Goal Attainment Scaling Research Articles

Effects of Telerehabilitation-Based Structured Home Program on Activity, Participation and Goal Achievement in Preschool Children with Cerebral Palsy: A Triple-Blinded Randomized Controlled Trial

A home program is implemented as an evidence-based mode of delivering services for physiotherapy and rehabilitation. Telerehabilitation is a method applied in physiotherapy modalities for children. This study aims to determine the effectiveness of usual care plus a Telerehabilitation-Based Structured Home Program on preschool children with cerebral palsy (CP) compared to usual care. Forty-three children aged 3-6 years (mean age 4.66 ± 1.08 years) with CP were randomly assigned to the Telerehabilitation-Based Structured Home Program and usual care groups. Their motor function was assessed with the Gross Motor Function Measure (GMFM); performance and satisfaction were evaluated with the Canadian Occupational Performance Measure (COPM); goal achievement was assessed with the Goal Attainment Scale (GAS); and activity and participation were evaluated with Pediatric Evaluation of Disability Inventory (PEDI). Participants were evaluated at baseline, immediately post-intervention (12 weeks) and at follow-up (24 weeks). There was a statistically significant difference between pre- and post-test GMFM, COPM, GAS and PEDI scores in the intervention and control groups (p < 0.001). The Telerehabilitation-Based Structured Home Program showed statistically significant changes in activity, participation and goal achievement after 12 weeks of intervention (p < 0.001). However, significant results were not obtained in the usual care group. The Telerehabilitation-Based Structured Home Program may be an effective method for preschool children with CP. (Registration number: NCT04807790; no = KA-20124/26.01.2021).

Feasibility, reliability and validity of a modified approach to goal attainment scaling to measure goal outcomes following cognitive remediation in a residential substance use disorder rehabilitation setting

ABSTRACT Objective Although person-centred outcome measures have been recommended to evaluate cognitive rehabilitation interventions, few validated measures have been developed for this purpose. The current study examined aspects of feasibility, reliability and validity of a modified version of goal attainment scaling that uses a goal menu, calculator and control goals. Method Participants were N=25 female residents of a substance use disorder therapeutic community who were allocated to a four-week cognitive remediation (n=13) or treatment as usual (n=12) control group in a controlled sequential groups trial. Modified goal attainment scaling was used to set goals. Limited efficacy and efficiency, quality appraisal criteria, and convergent and discriminant validity of target and control goals were used to examine feasibility, reliability and content validity, and construct validity, respectively. Results Target goals were achieved at a higher rate than control goals for the Intervention, but not Control, group, with a medium effect size (r = 0.5). The approach was efficient and 44% of reliability and 75% of content validity criteria were met. Target goals correlated more strongly than control goals with the Behavior Rating Inventory of Executive Function - Adult version. Conclusions The modified approach to goal attainment scaling demonstrated aspects of feasibility, reliability and validity.

Measuring what gets done: Using goal attainment scaling in a vocational counseling program for survivors of childhood cancer.

Childhood cancer survivors face education and employment challenges due to physical, cognitive, and psychosocial effects of the disease and treatments, with few established programs to assist them. The objectives of this study were to describe the implementation of Goal Attainment Scaling (GAS) to evaluate an educational and vocational counseling program established for survivors of childhood cancer, and analyze patterns of program engagement and client outcomes, stratified by demographic and diagnostic characteristics. A population-based retrospective cohort study of childhood cancer survivors who were engaged with the Pediatric Oncology Group of Ontario's School and Work Transitions Program (SWTP) between January 2015 and December 2018 was utilized. Survivors were followed from SWTP engagement until May 30, 2019 to capture goal attainment. Individual goals were summarized across various demographic, disease, and treatment strata. In total, 470 childhood cancer survivors (median age=17.9, 58% male) set 4,208 goals in the SWTP during the study period. The mean length of observation was 130.8 weeks (SD=56.9). Overall, 68% of the goals were achieved. Eighty-three percent of the goals related to further education. Clients diagnosed with a solid tumor set the most goals on average, followed by those with central nervous system tumors and leukemia/lymphoma. The SWTP assists childhood cancer survivors in realizing their academic and vocational goals. Application of GAS in this setting is a feasible way to evaluate program outcomes. From the volume and breadth of the GAS goals set and achieved, the overall success of the SWTP appears strong.

Content and attainment of individual treatment goals in CBT

This study examined the attainment of individual treatment goals as an approach to measure treatment outcomes retrospectively in comparison to standardized self-report measures. In total, 189 outpatients defined 1–3 treatment goals (N = 455) using the Goal Attainment Scaling (GAS) at the beginning of cognitive behavioral therapy (CBT) and indicated the degree of their individual goal attainment post-treatment. The goals were assigned to content-related goal types of the Bern Inventory of Treatment Goals (BIT-T). The extent of goal attainment was, on average, between 1 (goal halfway attained) and 2 (goal attained) (M = 1.43), regardless of goal content and diagnostic group. Goals of the goal type “problems and symptoms” were chosen most frequently and showed significantly higher goal attainment than “interpersonal” goals, thereby indicating that outpatients in CBT mainly want to work on treatment goals like coping with problems and symptoms. Additionally, standardized self-report measures (Brief Symptom Inventory, Beck Depression Inventory-II) were assessed, and associations with the GAS were calculated. They correlated significantly but moderately, thus indicating that the two concepts complement each other and should ideally be implemented together.

THE RESULTS OF ENVIRONMENTAL ENRICHMENT AND NEURODEVELOPMENTAL TREATMENT IN SHPRINTZEN-GOLDBERG SYNDROME: A CASE REPORT

Purpose: This case report aimed to investigate the efficacy of the physiotherapy program in a child with Shprintzen-Goldberg syndrome (SGS). &#x0D; Methods: A 9-month-old case diagnosed with SGS by a medical geneticist was the focus of the study. Congenital pes varus, craniosynostosis and craniofacial deformities, mental, social, emotional and motor retardation, regulation and sensory hyperreactivity symptoms were observed in the case. The physiotherapy program consists of environmental enrichment and Neurodevelopmental Treatment (NDT) approaches twice a week for 12 months and was conducted by a physiotherapist at Karadeniz Ereğli Private Gökkuşağı Special Education and Rehabilitation Center. Gross motor function and disability level were assessed using Gross Motor Function Measurement-88 (GMFM-88) and Gross Motor Function Classification System (GMFCS), respectively. The success of physiotherapy goals was determined by Goal Attainment Scaling (GAS).&#x0D; Results: At 12 months follow-up, GMFM-88 score increased from 5.52% to 45.47% and GAS total scores increased from -6 to +6 point. &#x0D; Conclusions: The literature review shows that no previous study assessed the effectiveness of 12 months physiotherapy program in a child with SGS. We consider that physiotherapy including targeted NDT approaches can support motor development in rare cases, such as SGS characterized by severe motor involvement.

Evaluating the feasibility and preliminary efficacy of a Cognitive Occupation-Based programme for people with Multiple Sclerosis (COB-MS): an update to the protocol for a feasibility cluster-randomised controlled trial

BackgroundCognitive difficulties experienced by people with multiple sclerosis (MS) impact on quality of life and daily functioning, from childcare and work to social and self-care activities. The Cognitive Occupation-Based programme for people with MS (COB-MS) was developed as a holistic, individualised cognitive rehabilitation intervention to address the wide-ranging symptoms and functional difficulties that present in MS, including the ability to maintain employment, social activities, home management and self-care. The aim of the research is to evaluate the feasibility and preliminary efficacy of COB-MS for people with MS.MethodsDue to the impacts of COVID-19, trial activities that were planned for in-person delivery were completed remotely. One hundred and twenty people with MS will be assigned to participate in either the COB-MS programme or a treatment-as-usual, wait-list control group as part of this single-blind, cluster-randomised controlled feasibility and preliminary efficacy trial of the COB-MS programme. The COB-MS group will participate in an eight-session occupational-based cognitive rehabilitation programme over 9 weeks. The COB-MS intervention was planned for in-person delivery but was delivered online by occupational therapists to small groups of people with MS. The primary outcome measure is the Goal Attainment Scaling at 12 weeks. Participants will be assessed pre-intervention, post-intervention, 12 weeks post-intervention and 6 months post-intervention. Qualitative evaluations of participants’ perspectives will also be examined as part of the feasibility study. Data, due to be collected in-person, was collected online or by post. The original study design, including the statistical analysis plan, remains unchanged despite the shift to a remote trial conduct.DiscussionResults will provide recommendations for a future definitive trial of COB-MS, with respect to both feasibility and preliminary, clinical efficacy.Trial registrationISRCTN ISRCTN11462710. Registered on 9 September 2019 and updated on 23 September 2020 to account for changes outlined here.

Efficacy of self-management program associated with a spa therapy for knee osteoarthritis patients (GETT 2): a research protocol for a randomized trial

IntroductionOsteoarthritis is a chronic pathology that involves multidisciplinary management. Self-management for patients is an essential element, present in all international guidelines. During the time of the spa therapy, the patient is receptive to take the advantage of self-management workshops.The aim of this study is to assess the effects of 18 days spa therapy associated with a self-management intervention in patients with knee osteoarthritis in comparison with spa therapy alone on a priority objective, personalized and determined with the patient, chosen in the list of 5 objectives determined during the self-management initial assessment.Methods and analysisTwo hundred fifty participants with knee osteoarthritis will participate to this multicenter, prospective, randomized, controlled study. All patients will benefit 18 days of spa therapy and patients randomized in the intervention group will participate to 6 self-management workshops. Randomization will be centralized. The allocation ratio will be 1:1. Data analysts and assessor will be blinded. The primary outcome is the effectiveness of the educational workshops associated with spa therapy in comparison with spa therapy alone on a priority objective, measured by Goal Attainment Scaling (GAS). The secondary outcomes are disability, health-related quality of life, and pain intensity.Ethics and disseminationEthics were approved by the CPP Sud-Méditerranée II. The results will be disseminated in a peer-reviewed journal and disseminated at PRM, rheumatology, and orthopedics conferences. The results will also be disseminated to patients.Trial registrationTrial registration number NCT03550547. Registered 8 June 2018.Date and version identifier of the protocol. Version N°6 of March 12, 2018.

Effects of specialist care lower limb orthoses on personal goal attainment and walking ability in adults with neuromuscular disorders.

Lower limb orthoses intend to improve walking in adults with neuromuscular disorders (NMD). Yet, reported group effects of lower limb orthoses on treatment outcomes have generally been small and heterogeneous. We propose that guideline-based orthotic care within a multidisciplinary expert setting may improve treatment outcomes. To examine the effectiveness of specialist care orthoses compared to usual care orthoses on personal goal attainment and walking ability. Cohort study. Adults with NMD who experienced walking problems due to calf and/or quadriceps muscle weakness and were provided with a specialist care lower limb orthosis between October 2011 and January 2021. Three months after provision, the specialist care orthosis was compared to the usual care orthosis worn at baseline in terms of personal goal attainment (Goal Attainment Scaling (GAS)), comfortable walking speed (m/s), net energy cost (J/kg/m) (both assessed during a 6-minute walk test), perceived walking ability and satisfaction. Sixty-four adults with NMD were eligible for analysis. The specialist care orthoses comprised 19 dorsiflexion-restricting ankle-foot orthoses (AFOs), 22 stance-control knee-ankle-foot orthoses (KAFOs) and 23 locked KAFOs. Overall, 61% of subjects showed a clinically relevant improvement in GAS score. Perceived safety, stability, intensity, fear of falling and satisfaction while walking all improved (p≤0.002), and subjects were satisfied with their specialist care orthosis and the services provided. Although no effects on walking speed or net energy cost were found in combined orthosis groups, specialist care AFOs significantly reduced net energy cost (by 9.5%) compared to usual care orthoses (from mean (SD) 3.81 (0.97) to 3.45 (0.80) J/kg/m, p = 0.004). Guideline-based orthotic care within a multidisciplinary expertise setting could improve treatment outcomes in adults with NMD compared to usual orthotic care by improvements in goal attainment and walking ability. A randomized controlled trial is now warranted to confirm these results.

The Effectiveness of Exercise Physiology Services During the COVID-19 Pandemic: A Pragmatic Cohort Study

BackgroundThe COVID-19 pandemic markedly changed how healthcare services are delivered and telehealth delivery has increased worldwide. Whether changes in healthcare delivery borne from the COVID-19 pandemic impact effectiveness is unknown. Therefore, we examined the effectiveness of exercise physiology services provided during the COVID-19 pandemic.MethodsThis prospective cohort study included 138 clients who received exercise physiology services during the initial COVID-19 pandemic. Outcome measures of interest were EQ-5D-5L, EQ-VAS, patient-specific functional scale, numeric pain rating scale and goal attainment scaling.ResultsMost (59%, n = 82) clients received in-person delivery only, whereas 8% (n = 11) received telehealth delivery only and 33% (n = 45) received a combination of delivery modes. Mean (SD) treatment duration was 11 (7) weeks and included 12 (6) sessions lasting 48 (9) minutes. The majority (73%, n = 101) of clients completed > 80% of exercise sessions. Exercise physiology improved mobility by 14% (β = 0.23, P = 0.003), capacity to complete usual activities by 18% (β = 0.29, P < 0.001), capacity to complete important activities that the client was unable to do or having difficulty performing by 54% (β = 2.46, P < 0.001), current pain intensity by 16% (β = − 0.55, P = 0.038) and goal attainment scaling t-scores by 50% (β = 18.37, P < 0.001). Effectiveness did not differ between delivery modes (all: P > 0.087).ConclusionsExercise physiology services provided during the COVID-19 pandemic improved a range of client-reported outcomes regardless of delivery mode. Further exploration of cost-effectiveness is warranted.

Long-Term Exercise Assistance: Group and One-on-One Interactions between a Social Robot and Seniors

For older adults, regular exercises can provide both physical and mental benefits, increase their independence, and reduce the risks of diseases associated with aging. However, only a small portion of older adults regularly engage in physical activity. Therefore, it is important to promote exercise among older adults to help maintain overall health. In this paper, we present the first exploratory long-term human–robot interaction (HRI) study conducted at a local long-term care facility to investigate the benefits of one-on-one and group exercise interactions with an autonomous socially assistive robot and older adults. To provide targeted facilitation, our robot utilizes a unique emotion model that can adapt its assistive behaviors to users’ affect and track their progress towards exercise goals through repeated sessions using the Goal Attainment Scale (GAS), while also monitoring heart rate to prevent overexertion. Results of the study show that users had positive valence and high engagement towards the robot and were able to maintain their exercise performance throughout the study. Questionnaire results showed high robot acceptance for both types of interactions. However, users in the one-on-one sessions perceived the robot as more sociable and intelligent, and had more positive perception of the robot’s appearance and movements.

The Effect of Occupation-Based Modified Constraint-Induced Movement Therapy on the Participation of Children with Cerebral Palsy: A Single-Blind Randomized Controlled Trial.

This study investigates the impact of modified constraint-induced movement therapy (m-CIMT), accompanied by occupation-based and activity analysis, on the participation of children with hemiplegia. Twenty-three participants were randomly assigned to the intervention and control groups. The intervention group received occupation-based m-CIMT (m-CIMT along with occupation-based and activity analysis), while the control group received m-CIMT without occupation-based and activity analysis. The intervention was conducted one hour per day, three days a week, for four weeks. The primary outcomes revealed no significant differences between groups in promoting the participation of children with hemiplegia in the activities of daily living (ADL). However, scores were higher in the intervention group with a medium to large effect size (Canadian occupational performance measure: F(1,19)=2.14, P=0.160, η2P=0.101, Canadian occupational performance measure-satisfaction: F(1,19)=1.53, P=0.231, η2P=0.075, Goal attainment scaling: F(1,19)=5.55, P=0.029, η2P=0.226). This effect remained during the follow-up period. The secondary outcomes indicated no significant differences between groups in improving the manual ability of the children. However, scores were higher in the intervention group with a medium to large effect size (ABILHAND-Kids: F(1,19)=0.64, P=0.434, η2P=0.033, pediatric motor activity log- how long: F(1,19)=3.53, P=0.076, η2P=0.157, pediatric motor activity log- how well: F(1,19)=2.59, P=0.124, η2P=0.120). This effect was sustainable during the follow-up period. m-CIMT accompanied by occupation-based and activity analysis and the client-centered paradigm substantially enhances the manual ability of children with hemiplegia and their participation in the ADL.

Evaluation of Selective Tibial Neurotomy for the Spastic Foot Treatment Using a Personal Goal-Centered Approach: A 1-Year Cohort Study.

Selective tibial neurotomy (STN) has already demonstrated its effectiveness to reduce foot deformities and spasticity, but assessment according to a goal-centered approach is missing. To evaluate the effectiveness of STN associated with a postoperative rehabilitation program for the treatment of the spastic foot, according to a goal-centered approach. Interventional study (before-after STN and rehabilitation program) with observational design including consecutive adult patients with spastic foot, who received STN followed by a rehabilitation program, was performed. The primary outcome measure was the achievement of individual goals at the 1-year follow-up using the Goal Attainment Scaling methodology (with T-score). The secondary outcomes measures were the Modified Ashworth Scale and the modified Rankin Score. A total of 104 patients were included. At the 1-year follow-up, 228/252 (90.5%) goals were achieved: 62/252 (24.6%) were achieved as initially expected, 86/252 (34.1%) were achieved better than initially expected, and 80 (31.7%) were achieved much better than initially expected. The mean T-score was significantly increased at the 1-year follow-up (61.5 ± 10.5) compared with the preoperative period (38.1 ± 2.9, P < .00001), and 95/104 (91.3%) patients had a T-score ≥50, meaning that these patients have achieved their goals. At follow-up, spastic deformities were all significantly decreased ( P < .0001), the Modified Ashworth Scale was significantly lower for each muscle targeted ( P < .0001), and the modified Rankin Score was significantly decreased ( P < .0001) allowing the patient population to improve from a moderate to a slight disability status. This study showed that STN, associated with a postoperative rehabilitation program, successfully achieve personal goals in patients with spastic foot.

INTERVENTION FIDELITY IN THE FAMILY-CENTERED FUNCTION-FOCUSED CARE INTERVENTION

Abstract This session will provide a description of the treatment fidelity (TF) plan from the Family-centered Function-focused Care (Fam-FFC) trial. Components of the TF plan, measures, procedures for implementation, and findings will be presented, and discussed within the context of the COVID-19 pandemic. The components of the Fam-FFC TF plan and results include: 1) Delivery based on completion of the steps in Fam-FFC ; 2) Receipt based on evidence of Staff knowledge of Fam-FFC (percentage of nursing staff that demonstrated test scores above 80%); 3) Enactment based on achievement of goals using the Goal Attainment Scale ; completion of the Fam-Path Audit of bedside goals and treatment plans, post-acute follow-up and plan update ; and evidence of Fam-FFC based on the Fam-FFC Behavior Checklist (80% staff performance of Fam-FFC). The TF plan demonstrated evidence of delivery, receipt and enactment of study activities. Findings will be used to develop an implementation trial.

The feasibility of a flexible exercise participation programme (FEPP) for individuals with multiple sclerosis.

Individuals with multiple sclerosis (MS) want health advice regarding participation in their choice of exercise. To address this need, a flexible exercise participation programme (FEPP) was developed, underpinned by the MS aerobic exercise guidelines and supported by a physiotherapist using behaviour change techniques. The aim of this study was to investigate the feasibility of the FEPP for individuals with minimal disability from MS. A feasibility study utilising a single group pre/post-intervention design was conducted. The 12-week FEPP was completed by 10 individuals with MS (EDSS 0-3.5). Exercise progression in duration, intensity or frequency of exercise (in line with MS exercise guidelines) was guided by a self-perceived weekly energy level score, and weekly telephone coaching sessions using behavioural change techniques. Trial feasibility was assessed via measures of process (recruitment and retention), resources/management (communication time; data entry) and scientific feasibility (safety; compliance). Secondary FEPP feasibility outcomes included the Goal Attainment Scale (GAS) T-score, exercise participation (weekly exercise diary), high-level mobility (HiMAT), vitality (Subjective Vitality Scale), biomarkers for inflammation (cytokines levels [IL2, IL4, IL6, IL10, TNF and IFNγ]), and acceptability (participant survey). Process: In total, 11 (85%) of 13 eligible participants enroled at baseline with 10 (91%) completing the study. Resources/management: Coaching sessions included a baseline interview-mean 39min (SD: 6.6) and telephone coaching-mean 10min (SD: 3.8) per week. Outcome measure data collection time-mean 44min (SD: 2.1). Scientific feasibility: Two participants experienced a fall during their exercise participation. Self-reported compliance was high (99%). GAS T-scores increased significantly, indicating achievement of exercise participation goals. Secondary outcomes showed trends towards improvement. The FEPP was feasible, safe and highly acceptable for use with individuals with MS and warrants a larger trial to explore effectiveness.

Goal attainment in individual and group-based neuropsychological interventions for young adults with dyslexia in a randomized controlled trial

In this study, we investigated the outcome of goal attainment in individual and group-based neuropsychological intervention for young adults with dyslexia. Participants (N = 120) were randomly assigned to individual intervention, group intervention, or wait-list control group. Attainment of goals set personally before the intervention was evaluated after the intervention using Goal Attainment Scaling. The participants were asked to report the number of concrete changes made in their study or working habits at five months (after intervention or wait-list control time), at ten months, and 15 months post-interventions. Potential predictive factors for predicting goal attainment were evaluated including background and cognitive variables, self-reported psychological variables (mood, cognitive and behavioral strategies, self-esteem, quality of life), and self-reported behavioral variables (concrete changes made, time used to work on intervention matters). Based on the results, personal goals were, on average, met especially in individual intervention. Nearly 50% of goal attainment was explained by intervention type, behavioral and cognitive strategies, concrete changes made, cognitive capacity, and used time. Concrete changes, e.g., adopting new strategies and methods in study or working habits, were reported in both interventions and more than during the wait-list control period. The changes seemed long-lasting as fifteen months post-intervention, 76.4% reported still using the learned strategies. A structured and relatively short neuropsychological intervention for dyslexia in either individual or group format can lead to attaining pre-set personal goals and positive behavioral changes that can last up to over a year post-intervention.

The feasibility of Goal Attainment Scaling in adults with Down syndrome: early results from the LIFE‐DSR‐GAS sub‐study

AbstractBackgroundAdults with Down syndrome (DS) face a markedly increased risk of Alzheimer’s disease (AD). LIFE‐DSR is a 32‐month longitudinal study of adults with DS aiming to characterize and assess change in cognition, behaviour, function, and health. Goal Attainment Scaling (GAS) is a patient‐centric outcome measure. It evaluates change in goals that are meaningful to participants/caregivers and has proven a valuable and sensitive instrument in dementia clinical trials. To assess the feasibility of using GAS in the DS population and to better understand what is most important to them, the LIFE‐DSR‐GAS sub‐study is enrolling and following participants and their caregivers over 16 months.MethodParticipants with DS and their caregivers who consented to the LIFE‐DSR‐GAS sub‐study took part in an initial goal‐setting interview with a trained GAS rater to identify meaningful goal areas related to the participant’s condition. Change in these goals was then rated at two follow‐up interviews at 3 and 16 months. To aid in goal setting, a menu of 58 common DS‐AD goal areas was developed pre‐study through interviews with expert clinicians and caregivers. A secondary objective is to evaluate this menu in both its comprehensiveness and usefulness as a resource to improve GAS implementation in DS‐AD studies.ResultTo date, 31 (of 45) participants with DS and their caregivers have enrolled across three U.S. study sites. All participants have set three goals (the recommended minimum). The mean (standard deviation) time to conduct the interviews was 43.2 (7.5) minutes. 20 participants have completed a 3‐month follow‐up interview; the mean time taken was 18.2 (10.4) minutes. Most goals (83/93) set to date were selected from the DS‐AD menu. The most frequent goals concerned aspects of daily function: Household Chores (8 goals), Personal Care and Hygiene (8), and Meal‐Time Preparation and Activities (7).ConclusionEvaluating health outcomes in people with DS is challenging due to varying levels of baseline cognition and function, further complicated by the onset of dementia. There is a critical need for sensitive and valid measures that capture this heterogeneity. Our early data suggest that GAS may offer a feasible solution.

Effects of a Goal-Oriented and Home-Based Intervention in the Chronic Phase of Traumatic Brain Injury

<h3>Research Objectives</h3> To evaluate the efficacy of a goal-oriented and home-based rehabilitation intervention in the chronic phase of traumatic brain injury (TBI). As the expected sequelae after TBI are highly heterogeneous, there is a need to tailor rehabilitation interventions to meet the specific need of each patient. <h3>Design</h3> Parallel two-group randomized controlled trial. Pre-test at baseline (T1), follow-up immediately post-intervention (4-5 months; T2) and at 12 months post inclusion (T3). <h3>Setting</h3> Ambulatory resources (physiotherapist, medical doctor, neuropsychologist) from specialized health care services delivered in-home in South-Eastern Norway. <h3>Participants</h3> 120 participants were randomized 1:1 to either treatment or control groups. Inclusion criteria were a TBI diagnosis with radiologically verified intracranial abnormalities, age 18-72 years, time since injury ≥ 2 years and experiencing ongoing TBI-related cognitive, emotional, and/or physical problems, and/or reduced physical and mental health, and/or difficulties with participation in activities with family, friends, and/or in the community. <h3>Interventions</h3> An individualized, goal-oriented intervention delivered in-home or by videoconference, with eight intervention sessions delivered over a 4-month period. Main elements of the intervention were (i) identification of target problem areas, (ii) establishment of SMART-goals and Goal Attainment Scaling, and (iii) development of an action plan with evidence-based strategies to achieve goal and mitigate difficulties. The control group received standard care in the municipalities. <h3>Main Outcome Measures</h3> Primary outcomes were health-related quality of life (HRQoL) and participation. Secondary outcomes included difficulty handling target outcome areas, post-concussive symptoms, anxiety, depression, and functional competency. <h3>Results</h3> The intervention group displayed significantly higher HRQoL, as well as lower rates of post-concussive symptoms and anxiety-related symptoms at T3.The time X group model was not significant for other outcomes. Detailed results will be presented at the conference. <h3>Conclusions</h3> This intervention shows promise as a potential treatment option for a broad range of problem profiles found in the chronic phase of TBI. The intervention might be relevant for other acquired brain injuries, but more research is needed. <h3>Author(s) Disclosures</h3> The authors have nothing to disclose.

Evaluating the Effectiveness and Safety of AbobotulinumtoxinA in Pediatric Lower Limb Spasticity (PLLS)

<h3>Research Objectives</h3> To assess the longitudinal attainment of patient-centered, function-related goal attainment scale (GAS) T-score after repeated abobotulinumtoxinA (aboBoNT-A) injections. <h3>Design</h3> Phase IV, prospective, observational study. <h3>Setting</h3> 28 US-based centers. <h3>Participants</h3> Patients aged 2–17 years with PLLS. <h3>Interventions</h3> AboBoNT-A injections over ≤30 months (≤10 cycles). <h3>Main Outcome Measures</h3> Assessment of cumulated GAS T-scores across all cycles to measure progress of therapy goals; scores of ≥50 reflect goal achievement. <h3>Results</h3> Of 210 patients, 77.6% (n=163) were previously treated with botulinum neurotoxin. Available Gross Motor Function Classification System (GMFCS) levels showed that 31.3% (n=61/195) of patients were non-ambulatory (GMFCS IV/V). Mean±SD cumulated GAS T-score was 51.1±9.3. Overall, 75.2% of patients achieved their primary goals. Across all cycles, the number of muscles injected ranged from 5.5±2.9‒7.0±3.7; the number of injection points ranged from 8.1±2.7‒9.9±5.7, with gastrocnemius injections being most common (85.7%). Injection guidance techniques were used in >70% of patients in Cycles 1‒6; electrostimulation was most frequent (>50%). In the safety population (N=242), 102 patients (42.1%) reported 392 treatment-emergent AEs, which were mostly mild to moderate. Overall, a total of 35 AEs in 15 patients (6.2%) were deemed treatment-related. <h3>Conclusions</h3> Overall, goals were achieved as or better than expected in the majority of patients. AboBoNT-A was well tolerated, with a low incidence of treatment-related AEs. These results confirm aboBoNT-A as an effective treatment option, with a positive risk-benefit profile. <h3>Author(s) Disclosures</h3> MG: Received consultancy fees/research support from Ipsen; ED: Received consultancy fees and/or research support from Allergan, Ipsen, Solstice, Merz; AT: Received consultancy fees/research support from Ipsen; AC: Received research support from Ipsen; SHE: Received consultancy fees from Biogen and Ipsen; FCG and JG: Employees of Ipsen.

The Effects of the Pragmatic Intervention Programme in Children with Autism Spectrum Disorder and Developmental Language Disorder.

Children with Autism Spectrum Disorder (ASD) and Developmental Language Disorder (DLD) frequently face pragmatic impairments which may result in learning, socialization, and mental health difficulties, therefore early intervention is crucial. In Portugal, the Pragmatic Intervention Programme (PICP) has been recently developed and validated, but its effects are unknown. This study aims to determine the effects of the PICP on preschool-age children with ASD or DLD with pragmatic impairments. A non-randomized controlled trial has been conducted. The children (n = 20) were assigned to the intervention (n = 11) or the control group (waiting list) (n = 9). Each child attended 24 PICP-based intervention sessions provided by a Speech and Language Therapist in kindergarten. The primary outcome measure was a Goal Attainment Scale (GAS) rated by parents and kindergarten teachers. Secondary outcomes include parent/teacher-reported communication skills (Escala de Avaliação de Competências Comunicativas) and an assessment of the child's general language ability (Teste de Linguagem-Avaliação da Linguagem Pré-Escolar). GAS results show that all the children in the intervention group made progress. Statistically significant differences between pre- and post-intervention assessments were found for all secondary outcomes. The main findings suggest that the PICP improves language in preschool-age children with ASD and DLD with pragmatic difficulties. Further research is needed to analyse the effects of the PICP for each neurodevelopmental disorder individually. These results are crucial and will contribute to future research and evidence-based practice.

Approaches to the Assessment of Clinical Benefit of Treatments for Conditions That Have Heterogeneous Symptoms and Impacts: Potential Applications in Rare Disease

ObjectivesEvaluating the clinical benefit of interventions for conditions with heterogeneous symptom and impact presentations is challenging. The same condition can present differently across and within individuals over time. This occurs frequently in rare diseases. The purpose of this review was to identify (1) assessment approaches used in clinical trials to address heterogeneous manifestations that could be relevant in rare disease research and (2) US Food and Drug Administration (FDA)–approved labeling claims that used these approaches. MethodsA targeted literature review was conducted examining peer-reviewed publications and FDA-approved labeling claims from January 2002 to July 2020, focusing on claims incorporating clinical outcome assessments. Approaches were then assessed for their potential application in rare diseases. ResultsA total of 6 assessment approaches were identified: composite or other multicomponent endpoints, multidomain responder index, most bothersome symptom (MBS), goal attainment scaling, sliding dichotomy, and adequate relief. A total of 59 FDA-approved labeling claims associated with these approaches were identified: composite or other multicomponent endpoints (n=49), MBS (n=9), and adequate relief (n=1). A total of 10 FDA-approved labeling claims, all using multicomponent endpoints, were identified for rare diseases. ConclusionsMulticomponent, MBS, and adequate relief have been included in FDA-approved labeling claims. Multicomponent endpoints, including composite endpoints, were the most frequent way to address heterogeneous manifestations of both common and rare diseases. MBS may be acceptable to regulators, whereas multidomain responder index is unlikely to be. The goal attainment scaling and adequate relief approaches may have potential utility in rare disease trials, assuming the theoretical and statistical challenges inherent in each approach are managed.