Validation of the Italian version of a patient-reported outcome measure for Hereditary Spastic Paraplegia.

BACKGROUND AND AIM: Patient-reported outcome measures (PROMs) are recognized as valuable measures in the clinical setting. In 2018 we developed the Italian version of the "Hereditary Spastic Paraplegia-Self Notion and Perception Questionnaire" (HSP-SNAP), a disease-specific questionnaire that collects personal perception on motor symptoms related to HSP such as stiffness, weakness, imbalance, reduced endurance, fatigue and pain. In this study our primary aim was to assess the questionnaire validity and reliability. Our secondary aim was to characterize the symptoms "perceived" by patients with HSP and compare them with those "perceived" by age-matched healthy subjects. METHODS: The 12-item HSP-SNAP questionnaire was submitted to 20 external judges for comprehensibility and to 15 external judges for content validity assessment. We recruited 40 subjects with HSP and asked them to fill the questionnaire twice for test-retest procedure. They also completed the Medical Outcome Survey Short Form (SF-36) and were evaluated by the Spastic Paraplegia Rating Scale and the Six-Minute Walk Test. We also recruited 44 healthy subjects who completed the HSP-SNAP once to test score variability. RESULTS: The HSP-SNAP content validity index was high (0.8±0.1) and the test-retest analysis showed high reliability (ICC = 0.94). The mean HSP-SNAP score (score range 0-48) of the HSP group was 22.2±7.8, which was significantly lower than healthy subjects (43.1±6.3). The most commonly perceived symptom was stiffness, followed by weakness and imbalance. CONCLUSION: Although HSP-SNAP does not investigate non-motor symptoms and we validated only its Italian version, it showed good validity and reliability and it could be used in combination with other objective outcome measures for clinical purposes or as endpoints for future clinical rehabilitation studies. TRIAL REGISTRATION: Trial Registration: ClinicalTrial.gov, NCT04256681. Registered 3 February 2020.

Minimum Clinically Important Difference of Gross Motor Function and Gait Endurance in Children with Motor Impairment: A Comparison of Distribution-Based Approaches.

OBJECTIVE: The minimum clinically important difference (MCID) is a standard way of measuring clinical relevance. The objective of this work was to establish the MCID for the 6-minute walking test (6minWT) and the Gross Motor Function Measure (GMFM-88) in pediatric gait disorders. METHODS: A cohort, pretest-posttest study was conducted in a hospitalized care setting. A total of 182 patients with acquired brain injury (ABI) or cerebral palsy (CP) performed 20 robot-assisted gait training sessions complemented with 20 sessions of physical therapy over 4 weeks. Separate MCIDs were calculated using 5 distribution-based approaches, complemented with an anonymized survey completed by clinical professionals. RESULTS: The MCID range for the 6minWT was 20-38 m in the ABI cohort, with subgroup ranges of 20-36 m for GMFCS I-II, 23-46 m for GMFCS III, and 24-46 m for GMFCS IV. MCIDs for the CP population were 6-23 m, with subgroup ranges of 4-28 m for GMFCS I-II, 9-19 m for GMFCS III, and 10-27 m for GMFCS IV. For GMFM-88 total score, MCID values were 1.1%-5.3% for the ABI cohort and 0.1%-3.0% for the CP population. For dimension "D" of the GMFM, MCID ranges were 2.3%-6.5% and 0.8%-5.2% for ABI and CP populations, respectively. For dimension "E," MCID ranges were 2.8%-6.5% and 0.3%-4.9% for ABI and CP cohorts, respectively. The survey showed a large interquartile range, but the results well mimicked the distribution-based methods. CONCLUSIONS: This study identified for the first time MCID ranges for 6minWT and GMFM-88 in pediatric patients with neurological impairments, offering useful insights for clinicians to evaluate the impact of treatments. Distribution-based methods should be used with caution: methods based on pre-post correlation may underestimate MCID when applied to patients with small improvements over the treatment period. Our results should be complemented with estimates obtained using consensus- and anchor-based approaches.

Effect of Robot-Assisted Gait Training in a Large Population of Children With Motor Impairment Due to Cerebral Palsy or Acquired Brain Injury.

OBJECTIVE: To evaluate retrospectively the effect of robotic rehabilitation in a large group of children with motor impairment; an additional goal was to identify the effects in children with cerebral palsy (CP) and acquired brain injury (ABI) and with different levels of motor impairment according to the Gross Motor Function Classification System. Finally, we examined the effect of time elapsed from injury on children's functions. DESIGN: A cohort, pretest-posttest retrospective study was conducted. SETTING: Hospitalized care. PARTICIPANTS: A total of 182 children, 110 with ABI and 72 with CP and with Gross Motor Function Classification System (GMFCS) levels I-IV, were evaluated retrospectively. INTERVENTIONS: Patients underwent a combined treatment of robot-assisted gait training and physical therapy. MAIN OUTCOME MEASURES: All the patients were evaluated before and after the training using the 6-minute walk test and the Gross Motor Function Measure. A linear mixed model with 3 fixed factors and 1 random factor was used to evaluate improvements. RESULTS: The 6-minute walk test showed improvement in the whole group and in both ABI and CP. The Gross Motor Function Measure showed improvement in the whole group and in the patients with ABI but not in children with CP. The GMFCS analysis showed that all outcomes improved significantly in all classes within the ABI subgroup, whereas improvements were significant only for GMFCS III in children with CP. CONCLUSIONS: Children with motor impairment can benefit from a combination of robotic rehabilitation and physical therapy. Our data suggest positive results for the whole group and substantial differences between ABI and CP subgroups, with better results for children with ABI, that seem to be consistently related to time elapsed from injury.

Robotically-driven orthoses exert proximal-to-distal differential recovery on the lower limbs in children with hemiplegia, early after acquired brain injury.

Robotically-driven orthoses (RDO) are promising for treating gait impairment in children with hemiplegia after acquired brain injury (ABI). Despite this, existing literature on the employment of RDO in ABI is scanty, and cohorts' age spans throughout the adult age, with no specific focus on the developmental age. We aim to compare a treatment solely based on conventional physiotherapy (CP) with a program combining RDO training with CP, and to examine the effect of time following ABI on recovery. A prospective two-cohorts study was conducted in a rehabilitation hospital. Post-acute and chronic children with hemiplegia due to ABI underwent either: (i) 20 sessions of RDO plus 20 sessions of CP (n = 29), or (ii) 40 of CP (n = 12). Gross Motor Function Measures (GMFM), Functional Assessment Questionnaire (FAQ), 6 Minutes Walk Test and gait analysis (GA) parameters were recorded before and after training. Over all the patients in RDO + CP group, all GMFM domains and FAQ improved after RDO training (p < 0.05). The unaffected limb showed significantly decreased stance, increased step length and reduced anteroposterior center of pressure oscillation; the affected side increased the stride length. ROM hip and knee flex-extension increased bilaterally (p < 0.05 for all). RDO training during the acute/subacute post-injury phase increased motor functional abilities, cadence and velocity of gait (p < 0.05). We conclude that RDO imposes a proximal-to-distal differential effect on the lower limbs, with the hip joint being the most stimulated. RDO training fostered recovery, increasing the quality of gait on the unaffected side. Planning RDO early in the rehabilitation course of pediatric ABI is advantageous. RDO + CP may extend rehabilitation efficacy to the proximal segment of leg and to gait velocity.

Effects of dose and duration of Robot-Assisted Gait Training on walking ability of children affected by cerebral palsy.

BACKGROUND: Robot-Assisted Gait Training (RAGT) is a widespread approach for locomotion rehabilitation but information about intervention frequency and duration is still lacking. OBJECTIVE: To evaluate the effect of frequency and duration of a RAGT on motor outcome of children affected by Cerebral Palsy (CP). METHODS: Forty-four CP children (age 4-17) underwent one among four different intensive trainings with equal dose of intervention, combining Task-Oriented Physiotherapy (TOP) and RAGT: 40 sessions (4 sessions/week) over 10 weeks of sole TOP (group1) or RAGT (group2) or RAGT and TOP (2 + 2 sessions/week; group3); 40 sessions in shorter period (4 weeks) of RAGT and TOP (5 + 5 sessions/week; group4). Each child was assessed before, after the training and after 3 months with: Ashworth, gross motor function measure (GMFM)-88, GMFM-66, six minutes walking test and gait analysis. RESULTS: No differences among the 4 protocols were highlighted although both groups with exclusive physiotherapy and RAGT obtained significant improvements in GMFM-88, GMFM-E and GMFM-66 while the mixed approaches did not show significant changes. CONCLUSION: Single-treatment approaches seem to be more effective than mixed approaches, independently from the duration (4 or 10 weeks). RAGT seems to have similar effect with respect to the traditional TOP, at least over 10 weeks.