcare professionals. In the United States it is used for small cell lung cancer that initially responded to first-line chemotherapy and has come back, and — together with cisplatin — for cervical cancer that is stage IV-B, recurrent, or persistent and cannot be cured with surgery or radiotherapy. Because topotecan strongly suppresses the bone marrow, it carries a boxed warning: it is only given when the neutrophil count is high enough, and blood counts are checked frequently throughout treatment. (1)
What topotecan is used for
Small cell lung cancer (relapsed, sensitive disease)
For small cell lung cancer with sensitive disease — disease that responded to first-line chemotherapy — after it has progressed. Given as 1.5 mg/m² IV over 30 minutes daily for 5 consecutive days of each 21-day course. (1)
Cervical cancer (with cisplatin)
For stage IV-B, recurrent, or persistent carcinoma of the cervix not amenable to curative surgery and/or radiation: 0.75 mg/m² IV over 30 minutes on days 1–3, with cisplatin on day 1, every 21 days. (1)
Before taking topotecan
Do not take / not appropriate for
- Topotecan is not given to anyone with a history of severe hypersensitivity (such as anaphylactoid reactions) to topotecan or its ingredients. (United States) (1)
- Not given with severe bone marrow depression, and never started when the baseline neutrophil count is below 1,500 cells/mm³ (boxed warning). () (1)
Tell your clinician about
(1)How to take topotecan
- Topotecan is prepared and given by the oncology team as an intravenous infusion over 30 minutes; you do not take or handle it yourself.
- Blood counts are checked frequently; each cycle only proceeds when counts have recovered sufficiently.
- The schedule (daily for 5 days for lung cancer, or days 1–3 with cisplatin for cervical cancer, both in 21-day cycles) is set and adjusted by the oncology team.
Procedures and treatment changes
Tell the treating team before surgery, procedures, or any medicine change. Do not alter treatment without the responsible clinician. (1)
Missed dose
Doses are scheduled and given by the oncology team; patients do not manage missed doses. If an appointment is missed or delayed, the team adjusts the schedule.
Overdose
Overdose is managed in hospital by the treating team with supportive care; increased bone marrow suppression is the main expected effect. (1)
Side effects
Side effects and their urgency vary; report severe, persistent, or concerning symptoms.
Low blood counts (neutropenia, anemia, thrombocytopenia, leukopenia)
What to notice: Neutropenia 97%, leukopenia 97%, anemia 89%, thrombocytopenia 69% in small-cell-lung-cancer trials; usually found on blood tests.
What to do: The team monitors counts every cycle; report fever, bruising, bleeding, or marked tiredness promptly. (1)
Nausea and vomiting
What to notice: Nausea 64% and vomiting 45% in lung-cancer trials.
What to do: The team can give anti-sickness medicines; report persistent vomiting. (1)
Hair loss (alopecia)
What to notice: About 49% of patients in trials.
What to do: Usually temporary; discuss support options with the care team. (1)
Diarrhea and constipation
What to notice: Diarrhea 22%, constipation 21% in trials.
What to do: Report persistent changes, especially diarrhea with fever. (1)
Possible infection during low counts
What to notice: Fever, chills, sore throat, or feeling unwell.
What to do: Contact the oncology team urgently — this can be an emergency during chemotherapy. (1)
Breathing problems
What to notice: New cough, breathlessness, fever.
What to do: Prompt review for interstitial lung disease. (1)
Serious warnings
Bone marrow suppression (boxed warning) — emergency
Symptoms: Fever, chills, or signs of infection; unusual bruising or bleeding; marked tiredness or pallor. Grade 4 neutropenia occurred in 78% of small-cell-lung-cancer patients.
Action: Contact the oncology team immediately about any fever or signs of infection or bleeding — suppression can be severe and result in infection and death. Blood counts are monitored frequently and treatment is only given when counts allow. (1)
Neutropenic colitis — emergency
Symptoms: Fever together with abdominal pain, especially during periods of low neutrophil counts.
Action: Report fever with abdominal pain to the oncology team urgently — fatalities from neutropenic colitis have been reported. (1)
Interstitial lung disease — serious
Symptoms: New or worsening cough, breathlessness, or fever.
Action: Report promptly; the team investigates and discontinues treatment if interstitial lung disease is confirmed. Some cases have been fatal. (1)
Infusion-site extravasation — serious
Symptoms: Pain, stinging, redness, or swelling at the drip site during or after the infusion.
Action: Tell the infusion staff straight away — most reactions are mild but severe cases have been reported. (1)
Harm to a developing baby (embryo-fetal toxicity) — serious
Symptoms: Not a symptom — a risk with any exposure during pregnancy.
Action: Use effective contraception during treatment and tell the team immediately about a possible pregnancy; topotecan can cause fetal harm. (1)
Interactions
| Medicine or test | Effect | Action |
|---|---|---|
| Cisplatin (when used in the cervical-cancer combination) | The combination regimen has greater blood-count toxicity than topotecan alone; anemia occurred in 94% and neutropenia in 89% of combination-treated patients. | The oncology team schedules and monitors the combination; report all symptoms between visits. (1) |
| Other bone-marrow-suppressing treatments (chemotherapy, radiotherapy) | Additive suppression of blood counts. | The team plans timing and doses around prior and concurrent treatment; give them a full treatment history. (1) |
Pregnancy, breastfeeding, kidney/liver function, age
Pregnancy
Topotecan can cause fetal harm (embryolethality, fetotoxicity, teratogenicity). It is not used in pregnancy; effective contraception is used during treatment. (1)
Breastfeeding
Breastfeeding is discontinued during topotecan treatment. (1)
Children
This label covers adult use in small cell lung cancer and cervical cancer. (1)
Kidney_impairment
No adjustment for creatinine clearance 40–60 mL/min; dose reduced to 0.75 mg/m² for 20–39 mL/min; insufficient data below 20 mL/min. (1)
Liver_impairment
Clearance is reduced with elevated bilirubin, but the usual regimen was tolerated in studies; the team monitors liver function. (1)
Monitoring
Before starting
- Baseline blood counts — neutrophils at least 1,500 cells/mm³ and platelets at least 100,000/mm³ before the first cycle (boxed warning)
- Kidney and liver function review; pregnancy status where applicable
During treatment
- Frequent peripheral blood counts throughout treatment
- Watch for fever, infection signs, abdominal pain, and respiratory symptoms between cycles
Storage
Topotecan is a hazardous drug stored and prepared by the hospital pharmacy (vials at 20–25°C protected from light; prepared under a vertical laminar flow hood with protective equipment; diluted solution used within 4 hours at room temperature or 24 hours refrigerated). Patients never store or handle it.
Professional information
Clinician-facing context only. This section does not replace current product labeling.
United States
Indication: Relapsed sensitive small cell lung cancer; stage IV-B/recurrent/persistent cervical carcinoma (with cisplatin)
Dose summary: SCLC: 1.5 mg/m² IV over 30 min daily × 5 days, q21d. Cervical: 0.75 mg/m² IV over 30 min days 1–3 + cisplatin 50 mg/m² day 1, q21d. First cycle requires ANC ≥1,500/mm³ and platelets ≥100,000/mm³.
Renal context: CrCl 40–60 mL/min: no adjustment. CrCl 20–39 mL/min: reduce to 0.75 mg/m². CrCl <20 mL/min: insufficient data. Hepatic: elevated bilirubin reduces clearance (~67% of normal) but usual regimen tolerated. (1)
Frequently asked questions
Is topotecan a tablet I take at home?
This page covers topotecan injection, which is given intravenously by the oncology team in a healthcare setting. (A separate capsule form of topotecan exists under the Hycamtin brand, with its own labeling.) (1)
Why are my blood tests checked so often?
Topotecan carries a boxed warning for bone marrow suppression: it can sharply lower infection-fighting white cells, platelets, and red cells. Counts are checked frequently, and each cycle only goes ahead once your counts have recovered enough. (1)
What symptoms need urgent attention during treatment?
Fever, chills, or any sign of infection; fever with abdominal pain; unusual bleeding or bruising; and new cough or breathlessness. Contact the oncology team urgently about any of these — infections during low blood counts can be life-threatening, and lung inflammation (interstitial lung disease) has been reported. (1)
Can I become pregnant or breastfeed during treatment?
No — topotecan can harm a developing baby, so effective contraception is used during treatment, and breastfeeding is stopped while receiving it. Discuss timing and options with the oncology team. (1)
What is the generic name of Topotecan?
The generic name is topotecan. It is sold under brand names including Hycamtin.
What class of drug is topotecan?
Topotecan is classed as: Topoisomerase I inhibitor (chemotherapy).
References
- TOPOTECAN injection (Accord Healthcare) — prescribing informationU.S. National Library of Medicine (DailyMed) · United States · dated 2023-12 · accessed 2026-08-08 · source 1
- TOPOTECAN HYDROCHLORIDE INJECTION, POWDER, LYOPHILIZED, FOR SOLUTION [FRESENIUS KABI USA, LLC] — US prescribing information (DailyMed SPL)U.S. National Library of Medicine (DailyMed) · US · accessed 2026-08-14 · source 2
- Topotecan — FDA approval record (Drugs@FDA NDA200582)U.S. Food and Drug Administration · US · accessed 2026-08-14 · source 3
- topotecan — RxNorm normalized drug concept (RXCUI 266573)U.S. National Library of Medicine (RxNorm) · US · accessed 2026-08-14 · source 4
Brand names by country
Single-ingredient topotecan names are shown separately by country. Product availability, strengths, and supply rules can change; combination products are not included here.
Priority countries
United States
- HYCAMTIN4 mg/4mL, .25 mg, 1 mg · novartis pharmaceuticals corporation
Canada
- HYCAMTIN