Gillette Functional Assessment Questionnaire Research Articles

Are tactile function and body awareness of the foot related to motor outcomes in children with upper motor neuron lesions?

Somatosensory function can be reduced in children with Upper Motor Neuron (UMN) lesions. Therefore, we investigated relationships between somatosensory functions of the foot and motor outcomes in children with UMN lesions. In this cross-sectional study, we assessed the Tactile Threshold (TT) with monofilaments and body awareness with Tactile Localisation Tasks for spatial-related action (TLTaction) and structural-related perception (TLTperception) body representation at the foot sole. Furthermore, we assessed four motor outcomes: the Selective Control Assessment of the Lower Extremity (SCALE), the modified Timed Up and Go test (mTUG), the Gillette Functional Assessment Questionnaire (FAQ), and the Functional Mobility Scale (FMS). Spearman's correlations (ρ) were applied to assess relationships between the somatosensory function of the foot sole and the applied motor outcomes. Thirty-five children with UMN lesions, on average 11.7 ± 3.4 years old, participated. TLTperception correlated significantly with all lower limb motor outcomes (|ρ|=0.36-0.57; p < 0.05), but TLTaction (|ρ|=0.00-0.27; p = 0.15-0.97, and TT did not (|ρ|=0.01-0.83; p = 0.73-0.94). TLTperception correlated strongly with the Gross Motor Function Classification System (|ρ|=0.62; p = 0.001) in children with cerebral palsy (n = 24). Assessing structural body representation of the foot sole should be considered when addressing lower limb motor impairments, including gait, in children with upper motor neuron lesions. Our results suggest that the assessment of tactile function and spatial body representation may be less related to lower limb motor function.

The validity and reliability of the Turkish version of the functional mobility scale in patients with cerebral palsy.

Objectives: The aim of this study was to investigate the validity and reliability of the Turkish version of the Functional Mobility Scale (FMS) in patients with cerebral palsy. Patients and methods: The validity and reliability study was conducted with 100 cerebral palsy patients (66 males, 34 females; mean age: 6.4±2.7 years; range, 2 to 18 years) between July 2015 and July 2018. The translation of the FMS was performed according to international standards. For test-retest reliability, 54 patients were reevaluated one week after the initial test with the Turkish version of the FMS, and Cohen's weighted kappa values were analyzed. The validity of the scale was assessed by correlating the FMS with the Gross Motor Function Classification System and the Gillette Functional Assessment Questionnaire Walking Scale. Twenty patients were evaluated by two researchers for interobserver reliability. Results: The kappa coefficients for test-retest reliability were 0.90 for FMS 5 m, 0.92 for FMS 50 m, and 0.91 for FMS 500 m. An evaluation of the validity revealed a significant correlation between FMS and the Gross Motor Function Classification System for all distances (r=-0.95, r=-0.96, and r=-0.92 for 5, 50, and 500 m, respectively; p <0.001), as well as the Gillette Functional Assessment Questionnaire Walking Scale (r=-0.95, r=-0.94, and r=-0.91 for 5, 50, and 500 meters, respectively; p <0.001). The kappa coefficients related to interobserver reliability were 0.73 for 5 m, 0.69 for FMS 50 m, and 0.81 for FMS 500 m. Conclusion: The Turkish version of the FMS can be considered a valid and reliable instrument for the assessment of cerebral palsy patients.

Adaptive functioning and neurodevelopment in patients with Dravet syndrome: 12-month interim analysis of the BUTTERFLY observational study

ObjectiveThe BUTTERFLY observational study aims to elucidate the natural trajectory of Dravet syndrome (DS) and associated comorbidities in order to establish a baseline for clinical therapies. We present the 12-month interim analysis of the study. Materials and methodsPatients with a genetically confirmed diagnosis of DS were enrolled in the study. Adaptive functioning and neurodevelopmental status were measured using the Vineland Adaptive Behavior Scale, Third Edition (Vineland-III), Bayley Scales of Infant Development, Third Edition (BSID-III), and Wechsler Preschool and Primary Scale of Intelligence, Fourth Edition (WPPSI-IV). Executive function, ambulatory function and locomotor activities, and overall clinical status were measured using the Behavior Rating Inventory of Executive Function – Preschool Version (BRIEF-P) scale, Gillette Functional Assessment Questionnaire (Gillette FAQ), and Clinician or Caregiver Global Impression of Change scales (CGI-C or CaGI-C) respectively. ResultsOverall, 36 patients were enrolled across three age groups, with 35 patients completing at least part or all of one post-baseline visit through Month 12. Significant improvements in receptive communication, as assessed by Vineland-III and BSID-III raw scores, and in verbal comprehension subtests, as assessed by WPPSI-IV raw scores, were observed in BUTTERFLY patients for the all-patient group. Many patients performed on the impaired end of the BRIEF-P Global Executive Composite scale at baseline suggesting difficulties in executive function, and no significant change was observed in BRIEF-P scores for the all-patient group. Most patients performed in the dynamic range of the Gillette FAQ at baseline, and no significant change was observed in Gillette FAQ scores for the all-patient group. Lastly, there was significant improvement observed in the CaGI-C scores for the all-patient group. SignificanceThis BUTTERFLY interim analysis shows small improvements in communication skills along with stability in other developmental abilities across patients with DS enrolled in the study from baseline to Month 12.

Psychometric properties of the Edinburgh Visual Gait Score in children with spastic cerebral palsy

BackgroundTo our knowledge, no other studies investigated the internal consistency of the Edinburgh Visual Gait Score (EVGS). The aim of our study was to determine the reliability and construct validity of the EVGS in children with cerebral palsy (CP).ResultsA total of fifty children with CP were enrolled in the study. Participants were evaluated with Gross Motor Function Classification System (GMFCS), Wisconsin Gait Scale (WGS), Gillette Functional Assessment Questionnaire (Gillette FAQ), Timed Up and Go Test (TUG), and EVGS (Rater-A and Rater-B). Slow-motion video analysis was used for the visual gait analysis. The inter-rater reliability, internal consistency, and construct validity of the EVGS were analyzed. The intraclass correlation coefficient (ICC) of the EVGS total score was 0.947 (CI: 0.90–0.97). Inter-rater reliability was excellent (ICC>0.80). The Cronbach’s alpha value was 0.936, within the acceptable range (0.70<α<0.95). In addition, the standard error of measurement (SEM95) and minimal detectable change (MDC95) scores of EVGS were 1.72 and 4.78, respectively. The correlation values of EVGS (Rater-A) and GMFCS, TUG, GFAQ, and WGS were 0.494, 0.661, −0.663, and 0.611, respectively. On the other hand, the correlation values of EVGS (Rater-B) and GMFCS, TUG, GFAQ, and WGS were 0.492, 0.664, −0.714, and 0.757, respectively. Except for comparison with GMFC, EVGS was highly valid in all other correlational analyzes (r>0.50). EVGS had moderate validity with GMFCS for both raters.ConclusionThe EVGS was reliable and valid. Internal consistency of the EVGS is high, indicating a consistent structure to assess gait in children with CP.

Cross-cultural adaptation, reliability and validation of the Gillette Functional Assessment Questionnaire (FAQ) into Brazilian Portuguese in patients with cerebral palsy

BackgroundThe purpose of this study was to translate, cross-culturally adapt and validate the Gillette Functional Assessment Questionnaire (FAQ) into Brazilian Portuguese.MethodsThe translation and cross-cultural adaptation was carried out in accordance with international recommendations. The FAQ was applied to a sample of 102 patients diagnosed with cerebral palsy (CP). Construct validity was assessed using Spearman’s correlation coefficient (rho), and the FAQ score was correlated with the Functional Mobility Scale (FMS) and Gross Motor Function Classification Scale (GMFCS). A subsample of 50 patients was used to assess reliability using intraclass correlation coefficient (ICC), standard error of measurement (SEM) and minimum detectable difference (MDD). Ceiling and floor effects were also evaluated.ResultsThe Brazilian version of the FAQ showed excellent test-retest reliability by the assessment of the physiotherapist (ICC = 0.99) and respondent (ICC = 0.97), as well as excellent inter-examiner reliability (ICC = 0.94). The SEM was 0.23 (physiotherapist), 0.47 (respondent) and 0.64 (inter-examiner), while the MDD was 0.64 (physiotherapist), 1.29 (respondent) and 1.76 (inter-examiner). The classification of gross motor function showed a high correlation with the FAQ applied by the physiotherapist (rho = -0.89) and by the respondent (rho = -0.87). The FMS-5 m was highly correlated with the FAQ applied by the physiotherapist and the respondent (rho = 0.88 and rho = 0.87, respectively). The FMS-50 and FMS-500 presented very high correlation with the FAQ applied by the physiotherapist (rho = 0.91 for both) and high correlation with the FAQ applied by the respondent (rho = 0.89 and rho = 0.88, respectively). The Brazilian version of the FAQ did not present the ceiling and floor effects.ConclusionThe FAQ presented adequate psychometric properties in patients with CP, indicating that it is possible to use it as a measure of functional gait mobility in Brazil.

Comparison of the use of intramedullary telescopic system and titanium elastic rods in children with osteogenesis imperfecta I and III types

Comparison of the use of intramedullary telescopic system and titanium elastic rods in children with osteogenesis imperfecta I and III types

The relationship between the backward walking and proprioception, trunk control, and muscle strength in children with cerebral palsy.

This study was planned to determine the factors affecting backward walking in children with cerebral palsy (CP). The study included 30 children with CP, with a mean age of 10.43 ± 2.76years. Backward walking abilities were evaluated with the 3-Meter Back Walk Test (3MBWT). A digital goniometer was used to evaluate proprioception, the Trunk Control Measurement Scale (TCMS) was used for trunk control, a digital muscle dynamometer was used for muscle strength, and the Gillette Functional Assessment Questionnaire (FAQ) was used for gait evaluation. When the spasticity of children at levels I and II according to the Gross Motor Function Classification System (GMFCS) was compared, a significant difference was found in favor of level I in hamstring, gastro-soleus, and gastrocnemius spasticity (p < 0.05). When the results of the 3MBWT, TCMS, and FAQ were compared, a significant difference was found in favor of level I (p < 0.05). No significant relationship was revealed between the 3MBWT and lower extremity proprioception and TCMS (p > 0.05). A significant negative correlation was observed between the 3MBWT and FAQ (p < 0.05). No significant correlation was found between the 3MBWT and lower extremity muscle strengths (p > 0.05). A significant positive correlation was found only between hip extension proprioception and iliopsoas muscle strength (p = 0.023). There was no significant correlation between the FAQ and lower extremity muscle strength (p > 0.05). It was revealed that the backward walking ability increased as the forward walking function improved in children with CP, but it was not affected by proprioception, trunk control, and muscle strength. NCT05088629 (10/11/2021).

Parental Distress: A Result of Functional and Behavioral Difficulties in Children with Cerebral palsy

Background: Children with cerebral palsy (CP) may have risk factors of Functional and Behavioral difficulties. Aims: To Determine the parental distress as a result of functional and behavioral difficulties of children with C.P. Methodology: The population was based in Pakistan, cross-sectional survey was conducted in which caregivers of 40 children with CP aged 3-8 Years were involved by online “ Survey System” software and completed the Agitated Behavior Scale(ABS), The Gillette Functional Assessment Questionnaire and Parental Stress Scale to determine the child’s behavior, functional difficulties, and parental distress. Correlation paired 2-tail and Linear regression was used to analyze the data. Data were analyzed using IBM SPSS 20.Results: The demographic data (Age and Gender),non-significant regression equation f=2 (37), P &gt; 0.01 with an Power calculation r2 = 0.45. Functional difficulties (F.A and L. A), not-significant regression equation was found df=4(35) , P &gt; 0.01 with an Power calculation r2 = 0.07 while, not-significant regression equation was found df=5(34) , P &gt; 0.01 with an Power calculation r2 = 0.03 and findings indicate that parental distress has no statistically significant relationship (|r| n=40, p&gt; 0.05) to Functional Abilities , Locomotors Abilities and Behavior Difficulties. Conclusion and Implications: Evidence shows that psychological, behavioral, emotional, and communication problems are frequent in children with CP. It’s not compulsory to have highly rated parental stress in case of children having any disability and child’s effect agitated behaviors. Effective rehabilitation programs should provide not only sufficient opportunities for the children’s disabilities but also psychological support for the mothers. Key Words: Cerebral Palsy, Children, Stress, Behavior, Parental Distress, Functional Difficulties, Locomotor Abilities

Retrospective case series of inverted V-shape tenotomy of gastrocnemius and recession of soleus at the musculotendinous junction for the treatment of fixed equinus deformity in children with cerebral palsy

Background: The authors describe results of a surgical percutaneous procedure for gastrocnemius-soleus lengthening at musculotendinous junction using a one-cut inverted V-shape technique for equinus correction in patients with cerebral palsy (CP). Methods: This was a retrospective case-series study of 52 patients with spastic CP between November 2001 and November 2016. Gross Motor Function Classification System (GMFCS) levels (II-IV) were involved. Statistical analysis was carried out for ankle dorsiflexion angles with knee extended and flexed, popliteal angle, and Gillette Functional Assessment Questionnaire (FAQ). Failure was determined by recurrence of equinus deformity or developing crouch gait due to overlengthening. Results: A mean age of 7 yr was recorded, with a minimum follow-up of 1 yr. A total number of 88 feet were operated upon, including several children who had bilateral deformities. Twelve (23%) patients were operated previously by either percutaneous or open tendo-Achilles lengthening. Success was achieved in 44 patients (85%), and eight patients (15%) had failure. Noncompliance with physical therapy and night splinting were the determined causes of failure for patients who had recurrence. GMFCS level-IV was a particular risk of failure (75%). No patient developed postoperative crouch gait caused by an over-lengthened tendo-Achilles. The improvement in the outcome measures were clinically significant in the successful group. Conclusions: The authors’ percutaneous gastrocnemius-soleus lengthening one-cut technique is a safe, cosmetic, and economic day-case surgery. It has low recurrence rate, with minimal risk of developing a crouched gait from overlengthening. Level of Evidence: Level IV.

The Effectiveness of Robot- vs. Virtual Reality-Based Gait Rehabilitation: A Propensity Score Matched Cohort.

Robot assisted gait training (RAGT) and virtual reality plus treadmill training (VRTT) are two technologies that can support locomotion rehabilitation in children and adolescents affected by acquired brain injury (ABI). The literature provides evidence of their effectiveness in this population. However, a comparison between these methods is not available. This study aims at comparing the effectiveness of RAGT and VRTT for the gait rehabilitation of children and adolescents suffering from ABI. This is a prospective cohort study with propensity score matching. Between October 2016 and September 2018, all patients undergoing an intensive gait rehabilitation treatment based on RAGT or VRTT were prospectively observed. To minimize selection bias associated with the study design, patients who underwent RAGT or VRTT were retrospectively matched for age, gender, time elapsed from injury, level of impairment, and motor impairment using propensity score in a matching ratio of 1:1. Outcome measures were Gross Motor Function Mesure-88 (GMFM-88), six-min walking test (6MWT), Gillette Functional Assessment Questionnaire (FAQ), and three-dimensional gait analysis (GA). The FAQ and the GMFM-88 had a statistically significant increase in both groups while the 6MWT improved in the RAGT group only. GA highlighted changes at the proximal level in the RAGT group, and at the distal district in the VRTT group. Although preliminary, this work suggests that RAGT and VRTT protocols foster different motor improvements, thus recommending to couple the two therapies in the paediatric population with ABI.

Health-related quality of life in adults with osteogenesis imperfecta.

Patient-reported outcome measures (PROMs) are increasingly utilized as endpoints in clinical trials. The Short Form Health Survey-12 (SF-12v2) is a generic PROM for adults. We sought to evaluate the validity of SF-12v2 in adults with osteogenesis imperfecta (OI). Physical and mental health-related quality of life (HRQoL) were assessed in a large cohort of adults in a multicenter, observational, natural history study. Physical HRQoL scores were correlated with the Gillette Functional Assessment Questionnaire (GFAQ). We calculated sample sizes required in clinical trials with crossover and parallel-group designs to detect clinically meaningful changes in physical HRQoL. Three hundred and two adults with OI types I, III, and IV were enrolled. Physical HRQoL scores in the study population were lower than population norms. Physical HRQoL scores moderately correlated with GFAQ for OI types I and IV. We found no correlations between mental and physical HRQoL. From a clinical trial readiness perspective, we show that SF-12v2 reliably measures physical function in adults with OI and can be utilized in crossover trials to detect meaningful physical HRQoL changes with small sample sizes. This study shows that SF-12v2 can be used to measure changes in physical HRQoL in response to interventions in OI.

Falls and Associated Factors among Adolescents and Young Adults with Arthrogryposis Multiplex Congenita.

Background:Falls research among individuals with arthrogryposis multiplex congenita (AMC), a group of congenital conditions characterized by joint contractures in two or more body regions, is sparse. The primary objectives of this study were to estimate the prevalence of single, multiple, and injurious falls among adolescents and adults with AMC and identify factors associated with multiple and injurious falls.Methods:Individuals, aged 10–50 years, with a diagnosis of AMC completed questionnaires obtaining demographic and AMC-specific information, falls history (e.g., number, injurious/non-injurious), markers of bone health, orthopedic surgical history, and mobility aid use, as well as the Gillette Functional Assessment Questionnaire and the Saltin-Grimby Physical Activity Level Scale. Falls were defined as “any body part above the ankle coming to rest on the ground, floor, or a lower level”. Falling was defined as ≥ 1 fall, while multiple falls were defined as ≥ 2 falls in the past year.Differences in falling, multiple falls, and injurious falls rates between adolescents (10–17 years) and adults (aged 18–50 years) were evaluated. Using univariate binary logistic regression models, associations between participant characteristics and multiple falls, as well as injurious falls, were evaluated, while considering age as a covariate (p ≤ 0.050); odds ratios (ORs) and 95% confidence intervals (CIs) were calculated.Results:Adolescents (N = 28; median age = 14 years) and adults (N = 40; median age = 32 years) with AMC had similar falling, i.e., 89.3% versus 70.0%, and injurious fall rates, i.e., 32.1% versus 27.5%, respectively (p > 0.050).Adolescents with AMC, however, were more likely to report multiple falls in the past year, i.e., 89.3%, when compared to adults with AMC, i.e., 57.5% (p = 0.005). Beyond age, multiple falls were associated with a greater number of lower-limb surgeries [p = 0.036, OR (95%CI): 1.18 (1.01–1.39)], ability to transfer floor-to-stand with support [p = 0.002, OR (95%CI): 8.98 (2.30–35.06)], and increased mobility per the Gillette Functional Assessment Questionnaire [p = 0.004, OR (95%CI): 1.48 (1.13–1.92)]. Factors associated with a reduced odds of multiple falls were spinal involvement [p = 0.025, OR (95%CI): 0.23 (0.07–0.84)], history of spinal surgery [p = 0.018, OR (95%CI: 0.18 (0.04–0.74)], greater upper extremity involvement [OR (95%CI): 0.65 (0.44–0.95)], home assistive device use [p = 0.010, OR (95%CI): 0.15 (0.03–0.63)], and community wheelchair use [p = 0.006, OR (95%CI): 0.16 (0.04–0.59)]. None of the explored characteristics were associated with injurious falls in the past year (p > 0.050).Conclusion:Falls are exceedingly common among adolescents and adults with AMC; potential risk and protective factors for multiple falls are identified for future prospective falls research.

FAMILY-CENTRED, GOAL-DIRECTED MULTIDISCIPLINARY APPROACH FOR LOWER EXTREMITY BOTULINUM TOXIN WITH PHYSICAL THERAPY AND REHABILITATION IN CEREBRAL PALSY

Purpose: This study aimed to investigate lower extremity botulinum toxin (BT) and physical therapy and rehabilitation (PTR) application scoping “family-centered, goal-directed multidisciplinary approach (FGMA)” in the children with cerebral palsy (CP) and to assess the satisfaction of parents and children from this approach. Methods: A physician and physiotherapist evaluated 30 children (age=6.33±2.38 years) with ambulatory CP and their parents using the FGMA. Gross Motor Function Classification System (GMFCS), Manual Ability Classification System (MACS), and Communication Function Classification System (CFCS) were used to define the functionality of children. Selectivity was assessed using the Selective Control Assessment of the Lower Extremity (SCALE). Walking was evaluated using the Observational Gait Scale (OGS) and the Gillette Functional Assessment Questionnaire (FAQ). Satisfaction levels marked on the Visual Analogue Scale. Results: Nineteen (63%) children were GMFCS level III, 16 (53%) children were MACS level I, 19 (63.33%) children were CFCS level I. Half of children had visual problems. While the most preferred muscles were hamstring and gastrocnemius for BT, the most common device was ankle-foot orthosis. The median score of SCALE, OGS, and FAQ, and the satisfaction of parents and children were 8 (4-17), 12 (2-24), 2 (1-10), 9 (7-10), and 7 (6-10) points, respectively. The satisfaction level of parents with the new approach was higher than the previous traditional approach (p&amp;lt;0.001). Conclusions: Both the parents and children may be satisfied with the FGMA for BT with the PTR program. Clinicians should take into account lower extremity selectivity, walking performance, and satisfaction levels as much as muscle tone or range of motion. Key Words: Botulinum Toxin; Cerebral Palsy; Parent; Physical Therapy.

Long-term Outcomes Following Multilevel Surgery in Cerebral Palsy.

This long-term consecutive, retrospective single-center cohort study evaluates long-term outcomes of single-event multilevel surgery in diplegic cerebral palsy with respect to functional status, gait, and patient satisfaction. All patients with diplegic cerebral palsy who underwent single-event multilevel surgery >10 years previously were included. Retrospective gait assessment was performed using the Edinburgh Visual Gait Score (EVGS) and Gillette Functional Assessment Questionnaire Walking Scale (FAQWS) preoperatively and at midterm postoperative follow-up (median 2.6 y) and prospectively at most recent review [median 18 y, interquartile (IQ) range: 14.4 to 20.5 y]. The Short Form-36 (SF-36) was used prospectively to evaluate outcome compared with population norms for adults from the UK. The complete assessment was possible in 26 of 39 patients who met the inclusion criteria. There was a statistically significant improvement at most recent follow-up compared with the preoperative assessment for EVGS and FAQWS [Wilcoxon Matched Pairs Signed Rank test -4.42 (P<0.0001) and 3.98 (P=0.0001), respectively]. The median Physical Health and Mental Health Component Summary scores for the SF-36 were 43.0 (IQ range: 32.8 to 46.8) and 55.3 (IQ range: 46.1 to 61.1), respectively. This study demonstrates significant long-term improvements in function, independence and patient satisfaction that continue into adulthood. This will inform discussions with patients and families considering this treatment option. Level IV-Retrospective cohort study.

A Multicenter Study of Intramedullary Rodding in Osteogenesis Imperfecta.

Background:Osteogenesis imperfecta (OI), a heritable connective tissue disorder with wide clinical variability, predisposes to recurrent fractures and bone deformity. Management requires a multidisciplinary approach in which intramedullary rodding plays an important role, especially for moderate and severe forms. We investigated the patterns of surgical procedures in OI in order to establish the benefits of rodding. The main hypothesis that guided this study was that rodded participants with moderate and severe OI would have lower fracture rates and better mobility.Methods:With data from the Linked Clinical Research Centers, we analyzed rodding status in 558 individuals. Mobility and fracture data in OI Types III and IV were compared between rodded and non-rodded groups. Univariate regression analyses were used to test the association of mobility outcomes with various covariates pertinent to rodding.Results:Of the individuals with OI, 42.1% had undergone rodding (10.7% of those with Type I, 66.4% with Type III, and 67.3% with Type IV). Rodding was performed more frequently and at a younger age in femora compared with tibiae. Expanding intramedullary rods were used more frequently in femora. In Type III, the rate of fractures per year was significantly lower (p ≤ 0.05) for rodded bones. In Type III, the mean scores on the Gillette Functional Assessment Questionnaire (GFAQ) and Brief Assessment of Motor Function (BAMF) were higher in the rodded group. However, Type-IV non-rodded subjects had higher mean scores in nearly all mobility outcomes. OI type, the use of expanding rods in tibiae, and anthropometric measurements were associated with mobility outcomes scores.Conclusions:Current practice in 5 orthopaedic centers with extensive experience treating OI demonstrates that most individuals with moderate and severe types of OI undergo rodding procedures. Individuals with severe OI have improved mobility outcomes and lower fracture rates compared with their non-rodded peers, which suggests that early bilateral rodding benefits OI Type III. Our analysis showed a change in practice patterns in the final years of the study in the severe forms, with earlier and more simultaneous rodding procedures performed.Level of Evidence:Therapeutic Level III. See Instructions for Authors for a complete description of levels of evidence.

How does the gait profile score relate to the gillette functional assessment questionnaire 22-item skill set, a preliminary study.

How does the gait profile score relate to the gillette functional assessment questionnaire 22-item skill set, a preliminary study.

Surgical treatment of comminuted intraarticular distal femur fracture in patient with osteogenesis imperfecta type I

Aim. Osteogenesis imperfecta (OI) is characterized by bone fragility and long bones deformities. Most studies are dedicated to surgical treatment of diaphyseal fractures. To our knowledge, there are no reports giving recommendations about surgical treatment of distal femur intraarticular fractures.&#x0D; Clinical case. We describe the surgical treatment of a 14-year-old girl with OI who had intraarticular fracture of the left distal femur and fracture of a right femur diaphysis. Surgical treatment was complicated by migration of a titanium elastic nail and impaired consolidation, which had to be fixed with a plate and led to peri-implant fracture. Results were assessed before trauma and at 1 and 2 years after trauma with Gillette Functional Assessment Questionnaire (GFAQ) and Bleck score.&#x0D; Discussion. During surgical treatment of comminuted intraarticular distal femur fractures in patients with OI, we had to use big cancellous screw that made implantation in an intramedullary fixator more difficult. Internal fixation with a plate in patients with OI is associated with high risks of peri-implant fracture.&#x0D; Conclusion. For treatment of comminuted intraarticular fracture of the distal femur, it is necessary to have large variety of internal fixators, follow the principles of absolute and relative stability, and be familiar with minimally-invasive techniques.

Impact of Orthotic Prescription in Pediatric Partial Foot Amputation: A Case Study

ABSTRACT Introduction There is little information available on outcomes after prosthetic/orthotic management in partial foot amputation (PFA) in children. Methods This case study evaluates walking speed, community activity level, and physical function in a child with bilateral PFA after a change from an orthosis finishing distal to the ankle to one finishing proximal to the ankle. Outcome measures used were walking speed (10-meter walk test), the Gillette Functional Assessment Questionnaire (GFAQ), and the Oxford Ankle Foot Questionnaire for Children (OxAFQ-C) physical domain. Results At 3-month review GFAQ level increased by 3, OxAFQ-C physical domain increased 50 percentage points, and self-selected velocity increased 0.25 m/s. Conclusions This case study suggests that improvements in walking speed, community activity level, and physical function are possible in children with bilateral PFA when changing to orthoses, which compensate for mechanical deficits caused by the reduced lever arm of the foot.

Clinical Trial Designs and Measures in Hereditary Spastic Paraplegias.

Hereditary spastic paraplegias (HSPs) are a large group of genetically-diverse neurologic disorders characterized clinically by a common feature of lower extremity spasticity and gait difficulties. Current therapies are predominantly symptomatic, and even then usually provide inadequate relief of symptoms. Going forward, HSP therapeutics development requires a systematic analysis of quantifiable measures and tools to assess treatment response. This review summarizes promising therapeutic targets, assessment measures, and previous clinical trials for the HSPs. Oxidative stress, signaling pathways, microtubule dynamics, and gene rescue/replacement have been proposed as potential treatment targets or modalities. Quantitative evaluation of pre-clinical rodent HSP models emphasize rotarod performance, foot base angle, grip strength, stride length, beam walking, critical speed, and body weight. Clinical measures of HSP in humans include 10-m gait velocity, the Spastic Paraplegia Rating Scale (SPRS), Ashworth Spasticity Scale, Fugl-Meyer Scale, timed up-and-go, and the Gillette Functional Assessment Questionnaire. We conducted a broad search for past clinical trials in HSPs and identified trials that investigated pharmacological agents including atorvastatin, gabapentin, L-threonine, botulinum toxin, dalfampridine, methylphenidate, and baclofen. We provide recommendations for future HSP treatment directions based on these prior research experiences as well as regulatory insight.

Evaluating the Gillette Functional Assessment Questionnaire and the Functional Mobility Scale in a pediatric inpatient setting.

This commentary is on the original article by Ammann‐Reiffer et al. on pages 717–724 of this issue.