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Randomized Controlled Trial
. 2021 Jul;36(7):1654-1663.
doi: 10.1002/mds.28523. Epub 2021 Feb 17.

Randomized Trial of Botulinum Toxin Type A in Hereditary Spastic Paraplegia - The SPASTOX Trial

Fabricio Diniz de Lima  1 , Ingrid Faber  1 , Katiane R Servelhere  1 , Maria Fernanda R Bittar  1 , Alberto R M Martinez  1 , Luiza G Piovesana  1 , Melina P Martins  1 , Carlos Roberto Martins Jr  1 , Tatiana Benaglia  2 , Benilton de Sá Carvalho  2 , Anamarli Nucci  1 , Marcondes C França Jr  1
Affiliations
Randomized Controlled Trial

Randomized Trial of Botulinum Toxin Type A in Hereditary Spastic Paraplegia - The SPASTOX Trial

Fabricio Diniz de Lima et al. Mov Disord. 2021 Jul.

Abstract

Background: Hereditary spastic paraplegia presents spasticity as the main clinical manifestation, reducing gait quality and producing incapacity. Management with botulinum toxin type A (BoNT-A) is not well elucidated. The objective of the current study was to evaluate the efficacy and safety of BoNT-A in patients with hereditary spastic paraplegias.

Methods: This was a double-blind, randomized, placebo-controlled crossover trial. Each participant was randomly assigned to receive 1 injection session of either BoNT-A (100 IU/2 mL of Prosigne in each adductor magnus and each triceps surae) or saline 0.9% (2 mL). The primary outcome measure was change from baseline in maximal gait velocity, and secondary outcome measures included changes in gait at self-selected velocity, spasticity, muscle strength, Spastic Paraplegia Rating Scale, pain, fatigue, and subjective perception of improvement. We also looked at adverse events reported by the patients.

Results: We enrolled 55 patients, 36 of whom were men and 41 with the pure phenotype. Mean age was 43 ± 13.4 years (range, 19-72 years), mean age of onset waws 27 ± 13.1 years (range, <1 to 55 yars), and mean disease duration was 17 ± 12.7 years (range, 1-62 years). Compared with baseline, we did not find significant differences between groups in primary and secondary outcomes, except for reduction in adductor tone (P = 0.01). The adverse events were transient and tolerable, and their incidence did not significantly differ between treatments (P = 0.17).

Conclusions: BoNT-A was safe in patients with hereditary spastic paraplegias and reduced the adductor tone, but it was not able to produce functional improvement considering the doses, injection protocol, measures, and instruments used. © 2021 International Parkinson and Movement Disorder Society.

Keywords: botulinum toxin A; clinical trial; gait; hereditary spastic paraplegia; spasticity.

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References

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