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Effects of acetyl-DL-leucine in patients with cerebellar ataxia: a case series.

Strupp M, Teufel J, Habs M, Feuerecker R, Muth C, van de Warrenburg BP, Klopstock T, Feil K - J. Neurol. (2013)

Bottom Line: Mean total SARA decreased remarkably (p = 0.002) from a baseline of 16.1 ± 7.1 to 12.8 ± 6.8 (mean ± SD) on medication.No side effects were reported.In conclusion, acetyl-DL-leucine significantly improved ataxic symptoms without side effects and therefore showed a good risk-benefit profile.

View Article: PubMed Central - PubMed

Affiliation: Department of Neurology, University Hospital Munich and German Center for Vertigo and Balance Disorders, Campus Grosshadern, Marchioninistrasse 15, 81377, Munich, Germany, michael.strupp@med.uni-muenchen.de.

ABSTRACT
No existing medication has yet been shown to convincingly improve cerebellar ataxia. Therefore, the identification of new drugs for its symptomatic treatment is desirable. The objective of this case series was to evaluate the efficacy of treatment of cerebellar ataxia with the amino acid acetyl-DL-leucine (Tanganil). Thirteen patients (eight males, median age 51 years) with degenerative cerebellar ataxia of different etiologies (SCA1/2, ADCA, AOA, SAOA) were treated with acetyl-DL-leucine (5 g/day) without titration for 1 week. Motor function was evaluated by changes in the Scale for the Rating and Assessment of Ataxia (SARA) and in the Spinocerebellar Ataxia Functional Index (SCAFI) during treatment compared to a baseline examination. Quality of life (EuroQol-5D-3L) and side effects were also assessed. Mean total SARA decreased remarkably (p = 0.002) from a baseline of 16.1 ± 7.1 to 12.8 ± 6.8 (mean ± SD) on medication. There were also significant improvements in sub-scores for gait (p = 0.022), speech (p = 0.007), finger-chase (p = 0.042), nose-finger-test (p = 0.035), rapid-alternating-movements (p = 0.002) and heel-to-shin (p = 0.018). Furthermore, patients showed better performance in the SCAFI consisting of the 8-m-walking-time (8 MW, p = 0.003), 9-Hole-Peg-Test of the dominant hand (9HPTD, p = 0.011) and the PATA rate (p = 0.005). Quality of life increased during treatment (p = 0.003). No side effects were reported. In conclusion, acetyl-DL-leucine significantly improved ataxic symptoms without side effects and therefore showed a good risk-benefit profile. These findings need to be confirmed in placebo-controlled trials.

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Value changes on a Scale for the Assessment and Rating of Ataxia (SARA) and Spinocerebellar Ataxia Functional Index (SCAFI) sub-score items in terms of b 8 m walk (8 MW), c PATA word count in 10 s and d 9-hole-peg-test (9HPT) of the dominant and non-dominant hand before and during the therapy with acetyl-dl-leucine (5 g/day) (mean ± SD)
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Fig2: Value changes on a Scale for the Assessment and Rating of Ataxia (SARA) and Spinocerebellar Ataxia Functional Index (SCAFI) sub-score items in terms of b 8 m walk (8 MW), c PATA word count in 10 s and d 9-hole-peg-test (9HPT) of the dominant and non-dominant hand before and during the therapy with acetyl-dl-leucine (5 g/day) (mean ± SD)

Mentions: The clinical characteristics of the patients are given in Table 1. Thirteen patients with cerebellar disorders of different etiologies were observed (since patient no. 1 did not perform the SCAFI, he was excluded from the statistical analysis of this test). Mean duration of cerebellar symptoms was 8.5 ± 6.8 years. Mean total SARA decreased from a baseline of 16.1 ± 7.1 to 12.8 ± 6.8 (p = 0.002, Wilcoxon signed-rank test). During treatment the SARA subsection scores were notably lower for gait (p = 0.022), speech disturbance (p = 0.007), finger-chase (p = 0.042), nose-finger-test (p = 0.035), rapid-alternating-hand-movements (p = 0.002) and heel–shin-slide (p = 0.018) (Table 2; Figs. 1, 2). The SCAFI items also improved, represented by the 8 MW (p = 0.003), the 9HPT of the dominant hand (p = 0.011) and the PATA rate (p = 0.005). Moreover, the EQ-5D-3L changed from a baseline of 10.7 ± 1.6 to 9.5 ± 2.0 (p = 0.003), indicating that quality of life was higher during treatment. The visual analogue scale (VAS)—as part of the EQ-5D-3L—rose from 0.57 ± 0.21 to 0.63 ± 0.19 (p = 0.241). Eleven out of 13 patients reported a subjective improvement on therapy (nos. 1, 3, 4, 6, 7, 8, 9, 10, 11, 12, 13). The patients did not report any side effects.Table 2


Effects of acetyl-DL-leucine in patients with cerebellar ataxia: a case series.

Strupp M, Teufel J, Habs M, Feuerecker R, Muth C, van de Warrenburg BP, Klopstock T, Feil K - J. Neurol. (2013)

Value changes on a Scale for the Assessment and Rating of Ataxia (SARA) and Spinocerebellar Ataxia Functional Index (SCAFI) sub-score items in terms of b 8 m walk (8 MW), c PATA word count in 10 s and d 9-hole-peg-test (9HPT) of the dominant and non-dominant hand before and during the therapy with acetyl-dl-leucine (5 g/day) (mean ± SD)
© Copyright Policy - OpenAccess
Related In: Results  -  Collection

Show All Figures
getmorefigures.php?uid=PMC3824630&req=5

Fig2: Value changes on a Scale for the Assessment and Rating of Ataxia (SARA) and Spinocerebellar Ataxia Functional Index (SCAFI) sub-score items in terms of b 8 m walk (8 MW), c PATA word count in 10 s and d 9-hole-peg-test (9HPT) of the dominant and non-dominant hand before and during the therapy with acetyl-dl-leucine (5 g/day) (mean ± SD)
Mentions: The clinical characteristics of the patients are given in Table 1. Thirteen patients with cerebellar disorders of different etiologies were observed (since patient no. 1 did not perform the SCAFI, he was excluded from the statistical analysis of this test). Mean duration of cerebellar symptoms was 8.5 ± 6.8 years. Mean total SARA decreased from a baseline of 16.1 ± 7.1 to 12.8 ± 6.8 (p = 0.002, Wilcoxon signed-rank test). During treatment the SARA subsection scores were notably lower for gait (p = 0.022), speech disturbance (p = 0.007), finger-chase (p = 0.042), nose-finger-test (p = 0.035), rapid-alternating-hand-movements (p = 0.002) and heel–shin-slide (p = 0.018) (Table 2; Figs. 1, 2). The SCAFI items also improved, represented by the 8 MW (p = 0.003), the 9HPT of the dominant hand (p = 0.011) and the PATA rate (p = 0.005). Moreover, the EQ-5D-3L changed from a baseline of 10.7 ± 1.6 to 9.5 ± 2.0 (p = 0.003), indicating that quality of life was higher during treatment. The visual analogue scale (VAS)—as part of the EQ-5D-3L—rose from 0.57 ± 0.21 to 0.63 ± 0.19 (p = 0.241). Eleven out of 13 patients reported a subjective improvement on therapy (nos. 1, 3, 4, 6, 7, 8, 9, 10, 11, 12, 13). The patients did not report any side effects.Table 2

Bottom Line: Mean total SARA decreased remarkably (p = 0.002) from a baseline of 16.1 ± 7.1 to 12.8 ± 6.8 (mean ± SD) on medication.No side effects were reported.In conclusion, acetyl-DL-leucine significantly improved ataxic symptoms without side effects and therefore showed a good risk-benefit profile.

View Article: PubMed Central - PubMed

Affiliation: Department of Neurology, University Hospital Munich and German Center for Vertigo and Balance Disorders, Campus Grosshadern, Marchioninistrasse 15, 81377, Munich, Germany, michael.strupp@med.uni-muenchen.de.

ABSTRACT
No existing medication has yet been shown to convincingly improve cerebellar ataxia. Therefore, the identification of new drugs for its symptomatic treatment is desirable. The objective of this case series was to evaluate the efficacy of treatment of cerebellar ataxia with the amino acid acetyl-DL-leucine (Tanganil). Thirteen patients (eight males, median age 51 years) with degenerative cerebellar ataxia of different etiologies (SCA1/2, ADCA, AOA, SAOA) were treated with acetyl-DL-leucine (5 g/day) without titration for 1 week. Motor function was evaluated by changes in the Scale for the Rating and Assessment of Ataxia (SARA) and in the Spinocerebellar Ataxia Functional Index (SCAFI) during treatment compared to a baseline examination. Quality of life (EuroQol-5D-3L) and side effects were also assessed. Mean total SARA decreased remarkably (p = 0.002) from a baseline of 16.1 ± 7.1 to 12.8 ± 6.8 (mean ± SD) on medication. There were also significant improvements in sub-scores for gait (p = 0.022), speech (p = 0.007), finger-chase (p = 0.042), nose-finger-test (p = 0.035), rapid-alternating-movements (p = 0.002) and heel-to-shin (p = 0.018). Furthermore, patients showed better performance in the SCAFI consisting of the 8-m-walking-time (8 MW, p = 0.003), 9-Hole-Peg-Test of the dominant hand (9HPTD, p = 0.011) and the PATA rate (p = 0.005). Quality of life increased during treatment (p = 0.003). No side effects were reported. In conclusion, acetyl-DL-leucine significantly improved ataxic symptoms without side effects and therefore showed a good risk-benefit profile. These findings need to be confirmed in placebo-controlled trials.

Show MeSH
Related in: MedlinePlus