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Thorsten Schulte, MD; Rebekka Mattern, MD; Klaus Berger, MD, MPH, MSc; Sandra Szymanski, MD; Peter Klotz; Peter H. Kraus, MD; Horst Przuntek, MD; Ludger Schöls, MD

Arch Neurol. 2001;58:1451-1457.

Objective  To evaluate the efficiency of a combination of trimethoprim and sulfamethoxazole in patients with spinocerebellar ataxia type 3/Machado-Joseph disease (SCA3/MJD).

Design  Placebo-controlled, double-blind crossover trial in 22 patients with genetically confirmed SCA3/MJD. Study phases of 6 months were separated by a washout period of 4 weeks. Dosages were a combination of trimethoprim, 160 mg, and sulfamethoxazole, 800 mg, twice daily for 2 weeks, followed by a combination of trimethoprim, 80 mg, and sulfamethoxazole, 400 mg, twice daily for 5.5 months.

Setting  Outpatient department of the Neurological Clinic, Ruhr-University, Bochum, Germany.

Main Outcome Measures  Ataxia ranking scale, self-assessment score, static posturography, and results of motor performance testing. Effects on the visual system were studied using the achromatic Vision Contrast Test System and the Farnsworth-Munsell 100-hue test for color discrimination. Physical and mental health were documented using the Medical Outcomes Study 36-Item Short-Form Health Survey. Subgroup analyses assessed the influence of age, sex, age at onset, duration of the disease, phenotype, and CAG repeat length on test performance.

Results  Twenty of 22 patients completed the study. Dropouts were due to a rash (placebo phase) and an attempted suicide in a family conflict. Trimethoprim-sulfamethoxazole therapy had no significant effect in SCA3/MJD patients in the short-term analysis (2 weeks) or in the long-term interval (6 months).

Conclusions  In contrast to previous reports that studied smaller groups of patients, treatment with trimethoprim-sulfamethoxazole did not improve the diverse and complex movement disorders caused by SCA3/MJD. Trimethoprim-sulfamethoxazole had no effect on the visual system and cannot be recommended as a continuous treatment for SCA3/MJD patients.

From the Department of Neurology, St Josef Hospital, Ruhr-University, Bochum (Drs Schulte, Mattern, Szymanski, Kraus, Przuntek, and Schöls and Mr Klotz), and the Institute of Epidemiology and Social Medicine, University of Münster, Münster (Dr Berger), Germany.

Corresponding author and reprints: Ludger Schöls, MD, Neurologische Klinik der Ruhr-Universität, St Josef Hospital, Gudrunstraße 56, D-44791 Bochum, Germany (e-mail: Ludger.Schoels{at}ruhr-uni-bochum.de).

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