Topotecan in the treatment of brain metastases. A phase II study of GOIM (Gruppo Oncologico dell'Italia Meridionale)

Vito Lorusso, Domenico Galetta, Francesco Giotta, Antonio Rinaldi, Sante Romito, Cosimo Brunetti, Nicola Silvestris, Giuseppe Colucci

Research output: Contribution to journalArticlepeer-review


Background: Topotecan is able to cross the blood-brain barrier (BBB) and has been demonstrated to be active in brain metastases from small cell lung cancer (SCLC). Patients and Methods: The aim of this study was to evaluate the efficacy and toxicity of topotecan at a dosage of 1.5 mg/m2 for 5 consecutive days every 3 weeks in patients with brain metastases from various neoplasms. Results: Among the 19 patients enrolled, objective responses were observed in 2 out of 3 patients affected by brain metastases from SCLC. Stable disease was observed in 8 more patients (4 breast, 3 non-SCLC and 1 colon). According to the Simon two-step design, patient accrual was stopped because of the low response rate observed in the first 19 patients treated (early stopping rule). The G 3/4 haematological toxicity was severe, with 37% neutropenia, 21% thrombocytopenia and 16% anaemia, respectively. Conclusion: Our data do not support the use of topotecan in patients with brain metastases, except for SCLC. In addition, the haematological toxicity prevents the use of this regimen in elderly and poor performance status patients.

Original languageEnglish
Pages (from-to)2259-2263
Number of pages5
JournalAnticancer Research
Issue number3 B
Publication statusPublished - May 2006


  • Brain metastases
  • Topotecan

ASJC Scopus subject areas

  • Cancer Research
  • Oncology


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