Steroid treatment in four cases of anti-GAD cerebellar ataxia

Introduction. - Few neurological diseases are linked with anti-glutamic acid decarboxylase antibodies (GAD-ab); stiff man syndrome is an example. Cerebellar ataxia is a new feature of this expanding spectrum. No therapeutic trial is yet available in these diseases. We here report on four patients suffering from cerebellar ataxia linked with GAD-ab and review the data in the literature on this recently described syndrome. Method. - We conducted an open trial with monthly pulsed steroids. Steroid pulses were given six months followed with placebo for another six months. Main clinical and biological parameters were monitored monthly (International Cooperative Cerebellar Ataxia Rating Scale [ICARS] and GAD-ab). Result. - The clinical response was found limited and inconstant. Transient decline in GAD-ab level was noted in two patients. Moreover, GAD-ab level was found highly variable and did not correlate with clinical parameters. Discussion. - Cerebellar ataxia with GAD-ab is an increasingly described syndrome. Outcome can be severe, leading to definitive cerebellar atrophy. Diagnosis is supported by high level of serum GAD-ab with intrathecal secretion. Experimental data have suggested a direct excito-toxic effect of GAD-ab on Purkinje cells. Response to various treatments is often disappointing. Improvement has been obtained with veinoglobulins in individual patients. A weak clinical and biological response was associated with monthly steroid pulses. (C) 2008 Elsevier Masson SAS. Tous droits reserves.