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Neuro-Oncology 2004 6(1):55-62; doi:10.1215/S1152851703000395
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© 2004 by the Society forNeuro-Oncology

Immunomodulatory treatment trial for paraneoplastic neurologicaldisorders

Steven Vernino1, Brian Patrick O'Neill, Randolph S. Marks, Judith R. O'Fallon and David W. Kimmel

Departments of Neurology (S.V., B.P.O., D.W.K.), Oncology (R.S.M.), and HealthSciences Research (J.R.O.), Mayo Clinic, 200 First Street SW, Rochester, MN55905, USA

1 Address correspondence to Steven Vernino, Department of Neurology, MayoClinic, 200 First Street SW, Rochester, MN 55905, USA(verns{at}mayo.edu).


  Abstract

Paraneoplastic neurological disorders are devastating remote effects ofmalignancy. Despite compelling evidence of an autoimmune pathogenesis, empiricimmunomodulatory treatment of these disorders is often ineffective. However,very few systematic studies have been conducted, and the treatment of patientswithout active malignancy has not been addressed. We conducted a prospectiveopen-label treatment study of plasma exchange plus conventional cancerchemotherapy (10 patients) or plasma exchange plus continuous oralcyclophosphamide (10 patients). All patients had progressive symptoms and atleast moderate disability at enrollment (mean Rankin score, 3.4). Patients whohad experienced symptoms for more than 12 months were excluded (mean durationof symptoms at enrollment, 3.6 months). The primary outcome measure was changein quantitative disability measures (Rankin and Barthel scores) after 6 monthsof treatment; a positive response was defined as stability or improvement indisability. Overall, 50% of patients had a positive response at 6 months (6patients had improved by at least 1 Rankin grade). Patients with good outcometended to be those with less disability at time of enrollment. Hematologictoxicity was common among those receiving cyclophosphamide. Aggressiveimmunosuppression early in the clinical course should be considered inpatients who have paraneoplastic neurological disorders, even when there is noevidence of active malignancy.

Received July 22, 2003; Accepted September 22, 2003


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