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Immunomodulatory
treatment trial for paraneoplastic neurological disorders
Steven Vernino,
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 Health Sciences Research (J.R.O.), Mayo Clinic, 200 First Street
SW, Rochester, MN 55905, USA
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.
Source: http://lysander.ingentaselect.com/cgi-bin/linker?ini=dup_no&reqidx=/cw/dup/15228517/v6n1/s9/p55
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