Drug · FAERS side-effect profile with label screen
Topotecan Hydrochloride side effects: what 2,410 FDA adverse event reports show
Who reports
2,410 reports · 85% serious · 720 with death outcome · 58% female
openFDA FAERS counts, all years, deduplication not applied
Used for
Topotecan Injection is indicated for the treatment of: small cell lung cancer sensitive disease after failure of first-line chemotherapy. In clinical studies submitted to support approval, sensitive disease was defined as disease responding to chemothe.
FDA label, Indications and Usage
What it is
Topotecan is a semi-synthetic derivative of camptothecin and is an anti-tumor drug with topoisomerase I-inhibitory activity.
FDA label, Description section
Boxed warning
BONE MARROW SUPPRESSION Do not give Topotecan Injection to patients with baseline neutrophil counts less than 1,500 cells/mm 3 . In order to monitor the occurrence of bone marrow suppression, primarily neutropenia, w.
FDA label
How to read this page. FAERS is a spontaneous-reporting system: counts are not incidence rates and a signal is not proof of causation.
Each reaction below is run through an automated screen — is the term in the FDA label (known), in the indications (the disease being treated), an administrative/coding term, or none of these (unlabeled)?
That screen is mechanical text matching, not a causality assessment. The 30 hand-written Bradford Hill assessments are on the causality assessment page. Not medical advice.
Causality screen of the 40 strongest signals
Labeled — boxed warning 10Labeled adverse reaction 2Labeled (related wording) 4Likely indication confounding 1Reporting / coding artifact 3Lab value / disease marker 8Not in label — unassessed 12
What the badges mean
- Labeled — boxed warning The term (or its clinical synonym) appears in the FDA boxed warning: causality is regulator-recognised and considered serious.
- Labeled adverse reaction The exact term appears in the label's warnings or adverse-reactions sections: a known, expected effect. The FAERS signal confirms the label rather than discovering anything.
- Labeled (related wording) The label describes this effect under different wording (e.g. 'neutropenia' for 'neutrophil count decreased'). Treated as known.
- Likely indication confounding The term appears in the label's indications: reports probably describe the disease being treated (or its progression), not a drug effect. Classic confounding by indication.
- Reporting / coding artifact An administrative, monitoring or exposure term (product issue, off-label use, 'test normal', overdose...). Not a biological reaction; a high PRR here reflects how reports are coded.
- Lab value / disease marker A laboratory result, investigation or disease-activity marker (antibody positive, CRP abnormal, swollen joint count) not described in the label. These usually mirror the monitored disease or testing protocol rather than a drug effect; not screened further.
- Not in label — unassessed Not found in the label text, not an artifact pattern. This is a genuine candidate for a formal causality assessment (Bradford Hill / WHO-UMC); it has NOT had one here.
Strongest disproportionality signals
Flagged terms (PRR ≥ 2, χ² ≥ 4, n ≥ 10), ranked by PRR; infinite PRRs (no comparator reports) excluded. The EBGM column is a second, shrinkage-based estimate (MGPS) with its 5th percentile EB05 and the BCPNN IC025; a pair flagged by PRR alone is marked PRR-only and should be read as fragile. The strata column recomputes the PRR on serious reports only and flags pairs whose reports are unusually skewed by sex or come mostly from consumers rather than clinicians. 24 further terms are in the interactive explorer. Reaction links open the term's cross-drug page.
Show:
| Reaction | Reports | PRR 95% CI · ROR · χ²/p | EBGM EB05 · IC025 | Label screen | Serious-only & strata | Trial cross-check |
|---|---|---|---|---|---|---|
| Febrile neutropenia | 257 | 33.9 30.2–38.1ROR 37.8 · χ² 8,165 / p <1e-300 | 33.7 EB05 30.4 · IC025 +4.8 3/3 methods agree | Labeled — boxed warning exact term | serious PRR 22.7 | no trial data cached |
| Neutrophil count decreased | 126 | 27.1 22.9–32.2ROR 28.6 · χ² 3,143 / p <1e-300 | 27.0 EB05 23.2 · IC025 +4.3 3/3 methods agree | Labeled — boxed warning synonym match | serious PRR 20.4 · 91% clinician | no trial data cached |
| Accident | 37 | 27.1 19.7–37.4ROR 27.5 · χ² 903 / p 1e-198 | 26.7 EB05 20.2 · IC025 +3.8 3/3 methods agree | Not in label — unassessed | serious PRR 21.8 · ♀ 0% (drug 58%) · 95% clinician | no trial data cached |
| Red blood cell count decreased | 87 | 26.5 21.6–32.6ROR 27.5 · χ² 2,107 / p <1e-300 | 26.3 EB05 22.0 · IC025 +4.2 3/3 methods agree | Likely indication confounding term in indications; also in warnings | serious PRR 22.2 · 91% clinician | no trial data cached |
| Haematotoxicity | 24 | 22.6 15.2–33.6ROR 22.8 · χ² 473 / p 4e-105 | 22.2 EB05 15.6 · IC025 +3.3 3/3 methods agree | Not in label — unassessed | serious PRR 15.0 · 100% clinician | no trial data cached |
| Body temperature increased | 49 | 20.9 15.8–27.5ROR 21.3 · χ² 906 / p 2e-199 | 20.7 EB05 16.2 · IC025 +3.6 3/3 methods agree | Lab value / disease marker | serious PRR 17.4 · ♀ 8% (drug 58%) | no trial data cached |
| Metastases to central nervous system | 27 | 18.4 12.6–26.7ROR 18.6 · χ² 426 / p 7e-95 | 18.1 EB05 12.9 · IC025 +3.2 3/3 methods agree | Not in label — unassessed | serious PRR 12.5 · ♀ 85% (drug 58%) | no trial data cached |
| Haemorrhoids | 38 | 16.9 12.4–23.2ROR 17.2 · χ² 554 / p 9e-123 | 16.7 EB05 12.6 · IC025 +3.3 3/3 methods agree | Not in label — unassessed | serious PRR 14.2 · ♀ 0% (drug 58%) · 95% clinician | no trial data cached |
| White blood cell count decreased | 204 | 16.4 14.4–18.7ROR 17.8 · χ² 2,942 / p <1e-300 | 16.3 EB05 14.5 · IC025 +3.7 3/3 methods agree | Labeled (related wording) related wording | serious PRR 14.4 | no trial data cached |
| Neoplasm progression | 65 | 16.1 12.7–20.5ROR 16.6 · χ² 908 / p 1e-199 | 16.0 EB05 13.0 · IC025 +3.4 3/3 methods agree | Not in label — unassessed | serious PRR 12.7 | no trial data cached |
| Ovarian cancer | 17 | 16.0 10.0–25.7ROR 16.1 · χ² 224 / p 6e-51 | 15.3 EB05 9.6 · IC025 +2.7 3/3 methods agree | Not in label — unassessed | serious PRR 10.8 | no trial data cached |
| Hospice care | 18 | 15.9 10.0–25.2ROR 16.0 · χ² 237 / p 1e-53 | 15.3 EB05 9.8 · IC025 +2.7 3/3 methods agree | Not in label — unassessed | serious PRR 6.1 | no trial data cached |
| Pancytopenia (all blood cells low) | 99 | 15.5 12.8–18.8ROR 16.1 · χ² 1,328 / p 5e-291 | 15.4 EB05 13.0 · IC025 +3.5 3/3 methods agree | Labeled — boxed warning exact term | serious PRR 10.3 | no trial data cached |
| Platelet count decreased | 184 | 15.1 13.1–17.3ROR 16.2 · χ² 2,408 / p <1e-300 | 15.0 EB05 13.3 · IC025 +3.6 3/3 methods agree | Labeled — boxed warning synonym match | serious PRR 11.9 | no trial data cached |
| Ileus | 19 | 13.5 8.6–21.1ROR 13.6 · χ² 207 / p 4e-47 | 12.7 EB05 8.1 · IC025 +2.6 3/3 methods agree | Not in label — unassessed | serious PRR 9.1 | no trial data cached |
| Metastases to liver | 27 | 13.4 9.2–19.5ROR 13.6 · χ² 298 / p 5e-67 | 12.9 EB05 9.0 · IC025 +2.8 3/3 methods agree | Not in label — unassessed | serious PRR 9.1 · ♀ 89% (drug 58%) | no trial data cached |
| Blood lactate dehydrogenase increased | 24 | 13.1 8.8–19.5ROR 13.2 · χ² 256 / p 5e-58 | 12.5 EB05 8.5 · IC025 +2.7 3/3 methods agree | Lab value / disease marker | serious PRR 9.0 · ♀ 17% (drug 58%) · 92% clinician | no trial data cached |
| Drug toxicity | 19 | 12.0 7.7–18.8ROR 12.1 · χ² 180 / p 2e-41 | 11.0 EB05 7.0 · IC025 +2.5 3/3 methods agree | Labeled (related wording) related wording | serious PRR 8.4 | no trial data cached |
| Bone marrow failure | 28 | 11.9 8.2–17.2ROR 12.1 · χ² 269 / p 8e-61 | 11.3 EB05 7.9 · IC025 +2.7 3/3 methods agree | Labeled (related wording) related wording | serious PRR 7.8 | no trial data cached |
| Lymphocyte count decreased | 24 | 11.8 7.9–17.6ROR 11.9 · χ² 227 / p 1e-51 | 11.1 EB05 7.5 · IC025 +2.6 3/3 methods agree | Lab value / disease marker | serious PRR 9.8 | no trial data cached |
| Metastases to bone | 22 | 11.7 7.7–17.8ROR 11.8 · χ² 205 / p 9e-47 | 10.9 EB05 7.2 · IC025 +2.5 3/3 methods agree | Not in label — unassessed | serious PRR 8.0 · ♀ 86% (drug 58%) | no trial data cached |
| Thrombocytopenia (low platelets) | 140 | 10.9 9.3–12.8ROR 11.5 · χ² 1,259 / p 4e-276 | 10.8 EB05 9.4 · IC025 +3.1 3/3 methods agree | Labeled — boxed warning exact term | serious PRR 7.6 | no trial data cached |
| Neutropenia (low neutrophils (infection-fighting cells)) | 169 | 10.9 9.4–12.6ROR 11.6 · χ² 1,512 / p <1e-300 | 10.8 EB05 9.4 · IC025 +3.1 3/3 methods agree | Labeled — boxed warning exact term | serious PRR 7.5 | no trial data cached |
| Anaemia (low red blood cells) | 235 | 10.7 9.5–12.1ROR 11.8 · χ² 2,079 / p <1e-300 | 10.6 EB05 9.5 · IC025 +3.2 3/3 methods agree | Labeled — boxed warning exact term | serious PRR 7.8 | no trial data cached |
| Anorexia | 16 | 10.7 6.5–17.4ROR 10.7 · χ² 131 / p 2e-30 | 9.4 EB05 5.7 · IC025 +2.2 3/3 methods agree | Labeled adverse reaction exact term | serious PRR 9.5 | no trial data cached |
| Blood urea increased | 19 | 9.8 6.3–15.4ROR 9.9 · χ² 142 / p 5e-33 | 8.7 EB05 5.6 · IC025 +2.2 3/3 methods agree | Lab value / disease marker | serious PRR 7.4 | no trial data cached |
| Blood sodium decreased | 21 | 9.7 6.4–14.9ROR 9.8 · χ² 156 / p 5e-36 | 8.7 EB05 5.8 · IC025 +2.3 3/3 methods agree | Lab value / disease marker | serious PRR 7.1 · ♀ 19% (drug 58%) | no trial data cached |
| Blood bilirubin increased | 27 | 8.7 6.0–12.6ROR 8.8 · χ² 176 / p 2e-40 | 7.9 EB05 5.6 · IC025 +2.3 3/3 methods agree | Lab value / disease marker | serious PRR 6.7 | no trial data cached |
| Leukopenia (low white blood cells) | 47 | 8.2 6.2–10.9ROR 8.3 · χ² 291 / p 2e-65 | 7.8 EB05 6.0 · IC025 +2.4 3/3 methods agree | Labeled adverse reaction exact term | serious PRR 5.8 | no trial data cached |
| Full blood count decreased | 18 | 7.8 4.9–12.3ROR 7.8 · χ² 100 / p 9e-24 | 6.7 EB05 4.4 · IC025 +1.9 3/3 methods agree | Lab value / disease marker | serious PRR 5.9 | no trial data cached |
| Blood test abnormal | 12 | 7.4 4.2–13.1ROR 7.5 · χ² 61 / p 3e-15 | 6.0 EB05 3.6 · IC025 +1.6 3/3 methods agree | Reporting / coding artifact | serious PRR 4.5 | no trial data cached |
| Disease progression | 95 | 7.0 5.8–8.5ROR 7.3 · χ² 486 / p 5e-108 | 6.8 EB05 5.7 · IC025 +2.4 3/3 methods agree | Reporting / coding artifact | serious PRR 4.9 | no trial data cached |
| Interstitial lung disease | 38 | 6.8 5.0–9.4ROR 6.9 · χ² 184 / p 3e-42 | 6.4 EB05 4.8 · IC025 +2.1 3/3 methods agree | Labeled — boxed warning exact term | serious PRR 4.7 · ♀ 28% (drug 58%) | no trial data cached |
| Agranulocytosis (severe loss of white cells) | 12 | 5.7 3.2–9.9ROR 5.7 · χ² 41 / p 6e-11 | 4.7 EB05 2.9 · IC025 +1.3 3/3 methods agree | Labeled — boxed warning synonym match | serious PRR 3.8 | no trial data cached |
| Acute myeloid leukaemia | 10 | 5.6 3.0–10.4ROR 5.6 · χ² 33 / p 4e-9 | 4.5 EB05 2.7 · IC025 +1.1 3/3 methods agree | Not in label — unassessed | serious PRR 3.8 | no trial data cached |
| Haemoglobin decreased | 64 | 5.4 4.3–6.9ROR 5.5 · χ² 227 / p 1e-51 | 5.2 EB05 4.2 · IC025 +2.0 3/3 methods agree | Labeled — boxed warning synonym match | serious PRR 3.7 | no trial data cached |
| C-reactive protein increased | 22 | 5.3 3.5–8.0ROR 5.3 · χ² 72 / p 1e-17 | 4.8 EB05 3.3 · IC025 +1.6 3/3 methods agree | Lab value / disease marker | serious PRR 3.7 · ♀ 32% (drug 58%) | no trial data cached |
| Septic shock | 25 | 5.0 3.4–7.4ROR 5.1 · χ² 77 / p 9e-19 | 4.6 EB05 3.3 · IC025 +1.6 3/3 methods agree | Not in label — unassessed | serious PRR 3.4 | no trial data cached |
| Mucosal inflammation | 15 | 5.0 3.0–8.3ROR 5.0 · χ² 44 / p 1e-11 | 4.4 EB05 2.8 · IC025 +1.3 3/3 methods agree | Labeled (related wording) related wording | serious PRR 3.6 | no trial data cached |
| Malignant neoplasm progression | 48 | 4.1 3.1–5.4ROR 4.2 · χ² 110 / p 5e-26 | 4.0 EB05 3.1 · IC025 +1.5 3/3 methods agree | Reporting / coding artifact | serious PRR 2.8 | no trial data cached |
Trial evidence (ClinicalTrials.gov)
Trial cross-check not yet run for this drug (ClinicalTrials.gov pull covers the 800 most-reported drugs first).
Most frequently reported reactions
By raw co-report count, regardless of disproportionality. Common terms here are often the illness itself or generic outcomes.
| Reaction | Reports | PRR 95% CI · ROR · χ²/p | EBGM EB05 · IC025 | Label screen | Serious-only & strata | Trial cross-check |
|---|---|---|---|---|---|---|
| Death | 384 | 3.9 3.5–4.3ROR 4.4 · χ² 853 / p 8e-188 | 3.9 EB05 3.5 · IC025 +1.8 3/3 methods agree | Outcome field, not a reaction boxed warning mentions mortality | serious PRR 2.6 | no trial data cached |
| Febrile neutropenia | 257 | 33.9 30.2–38.1ROR 37.8 · χ² 8,165 / p <1e-300 | 33.7 EB05 30.4 · IC025 +4.8 3/3 methods agree | Labeled — boxed warning exact term | serious PRR 22.7 | no trial data cached |
| Anaemia (low red blood cells) | 235 | 10.7 9.5–12.1ROR 11.8 · χ² 2,079 / p <1e-300 | 10.6 EB05 9.5 · IC025 +3.2 3/3 methods agree | Labeled — boxed warning exact term | serious PRR 7.8 | no trial data cached |
| White blood cell count decreased | 204 | 16.4 14.4–18.7ROR 17.8 · χ² 2,942 / p <1e-300 | 16.3 EB05 14.5 · IC025 +3.7 3/3 methods agree | Labeled (related wording) related wording | serious PRR 14.4 | no trial data cached |
| Platelet count decreased | 184 | 15.1 13.1–17.3ROR 16.2 · χ² 2,408 / p <1e-300 | 15.0 EB05 13.3 · IC025 +3.6 3/3 methods agree | Labeled — boxed warning synonym match | serious PRR 11.9 | no trial data cached |
| Neutropenia (low neutrophils (infection-fighting cells)) | 169 | 10.9 9.4–12.6ROR 11.6 · χ² 1,512 / p <1e-300 | 10.8 EB05 9.4 · IC025 +3.1 3/3 methods agree | Labeled — boxed warning exact term | serious PRR 7.5 | no trial data cached |
| Thrombocytopenia (low platelets) | 140 | 10.9 9.3–12.8ROR 11.5 · χ² 1,259 / p 4e-276 | 10.8 EB05 9.4 · IC025 +3.1 3/3 methods agree | Labeled — boxed warning exact term | serious PRR 7.6 | no trial data cached |
| Neutrophil count decreased | 126 | 27.1 22.9–32.2ROR 28.6 · χ² 3,143 / p <1e-300 | 27.0 EB05 23.2 · IC025 +4.3 3/3 methods agree | Labeled — boxed warning synonym match | serious PRR 20.4 · 91% clinician | no trial data cached |
| Pancytopenia (all blood cells low) | 99 | 15.5 12.8–18.8ROR 16.1 · χ² 1,328 / p 5e-291 | 15.4 EB05 13.0 · IC025 +3.5 3/3 methods agree | Labeled — boxed warning exact term | serious PRR 10.3 | no trial data cached |
| Disease progression | 95 | 7.0 5.8–8.5ROR 7.3 · χ² 486 / p 5e-108 | 6.8 EB05 5.7 · IC025 +2.4 3/3 methods agree | Reporting / coding artifact | serious PRR 4.9 | no trial data cached |
| Red blood cell count decreased | 87 | 26.5 21.6–32.6ROR 27.5 · χ² 2,107 / p <1e-300 | 26.3 EB05 22.0 · IC025 +4.2 3/3 methods agree | Likely indication confounding term in indications; also in warnings | serious PRR 22.2 · 91% clinician | no trial data cached |
| Neoplasm progression | 65 | 16.1 12.7–20.5ROR 16.6 · χ² 908 / p 1e-199 | 16.0 EB05 13.0 · IC025 +3.4 3/3 methods agree | Not in label — unassessed | serious PRR 12.7 | no trial data cached |
| Haemoglobin decreased | 64 | 5.4 4.3–6.9ROR 5.5 · χ² 227 / p 1e-51 | 5.2 EB05 4.2 · IC025 +2.0 3/3 methods agree | Labeled — boxed warning synonym match | serious PRR 3.7 | no trial data cached |
| Body temperature increased | 49 | 20.9 15.8–27.5ROR 21.3 · χ² 906 / p 2e-199 | 20.7 EB05 16.2 · IC025 +3.6 3/3 methods agree | Lab value / disease marker | serious PRR 17.4 · ♀ 8% (drug 58%) | no trial data cached |
| Malignant neoplasm progression | 48 | 4.1 3.1–5.4ROR 4.2 · χ² 110 / p 5e-26 | 4.0 EB05 3.1 · IC025 +1.5 3/3 methods agree | Reporting / coding artifact | serious PRR 2.8 | no trial data cached |
Unlabeled candidate signals
These flagged terms were not found in the FDA label text and do not match an artifact pattern. They are the pairs that would merit a structured assessment (temporality, dose-response, biological plausibility, consistency across databases, trial or cohort evidence). The screen has not done that; string-matching misses label wording, and a strong PRR can still be stimulated reporting, co-medication, or the disease itself.
- Accident — n=37, PRR 27.1
- Haematotoxicity — n=24, PRR 22.6
- Metastases to central nervous system — n=27, PRR 18.4
- Haemorrhoids — n=38, PRR 16.9
- Neoplasm progression — n=65, PRR 16.1
- Metastases to liver — n=27, PRR 13.4
- Metastases to bone — n=22, PRR 11.7
- Septic shock — n=25, PRR 5.0
Want to assess one of these properly? The runbook describes the Bradford Hill / trial-evidence workflow used on the assessment page, and handoff_top1000_signals.csv lists the strongest unassessed pairs across all drugs.
Common questions
What are the most reported side effects of Topotecan?
In FDA FAERS, the most frequently co-reported reaction terms for Topotecan are febrile neutropenia, anaemia, white blood cell count decreased, platelet count decreased, neutropenia, thrombocytopenia. These are report counts, not rates: FAERS has no denominator, reporting is voluntary and uneven, and a term can be reported because of the illness being treated rather than the drug.
Does Topotecan have an FDA boxed warning?
Yes. The current FDA label carries a boxed warning: BONE MARROW SUPPRESSION Do not give Topotecan Injection to patients with baseline neutrophil counts less than 1,500 cells/mm 3 . In order to monitor the occurrence of bone marrow suppression, primarily neutropenia, w.
Do FAERS signals prove that Topotecan causes these side effects?
No. A disproportionality signal (PRR, ROR) means a reaction is reported more often with this drug than with others in the database. That can reflect a real effect, but also the disease being treated, stimulated reporting after news or litigation, co-medications, or how reports are coded. Causality needs temporality, dose-response, biological plausibility, consistency across data sources and, ideally, trial or cohort evidence. This page screens each term against the FDA label and ClinicalTrials.gov results; it does not establish causation.
Sources & methods
- FDA label via openFDA (set ID 7636bc1e-77b6-46ad-9df0-c8bb1c5e503f, effective 20251217; matched on
openfda.generic_name.exact:"TOPOTECAN") - Bayesian shrinkage measures computed offline over all 1,317,300 cached pairs: BCPNN information component (IC, with Norén closed-form IC025) and MGPS empirical-Bayes geometric mean (EBGM, EB05) with a two-component gamma prior fitted by maximum marginal likelihood (α₁=0.57, β₁=0.02, α₂=1.42, β₂=0.70, w=0.17); unstratified
- FAERS pair statistics from the OpenPV build of 2026-09-14 (openFDA
count=aggregation, fieldpatient.drug.openfda.generic_name.exact, 64 reaction terms, min 3 co-reports); PRR/ROR with 95% CI, chi-square with Yates correction - Serious-only and stratified counts (serious:1, patient.patientsex, primarysource.qualification) from openFDA count queries; serious-only PRR uses the drug serious report total and the term global serious total (N_serious=11,882,979); strata shown only when n ≥ 20
- Seriousness and sex counts from openFDA
count=serious,patient.patientsex,seriousnessdeath
Page generated 2026-10-08. Label and PubChem text are retrieved automatically and may lag the current label; always check the current prescribing information. OpenPV is a beta research tool of the Open Source Medicine Foundation.