Prospective Trial on Topotecan Salvage Therapy in Primary CNS Lymphoma

Ann Oncol. 2006 Jul;17(7):1141-5. doi: 10.1093/annonc/mdl070. Epub 2006 Apr 7.


Background: Standard salvage therapy has not been established for recurrent primary central nervous system lymphoma (PCNSL). We report the final results of a prospective study on topotecan chemotherapy in relapsed or refractory PCNSL.

Patients and methods: The study included 27 patients with a median age of 51 years and an ECOG performance status of 2. Fourteen patients were refractory to the last therapy, and 13 relapsed after a median period of 6.0 months. Pretreatment with up to four regimens included chemotherapy in 26 patients and whole brain irradiation in 14. A 30-min daily topotecan infusion of 1.5 mg/m(2) for 5 days was repeated every 3 weeks.

Results: The response rate was 33% with five complete (CR) and four partial remissions (PR). The median follow-up was 37.7 months. All complete responders had sustained remissions lasting for 9 to 28 months. The median event-free survival (EFS) was 2.0 months (9.1 months in responders), the overall survival (OAS) was 8.4 months. CTC grade 3-4 leukopenia occurred in 26% and thrombocytopenia in 11% of the patients. Eight of 12 patients alive without cerebral lymphoma > or = six months after topotecan exhibited deficits attributable to late neurotoxicity.

Conclusion: Topotecan as monotherapy is active in relapsed and refractory PCNSL with tolerable toxicity.

Publication types

  • Clinical Trial, Phase II
  • Multicenter Study

MeSH terms

  • Adult
  • Aged
  • Antineoplastic Agents / therapeutic use*
  • Central Nervous System Neoplasms / drug therapy*
  • Central Nervous System Neoplasms / mortality
  • Central Nervous System Neoplasms / radiotherapy
  • Combined Modality Therapy
  • Disease-Free Survival
  • Female
  • Humans
  • Lymphoma / drug therapy*
  • Lymphoma / mortality
  • Lymphoma / radiotherapy
  • Male
  • Middle Aged
  • Neoplasm Recurrence, Local / drug therapy
  • Neoplasm Recurrence, Local / mortality
  • Prospective Studies
  • Remission Induction
  • Salvage Therapy*
  • Survival Analysis
  • Topotecan / therapeutic use*


  • Antineoplastic Agents
  • Topotecan