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Topotecan

Drugs and Chemicals · NLM MeSH · updated 2026-10-31 · version 1

Citations verified against PubMedNot yet clinician-reviewed10 referencesApproximately 5 min read

An antineoplastic agent used to treat ovarian cancer.

Table of Contents

1. Definition2. Research landscape3. Reading the evidence4. Open-access studies5. Key reviews6. References7. About this article

Definition and research guide

This page combines a MeSH definition, publication statistics and selected research abstracts. It is a starting point for reading the literature, not a medical textbook or a systematic evidence review.

Content available: 4 complete abstracts (1,058 words), 10 references. Full article text is not stored or reproduced here; follow the PMID, DOI or PMC links to read the original sources and their access terms.

Definition

An antineoplastic agent used to treat ovarian cancer. It works by inhibiting dna topoisomerases, TYPE I.

Definition: NLM Medical Subject Headings (MeSH) scope note (Public domain (U.S. National Library of Medicine); attribution requested). View the MeSH record.

Also known as: 9-Dimethylaminomethyl-10-hydroxycamptothecin; 9 Dimethylaminomethyl 10 hydroxycamptothecin; Topotecan Hydrochloride; Hydrochloride, Topotecan; Topotecan Monohydrochloride, (S)-Isomer; Nogitecan Hydrochloride; Hydrochloride, Nogitecan; Hycamtin.

Where it fits in the MeSH classification

Broader topics
Camptothecin

Related concepts

  • Dna Topoisomerases

Research landscape

PubMed indexes 2,405 records tagged with the MeSH heading “Topotecan” (counts retrieved 2026-10-31). 269 of them (11.2%) were published from 2020 onward, and the busiest period so far is 2000-2009 with 1,052 records.

Reviews account for 273 records (11.4%) and meta-analyses for 20. Randomized controlled trials number 145 (6.0%) as a share of all records on the topic.

PubMed records by publication periodthrough 1999: 381 records; 2000-2009: 1,052 records; 2010-2019: 703 records; 2020 onward: 269 records381through 19991,0522000-20097032010-20192692020 onward
Records in PubMed indexed with this heading, by publication period. Real counts from NCBI E-utilities; the final period covers only part of the current year. Volume reflects how much has been published, not how strong the evidence is.
Show the data
PeriodRecords
through 1999381
2000-20091,052
2010-2019703
2020 onward269
Share of records by publication typeReviews: 11.4 percent; Meta-analyses: 0.8 percent; Randomized controlled trials: 6.0 percentReviews11.4% (273)Meta-analyses0.8% (20)Randomized controlled trials6.0% (145)
Share of all PubMed records on this heading that carry each publication type. Types overlap with other records, so the shares do not sum to 100%.
Publication-type chart data
Publication types among 2,405 PubMed records
TypeRecordsShare
Reviews27311.4%
Meta-analyses200.8%
Randomized controlled trials1456.0%

Method: PubMed query "Topotecan"[MeSH Terms] with publication-type and date filters, retrieved 2026-10-31.

Reading the evidence in drugs and chemicals

Studies of a chemical or drug can answer very different questions: what it is and how it behaves in cells and animals, how the body handles it, and whether it changes outcomes in people. Laboratory and animal findings often do not carry over to patients, and observational studies of exposures are confounded by who uses a substance and why. Nothing on this page is a recommendation to use, avoid, start or stop any substance or medicine; talk to a qualified clinician or pharmacist about your own situation. Check the dose, the species, the comparison group and whether a patient-relevant outcome was measured.

Evidence from open-access studies

These open-access studies come from PubMed’s best-match results for this heading (papers whose main topic it is), ranked here by citation count, with one recent addition. Each abstract is reproduced in full under its recorded open licence so that you can read the authors’ own words. They illustrate the literature; they are not a systematic summary of it. Citation counts measure attention, not reliability or clinical benefit.

Study comparison — selected abstracts, not a systematic review
Study / authorsYearSource label / designEurope PMC citationsLicence / source
Topotecan for Relapsed Small-cell Lung Cancer: Systematic Review and Meta-Analysis of 1347 Patients
Horita N, Yamamoto M, Sato T, Tsukahara T, Nagakura H, Tashiro K, et al.
2015Meta-analysis
Design not recorded in structured metadata
85cc by
PMID 26486755 · DOI · PMC4614251
A phase III trial of topotecan and whole brain radiation therapy for patients with CNS-metastases due to lung cancer
Neuhaus T, Ko Y, Muller RP, Grabenbauer GG, Hedde JP, Schueller H, et al.
2009Randomized controlled trial
Design not recorded in structured metadata
68cc by
PMID 19127261 · DOI · PMC2634726
Topoisomerase I inhibitors, shikonin and topotecan, inhibit growth and induce apoptosis of glioma cells and glioma stem cells
Zhang FL, Wang P, Liu YH, Liu LB, Liu XB, Li Z, et al.
2013Research article
Design not recorded in structured metadata
57cc by
PMID 24303074 · DOI · PMC3841142
Quercetin Enhances Topotecan Cytotoxicity in Retinoblastoma Cells Through ROS-Associated Stress and Apoptotic Signaling
Maçin A, Duman E, Özdemir İ, Tuncer MC
2026Research article
Design not recorded in structured metadata
0cc by
PMID 42072718 · DOI · PMC13115432

Topotecan for Relapsed Small-cell Lung Cancer: Systematic Review and Meta-Analysis of 1347 Patients [1]

Meta-analysis Horita N, Yamamoto M, Sato T, Tsukahara T, Nagakura H, Tashiro K, et al. · · 2015 · cited 85 times (Europe PMC)

Abstract reading summary — source excerpts
Opening excerpt
Topotecan is the most reliable chemotherapy regimen for relapsed small-cell lung carcinoma (SCLC).
Closing excerpt
Adverse events were mainly hematological.
Interpretation limits
Verbatim excerpts from the authors’ abstract; the closing excerpt may describe a finding, limitation or recommendation. Design, population, effect size and uncertainty have not been independently extracted or appraised. Read the complete abstract and linked paper before interpreting the findings.
Read complete abstract (197 words)

Topotecan is the most reliable chemotherapy regimen for relapsed small-cell lung carcinoma (SCLC). The efficacy and adverse effects of topotecan as reported by previous studies varied greatly. The inclusion criterion was a prospective study that was able to provide data for 6-month over-all survival (OS) rate, 1-year OS rate, objective responses, and/or adverse effects of single agent topotecan as a second line chemotherapy for SCLC, written in English language as a full article. Any topotecan regimen were allowed. Binary data were meta-analyzed with the random-model generic inverse variance method. We included 14 articles consisted of 1347 patients. Pooled values were estimated as follows. Six-month OS rate: 37% (95% CI: 28-46%). One-year OS rate: 9% (95% CI: 5-13%). Response rate: 5% (95% CI: 1-8%). Six-month OS rate: 57% (95% CI: 50-64%). One-year OS rate: 27% (95% CI: 22-32%). Response rate: 17% (95% CI: 11-23%). Grade III/IV neutropenia 69% (95% CI: 58-80%). Grade III/IV thrombopenia 41% (95% CI: 34-48%). Grade III/IV anemia 24% (95% CI: 17-30%). Non-hematorogical events were rare. Chemotherapy-related death 2% (95% CI: 1-3%). In conclusion, Topotecan provided a possibly promising outcome for sensitive-relapse SCLC and poor outcome for refractory relapse SCLC. Adverse events were mainly hematological.

Abstract reproduced from the open-access article under the recorded cc by licence; © the authors. PMID 26486755 · DOI · PMC4614251

A phase III trial of topotecan and whole brain radiation therapy for patients with CNS-metastases due to lung cancer [2]

Randomized controlled trial Neuhaus T, Ko Y, Muller RP, Grabenbauer GG, Hedde JP, Schueller H, et al. · · 2009 · cited 68 times (Europe PMC)

Abstract reading summary — source excerpts
Opening excerpt
Brain metastases represent an important cause of morbidity in patients with lung cancer and are associated with a mean survival of less than 6 months.
Closing excerpt
The available data show no significant advantage for concurrent radiochemotherapy for patients with lung cancer; however, the recruited number of patients is too low to exhibit a small advantage of combined treatment.
Interpretation limits
Verbatim excerpts from the authors’ abstract; the closing excerpt may describe a finding, limitation or recommendation. Design, population, effect size and uncertainty have not been independently extracted or appraised. Read the complete abstract and linked paper before interpreting the findings.
Read complete abstract (317 words)

Brain metastases represent an important cause of morbidity in patients with lung cancer and are associated with a mean survival of less than 6 months. Thus, new regimens improving the outcome of these patients are urgently needed. On the basis of promising data raised in a phase I/II trial, we initiated an open, randomised, prospective, multicentric phase III trial, comparing whole brain radiation therapy (WBRT; 20 x 2 Gy) alone with WBRT+topotecan (RCT; 0.4 mg m(-2) day(-1) x 20). A total of 320 patients with CNS-metastases due to SCLC or NSCLC were projected. The primary end point was overall survival, whereas second end points were local response and progression-free survival. However, until the cutoff date of study completion (i.e., a study duration of 34 months), only a total of 96 (RCT:47, WBRT:49) patients had been recruited, and so an analysis was performed at that time point. Although the numbers of grade 3/4 non-haematological toxicities (besides alopecia 115 (RCT/WBRT: 55 out of 60) were evenly distributed, the 25 haematological events occurred mainly in the combined treatment arm (24 out of 1). Local response, evaluated 2 weeks after treatment, was assessable in 44 (RCT/WBRT: 23 out of 21) patients, showing CR in eight (3 out of 5), PR in 17 (11 out of 6), SD in 14 (8 out of 6) and PD in five (1 out of 4) patients (all differences n.s.). Neither OAS (RCT/WBRT: median (days)): 87 out of 95, range 3-752/4-433; HR 1.32; 95% CI (0.83; 2.10)) nor PFS (median (days)): 71 out of 66, range, 3-399/4-228; HR 1.28, 95% CI (0.73; 2.43) differed significantly. On the basis of these results and the slow recruitment, a continuation of the study did not seem reasonable. The available data show no significant advantage for concurrent radiochemotherapy for patients with lung cancer; however, the recruited number of patients is too low to exhibit a small advantage of combined treatment.

Abstract reproduced from the open-access article under the recorded cc by licence; © the authors. PMID 19127261 · DOI · PMC2634726

Topoisomerase I inhibitors, shikonin and topotecan, inhibit growth and induce apoptosis of glioma cells and glioma stem cells [3]

Research article Zhang FL, Wang P, Liu YH, Liu LB, Liu XB, Li Z, et al. · · 2013 · cited 57 times (Europe PMC)

Abstract reading summary — source excerpts
Opening excerpt
Gliomas, the most malignant form of brain tumors, contain a small subpopulation of glioma stem cells (GSCs) that are implicated in therapeutic resistance and tumor recurrence.
Closing excerpt
Therefore, the above results showed that topoisomerase I inhibitors, shikonin and topotecan, inhibited growth and induced apoptosis of GSCs as well as glioma cells, which suggested that they might be the potential anticancer agents targeting gliomas to provide a novel therapeutic strategy.
Interpretation limits
Verbatim excerpts from the authors’ abstract; the closing excerpt may describe a finding, limitation or recommendation. Design, population, effect size and uncertainty have not been independently extracted or appraised. Read the complete abstract and linked paper before interpreting the findings.
Read complete abstract (229 words)

Gliomas, the most malignant form of brain tumors, contain a small subpopulation of glioma stem cells (GSCs) that are implicated in therapeutic resistance and tumor recurrence. Topoisomerase I inhibitors, shikonin and topotecan, play a crucial role in anti-cancer therapies. After isolated and identified the GSCs from glioma cells successfully, U251, U87, GSCs-U251 and GSCs-U87 cells were administrated with various concentrations of shikonin or topotecan at different time points to seek for the optimal administration concentration and time point. The cell viability, cell cycle and apoptosis were detected using cell counting kit-8 and flow cytometer to observe the inhibitory effects on glioma cells and GSCs. We demonstrated that shikonin and topotecan obviously inhibited proliferation of not only human glioma cells but also GSCs in a dose- and time-dependent manner. According to the IC50 values at 24 h, 2 μmol/L of shikonin and 3 μmol/L of topotecan were selected as the optimal administration concentration. In addition, shikonin and topotecan induced cell cycle arrest in G0/G1 and S phases and promoted apoptosis. The down-regulation of Bcl-2 expression with the activation of caspase 9/3-dependent pathway was involved in the apoptosis process. Therefore, the above results showed that topoisomerase I inhibitors, shikonin and topotecan, inhibited growth and induced apoptosis of GSCs as well as glioma cells, which suggested that they might be the potential anticancer agents targeting gliomas to provide a novel therapeutic strategy.

Abstract reproduced from the open-access article under the recorded cc by licence; © the authors. PMID 24303074 · DOI · PMC3841142

Quercetin Enhances Topotecan Cytotoxicity in Retinoblastoma Cells Through ROS-Associated Stress and Apoptotic Signaling [4]

Research article Maçin A, Duman E, Özdemir İ, Tuncer MC · · 2026

Abstract reading summary — source excerpts
Opening excerpt
Quercetin, a naturally occurring flavonoid, exhibits antiproliferative and pro-apoptotic effects across various cancer models.
Closing excerpt
These findings should be interpreted within a preclinical context and suggest that quercetin may represent a potential adjunct strategy warranting further validation in translational and in vivo models.
Interpretation limits
Verbatim excerpts from the authors’ abstract; the closing excerpt may describe a finding, limitation or recommendation. Design, population, effect size and uncertainty have not been independently extracted or appraised. Read the complete abstract and linked paper before interpreting the findings.
Read complete abstract (315 words)

Quercetin, a naturally occurring flavonoid, exhibits antiproliferative and pro-apoptotic effects across various cancer models. Topotecan, a topoisomerase I inhibitor, is used in the treatment of retinoblastoma; however, its clinical utility is limited by dose-dependent toxicity. This study aimed to investigate whether quercetin is associated with enhanced topotecan-induced cytotoxicity in retinoblastoma and to explore the underlying mechanisms under both two-dimensional (2D) and three-dimensional (3D) conditions. Cell viability was assessed using the MTT assay, and drug interactions were evaluated using the combination index (CI) based on the Chou-Talalay method. Apoptosis was analyzed by Annexin V-FITC/PI staining and flow cytometry. Reactive oxygen species (ROS) levels and mitochondrial membrane potential were evaluated using fluorometric methods, and N-acetyl-L-cysteine (NAC) was used for functional modulation of oxidative stress. Three-dimensional tumor spheroid models were used to assess treatment effects under conditions that partially recapitulate tumor architecture. Gene expression levels of apoptosis-related markers and PI3K/Akt/mTOR pathway components were analyzed by quantitative real-time polymerase chain reaction (qRT-PCR). The combination of quercetin and topotecan was associated with synergistic cytotoxic effects in Y79 cells (CI < 1), accompanied by increased ROS levels, mitochondrial membrane depolarization, and elevated apoptotic cell death. NAC co-treatment partially attenuated ROS levels and restored cell viability. In 3D spheroid models, combination treatment induced structural disruption, reduced viability, and increased cell death, effects that were partially reversed by NAC. Gene expression analysis revealed upregulation of pro-apoptotic genes and downregulation of survival-related genes, along with increased PTEN expression. Quercetin is associated with enhanced topotecan-induced cytotoxicity in retinoblastoma cells under both 2D and 3D conditions. These effects were associated with ROS-associated cellular stress, mitochondrial dysfunction, and modulation of apoptotic and survival-related pathways. The partial rescue by NAC supports a contributory, but not exclusive, role of oxidative stress. These findings should be interpreted within a preclinical context and suggest that quercetin may represent a potential adjunct strategy warranting further validation in translational and in vivo models.

Abstract reproduced from the open-access article under the recorded cc by licence; © the authors. PMID 42072718 · DOI · PMC13115432

Key reviews and meta-analyses

The most cited review-type papers among PubMed’s best-match results for this heading, listed with bibliographic details only. Follow the links for the full text and access terms.

  • Research article Topotecan - A novel topoisomerase I inhibitor: pharmacology and clinical experience [5]
    Kollmannsberger C, Mross K, Jakob A, Kanz L, Bokemeyer C · Oncology · 1999 · cited 137 times (Europe PMC)
  • Research article Pharmacology of topoisomerase I inhibitors irinotecan (CPT-11) and topotecan [6]
    Mathijssen RH, Loos WJ, Verweij J, Sparreboom A · Curr Cancer Drug Targets · 2002 · cited 101 times (Europe PMC)
  • Research article Integrated computational strategies for UV/vis spectra of large molecules in solution [7]
    Barone V, Polimeno A · Chem Soc Rev · 2007 · cited 82 times (Europe PMC)
  • Research article Extending the platinum-free interval in recurrent ovarian cancer: the role of topotecan in second-line chemotherapy [8]
    Bookman MA · Oncologist · 1999 · cited 74 times (Europe PMC)
  • Research article Population pharmacokinetic and adverse event analysis of topotecan in patients with solid tumors [9]
    Mould DR, Holford NH, Schellens JH, Beijnen JH, Hutson PR, Rosing H, et al. · Clin Pharmacol Ther · 2002 · cited 59 times (Europe PMC)
  • Research article The role of topotecan in the treatment of brain metastases [10]
    Wong ET, Berkenblit A · Oncologist · 2004 · cited 58 times (Europe PMC)

References

  1. Horita N, Yamamoto M, Sato T, Tsukahara T, Nagakura H, Tashiro K, et al.. Topotecan for Relapsed Small-cell Lung Cancer: Systematic Review and Meta-Analysis of 1347 Patients. . 2015. PMID 26486755 · DOI · PMC4614251
  2. Neuhaus T, Ko Y, Muller RP, Grabenbauer GG, Hedde JP, Schueller H, et al.. A phase III trial of topotecan and whole brain radiation therapy for patients with CNS-metastases due to lung cancer. . 2009. PMID 19127261 · DOI · PMC2634726
  3. Zhang FL, Wang P, Liu YH, Liu LB, Liu XB, Li Z, et al.. Topoisomerase I inhibitors, shikonin and topotecan, inhibit growth and induce apoptosis of glioma cells and glioma stem cells. . 2013. PMID 24303074 · DOI · PMC3841142
  4. Maçin A, Duman E, Özdemir İ, Tuncer MC. Quercetin Enhances Topotecan Cytotoxicity in Retinoblastoma Cells Through ROS-Associated Stress and Apoptotic Signaling. . 2026. PMID 42072718 · DOI · PMC13115432
  5. Kollmannsberger C, Mross K, Jakob A, Kanz L, Bokemeyer C. Topotecan - A novel topoisomerase I inhibitor: pharmacology and clinical experience. Oncology. 1999. PMID 9885371 · DOI
  6. Mathijssen RH, Loos WJ, Verweij J, Sparreboom A. Pharmacology of topoisomerase I inhibitors irinotecan (CPT-11) and topotecan. Curr Cancer Drug Targets. 2002. PMID 12188913 · DOI
  7. Barone V, Polimeno A. Integrated computational strategies for UV/vis spectra of large molecules in solution. Chem Soc Rev. 2007. PMID 18213981 · DOI
  8. Bookman MA. Extending the platinum-free interval in recurrent ovarian cancer: the role of topotecan in second-line chemotherapy. Oncologist. 1999. PMID 10337378 · DOI
  9. Mould DR, Holford NH, Schellens JH, Beijnen JH, Hutson PR, Rosing H, et al.. Population pharmacokinetic and adverse event analysis of topotecan in patients with solid tumors. Clin Pharmacol Ther. 2002. PMID 12011819 · DOI
  10. Wong ET, Berkenblit A. The role of topotecan in the treatment of brain metastases. Oncologist. 2004. PMID 14755016 · DOI

About this article

Sources
NLM Medical Subject Headings (definition, synonyms); NCBI PubMed (counts, identifiers); Europe PMC (open-access abstracts and citation counts).
Verification
Every reference was resolved against PubMed on 2026-10-31; title, year and DOI matched.
Review state
automated source verified; no human expert review has been recorded yet.
Version
1, last updated 2026-10-31.

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At a glance

2,405
PubMed records on this heading
11%
published since 2020
273
review articles
145
randomized trials
10
verified references below

Counts come from PubMed on 2026-10-31; they show how much has been written, not how strong the evidence is.

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