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

Abstract

A BS TRACT: 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 AE 13.4 years (range, 19-72 years), mean age of onset waws 27 AE 13.1 years (range, <1 to 55 yars), and mean disease duration was 17 AE 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.

Inertial Gait Sensors to Measure Mobility and Functioning in Hereditary Spastic Paraplegia

“…29 This is also in line with previous studies demonstrating (1) that the mental and physical component scores of the SF-36 questionnaire both correlated significantly with total SPRS values in a cohort of 143 German patients with HSP, 30 (2) that health-related quality of life as measured by the RAND-36 questionnaire was associated with subjective changes in health in a cohort of 49 Estonian patients with HSP, 31 (3) that spastic gait in HSP causes withdrawal from social activities and shame in 14 Dutch patients with HSP as determined by semistructured interviews, 32,33 and (4) that specific interventions to improve mobility also lead to improved quality of life 34 and fatigue. 35,36 Moreover, the maximum gait distance correlated with quality of life as measured with the EuroQol-5 dimension questionnaire in 118 SPG4 patients. 37 Thus, symptomatic treatment to improve gait and eventually also the prevention of progressive gait disability is an urgent need in clinical care and research for HSP.…”

Chinese patients with hereditary spastic paraplegias (HSPs): a protocol for a hospital-based cohort study

“…Neurological examinations include assessment of mental status, level of consciousness, sensory function, motor function, cranial nerve function and reflexes. 14 SPRS scores will be determined and assessed for every participant to quantify the severity of the disease. 15 16 The 6-Minute Walk Test will be performed, and if the participant is able to walk independently with or without assistance, a video of the test will be recorded.…”

Controversies and Clinical Applications of Non-Invasive Transspinal Magnetic Stimulation: A Critical Review and Exploratory Trial in Hereditary Spastic Paraplegia

“…Interest in spinal cord stimulation has gained traction since promising results were obtained in clinical experiments with invasive stimulation in Parkinson’s disease (PD) [ 4 ], motor recovery after spinal cord injury [ 5 ], and innovations in pain control [ 6 ]. Renewed interest in clinical applications of TsMS has arisen from the exciting possibility of offering similar neuromodulation with non-invasive alternatives combined with favorable safety and cost profiles.…”