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Attempts to Distinguish a Treatable Disorder

Ersin Tan, MD; D. Joanne Lynn, MD; Anthony A. Amato, MD; John T. Kissel, MD; Kottil W. Rammohan, MD; Zarife Sahenk, MD; John R. Warmolts, MD; Carlayne E. Jackson, MD; Richard J. Barohn, MD; Jerry R. Mendell, MD

Arch Neurol. 1994;51(2):194-200.

Abstract
Objective
To determine if response to immunosuppressive treatment in motor neuron syndromes could be predicted on the basis of clinical features, anti-GM₁ antibodies, or conduction block.

Design
Prospective, uncontrolled, treatment trial using prednisone for 4 months followed by intravenous cyclophosphamide (3 g/m²) continued orally for 6 months.

Setting
All patients were referred to university hospital medical centers.

Patients
Sixty-five patients with motor neuron syndromes were treated with prednisone; 11 patients had elevated GM₁ antibody titers, and 11 patients had conduction block. Forty-five patients received cyclophosphamide, eight of whom had elevated GM¹ antibodies and 10 had conduction block.

Results
One patient responded to prednisone, and five patients responded to cyclophosphamide treatment. Only patients with a lower motor neuron syndrome and conduction block improved with either treatment. Response to treatment did not correlate with GM¹ antibodies.

Conclusions
GM¹ antibodies did not serve as a marker for improvement in patients with motor neuron syndrome treated with immunosuppressive drugs. Patients with amyotrophic lateral sclerosis failed to improve irrespective of laboratory findings.

Author Affiliations
From the Departments of Neurology, The Ohio State University, Columbus (Drs Tan, Lynn, Amato, Kissel, Rammohan, Sahenk, Warmolts, and Mendell), and The University of Texas Health Science Center, San Antonio (Drs Jackson and Barohn).

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