Immunomodulatory treatment trial for paraneoplastic neurological disorders

Steven Vernino, Brian Patrick O'Neill, Randolph S. Marks, Judith R. O'Fallon, David W. Kimmel

Research output: Contribution to journalArticle

142 Citations (Scopus)

Abstract

Paraneoplastic neurological disorders are devastating remote effects of malignancy. Despite compelling evidence of an autoimmune pathogenesis, empiric immunomodulatory treatment of these disorders is often ineffective. However, very few systematic studies have been conducted, and the treatment of patients without active malignancy has not been addressed. We conducted a prospective open-label treatment study of plasma exchange plus conventional cancer chemotherapy (10 patients) or plasma exchange plus continuous oral cyclophosphamide (10 patients). All patients had progressive symptoms and at least moderate disability at enrollment (mean Rankin score, 3.4). Patients who had experienced symptoms for more than 12 months were excluded (mean duration of symptoms at enrollment, 3.6 months). The primary outcome measure was change in quantitative disability measures (Rankin and Barthel scores) after 6 months of treatment; a positive response was defined as stability or improvement in disability. Overall, 50% of patients had a positive response at 6 months (6 patients had improved by at least 1 Rankin grade). Patients with good outcome tended to be those with less disability at time of enrollment. Hematologic toxicity was common among those receiving cyclophosphamide. Aggressive immunosuppression early in the clinical course should be considered in patients who have paraneoplastic neurological disorders, even when there is no evidence of active malignancy.

Original languageEnglish (US)
Pages (from-to)55-62
Number of pages8
JournalNeuro-Oncology
Volume6
Issue number1
DOIs
StatePublished - Jan 2004

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Nervous System Diseases
Plasma Exchange
Therapeutics
Cyclophosphamide
Neoplasms
Immunosuppression
Outcome Assessment (Health Care)
Drug Therapy

ASJC Scopus subject areas

  • Cancer Research
  • Oncology
  • Clinical Neurology

Cite this

Immunomodulatory treatment trial for paraneoplastic neurological disorders. / Vernino, Steven; O'Neill, Brian Patrick; Marks, Randolph S.; O'Fallon, Judith R.; Kimmel, David W.

In: Neuro-Oncology, Vol. 6, No. 1, 01.2004, p. 55-62.

Research output: Contribution to journalArticle

Vernino, Steven ; O'Neill, Brian Patrick ; Marks, Randolph S. ; O'Fallon, Judith R. ; Kimmel, David W. / Immunomodulatory treatment trial for paraneoplastic neurological disorders. In: Neuro-Oncology. 2004 ; Vol. 6, No. 1. pp. 55-62.
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