Department of Neurology, University of Campinas (UNICAMP), Campinas, Brazil.
Department of Statistics, University of Campinas (UNICAMP), Campinas, Brazil.
Mov Disord. 2021 Jul;36(7):1654-1663. doi: 10.1002/mds.28523. Epub 2021 Feb 17.
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.
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.
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).
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.
遗传性痉挛性截瘫以痉挛为主要临床表现，降低步态质量并导致活动能力丧失。肉毒毒素 A 型（BoNT-A）的管理尚未得到充分阐明。本研究的目的是评估 BoNT-A 治疗遗传性痉挛性截瘫患者的疗效和安全性。
这是一项双盲、随机、安慰剂对照交叉试验。每位参与者随机接受 Prosigne 100IU/2mL（每个内收大肌和每个比目鱼肌各 1 剂）或 0.9%生理盐水（2mL）1 次注射。主要结局指标为最大步行速度自基线的变化，次要结局指标包括自主选择速度下的步行变化、痉挛、肌肉力量、痉挛性截瘫评定量表、疼痛、疲劳和主观改善感知。我们还观察了患者报告的不良事件。
我们共纳入 55 名患者，其中 36 名男性，41 名纯表型。平均年龄为 43±13.4 岁（范围，19-72 岁），平均发病年龄为 27±13.1 岁（范围，<1-55 岁），平均病程为 17±12.7 岁（范围，1-62 岁）。与基线相比，我们未发现两组在主要和次要结局方面存在显著差异，除了内收肌张力降低（P=0.01）。不良事件为一过性且可耐受，且两种治疗方法的发生率无显著差异（P=0.17）。
BoNT-A 治疗遗传性痉挛性截瘫安全有效，可降低内收肌张力，但考虑到剂量、注射方案、测量和使用的仪器，其无法产生功能改善。© 2021 国际帕金森病和运动障碍协会。
