Meaningful Functional Improvement Research Articles

The influence of scaffolding on intrinsic motivation and autonomous adherence to a game-based, unsupervised home rehabilitation program for people with upper extremity hemiparesis due to stroke. A randomized controlled trial.

This parallel, randomized controlled trial examines intrinsic motivation, adherence and motor function improvement demonstrated by two groups of subjects that performed a twelve-week, home-based upper extremity rehabilitation program. Seventeen subjects played games presenting eight to twelve discrete levels of increasing difficulty. Sixteen subjects performed the same activities controlled by success algorithms that modify game difficulty incrementally. 33 persons 20 to 80 years of age, at least six months post stroke with moderate to mild hemiparesis were randomized using a random number generator into the two groups. They were tested using the Action Research Arm Test, Upper Extremity Fugl Meyer Assessment, Stroke Impact Scale and Intrinsic Motivation Inventory pre and post training. Adherence was measured using timestamps generated by the system. Subjects had the Home Virtual Rehabilitation System [1]systems placed in their homes and were taught to perform rehabilitation games using it. Subjects were instructed to train twenty minutes per day but were allowed to train as much as they chose. Subjects trained for twelve weeks without appointments and received intermittent support from study staff. Group outcomes were compared using ANOVA. Correlations between subject demographics and adherence, as well as motor outcome, were evaluated using Pearson Correlation Coefficients. Classification and Regression Tree (CART) models were generated to predict responders using demographics and baseline measures. There were 5 dropouts and no adverse events. The main effect of time was statistically significant for four of the five clinical outcome measures. There were no significant training group by time interactions. Measures of adherence did not differ between groups. 21 subjects from both groups, demonstrated clinically important improvements in UEFMA score of at least 4.25 points. Subjects with pre training UEFMA scores below 53.5 averaged a seven-point UEFMA increase. IMI scores were stable pre to post training. Scaffolding did not have a meaningful impact on adherence or motor function improvement. A sparsely supervised program of game-based treatment in the home was sufficient to elicit meaningful improvements in motor function and activities of daily living. Common factors considered barriers to the utilization of telerehabilitation did not impact adherence or motor outcome. Clinical Trials.gov - NCT03985761, Registered June 14, 2019.

Nusinersen Treatment for Spinal Muscular Atrophy: Retrospective Multicenter Study of Pediatric and Adult Patients in Kuwait.

Spinal muscular atrophy is a neuromuscular genetic condition associated with progressive muscle weakness and atrophy. Nusinersen is an antisense oligonucleotide therapy approved for the treatment of 5q spinal muscular atrophy in pediatric and adult patients. The objective of this clinical case series is to describe the efficacy and safety of nusinersen in treating spinal muscular atrophy in 20 pediatric and 18 adult patients across six treatment centers in Kuwait. Functional motor assessments (Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders, Hammersmith Functional Motor Scale Expanded, and Revised Upper Limb Module) were used to assess changes in motor function following nusinersen treatment. The safety assessment involved clinical monitoring of adverse events. The results demonstrate clinically meaningful or considerable improvement in motor performance for nearly all patients, lasting over 4 years in some cases. A total of 70% of patients in the pediatric cohort and 72% of patients in the adult cohort achieved a clinically meaningful improvement in motor function following nusinersen treatment. Additionally, nusinersen was well-tolerated in both cohorts. These findings add to the growing body of evidence relating to the clinical efficacy and safety of nusinersen.

The effects of the addition of Mulligan mobilization with movement to exercise on elbow pain and function associated with lateral elbow tendinopathy

BackgroundMulligan mobilization with movement (MWM) and eccentric exercise (EE) have been shown to be effective for the treatment of chronic lateral elbow tendinopathy (LET). However, the addition of MWM to EE effects on LET remained unknown. The objective of the study was to determine the effects of adding of Mulligan mobilization with movement to eccentric exercise for the treatment of LET. MethodsIn this quasi-experimental study, thirty participants with unilateral LET were assigned into two groups: 15 in MWM (received MWM + EE) (7 M and 8 F, mean age 36.8 ± 8.3 years) and 15 in EE (9 M and 6 F, mean age 46 ± 8.1 years). Both groups received education about painful activities and eccentric exercise, while the MWM group received MWM with the same eccentric exercise as the EE group. The visual analog scale (VAS) and the patient-rated tennis elbow evaluation (PRTEE) were used to assess pain and upper limb function, respectively, at baseline and at the end of 4 weeks. The data was analyzed using an independent t-test and a paired t-test. ResultsThere was no significant difference (p > 0.05) between groups at baseline. At the end of 4 weeks, significant and clinically meaningful improvements in pain and function were observed for both groups (p < 0.001); however, MWM group showed much better results in VAS (p < 0.001, d = 1.3) and PRTEE (p < 0.001, d = 5.2) ConclusionThis study showed that adding MWM to eccentric exercise resulted in enhanced pain reduction and improved function of the upper limb.

Adaptive Functional Electrical Stimulation Delivers Stimulation Amplitudes Based on Real-Time Gait Biomechanics.

Functional electrical stimulation (FES) is often used in poststroke gait rehabilitation to decrease foot drop and increase forward propulsion. However, not all stroke survivors experience clinically meaningful improvements in gait function following training with FES. The purpose of this work was to develop and validate a novel adaptive FES (AFES) system to improve dorsiflexor (DF) and plantarflexor (PF) stimulation timing and iteratively adjust the stimulation amplitude at each stride based on measured gait biomechanics. Stimulation timing was determined by a series of bilateral footswitches. Stimulation amplitude was calculated based on measured dorsiflexion angle and peak propulsive force, where increased foot drop and decreased paretic propulsion resulted in increased stimulation amplitudes. Ten individuals with chronic poststroke hemiparesis walked on an adaptive treadmill with adaptive FES for three 2-min trials. Stimulation was delivered at the correct time to the dorsiflexor muscles during 95% of strides while stimulation was delivered to the plantarflexor muscles at the correct time during 84% of strides. Stimulation amplitudes were correctly calculated and delivered for all except two strides out of nearly 3000. The adaptive FES system responds to real-time gait biomechanics as intended, and further individualization to subject-specific impairments and rehabilitation goals may lead to improved rehabilitation outcomes.

EXERCISE PHYSIOLOGIST-LED PAIN SCIENCE COACHING VIA TELEHEALTH IMPROVED PAIN AND FUNCTION IN LIFE INSURANCE CLAIMANTS: A PRAGMATIC COHORT STUDY

INTRODUCTION &amp; AIMS Personal injury and illness compensation claimants are more likely to experience persistent pain and poorer clinical/vocational outcomes than non-compensated clients. Evidence supports the effectiveness of pain science coaching to help people understand and re-train their pain system. This consequently reduces pain intensity, improves function and quality of life. However, the effectiveness of telehealth-delivered, exercise physiologist-led pain science coaching is yet to be evaluated. This study aimed to determine the effectiveness of an accredited exercise physiologist-led pain science coaching intervention via telehealth in Australian life insurance claimants. METHODS This pragmatic cohort study included 1,275 claimants receiving a pain science coaching intervention supported by life insurance compensation. Ethics was approved via Deakin University Human Research Ethics Committee (2023-347). Outcome measures were pain intensity (11 item numerical pain rating scale, range: 0-10 points) and function (two 11-item patient-specific functional scale, range: 0-20 points). Linear mixed models determined within-group change over time. RESULTS Clients (female: 75%, male: 25%) had a mean (SD) age of 50 (10) years (range: 20-69 years) and claim duration of 2 (2) years (range: 0-20 years). Mean (SD) intervention duration was 10 (6) weeks (range: 3-41 weeks), financial cost was A$1,149 (A$202; range: A$510-A$2,040) and included 5 (1) hours (range: 2-11 hours) of pain science coaching. Following the intervention, pain intensity decreased 25% (estimated marginal mean change [95%CI]: -1.49 [-1.59, -1.40] points, P&amp;lt;0.001) and function increased 76% (4.41 [4.21, 4.62] points, P&amp;lt;0.001). Changes surpassed established clinically meaningful effect thresholds for pain intensity (1.17 points) and function (2.6 points). Client Net Promotor Score was +60 (Australian Healthcare Index benchmark ≥ +30) and 91% were satisfied with the intervention. CONCLUSION An exercise physiologist-led pain science coaching intervention resulted in clinically meaningful improvements in pain intensity and function in compensation claimants. Clients reported high satisfaction rates.

Time to Achieve the Minimal Clinically Important Difference in Primary Total Hip Arthroplasty: Comparison of Anterior and Posterior Surgical Approaches

BackgroundControversy remains over outcomes between total hip arthroplasty approaches. This study aimed to compare the time to achieve the minimal clinically important difference (MCID) for the Hip Disability and Osteoarthritis Outcome Score-Physical Function Short Form (HOOS-PS) and the Patient-Reported Outcomes Measurement Information System (PROMIS) Global-Physical for patients who underwent anterior and posterior surgical approaches in primary total hip arthroplasty. MethodsPatients from 2018 to 2021 with preoperative and postoperative HOOS-PS or PROMIS Global-Physical questionnaires were grouped by approach. Demographic and MCID achievement rates were compared, and survival curves with and without interval-censoring were used to assess the time to achieve the MCID by approach. Log-rank and weighted log-rank tests were used to compare groups, and Weibull regression analyses were performed to assess potential covariates. ResultsA total of 2,725 patients (1,054 anterior and 1,671 posterior) were analyzed. There were no significant differences in median MCID achievement times for either the HOOS-PS (anterior: 5.9 months, 95% confidence interval [CI]: 4.6 to 6.4; posterior: 4.4 months, 95% CI: 4.1 to 5.1, P = .65) or the PROMIS Global-Physical (anterior: 4.2 months, 95% CI: 3.5 to 5.3; posterior: 3.5 months, 95% CI: 3.4 to 3.8, P = .08) between approaches. Interval-censoring revealed earlier times of achieving the MCID for both the HOOS-PS (anterior: 1.509 to 1.511 months; posterior: 1.7 to 2.3 months, P = .87) and the PROMIS Global-Physical (anterior: 3.0 to 3.1 weeks; posterior: 2.7 to 3.3 weeks, P = .18) for both surgical approaches. ConclusionsThe time to achieve the MCID did not differ by surgical approach. Most patients will achieve clinically meaningful improvements in physical function much earlier than previously believed. Level of EvidenceLevel III, Retrospective Comparative Study.

Accelerated versus Standard Rehabilitation after Meniscus Allograft Transplantation in the Knee.

Meniscus allograft transplantation (MAT) is a proven treatment option for patients with symptomatic irreparable meniscus deficiency. When patients are adherent to prescribed post-operative restriction and rehabilitation protocols, outcomes after MAT are considered good to excellent. However, nonadherence to standard protocols is common and can be associated with undesirable outcomes and patient dissatisfaction. Based on demonstrated safety for early weightbearing following MAT in conjunction with significant advances in graft preservation and surgical techniques, our joint preservation center implemented a shift in practice towards accelerated weightbearing following MAT and designed this study to test the hypothesis that accelerated rehabilitation would be associated with superior adherence, patient-reported outcomes, and patient satisfaction, without diminishing patient safety, when compared to standard rehabilitation. Patients were included for analyses when they had a undergone fresh or fresh-frozen MAT using a double bone plug technique for treatment of medial or lateral meniscus deficiency and at least 1-year treatment outcomes recorded. The results of this study revealed that patients who were prescribed accelerated rehabilitation after MAT were significantly more adherent than patients who were prescribed standard rehabilitation and reported statistically significant and clinically meaningful improvements in knee pain and function for at least 1-year following MAT, whereas those in the standard cohort did not. While not statistically different, treatment failure rate was lower in the accelerated rehabilitation cohort when compared to the standard rehabilitation cohort (11% vs 29%). Importantly, initial outcomes for revision MAT were associated with short-term success in all of the patients who opted for this option in the study population. These data suggest that accelerated weightbearing after meniscus allograft transplantation is safe, promotes patient adherence, and is associated with statistically significant and clinically meaningful improvements in patient-reported knee pain and function at early and mid-term follow-up.

Improvements in Patient-Reported Outcomes Through Six Months of Guselkumab Treatment in Patients With Active Psoriatic Arthritis: Real-World Data From the CorEvitas Psoriatic Arthritis/Spondyloarthritis Registry.

Evaluate patient-reported outcomes after 6 months of on-label guselkumab use in patients with rheumatologist-diagnosed active psoriatic arthritis (PsA) enrolled in the CorEvitas PsA/Spondyloarthritis Registry. This analysis includes registry participants who initiated and persisted with on-label guselkumab (after US Food and Drug Administration approval for PsA; 100 mg at weeks 0, 4, and every 8 weeks) at their 6-month follow-up visit (On-Label Persisters). Among patients not meeting response criteria at baseline, responses at 6 months were determined for patient-reported outcomes, including patient-reported pain (0-100 mm visual analog scale), patient global assessment of arthritis + psoriasis (PtGA; 0-100 visual analog scale), and Health Assessment Questionnaire-Disability Index (HAQ-DI; 0-3). Unadjusted, nominal P values were calculated via single-proportion, one-sided test (H0 = 0%; α=0.05). Of 90 On-Label Persisters, most had treatment-resistant PsA (92.2% and 73.3% previously received ≥1 and ≥2 biologic/targeted synthetic disease-modifying antirheumatic drugs, respectively), with mean (SD) baseline patient-reported pain, PtGA, and HAQ-DI scores of 57.0 (24.6), 50.3 (24.4), and 0.9(0.6), respectively. Among those with patient-reported pain and PtGA scores of at least 15 at baseline, 40.2% (33/82) and 46.8% (36/77), respectively, achieved at least 15-mm reductions at 6 months; among those with HAQ-DI scoresof at least 0.35 and more than 0.5 at baseline, respectively, 30.4% (21/69) achieved improvements of at least 0.35 and 10.3% (6/58) achieved scores of 0.5 or lower at 6 months (all nominal P < 0.001). Pain and physical function are important contributors to health-related quality of life. In this real-world population of patients with treatment-resistant PsA and 6 months of persistent guselkumab treatment, clinically meaningful improvements in pain and physical function were achieved by approximately 40% and 30% of patients, respectively.

Supplementary Respiratory Therapy Improves Pulmonary Function in Pediatric Patients with Cerebral Palsy: A Systematic Review and Meta-Analysis

Background: Despite medical advances, individuals with cerebral palsy (CP) face significant respiratory challenges, leading to heightened hospitalization rates and early mortality among this population. We hypothesize that integrating supplementary respiratory therapy into standard rehabilitation will result in significant improvements in pulmonary function, enhanced respiratory muscle strength, and an overall increase in the quality of life among pediatric patients with CP. Methods: A systematic search of literature across five databases was conducted, and random-effects meta-analyses were performed to assess the impact of supplementary respiratory therapy on (a) pulmonary function: forced vital capacity (FVC), forced expiratory volume in 1 s (FEV1), FVC/FEV1 ratio, peak expiratory flow (PEF), and (b) respiratory muscle strength: maximal inspiratory and expiratory pressure (MIP, MEP), and (c) quality of life. Certainty of evidence was determined by the GRADE assessment. Results: Analysis of data from 11 eligible randomized controlled trials revealed clinically meaningful changes in pulmonary function. We found a relevant mean difference (MD) in absolute PEF of 0.50 L/s (95% confidence interval (CI): 0.19; 0.82 p = 0.0107). The certainty of the evidence ranged from moderate to high. Conclusions: This study presents current evidence on the impact of various supplementary respiratory therapies for CP patients classified under gross motor function classification level I–IV, demonstrating clinically meaningful improvements in pulmonary function and respiratory muscle strength. These improvements suggest the potential for an enhanced quality of life. Our findings hold the promise of serving as a foundational reference for potential revisions to conventional rehabilitation care, incorporating supplementary respiratory therapy.

Frontloading Home Physical Therapy Visits for Patients With Heart Failure: A Multi-center Randomized Controlled Trial.

Frontloading home care visits has been found to be effective in the nursing profession but has not been investigated in physical therapy (PT) practice. This study aimed to examine the impact of frontloading home PT visits on function in persons with heart failure (HF). This was a prospective multi-center randomized controlled trial with blinded raters. A total of 82 ambulatory patients with a primary diagnosis of HF discharged from an acute care facility to home care participated in the study. Subjects were randomly allocated to an experimental frontloaded group (FLG) or control group (CG) for 4 weeks. FLG visit frequencies were five sessions per week for 2 weeks, and three sessions per week for 2 weeks. The CG received two sessions per week for 4 weeks. Functional measures including the 2-minute step test (2MST), 2-minute walk test (2MWT), gait speed (GS), Timed Up and Go (TUG), and 30-second chair rise test (30-CRT) were collected at the onset of care, at the end of 2 weeks and 4 weeks. The groups were statistically similar at baseline for all measures. All subjects significantly improved scores in all functional measures over time, within-subject main effect (p < .01). Significant between-subject effects were noted for 30-CRT (p = .04). Interaction effects were noted for GS (p = .03) and TUG test (p = .02). This is the first study to report meaningful improvements in function in individuals with HF. Significant treatment effect differences between the FLG and CG were found for GS, TUG, and 30-CRT. Future studies should examine the use of a standardized intervention to validate the effectiveness of frontloading home visits on quality of life and readmission rates.

Frontloading Home Physical Therapy Visits for Patients With Heart Failure: A Multi-center Randomized Controlled Trial.

Frontloading home care visits has been found to be effective in the nursing profession but has not been investigated in physical therapy (PT) practice. This study aimed to examine the impact of frontloading home PT visits on function in persons with heart failure (HF). This was a prospective multi-center randomized controlled trial with blinded raters. A total of 82 ambulatory patients with a primary diagnosis of HF discharged from an acute care facility to home care participated in the study. Subjects were randomly allocated to an experimental frontloaded group (FLG) or control group (CG) for 4 weeks. FLG visit frequencies were five sessions per week for 2 weeks, and three sessions per week for 2 weeks. The CG received two sessions per week for 4 weeks. Functional measures including the 2-minute step test (2MST), 2-minute walk test (2MWT), gait speed (GS), Timed Up and Go (TUG), and 30-second chair rise test (30-CRT) were collected at the onset of care, at the end of 2 weeks and 4 weeks. The groups were statistically similar at baseline for all measures. All subjects significantly improved scores in all functional measures over time, within-subject main effect (p < .01). Significant between-subject effects were noted for 30-CRT (p = .04). Interaction effects were noted for GS (p = .03) and TUG test (p = .02). This is the first study to report meaningful improvements in function in individuals with HF. Significant treatment effect differences between the FLG and CG were found for GS, TUG, and 30-CRT. Future studies should examine the use of a standardized intervention to validate the effectiveness of frontloading home visits on quality of life and readmission rates.

Clinical Delivery of Overground Exoskeleton Gait Training in Persons With Spinal Cord Injury Across the Continuum of Care: A Retrospective Analysis.

After spinal cord injury (SCI), inpatient rehabilitation begins and continues through outpatient therapy. Overground exoskeleton gait training (OEGT) has been shown to be feasible in both settings, yet its use as an intervention across the continuum has not yet been reported. This study describes OEGT for patients with SCI across the continuum and its effects on clinical outcomes. Medical records of patients with SCI who completed at least one OEGT session during inpatient and outpatient rehabilitation from 2018 to 2021 were retrospectively reviewed. Demographic data, Walking Index for Spinal Cord Injury-II (WISCI-II) scores, and OEGT session details (frequency, "walk" time, "up" time, and step count) were extracted. Eighteen patients [male (83%), White (61%), aged 37.4 ± 15 years, with tetraplegia (50%), American Spinal Injury Association Impairment Scale A (28%), B (22%), C (39%), D (11%)] completed OEGT sessions (motor complete, 18.2 ± 10.3; motor incomplete, 16.7 ± 7.7) over approximately 18 weeks (motor complete, 15.1 ± 6.4; motor incomplete, 19.0 ± 8.2). Patients demonstrated improved OEGT session tolerance on device metrics including "walk" time (motor complete, 7:51 ± 4:42 to 24:50 ± 9:35 minutes; motor incomplete, 12:16 ± 6:01 to 20:01 ± 08:05 minutes), "up" time (motor complete, 16:03 ± 7:41 to 29:49 ± 12:44 minutes; motor incomplete, 16:38 ± 4:51 to 23:06 ± 08:50 minutes), and step count (motor complete, 340 ± 295.9 to 840.2 ± 379.4; motor incomplete, 372.3 ± 225.2 to 713.2 ± 272). Across therapy settings, patients with motor complete SCI experienced improvement in WISCI-II scores from 0 ± 0 at inpatient admission to 3 ± 4.6 by outpatient discharge, whereas the motor incomplete group demonstrated a change of 0.2 ± 0.4 to 9.0 ± 6.4. Patients completed OEGT across the therapy continuum. Patients with motor incomplete SCI experienced clinically meaningful improvements in walking function.

Effectiveness of an eHealth-Delivered Program to Empower People With Musculoskeletal Pain in Rural Australia: A Randomized Controlled Trial.

Our objective was to evaluate the effectiveness of a three-month physiotherapist-delivered eHealth physical activity program compared with usual care to improve function in adults with low back pain or knee osteoarthritis in rural Australia. This was a parallel, two-group, pragmatic, superiority, randomized controlled trial involving three- and six-month posttreatment follow-ups. There was a total of 156 adults with chronic nonspecific low back pain (n=97) or knee osteoarthritis (n=59) from rural Australia. The intervention involved an eHealth physical activity and an exercise program that included five to eight teleconsultations with a physiotherapist (primary time point three months) or usual care (eg, general practitioner, physiotherapy, and pain medication). The primary outcome was the Patient-Specific Functional Scale (0-30), with a three-point difference between groups being considered the minimum clinically important difference. Participants receiving the eHealth intervention (n=78) reported significantly greater and clinically worthwhile improvements in function (mean between-group difference 3.6; 95% confidence interval [CI] 1.3-5.9) compared to participants receiving usual care (n=78). Small but statistically significantly greater improvements in disability (7.2 of 100; 95% CI 2.1-12.3) and quality of life (4.5 of 100; 95% CI 0.0-9.0) also favored the eHealth group. No clinical or statistical differences between groups were found for the secondary outcomes of pain, coping skills, and physical activity levels. A physiotherapist-delivered eHealth intervention is effective and provides clinically meaningful improvements in function compared to usual care for people with musculoskeletal pain in rural communities. These findings highlight the potential for eHealth-based programs to improve access to evidence-based exercise interventions for people with musculoskeletal pain in rural communities.

Hip-focused physical therapy versus spine-focused physical therapy for older adults with chronic low back pain at risk for mobility decline (MASH): a multicentre, single-masked, randomised controlled trial

Previously, we identified a population of older adults with chronic low back pain, hip pain, and hip muscle weakness who had worse 12-month low back pain and functional outcomes than age-matched adults with only low back pain, indicating an increased risk for future mobility decline. We sought to determine whether tailored, hip-focused physical therapy reduced pain and functional limitations in this high-risk population compared with non-tailored, spine-focused physical therapy. We did a multicentre, single-masked, randomised controlled trial at three research-based sites in the USA. We recruited older adults (aged 60-85 years) with hip pain and weakness who reported moderate low back pain intensity at least half the days in the last 6 months. Patients were randomly assigned to hip-focused physical therapy or spine-focused physical therapy using permuted blocks with random block size, stratified by site and sex (ie, male or female). The primary outcomes were self-reported disability using the Quebec Back Pain Disability Scale (QBPDS) and performance-based 10-Meter Walk Test (10MWT) at 8 weeks. All analyses were done in the intention-to-treat population. Adverse events were collected by study staff via a possible adverse event reporting form and then adjudicated by site investigators. This trial was registered with ClinicalTrials.gov, NCT04009837. Between Nov 1, 2019, and April 30, 2022, 184 participants were randomly assigned to receive hip-focused (n=91) or spine-focused physical therapy (n=93) interventions. The mean age was 70·7 (SD 6·2) years. 121 (66%) of 184 participants were women, 63 (34%) were men, and 149 (81%) were White. At 8 weeks, the mean between-group difference on the QBPDS was 4·0 (95% CI 0·5 to 7·5), favouring hip-focused physical therapy. Both groups had similar, clinically meaningful gait speed improvements (10MWT) at 8 weeks (mean difference 0·004 m/s [95% CI -0·044 to 0·052]). No serious adverse events were related to study participation. Tailored hip-focused physical therapy demonstrated greater improvements in low back pain-related disability at 8 weeks. However, both hip-focused physical therapy and spine-focused physical therapy produced clinically meaningful improvements in disability and function for this high-risk population at 6 months. These findings warrant further investigation before clinical implementation. US National Institute on Aging of the National Institutes of Health.

Safety and Efficacy of Nusinersen and Risdiplam for Spinal Muscular Atrophy: A Systematic Review and Meta-Analysis of Randomized Controlled Trials.

We performed a systematic review and meta-analysis of the efficacy and safety of nusinersen and risdiplam in the treatment of spinal muscular disease (SMA). We screened the literature published in Pubmed, Web of Science, Embase, and Cochrane before July 2023 to conduct randomized controlled trials to test the treatment of SMA patients with nusinersen and risdiplam. The data were analyzed using Review Manager 5.4 software and Stata version 15.0 software. A total of six randomized controlled trials were included, involving 728 SMA patients, to synthesize evidence. It is reported that nusinersen treatment was beneficial for increasing the score of the Hammersmith Functional Motor Scale-Expanded (HFMSE) (WMD: 4.90; 95% CI: 3.17, 6.63; p < 0.00001), Revised Upper Limb Module (RULM) (WMD: 3.70; 95% CI: 3.30, 4.10; p < 0.00001), and Hammersmith Infant Neurological Evaluation Section 2 (HINE-2) (WMD: 5.21; 95% CI: 4.83, 5.60; p < 0.00001). In addition, the risdiplam treatment group also showed statistically significant improvements in the HFMSE score (WMD:0.87; 95% CI: 0.05, 1.68; p = 0.04), the 32-item Motor Function Measure (MFM32) (WMD:1.48; 95% CI: 0.58, 2.38; p = 0.001), and (WMD: 1.29; 95% CI: 0.57, 2.01; p = 0.0005). Nusinersen and risdiplam did not cause a statistically significant increase in the RULM score for adverse events (OR: 0.93; 95% CI: 0.51, 1.7; p = 0.82) and for severe adverse events (OR: 0.77; 95% CI: 0.47, 1.27; p = 0.31). Our analysis found that nusinersen and risdiplam treatment showed clinically meaningful improvement in motor function and a similar incidence rate of adverse events compared with the placebo. Further research should be carried out to provide a direct comparison between the two drugs in terms of safety and efficacy.

Achieving Functional Goals During a Skilled Nursing Facility Stay: A National Study of Medicare Beneficiaries.

The aims of the study are to describe the frequency that functional goals are documented on the Minimum Data Set and to identify resident characteristics associated with meeting or exceeding discharge goals. We selected Medicare fee-for-service beneficiaries admitted to a skilled nursing facility within 3 days of hospital discharge from October 1, 2018, to December 31, 2019 ( N = 1,228,913). The admission Minimum Data Set was used to describe the discharge goal scores for seven self-care and 16 mobility items. We used the eight self-care and mobility items originally included in a publicly reported quality measure to calculate total scores for discharge goals, admission performance, and discharge performance ( n = 371,801). For all self-care items, more than 70% of residents had a goal score of 1-6 points documented on the admission Minimum Data Set. Chair/bed-to-chair transfer had the highest percentage of residents with a score of 1-6 points (77.1%) and walking up/down 12 steps had the lowest (23.2%). Approximately 44% of residents had a discharge performance score that met or exceeded their goal score. Older age, urinary incontinence, and cognitive impairment had the lowest odds of meeting or exceeding discharge goals. Assessing a resident's functional goals is important to providing patient-centered care. This information may help skilled nursing facilities determine whether a resident has made meaningful functional improvements.

Feasibility and outcomes of supplemental gait training by robotic and conventional means in acute stroke rehabilitation

IntroductionPracticality of implementation and dosing of supplemental gait training in an acute stroke inpatient rehabilitation setting are not well studied but can have positive impact on outcomes.ObjectivesTo determine the feasibility of early, intense supplemental gait training in inpatient stroke rehabilitation, compare functional outcomes and the specific mode of delivery.Design and settingAssessor blinded, randomized controlled trial in a tertiary Inpatient Rehabilitation Facility.ParticipantsThirty acute post-stroke patients with unilateral hemiparesis (≥ 18 years of age with a lower limb MAS ≤ 3).InterventionLokomat® or conventional gait training (CGT) in addition to standard mandated therapy time.Main outcome measuresNumber of therapy sessions; adverse events; functional independence measure (FIM motor); functional ambulation category (FAC); passive range of motion (PROM); modified Ashworth scale (MAS); 5 times sit-to-stand (5x-STS); 10-m walk test (10MWT); 2-min walk test (2MWT) were assessed before (pre) and after training (post).ResultsThe desired supplemental therapy was implemented during normal care delivery hours and the patients generally tolerated the sessions well. Both groups improved markedly on several measures; the CGT group obtained nearly 45% more supplemental sessions (12.8) than the Lokomat® group (8.9). Both groups showed greater FIM improvement scores (discharge – admission) than those from a reference group receiving no supplemental therapy. An overarching statistical comparison between methods was skewed towards a differential benefit (but not significant) in the Lokomat® group with medium effect sizes. By observation, the robotic group completed a greater number of steps, on average. These results provide some evidence for Lokomat® being a more efficient tool for gait retraining by providing a more optimal therapy “dose”.ConclusionsWith careful planning, supplemental therapy was possible with minimal intrusion to schedules and was well tolerated. Participants showed meaningful functional improvement with relatively little supplemental therapy over a relatively short time in study.

The Role of Surgery in the Management of Radiation-Induced Brachial Plexopathy.

Radiation-induced brachial plexopathy (RIBP) is a rare long-term complication of radiation therapy often causing pain, motor deficit, and overall quality of life reduction for affected patients. While a standard treatment for RIBP is yet to be established, management consists mostly of symptom management through the use of medications and physical therapy. There is a lack of evidence regarding the efficacy of surgical treatment. Omentoplasties and other vascularized flaps are commonly discussed options for eliminating neuropathic pain associated with RIBP. However, these approaches show no meaningful improvement in motor function. While limited, current literature suggests that nerve transfers may be an option for relief of neuropathic pain as well as restoration of motor function. This review of literature explores the options available to those affected by RIBP with a focus on the role of surgery.

Dietary Interventions in Heart Failure with Preserved Ejection Fraction (HFpEF): a systematic scoping review and meta-analysis

Abstract Funding Acknowledgements Type of funding sources: Public Institution(s). Main funding source(s): Evelyn Trust. Background Pharmacotherapies have improved outcomes for patients with Heart Failure with reduced Ejection Fraction (HFrEF), but less so for patients with HFpEF. Limited treatments results in substantial burden at personal, clinical and economic levels. Diet has been implicated in the pathogenesis and prognosis of HFpEF, as such, dietary manipulation has the potential to be a useful management strategy. Purpose The aim of the scoping review was to establish which nutritional interventions have so far been tested in patients with HFpEF and establish how the dietary manipulation was achieved. The aim of the meta-analysis was to quantify the effects of interventions tested to determine whether there is evidence to support specific dietary recommendations in patients with HFpEF. Methods Key word searches were performed in five bibliographic databases to identify randomised or controlled studies of dietary interventions conducted in HFpEF or mixed heart failure (HF) samples. Study characteristics were summarised according to PICO (population, intervention, comparator, outcome) categories and intervention complexity was assessed. Studies included in the meta-analysis were appraised for bias and the quality of the body of evidence was assessed via the GRADE (Grading of Recommendations, Assessment, Development and Evaluations) framework. Results Twenty-five different interventions reported across 31 papers were identified. Only ten (40%) were conducted exclusively in HFpEF subjects. Studies employing highly tailored prescribed methods were most common (n=12, 48%). Dietary supplementation (n=10, 40%) and dietary pattern manipulations (n=3, 12%) were less represented. Seventeen studies were considered for meta-analysis; all but one were judged to be at high risk of bias. There was moderate quality evidence that caloric restriction led to clinically meaningful improvements in blood pressure and body weight. There was moderate quality evidence carbohydrate restriction resulted in meaningful reductions in blood pressure. There was very low quality evidence protein supplementation improved blood pressure and body weight and moderate quality evidence for clinically meaningful improvements in function. Conclusions Very few dietary interventions have been tested in HFpEF specifically. Most interventions tested were complex at the patient level, which may not be suitable for the older, multi-morbid patients who typify HFpEF. Results of meta-analyses performed were not of sufficient quality to make generalised conclusions. Some interventions delivered clinically meaningful changes that may be important to patients, therefore, clinicians could draw on these findings to inform personalised care plans and treatment goals.

The Impact of Implant Fixation Type on Reoperation Rates Following Endoprosthetic Reconstruction Surgery.

Prospective evidence supporting the use of cemented or uncemented implants in endoprosthetic reconstruction is lacking. The present study aimed to determine the effect of cemented fixation compared with uncemented fixation on the rate of all-cause reoperation at 1 year postoperatively. This is a secondary analysis of the Prophylactic Antibiotic Regimens In Tumor Surgery (PARITY) trial. A total of 503 patients who underwent surgical excision and endoprosthetic reconstruction of a lower-extremity bone tumor were included in this analysis. A multivariate Cox proportional hazards model was utilized to assess the independent relationship between fixation group and implant survivorship, with all-cause reoperation as the end point. There were 388 cemented implants and 115 uncemented implants. Participants had a mean age of 42.7 years (standard deviation, 22.0 years), and 59% were male. Overall, 131 reoperations were identified over the 1-year follow-up period. There were no significant differences found in all-cause reoperation (hazard ratio [HR], 1.05; 95% confidence interval [CI], 0.70 to 1.57; p = 0.761), septic reoperation, or aseptic reoperation between cemented and uncemented fixation at 1 year postoperatively. The Cox regression analysis demonstrated that total operative time (HR per hour, 1.10; 95% CI, 1.02 to 1.20; p = 0.019) was an independent predictor of reoperation. The risk of reoperation was lower in patients with benign aggressive disease (HR, 0.11; 95% CI, 0.02 to 0.80; p = 0.029) or metastatic bone disease (HR, 0.30; 95% CI, 0.11 to 0.85; p = 0.023). Patients who underwent cemented fixation showed clinically meaningful functional improvement at 1 year postoperatively. Cemented compared with uncemented fixation has no effect on 1-year all-cause reoperation rates in endoprosthetic reconstruction surgery. Further research is required to investigate the long-term survival of cemented versus uncemented implants. Prognostic Level IV. See Instructions for Authors for a complete description of levels of evidence.