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Topotecan Side Effects

In Summary

Commonly reported side effects of topotecan include: febrile neutropenia, sepsis, infection, abdominal pain, alopecia, anemia, asthenia, constipation, dyspnea, fatigue, fever, headache, leukopenia, nausea, neutropenia, stomatitis, thrombocytopenia, vomiting, granulocytopenia, diarrhea, and anorexia. Other side effects include: increased serum alanine aminotransferase, increased serum aspartate aminotransferase, intestinal obstruction, pain, and paresthesia. See below for a comprehensive list of adverse effects.

For the Consumer

Applies to topotecan: oral capsule

Along with its needed effects, topotecan may cause some unwanted effects. Although not all of these side effects may occur, if they do occur they may need medical attention.

Check with your doctor immediately if any of the following side effects occur while taking topotecan:

More common
  • Black, tarry stools
  • bleeding gums
  • blood in the urine or stools
  • chest pain
  • chills
  • cough
  • fever
  • lower back or side pain
  • painful or difficult urination
  • pale skin
  • pinpoint red spots on the skin
  • shortness of breath
  • sore throat
  • sores, ulcers, or white spots on the lips or in the mouth
  • swollen glands
  • troubled breathing with exertion
  • unusual bleeding or bruising
  • unusual tiredness or weakness

Some side effects of topotecan may occur that usually do not need medical attention. These side effects may go away during treatment as your body adjusts to the medicine. Also, your health care professional may be able to tell you about ways to prevent or reduce some of these side effects. Check with your health care professional if any of the following side effects continue or are bothersome or if you have any questions about them:

More common

For Healthcare Professionals

Applies to topotecan: intravenous powder for injection, intravenous solution, oral capsule


The most common adverse events were hematologic: neutropenia, leukopenia, anemia, and thrombocytopenia.[Ref]


Very common (10% or more): Neutropenia (up to 97%), leukopenia (97%), anemia (up to 94%), thrombocytopenia (up to 74%), pyrexia/grade 4 neutropenia (28%), febrile neutropenia (28%), hemorrhage (15%)
Common (1% to 10%): Pancytopenia, coagulation adverse event
Postmarketing reports: Severe bleeding (in association with thrombocytopenia)[Ref]


Very common (10% or more): Nausea (64%), other gastrointestinal adverse event (63%), vomiting (45%), diarrhea (32%), constipation (29%), abdominal pain (22%), stomatitis (18%)
Common (1% to 10%): Abdominal pain, intestinal obstruction, dyspepsia
Frequency not reported: Neutropenic colitis
Postmarketing reports: Abdominal pain potentially associated with neutropenic colitis[Ref]


Rash includes pruritus, rash erythematous, urticaria, dermatitis, bullous eruption, and maculopapular rash.[Ref]

Very common (10% or more): Alopecia (49%), dermatologic adverse reaction (48%), rash (16%)
Common (1% to 10%): Pruritus
Frequency not reported: Urticaria
Postmarketing reports: Angioedema, severe dermatitis, severe pruritus[Ref]


Very common (10% or more): Sepsis or pyrexia/infection with neutropenia (43%), infection-febrile neutropenia (28%), infection (27%)
Common (1% to 10%): Pneumonia, allergy-immunology adverse event, pharyngitis, rhinitis, sepsis


Pain includes abdominal pain or cramping, arthralgia, bone pain, chest pain (non-cardiac and non-pleuritic), dysmenorrhea, dyspareunia, earache, headache, hepatic pain, myalgia, neuropathic pain, pain due to radiation, pelvic pain, pleuritic pain, rectal or perirectal pain, tumor pain, body pain, back pain, and skeletal pain.
"Constitutional" is a composite term that includes fatigue (lethargy, malaise, asthenia), fever (in the absence of neutropenia), rigors, chills, sweating, and weight gain or loss.

Very common (10% or more): Constitutional (69%), pain (up to 59%), fatigue (29%), pyrexia (28%), asthenia (25%), mucositis (14%)
Common (1% to 10%): Treatment related death, malaise


Very common (10% or more): Dyspnea (22%), pulmonary adverse event (17%), coughing (15%)
Common (1% to 10%): Epistaxis, respiratory disorder
Rare (less than 0.1%): Interstitial lung disease
Postmarketing reports: Fatal interstitial lung disease[Ref]


Very common (10% or more): Hepatic adverse event (24%)
Common (1% to 10%): Transient elevations in hepatic enzymes, hyperbilirubinemia, elevated ALT, elevated AST[Ref]

Nervous system

Very common (10% or more): Non-neuropathy neurologic adverse event (35%), headache (18%)
Common (1% to 10%): Paresthesia, neuropathy, dizziness
Uncommon (0.1% to 1%): Hypoesthesia, peripheral neuropathy, paresis[Ref]


Very common (10% or more): Cardiovascular adverse event (25%)[Ref]


Very common (10% or more): Metabolic-laboratory adverse event (39%), anorexia (19%)
Frequency not reported: Dehydration


Very common (10% or more): Musculoskeletal adverse event (14%)[Ref]


Common (1% to 10%): Hypersensitivity (including rash)
Postmarketing reports: Allergic manifestations, anaphylactoid reactions[Ref]


Very common (10% or more): Genitourinary adverse event (36%)
Common (1% to 10%): Sexual reproduction function adverse event[Ref]


Rare (less than 0.1%): Extravasation
Postmarketing reports: Inadvertent extravasation


Common (1% to 10%): Endocrine adverse event


Common (1% to 10%): Ocular-visual adverse event


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Some side effects of topotecan may not be reported. Always consult your doctor or healthcare specialist for medical advice. You may also report side effects to the FDA.

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