Functional Outcome Measures Research Articles

The Predictive Validity of the Full Outline of UnResponsiveness Score Compared to the Glasgow Coma Scale in the Intensive Care Unit: A Systematic Review.

The Full Outline of UnResponsiveness (FOUR) score was developed to overcome the limitations of the Glasgow Coma Scale (GCS) when assessing individuals with impaired consciousness. We sought to review the evidence regarding the predictive validity of the GCS and FOUR score in intensive care unit (ICU) settings. This review was prospectively registered in PROSPERO (CRD42023420528). Systematic searches of CINAHL, MEDLINE, and Embase were undertaken. Prospective observational studies were included if both GCS and FOUR score were assessed in adults during ICU admission and if mortality and/or validated functional outcome measure scores were collected. Studies were excluded if they exclusively investigated patients with traumatic brain injury. Screening, data extraction, and quality assessment using the Quality in Prognosis Studies tool were conducted by two reviewers. Twenty studies of poor to moderate quality were included. Many studies only included patients with neurological illness and excluded sedated patients, despite high proportions of intubated patients. The FOUR score achieved higher area under the receiver operating characteristic curve values for mortality prediction compared with the GCS, and the FOUR score achieved significantly higher area under the receiver operating characteristic curve values for predictions of ICU mortality. Both coma scales showed similar accuracy in predicting "unfavorable" functional outcome. The FOUR score appeared to be more responsive than the GCS in the ICU, as most patients with a GCS score of 3 obtained FOUR scores between 1 and 8 due to preserved brainstem function. The FOUR score may be superior to the GCS for predicting mortality in ICU settings. Further adequately powered studies with clear, reliable methods for assessment of index and outcome scores are required to clarify the predictive performance of both coma scales in ICUs. Inclusion of sedated patients may improve generalizability of findings in general ICU populations.

Iron Chelators Augment Large Osteochondral Allograft Osseointegration in a Preclinical Canine Model.

Osteochondral allograft transplantation (OCAT) can be a successful joint restoration treatment option for large post-traumatic articular defects but is still associated with significant revision and failure rates. Despite recent advances that have improved OCAT success, insufficient osteochondral allograft (OCA) osseointegration remains a major cause of failure. Deferoxamine (DFO) is an effective angiogenic and osteo-anabolic iron chelator that consistently promotes bone neovascularization and regeneration. This study was designed to investigate local delivery of DFO for augmenting OCA osseointegration using a preclinical canine model for OCAT in the knee and hip as commonly affected joints. On Institutional Animal Care and Use Committee (IACUC) approval, 12 purpose-bred dogs underwent OCAT of the femoral head or femoral condyles with DFO or DFO-free (controls) microspheres in recipient sites. OCA revascularization, cellular repopulation, and integration were evaluated based on functional, diagnostic imaging, microcomputed tomography, histology, and immunohistochemistry outcome measures. Local delivery of DFO into OCAT recipient sites was associated with maintained or improved joint function, superior radiographic appearance, significantly greater trabecular thickness, higher bone volume, and new bone ingrowth compared with DFO-free controls. OCA osseointegration is dependent on cellular repopulation and neovascularization, resulting in new bone ingrowth through creeping substitution, and insufficient osseointegration with resorption and subsidence of the OCA remains a major cause of failure after transplantation. The results of this study suggest that local delivery of DFO using a controlled microsphere release system may reduce resorption and improve revascularization and cellular repopulation to increase new bone ingrowth, potentially expediting OCA osseointegration after transplantation.

Fidelity, acceptability, and feasibility of the revised functional autonomy measurement system for hospitalised people: An implementation study.

The revised functional autonomy measurement system (SMAF-R) is an outcome measure of activity performance and resource needs. This study aimed to measure the implementability of the SMAF-R as a routine outcome measure in acute and subacute inpatients. This was a mixed-methods implementation study. Occupational therapists were asked to complete the SMAF-R in hospitalised people with an anticipated length of stay greater than 48 hours over a 12-month period (n= 13,348). A baseline audit of SMAF-R completion rates was conducted, and behaviour change interventions were delivered over 12 months. The Implementability of Healthcare Interventions conceptual framework was used to evaluate fidelity (completion rates), acceptability (staff survey with quantitative and qualitative responses), and feasibility (change in SMAF-R scores from admission to discharge). Quantitative and qualitative (analysed thematically) data were integrated. Occupational therapists and other staff working to improve outcome measure completion within the electronic medical record and other allied health teams were consulted when developing and delivering behaviour change interventions. Peak completion of the SMAF-R did not meet an 80% fidelity success rate (admission: 32% in acute and 66% in subacute; discharge: 12% in acute and 48% in subacute). Two thirds of occupational therapists reported the SMAF-R was an acceptable measure to use, but only 38% liked using it. Thematic analysis revealed three themes: (1) A greater understanding of the relevance of the SMAF-R influenced acceptability; (2) using the SMAF-R within an electronic medical record may facilitate completion; and (3) it takes more time to complete SMAF. Patients made clinically significant improvements in SMAF-R scores during their hospital stay (median difference 16.5 [6.5-27.0], p< 0.001, n= 764). Although most occupational therapists reported the SMAF-R was acceptable, there were mixed opinions regarding the relevance of the SMAF-R in this setting, which may have impacted fidelity. Additional prompting and training (especially regarding understanding SMAF-R relevance to practice) may assist with improving SMAF-R completion. Further research is required to assess the psychometric properties of the SMAF-R in the hospital environment. The revised functional autonomy measurement system (SMAF-R) is an assessment tool used to measure performance in everyday activities (such as showering) and the resources needed to complete these tasks (e.g., a shower chair or someone's help). Occupational therapists can use it at the start of a patient admission and again at the end in different hospital settings to assess patients' needs and see if they improve. This study aimed to measure (1) how often occupational therapists use the SMAF-R with patients in hospital, (2) how acceptable occupational therapists find the SMAF-R, and (3) whether the SMAF-R shows changes in patients' abilities during their hospital stay. Before the study, usual completion of the SMAF-R was measured, and it was found to be completed in less than 1% of patients. Strategies were introduced to improve SMAF-R completion over 12 months. After this, completion of the SMAF-R improved to between 9% and 38% in different hospital settings. Changes in SMAF-R scores showed that patients improved during their time in hospital. Most occupational therapists reported the SMAF-R was an acceptable measure, but less than half liked using it. The low completion rates suggested that either the SMAF-R was not fit for purpose in this setting or that more support is needed to increase completion.

CD14 blockade preserves left ventricular structure and systolic function in a murine model of ST-elevation myocardial infarction

Abstract Background Cluster of differentiation-14 (CD14) facilitates the excessive pro-inflammatory monocyte-macrophage response to myocardial injury. Both acute and chronic infiltration of pro-inflammatory macrophages are implicated in the immunological progression of ischemic heart failure. However, investigation of clinically practicable strategies of CD14 blockade are yet to be reported. Purpose This fully blinded and randomised preclinical study evaluated the efficacy of a neutralising anti-CD14 antibody given at reperfusion in a murine model of reperfused ST-elevation myocardial infarction (STEMI) with key clinical features. Methods Large anterior STEMI was induced in 48 adult mice by left anterior descending coronary artery occlusion for 1 h prior to reperfusion and randomisation to receive anti-CD14 antibody, isotype control (IgG2a) or saline weekly for four weeks (independently blinded). Outcome measures included functional assessments by 9.4T cardiac magnetic resonance (CMR) imaging and ultra-high-resolution echocardiography at 21- and 28-days post-STEMI, respectively. Results Multi-plane short axis CMR at 21-days post-STEMI revealed greater left ventricular (LV) ejection fraction in the anti-CD14 antibody-treated group compared with saline- and isotype-treated controls (30±1 vs 19±1 and 20±1 %, respectively, p&amp;lt;0.001; Figure 1A). CMR also revealed reduced LV dilatation with anti-CD14-treatment compared to control groups (76±5 vs 102±8 and 107±7 µl EDV in saline- and isotype-treated controls, respectively, p&amp;lt;0.05). Parasternal long axis echocardiography at 28 days post-STEMI confirmed that anti-CD14 antibody treatment was associated with greater LV systolic function compared to saline and isotype controls (30±1 vs 24±1 and 25±2 % EF, respectively, p&amp;lt;0.05; Figure 1B). Preservation of LV end-diastolic (86±4 vs 111±6 and 101±5 µl, p&amp;lt;0.01) and end-systolic (61±3 vs 84±5 and 77±5 µl, p&amp;lt;0.01) volumes was also observed with anti-CD14 treatment. Global longitudinal strain (GLS) of the LV was also significantly greater at 28 days post-STEMI with anti-CD14 treatment relative to saline and isotype controls (-8.5±0.4 vs -6.8±0.3 and -6.6±0.4 %, respectively, p&amp;lt;0.05). Conclusion(s) The findings of this fully blinded and randomised preclinical study provide evidence of a clinically practicable strategy of CD14 blockade initiated at reperfusion to mitigate progressive LV systolic dysfunction and dilatation post-STEMI.Figure 1.Functional outcome measures

A 5-year natural history study in LAMA2-related muscular dystrophy and SELENON-related myopathy: the Extended LAST STRONG study

BackgroundSELENON-related myopathy (SELENON-RM) is a rare congenital myopathy characterized by slowly progressive axial muscle weakness, rigidity of the spine, scoliosis, and respiratory insufficiency. Laminin-a2-related muscular dystrophy (LAMA2-MD) has a similar clinical phenotype, which ranges from severe, early-onset congenital muscular dystrophy type 1A (MDC1A) to milder forms presenting as childhood- or adult-onset limb-girdle type muscular dystrophy. The first 1.5-year natural history follow-up showed that 90% of the patients had low bone quality, respiratory impairments were found in all SELENON-RM and most of the LAMA2-MD patients, and many had cardiac risk factors. However, further extensive knowledge on long-term natural history data, and clinical and functional outcome measures is needed to reach trial readiness. Therefore, we extended the natural history study with 3- and 5-year follow-up visits (Extended LAST STRONG).MethodsThe Extended LAST STRONG is a long-term natural history study in Dutch-speaking patients of all ages diagnosed with genetically confirmed SELENON-RM or LAMA2-MD, starting in September 2023. Patients visit our hospital twice over a period of 2 years to complete a 5-year follow up from the initial LAST-STRONG study. At both visits, they undergo standardized neurological examination, hand-held dynamometry (age ≥ 5 years), functional measurements, muscle ultrasound, respiratory assessments (spirometry, maximal inspiratory and expiratory pressure, sniff nasal inspiratory pressure; age ≥ 5 years), Dual-energy X-ray absorptiometry (DEXA-)scan (age ≥ 2 years), X-ray of the left hand (age ≤ 17 years), lower extremity MRI (age ≥ 10 years), accelerometry for 8 days (age ≥ 2 years), and questionnaires (patient report and/or parent proxy; age ≥ 2 years). All examinations are adapted to the patient’s age and functional abilities. Disease progression between all subsequent visits and relationships between outcome measures will be assessed.DiscussionThis study will provide valuable insights into the 5-year natural history of patients with SELENON-RM and LAMA2-MD and contribute to further selecting relevant and sensitive to change clinical and functional outcome measures. Furthermore, this data will help optimize natural history data collection in clinical care and help develop clinical care guidelines.Trial registrationThis study protocol including the patient information and consent forms has been approved by medical ethical reviewing committee (‘METC Oost-Nederland’; https://www.ccmo.nl/metcs/erkende-metcs/metc-oost-nederland, file number: 2023–16401). It is registered at ClinicalTrials.gov (NCT06132750; study registration date: 2023-10-05; study first passed date: 2023-11-15).

Cultural adaptation, validity and reliability of the Turkish version of north star ambulatory assessment.

The North Star Ambulatory Assessment (NSAA) is a functional motor outcome measure originally developed for patients with Duchenne muscular dystrophy (DMD). The aim of this study was to perform the cultural adaptation and investigate the validity and reliability of the Turkish version of the NSAA (T-NSAA) in DMD. After translation process, internal consistency, interrater and test-retest reliability of the NSAA were determined by using the Cronbach Alpha Coefficient and Intraclass Correlation Coefficient (ICC), respectively. Absolute reliability was determined by using the Standard Error of Measurement (SEM) with minimal detectable change at 95% limits of confidence (MDC95). Lower limb functionality of children was evaluated by Vignos Lower Extremity Scale (VLERS). To establish convergent validity, the correlations between T-NSAA and Motor Function Measure (MFM-32), 6-minute walk test (6-MWT), and VLERS were analyzed by using the Spearman's correlation coefficient. The study was completed with 86 patients with DMD whose age were mean 104.56 ± 24.66 months. The internal consistency (Cronbach's α = 0.94), intra-reliability (ICC = 0.977) and inter-reliability (ICC = 0.972) of T-NSAA were excellent. SEM and MDC values were low indicating satisfactory absolute agreement (< %10). The T-NSAA had strong correlations with the MFM-total score, 6-MWT, and VLERS (p < 0.01). T-NSAA is a valid and reliable tool to assess ambulatory status of Turkish-speaking DMD population. NCT05549999, Date of registration: September 19, 2022.

Multi-Characteristic Opsin Therapy to Functionalize Retina, Attenuate Retinal Degeneration, and Restore Vision in Mouse Models of Retinitis Pigmentosa.

Retinal degeneration 1 and 10 (rd1 and rd10) mice are useful animal models of retinitis pigmentosa (RP) with rapidly and slowly progressive pathologies, respectively. Our study aims were to determine the effect of adeno-associated viral vector 2 (AAV2)-delivered multi-characteristic opsin (MCO-010; under the control of a metabotropic glutamate receptor-6 promoter enhancer) on the morphological and functional characteristics of vision in both rd1 and rd10 mice. Various retinal measures of MCO-010 transduction and electrophysiological, behavioral, and other routine blood analyses were performed in the rd1 and/or rd10 mice after intravitreal injection of 1µL of MCO-010 or AAV2 vehicle. Functional tests included electroretinogram, visually evoked potential, and behavior assay (optomotor and water maze). Retinal thickness, intraocular pressure, and plasma cytokine levels were also determined. Following intravitreal MCO-010 injection, approximately 80% of bipolar cells were transduced in the retina, and no alterations in retinal thickness were observed at 4months post-injection. However, retinal thickness significantly decreased in control mice. MCO-010 treatment increased head movements and induced faster navigation of mice to the platform in a water-maze test. The MCO-010 gene therapy helped preserve visually evoked electrical response in the retina and visual cortex. No ocular toxicity, immunotoxicity, or phototoxicity was observed in the MCO-010-treated mice, even under chronic intense light conditions. Intravitreal MCO-010 was well tolerated in rd1 and rd10 mice models of RP, and it appeared to attenuate retinal photoreceptor degeneration based on retinal structure and functional outcome measures. As reported here, optogenetic treatment of the inner retina attenuates further retinal degeneration in addition to photosensitizing higher order neurons, and this disease-modifying aspect should be evaluated in optogenetic clinical trials.

Midterm Outcomes After Osteochondral Allograft Transplantation in the Knee Using High–Chondrocyte Viability Grafts

Background: Osteochondral allograft transplantation (OCAT) has become a standard-of-care treatment option for patients with large symptomatic articular defects. Recent advances in allograft science and OCAT protocols have been reported to result in consistently robust outcomes after OCAT in the knee. However, only short-term comparisons have been reported, and analyses are lacking for treatment failure risk factors that account for confounding variables. Hypothesis: Midterm functional graft survival rate would exceed 80% for all OCATs combined, with consideration of risk factors for lower survivorship including older patient age, higher body mass index (BMI), tibiofemoral bipolar OCAT, and nonadherence to prescribed postoperative rehabilitation protocols. Study Design: Case series; Level of evidence, 4. Methods: Patients with outcome data available at ≥5 years after primary OCAT using high chondrocyte–viability (HCV) osteochondral allografts were analyzed according to 2 clinically relevant definitions: (1) initial treatment failure, defined by revision or arthroplasty surgery performed for the primary OCAT at any time point during the study period; and (2) functional graft failure, defined by documented conversion to arthroplasty after primary or revision OCAT at any time point during the study period. Analyses were used to assess outcomes for each definition, separately for age group, sex, obesity status, tobacco use, type of OCAT surgery, osteotomy status, concurrent ligament surgery status, and adherence to postoperative protocols. Kaplan-Meier analyses were used to assess differences in survival rates, and Cox proportional hazards models were used to assess risk factors and multivariable relationships with survival. Patient-reported outcome measures for pain, function, mobility, and satisfaction were also analyzed. Results: Analysis included 137 primary knee OCATs performed in 134 patients with a mean follow-up of 66 months (59 female, 75 male; mean age, 37.8 years; mean BMI, 28.5). The midterm (5- to 8-year) functional graft survival rate for patients undergoing primary OCAT in the knee using HCV grafts was 82% for all cases combined, ranging from 69% for tibiofemoral bipolar HCV OCATs to 89% for patellofemoral bipolar, 94% for multisurface unipolar, and 97% for single-surface unipolar. Initial treatment failure rates (revision or arthroplasty after primary OCAT) and OCAT nonsurvival rates (arthroplasty after primary or revision OCAT) were greater for older patient age, concurrent ligament reconstruction, tibiofemoral bipolar OCAT, and nonadherence to the prescribed postoperative rehabilitation protocols. When adjusted for patients’ age, BMI, and tobacco use status, different surgery types did not demonstrate an increased risk for failure, while concurrent ligament reconstruction and nonadherence did. Patients who experienced functional graft survival after primary OCAT reported significantly greater improvements in PROMIS Physical Function and Mobility (Patient-Reported Outcomes Measurement Information System), International Knee Documentation Committee questionnaire, and Single Assessment Numeric Evaluation scores such that they were significantly higher at final follow-up as compared with patients who required arthroplasty. Patient-reported improvements in pain, function, and mobility exceeded minimal clinically important differences for ≥5 years after primary OCAT. When asked if they were satisfied with primary OCAT surgery, 76.2% of patients were very satisfied or satisfied with their results, while 8.5% were neutral and 15.4% were unsatisfied or very unsatisfied. Conclusion: With use of HCV osteochondral allografts, midterm (5- to 8-year) functional graft survival rates for patients undergoing primary OCAT in the knee were notably higher than previously reported midterm rates for traditional OCATs. When adjusted for patient characteristics, risk factors for nonsurvival included concurrent ligament reconstruction for knee instability and nonadherence to the prescribed postoperative rehabilitation protocols. Patients who experienced functional graft survival for ≥5 years after primary OCAT reported statistically significant and clinically meaningful improvements in pain, function, and mobility.

Long-Term Laryngeal Function and Quality of Life Following Treatment of Early Glottic Cancer: A Meta-Analysis.

To review the literature concerning long-term functional outcomes and quality of life (QoL) in patients undergoing transoral laser microsurgery (TLM) versus radiotherapy (RT) for early glottic cancer. A systematic search was conducted across PubMed, Scopus, and Cochrane Library from inception until April 2024. Articles considered were primary studies directly comparing the 2 treatment modalities in a population of T1 and T2 glottic cancer. Interest outcomes were patient report outcome measures (PROMs) of vocal function and QoL, clinician-reported measures, and acoustic analyses parameters. There was no significant difference between TLM and RT in grade, roughness, breathiness, asthenia, strain (relative risk, 1.11; 95% confidence interval [CI], 0.60-2.07; I2 = 90.96; P < .001), Voice Handicap Inventory-30 scores (standardized mean difference [SME] 0.51; 95% CI, -0.04-1.07; I2 = 89.72; P < .001), or fundamental frequency (SME 0.56; 95% CI, -0.14-1.25; I2 = 91.12; P < .001). The TLM group had significantly better performance with regards to jitter (SME 0.54; 95% CI, 0.08-1.00; I2 = 79.42; P < .001) and shimmer (SME 0.53; 95% CI, 0.11-0.95; I2 = 74.94; P < .001). The risk of bias was assessed to be serious. The findings suggest comparable long-term PROMs between TLM and RT in the treatment of early glottic carcinoma, with TLM showing better acoustic analysis outcomes. However, the available evidence remains scarce, of high heterogeneity, and at risk of bias. A direct comparison between TLM and RT through large randomized controlled trials is needed to provide more substantial evidence to determine the optimum treatment.

The relationship between ON-OFF function and OCT structural and angiographic parameters in early diabetic retinal disease.

This study measured associations between ON and OFF functional indicators and structural optical coherence tomography (OCT) and OCT angiography (OCTA) markers in diabetic retinal disease. Fifty-four participants with type 1 or type 2 diabetes (mean age = 34.1 years; range 18-60) and 48 age-matched controls (mean age = 35.4 years, range 18-59) underwent visual psychophysical testing, OCT and OCTA retinal imaging. Psychophysical tasks measuring (A) contrast increment and decrement sensitivity and (B) response times to increment and decrement targets were assessed as surrogate measures of ON and OFF retinal ganglion cell function. The group with diabetes had worse foveal contrast increment and decrement thresholds (p = 0.04) and were slower to search for increment and decrement targets relative to controls (p = 0.009). Individuals with diabetes had a less circular foveal avascular zone (FAZ) (p < 0.001) but did not differ from controls in foveal vessel density and FAZ area. Functional and structural outcome measures related to the peripheral retina were also comparable between those with and without diabetes. Functional responses to increments and decrements were not significantly correlated with FAZ circularity or vessel density in individuals with diabetes. Diabetic retinal disease results in impaired performance on measures of inferred ON and OFF pathway function in addition to vascular deficits measurable with OCTA. Future longitudinal studies may determine the temporal relationship between these deficits, and whether they predict future diabetic retinopathy.

The landscape of Functional Outcome Measures Used in Patients with Bone Sarcoma of the Lower Extremity or Pelvis: A Systematic Review.

This systematic review provides a structured overview of the measurement instruments of functional outcome used in lower extremity and pelvic bone sarcoma patients. We identified 42 unique instruments covering 18 distinct functional outcome constructs with most studies measuring constructs within the activity domain of the International Classification of Functioning, disability, and health. The MusculoSkeletal Tumor Society 1993 and 1987 score, Toronto Extremity Salvage Score, and range of motion instruments were the measurement instruments most commonly used.

Quantitative EEG Spectral Features Differentiate Genetic Epilepsies and Predict Neurologic Outcomes.

EEG plays an integral part in the diagnosis and management of children with genetic epilepsies. Nevertheless, how quantitative EEG features differ between genetic epilepsies and neurological outcomes remains largely unknown. Here, we aimed to identify quantitative EEG biomarkers in children with epilepsy and a genetic diagnosis in STXBP1 , SCN1A , or SYNGAP1 , and to assess how quantitative EEG features associate with neurological outcomes in genetic epilepsies more broadly. We analyzed individuals with pathogenic variants in STXBP1 (95 EEGs, n =20), SCN1A (154 EEGs, n =68), and SYNGAP1 (46 EEGs, n =21) and a control cohort of individuals without epilepsy or known cerebral disease (847 EEGs, n =806). After removing artifacts and epochs with excess noise or altered state from EEGs, we extracted spectral features. We validated our preprocessing pipeline by comparing automatically-detected posterior dominant rhythm (PDR) to annotations from clinical EEG reports. Next, as a coarse measure of pathological slowing, we compared the alpha-delta bandpower ratio between controls and the different genetic epilepsies. We then trained random forest models to predict a diagnosis of STXBP1 , SCN1A , and SYNGAP1 . Finally, to understand how EEG features vary with neurological outcomes, we trained random forest models to predict seizure frequency and motor function. There was strong agreement between the automatically-calculated PDR and clinical EEG reports ( R 2 =0.75). Individuals with STXBP1 -related epilepsy have a significantly lower alpha-delta ratio than controls ( P< 0.001) across all age groups. Additionally, individuals with a missense variant in STXBP1 have a significantly lower alpha-delta ratio than those with a protein-truncating variant in toddlers ( P< 0.001), children ( P =0.02), and adults ( P< 0.001). Models accurately predicted a diagnosis of STXBP1 (AUC=0.91), SYNGAP1 (AUC=0.82), and SCN1A (AUC=0.86) against controls and from each other in a three-class model (accuracy=0.74). From these models, we isolated highly correlated biomarkers for these respective genetic disorders, including alpha-theta ratio in frontal, occipital, and parietal electrodes with STXBP1 , SYNGAP1 , and SCN1A , respectively. Models were unable to predict seizure frequency (AUC=0.53). Random forest models predicted motor scores significantly better than age-based null models ( P< 0.001), suggesting spectral features contain information pertinent to gross motor function. In summary, we demonstrate that STXBP1 -, SYNGAP1- , and SCN1A -related epilepsies have distinct quantitative EEG signatures. Furthermore, EEG spectral features are predictive of some functional outcome measures in patients with genetic epilepsies. Large-scale retrospective quantitative analysis of clinical EEG has the potential to discover novel biomarkers and to quantify and track individuals' disease progression across development.

Comparison of pericapsular nerve group and lateral quadratus lumborum blocks on cumulative opioid consumption after primary total hip arthroplasty: a randomized controlled trial

IntroductionBoth the quadratus lumborum block (QLB) and the pericapsular nerve group (PENG) block provide effective postoperative analgesia after hip surgery while minimizing the impact on motor function. This study aimed...

Patient-reported disease burden in the Accelerate Clinical Trials in Charcot-Marie-Tooth Disease Study.

The Charcot-Marie-Tooth Disease Health Index (CMT-HI) is a disease-specific, patient-reported disease burden measure. As part of an international clinical trial readiness study, individuals with CMT1A (ages 18-75 years) underwent clinical outcome assessments (COAs), including the CMT-HI, to capture their longitudinal perspective on the disease burden. Two hundred and fifteen participants underwent serial COAs including the CMT-HI, CMT Functional Outcome Measure (CMT-FOM), CMT Neuropathy Score (CMTNSv2R), and CMT Exam Score (CMTES/CMTES-R). Correlations between the total and subscale scores for the CMT-HI and other COAs were determined. Changes in the CMT-HI scores over 12 months were assessed using paired t-tests. The minimum clinically important difference (MCID) for the CMT-HI and its subscales were calculated by anchoring to a participant global impression of change scale. At baseline, CMT1A participants were 44.5 ± 15 years old (range: 18-75) and 58% were women. The mean CMT-HI was 25.7 ± 18.8 (range: 0-91.9; 100 reflecting maximal disease burden). The CMT-HI correlated with the CMT-FOM (r = .54, p < .0001), CMTNSv2R (r = .48, p < .0001), and CMTES/CMTES-R (r = .52/r = .54, p < .0001). Disease burden was greater in women than in men (CMT-HI 29.1 ± 19.1 vs. 21.2 ± 17.3, p = .001). Over 12 months, there was a nonsignificant mean increase in CMT-HI of 0.40 ± 10.0 (n = 189, p = .89). The MCID for the CMT-HI total score was 3.8 points (95% CI: 1.7-5.9). Patient-reported disease burden in CMT1A as measured by the CMT-HI is associated with measures of neurologic impairment and physical functioning. Women reported a higher disease burden than men. These data will inform the design of clinical trials in CMT1A.

Arthroscopic reduction internal fixation for glenoid fractures: a systematic review of the outcomes and complications.

With the advanced arthroscopic technique, arthroscopic-assisted reduction and internal fixation (ARIF) is gaining popularity for Glenoid fractures with and without scapular involvement. ARIF offers a complete view of the articular surfaces and diagnoses and treats other associated injuries. ARIF provides less soft tissue trauma than open reduction internal fixation (ORIF). The aim of this systemic review is to look at the functional outcomes and complications of ARIF. A systematic review of the PubMed, Embase, and Scopus databases was performed. The search terms included "glenoid fracture" OR "scapula fracture" AND "arthroscopic fixation" OR "arthroscopy" OR "arthroscopic-assisted reduction and internal fixation". Studies were limited to English publications with reported functional outcome measures and complications. Patient demographic characteristics, clinical outcomes including range of motion, outcome performance scores including ROWE score, visual analogue scale and American shoulder and elbow surgeons score and complications were extracted. Five studies met the inclusion criteria. The participants ranged in age from 41 to 48, and the mean length of follow-up ranged from 12 to 41months postoperatively. The mean ROWE scores for the ARIF group were significantly better postoperatively. ARIF allows accurate diagnosis of the fracture pattern and associated injuries, in addition to representing a safe option to treat glenoid fractures with or without scapular fractures with fewer complications compared to ORIF. Level of Evidence: Level IV, Systematic review.

The Effect of Video-Based Action Observation Training and Live Action Observation Training on Motor Function, Activity Participation, and Secondary Outcome Measures in Children With Spastic Diparetic Cerebral Palsy: A Randomized Controlled Study.

This study aimed to investigate the effects of Video-Based Action Observation Training and Live Action Observation Training on motor function, activity participation, and secondary outcome measures in children with spastic diparetic cerebral palsy (CP). Thirty-nine children with spastic diparetic cerebral palsy, aged 5-14 years, with Gross Motor Function Classification System I-III, were distributed in equal numbers to any of the Video-Based Action Observation Training (conventional physiotherapy + Video-Based Action Observation Training), Live Action Observation Training (conventional physiotherapy + Live Action Observation Training), and control (conventional physiotherapy) groups through stratified randomization. For 8 weeks, action observation training groups received 20 minutes of conventional physiotherapy followed by 20 minutes of action observation training, and the control group received 40 minutes of conventional physiotherapy. Primary outcome measures were Gross Motor Function Measurement and Child and Adolescent Scale of Participation, secondary outcome measures were Pediatric Berg Balance Scale, timed-up-and-go test, five times sit-to-stand test (FTSST), Gillette Functional Assessment Questionnaire (GFAQ), and 1-minute walk test (1MWT). Improvements were observed in all other evaluation parameters of the groups except Gross Motor Function Measurement-lying and rolling (P = .066) in the Live Action Observation Training Group, and lying and rolling (P = .317) and crawling and kneeling (P = .063) motor subtests and Gillette Functional Assessment Questionnaire-walking scale (P = .513) in the control group. Comparisons of the increases in all other measurements between the groups, except for the dimensions of Gross Motor Function Measurement-lying and rolling (P = .172), were statistically significant (P < .05) and this difference was in favor of action observation training. It was found that 2 different AOTs applied in addition to conventional physiotherapy in children with spastic diparetic cerebral palsy were more effective on all outcomes than was conventional physiotherapy alone.

Subjective and functional outcome measures to evaluate performance of pediatric cochlear implant

BackgroundThe majority of research on the effects of cochlear implants has been on clinical evaluations of efficacy (hearing and speech skills and auditory thresholds). But it is unclear how much clinical efficacy metrics (speech, hearing, and language tests) actually demonstrate how beneficial cochlear implants (CI) are in everyday circumstances. In this study, we aim to assess the performance of a child’s current CI with both subjective and functional outcomes measures. Also, we aimed to assess factors affecting these outcomes after CI.MethodsThe study group included 45 cochlear-implanted children, and their chronological age ranges between 4 and 12 years. Children were divided according to their language age into three groups: group 1 (G1) includes15 children with a language age of less than 2 years, group 2 (G2) includes 15 children with a language age of 2 to less than 4 years, and group 3 (G3) includes 15 children with language age 4 to 6 years. Assessment was done in two sessions: one for phoniatric evaluation and another for audiological evaluation.ResultsCI enabled children to develop speech perception in a closed manner evidenced by the early speech perception (ESP) test with pattern perception being the easiest and monosyllabic words being the most difficult and the least to develop. Most children with CI were able to control their voice, to a lesser extent to use speech in meaningful use in social situations and to develop communication strategies in oral situations which were the least to develop, and this was evident by Meaningful Use of Speech Scale. Children with CI were able to acquire language, and their receptive language was better than expressive language, but their language age was below their chronological age.ConclusionThe cochlear implant is an effective rehabilitative tool for the treatment of prelingually deafened children, and this was evident by our results. A hierarchal approach of speech assessment according to the language age of the child and selection of age-appropriate speech perception test is very important to assess cochlear-implanted children Speech recognition had a high degree of variability. Early implantation age and increased duration of implant experience were the most important factors associated with good performance with CI, better speech perception, and better language outcomes.

Preoperative Osteoporosis Is Associated With Increased Health Care Utilization and Compromised Pain and Function Improvement After Primary Total Hip Arthroplasty: A Prospective Cohort Analysis

BackgroundOsteoporosis (OP) has been linked to complications after total hip arthroplasty (THA), but its impact on health care utilization and patient-reported outcomes remains unclear. This study aimed to evaluate the association between: 1) pre-THA OP and health care utilization as well as patient-reported pain and function outcome measures; and 2) dual energy X-ray absorptiometry (DEXA) scan-based T-scores and the aforementioned outcomes. MethodsA retrospective analysis of prospectively collected data of primary THA (2015 to 2018) was performed (n = 5,321) from a validated academic institutional database of a large North American tertiary health care system; of which 4,074 (76.6%) completed 1-year follow-up. Outcomes included prolonged length of stay [LOS] > three days, discharge disposition, 90-day readmission, and 1-year reoperation, as well as Hip Disability and Osteoarthritis Outcome Score (HOOS]) Pain, HOOS-function (PS), and minimal clinically important difference thresholds (MCID), and satisfaction. ResultsThe prevalence of OP pre-THA was 56.9%, of which 39.8% were not prescribed OP medications and 15.3% had a DEXA scan. Compared to those who did not have OP, those who had OP were independently associated with higher odds of prolonged LOS, nonhome discharge, 90-day readmission, and 1-year reoperation (P < 0.005). Furthermore, they had significantly higher odds of failing to achieve MCID (odds ratio: 1.41 (95% confidence interval: 1.06 to 1.89)) for HOOS-PS and satisfaction (odds ratio: 1.5 (95% confidence interval: 1.16 to 1.93)) at one year. Higher T-scores were associated with lower odds of prolonged LOS, nonhome discharge, failure to achieve MCID in HOOS-Pain, and HOOS-PS. ConclusionsOver half of patients had OP; however, only 15.3% of patients had a DEXA scan before THA. Patients who had OP were at higher risk of prolonged LOS, nonhome discharge, 90-day readmission, and 1-year reoperation in addition to poor pain/function improvement and dissatisfaction one year after THA.

Limb Salvage and Treatment of Posttraumatic Lymphedema in a Mangled Lower Extremity

Summary: Post traumatic lymphedema (PTL) is a known complication of extremity trauma that is detrimental to limb form and function, healing, and quality of life. In cases of complex lower extremity trauma with vascular and extensive soft tissue injury, the risk of PTL is increased. However, many trauma patients are lost to follow-up, making the risk and potential management of these patients’ lymphedema difficult to characterize. The purpose of this report is to describe the successful surgical management of PTL secondary to significant lower extremity trauma requiring complex limb salvage reconstruction. A 43-year-old woman involved in a motorcycle accident presented with a Gustilo IIIB right tibial fracture and single-vessel leg. She underwent successful limb salvage with serial debridement, bony fixation, creation of an arteriovenous loop with a contralateral saphenous vein graft, and a chimeric latissimus dorsi-serratus anterior muscle flap. At the 5-month follow-up, she presented with significant right lower extremity lymphedema. She underwent lymphovenous bypass surgery guided by preoperative indocyanine green lymphography, which resulted in a 62% improvement in functional outcome measures, eliminated her prior need for compression garments and inability to wear regular shoes, and sustained improvement at two years follow-up. This case illustrates a near circumferential traumatic defect reconstructed with a muscle flap and successful delayed lymphatic reconstruction with lymphovenous bypass in the same patient.

384P Functional Outcome Measures (FOMs) guiding clinical care of children with Duchenne muscular dystrophy

384P Functional Outcome Measures (FOMs) guiding clinical care of children with Duchenne muscular dystrophy