Topotecan in the treatment of acquired immunodeficiency syndrome-related progressive multifocal leukoencephalopathy

Walter Royal, B. Dupont, D. McGuire, L. Chang, K. Goodkin, T. Ernst, M. Judith Post, D. Fish, G. Pailloux, H. Poncelet, M. Concha, L. Apuzzo, E. Singer

Research output: Contribution to journalArticle

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Abstract

Progressive multifocal leukoencephalopathy (PML) affects about 1 in 20 individuals with the acquired immunodeficiency syndrome (AIDS) and has been associated with poor survival. This report describes the results of a phase II clinical trial using the drug topotecan, a semisynthetic analogue of camptothecan, administered to a cohort of subjects with AIDS-related PML. Data were evaluated on 11 of 12 subjects enrolled in the study. Three responded to therapy. Additionally, one patient was treated off-protocol and showed a response to treatment. Progression occurred after the first course; however, a partial response was noted after five courses. One study patient died from accidental overdose of topotecan. Overall, responders had higher pretreatment Karnofsky and lower Kurtzke expanded disability status scale scores than non-responders. The most frequent toxicities were hematologic (anemia, neutropenia, and thrombocytopenia). Five patients had dose delays; all delays were due to hematologic adverse events. This study demonstrates that topotecan treatment may be associated with decreased lesion size and prolonged survival from the infection. Because of the small number of subjects in the study, further studies are required to evaluate the efficacy of topotecan in treating this disease.

Original languageEnglish
Pages (from-to)411-419
Number of pages9
JournalJournal of NeuroVirology
Volume9
Issue number3
StatePublished - Jun 1 2003

Fingerprint

Progressive Multifocal Leukoencephalopathy
Topotecan
Acquired Immunodeficiency Syndrome
Phase II Clinical Trials
Survival
Therapeutics
Neutropenia
Thrombocytopenia
Anemia
Infection
Pharmaceutical Preparations

Keywords

  • HIV-1
  • MRI
  • Polyomavirus
  • Topoisomerases
  • Viral encephalitis

ASJC Scopus subject areas

  • Virology
  • Clinical Neurology

Cite this

Royal, W., Dupont, B., McGuire, D., Chang, L., Goodkin, K., Ernst, T., ... Singer, E. (2003). Topotecan in the treatment of acquired immunodeficiency syndrome-related progressive multifocal leukoencephalopathy. Journal of NeuroVirology, 9(3), 411-419.

Topotecan in the treatment of acquired immunodeficiency syndrome-related progressive multifocal leukoencephalopathy. / Royal, Walter; Dupont, B.; McGuire, D.; Chang, L.; Goodkin, K.; Ernst, T.; Judith Post, M.; Fish, D.; Pailloux, G.; Poncelet, H.; Concha, M.; Apuzzo, L.; Singer, E.

In: Journal of NeuroVirology, Vol. 9, No. 3, 01.06.2003, p. 411-419.

Research output: Contribution to journalArticle

Royal, W, Dupont, B, McGuire, D, Chang, L, Goodkin, K, Ernst, T, Judith Post, M, Fish, D, Pailloux, G, Poncelet, H, Concha, M, Apuzzo, L & Singer, E 2003, 'Topotecan in the treatment of acquired immunodeficiency syndrome-related progressive multifocal leukoencephalopathy', Journal of NeuroVirology, vol. 9, no. 3, pp. 411-419.
Royal W, Dupont B, McGuire D, Chang L, Goodkin K, Ernst T et al. Topotecan in the treatment of acquired immunodeficiency syndrome-related progressive multifocal leukoencephalopathy. Journal of NeuroVirology. 2003 Jun 1;9(3):411-419.
Royal, Walter ; Dupont, B. ; McGuire, D. ; Chang, L. ; Goodkin, K. ; Ernst, T. ; Judith Post, M. ; Fish, D. ; Pailloux, G. ; Poncelet, H. ; Concha, M. ; Apuzzo, L. ; Singer, E. / Topotecan in the treatment of acquired immunodeficiency syndrome-related progressive multifocal leukoencephalopathy. In: Journal of NeuroVirology. 2003 ; Vol. 9, No. 3. pp. 411-419.
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