Double-blind crossover trial of trimethoprim-sulfamethoxazole in spinocerebellar ataxia type 3/Machado-Joseph disease.

Abstract

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.

• Authors: T Schulte, R Mattern, K Berger, S Szymanski, P Klotz, P H Kraus, H Przuntek, L Schöls
• Journal: Archives of neurology (Arch Neurol) Vol. 58 Issue 9 Pg. 1451-7 (Sep 2001) ISSN: 0003-9942 [Print] United States
• PMID: 11559318 (Publication Type: Clinical Trial, Journal Article, Randomized Controlled Trial, Research Support, Non-U.S. Gov't)

Chemical References

• Anti-Infective Agents
• Trimethoprim, Sulfamethoxazole Drug Combination

Topics

• Adult
• Anti-Infective Agents (administration & dosage)
• Cross-Over Studies
• Double-Blind Method
• Female
• Humans
• Machado-Joseph Disease (drug therapy)
• Male
• Middle Aged
• Posture
• Treatment Outcome
• Trimethoprim, Sulfamethoxazole Drug Combination (administration & dosage)
• Vision Tests