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

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.

• Authors: Fabricio Diniz de Lima, Ingrid Faber, Katiane R Servelhere, Maria Fernanda R Bittar, Alberto R M Martinez, Luiza G Piovesana, Melina P Martins, Carlos Roberto Martins Jr, Tatiana Benaglia, Benilton de Sá Carvalho, Anamarli Nucci, Marcondes C França Jr
• Journal: Movement disorders : official journal of the Movement Disorder Society (Mov Disord) Vol. 36 Issue 7 Pg. 1654-1663 (07 2021) ISSN: 1531-8257 [Electronic] United States
• PMID: 33595142 (Publication Type: Journal Article, Randomized Controlled Trial, Research Support, Non-U.S. Gov't)
• Copyright: © 2021 International Parkinson and Movement Disorder Society.

Chemical References

• Neuromuscular Agents
• Botulinum Toxins, Type A

Topics

• Adolescent
• Adult
• Botulinum Toxins, Type A
• Child
• Child, Preschool
• Double-Blind Method
• Humans
• Infant
• Male
• Middle Aged
• Muscle Spasticity (drug therapy)
• Neuromuscular Agents (therapeutic use)
• Spastic Paraplegia, Hereditary (drug therapy)
• Treatment Outcome
• Young Adult