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INTRODUCTION: Magnetic Resonance-guided Focused Ultrasound (MRgFUS) thermal ablation is an effective noninvasive ultrasonic therapy to disrupt in vivo porcine tendon but is prone to inducing skin burns. We evaluated the safety profile of a novel hybrid protocol that minimizes thermal spread by combining long-pulse focused ultrasound followed by thermal ablation. METHODS: In-vivo Achilles tendons (hybrid N = 15, thermal ablation alone N = 21) from 15 to 20 kg Yorkshire pigs were randomly assigned to 6 treatment groups in two studies. The first (N = 21) was ablation (600, 900, or 1200 J). The second (N = 15) was hybrid: pulsed FUS (13.5 MPa peak negative pressure) followed by ablation (600, 900, or 1200 J). Measurements of ankle range of motion, tendon temperature, thermal dose (240 CEM43), and assessment of skin burn were performed in both groups. RESULTS: Rupture was comparable between the two protocols: 1/5 (20%), 5/5 (100%) and 5/5 (100%) for hybrid protocol, compared to 2/7 (29%), 6/7 (86%) and 7/7 (100%) for the ablation-only protocol with energies of 600, 900, and 1200 J, respectively. The hybrid protocol produced lower maximum temperatures, smaller areas of thermal dose, fewer thermal injuries to the skin, and fewer full-thickness skin burns. The standard deviation for the area of thermal injury was also smaller for the hybrid protocol, suggesting greater predictability. CONCLUSION: This study demonstrated a hybrid MRgFUS protocol combining long-pulse FUS followed by thermal ablation to be noninferior and safer than an ablation-only protocol for extracorporeal in-vivo tendon rupture for future clinical application for noninvasive release of contracted tendon.
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Imagen por Resonancia Magnética , Animales , Porcinos , Imagen por Resonancia Magnética/métodos , Ultrasonido Enfocado de Alta Intensidad de Ablación/métodos , Tendones/diagnóstico por imagen , Terapia por Ultrasonido/métodosRESUMEN
The potential of optical polarimetry is increasingly explored to unravel the tissue structure through several optical instrument configurations. Fiber-based solutions offer portability and are accommodative in an endoscopic examination environment. Here, we address the challenges in realizing a fiber-based optical polarimetry system through an approach involving an all-fiber polarization controller. The methods of device calibration and application in bulk tissues are discussed, and results are presented.
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AIM: We investigated the reliability and validity of the Danish child and parent versions of the Gait Outcomes Assessment List (GOAL) questionnaires for ambulatory children with cerebral palsy (CP). METHODS: Translation and cultural adaptations were performed and content validity evaluated. Participants were enrolled between 2016 and 2018 from Aarhus University Hospital, Denmark. Children and parents completed the GOAL questionnaires twice for test-retest reliability. Discriminative validity was evaluated by comparing the child and parent GOAL scores between children with Gross Motor Function Classification System (GMFCS) levels I and II. The concurrent validity of the GOAL questionnaires were investigated by comparing them with Challenge-20, which assesses motor skills in children with CP. RESULTS: We studied 59 children (57% boys) with CP and GMFCS I-II at a mean age of 10.6 years. Test-retest intra-class correlations were excellent for the children (0.91, 95% confidence interval (CI) 0.83-0.96) and good for the parents (0.83, 95% CI 0.67-0.91). GOAL scores decreased with increasing GMFCS (p < 0.05). Both versions correlated well. The mean children's scores were significantly (6.2/100) higher than the parents' (p < 0.001). The GOAL scores correlated positively with Challenge-20. CONCLUSION: The Danish GOAL child and parent questionnaires demonstrated good reliability and content and discriminative and concurrent validity.
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Parálisis Cerebral , Niño , Masculino , Humanos , Femenino , Parálisis Cerebral/diagnóstico , Reproducibilidad de los Resultados , Marcha , Encuestas y Cuestionarios , Padres , Evaluación de Resultado en la Atención de Salud , DinamarcaRESUMEN
BACKGROUND: Spinal fusion for scoliosis associated with cerebral palsy (CP) is challenging to study because specialized outcome measures are needed. Therefore, evidence in favor of the benefits of surgery has not been firmly established. This study aimed to determine if corrective spinal fusion improves health-related quality of life (HRQoL) in children with CP scoliosis at 2 years. METHODS: Children with CP and scoliosis who met the criteria for posterior spinal fusion were offered enrollment at 16 US and Canada centers. Participants' families selected either operative intervention (OP) or nonoperative treatment (NON) in discussion with their surgeon with no influence by the decision to participate in the research study. Demographic, clinical data (function level, magnitude of deformity, comorbidities), and HRQoL (CPCHILD Questionnaire) were collected at baseline and 2 years. Change (from baseline) in total CPCHIL scores was the primary outcome. RESULTS: Three hundred one OP and 34 NON subjects had complete baseline and 2-year data. At baseline, both groups were comparable in function level, comorbid status, and CPCHILD scores (52.1 ±15.3 vs. 53.4 ±14.5; P=0.66). The OP group had a larger spinal deformity magnitude (84.5Ë ± 21.8Ë vs. 66.3Ë ± 18.1Ë) (P=0.001). The total CPCHILD score improved in the OP group by 6.6 points (P<0.001). NON scores were unchanged (+1.2; P=0.65) during follow-up. There were also significant score increases in the OP group for 5 of 6 CPCHILD domains. The change in CPCHILD scores from enrollment to 2 years was more significant in the OP group (P=0.05). CONCLUSION: For children with CP who undergo spinal fusion, HRQoL improved over preoperative levels and an unchanged nonoperative control group. LEVEL OF EVIDENCE: Level II.
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BACKGROUND: Use of postoperative radiographs after surgical management of supracondylar humerus (SCH) fractures is often based on rote practice rather than evidence. The purpose of this study was to determine the frequency with which 3-week postoperative radiographs at the time of pin removal altered management plans in pediatric SCH fractures that were intraoperatively stable after closed reduction and percutaneous pinning (CRPP). METHODS: We prospectively recruited pediatric patients with SCH fractures managed by CRPP at our institution from June 2020 until June 2022, and reviewed retrospective data on pediatric SCH fractures managed surgically at our institution between April 2008 and March 2015. Patients were assessed for post-CRPP fracture alignment and stability. For prospective patients, we asked clinicians to document their management decision at the 3-week follow-up visit before evaluating the postoperative radiographs. Our primary outcome was change in management because of radiographic findings. RESULTS: Overall, 1066 patients in the retrospective data and 446 prospectively recruited patients met the inclusion criteria. In the prospective group, radiographic findings altered management for 2 patients (0.4%). One patient had slow callus formation and 1 patient was identified as having cubitus varus. Altered management included prolonged immobilization or additional radiographic follow-up. Radiographic findings altered management in 0 (0%) of 175 type II fractures, in 2 (0.9%) of 221 type III fractures, and in 0 (0%) of 44 type IV fractures. We obtained similar findings from retrospective data. CONCLUSION: Rote use of 3-week postoperative radiographs after surgical management of SCH fractures that are intraoperatively stable has minimal utility. Eliminating rote postoperative radiographs for SCH fractures can decrease the time and financial burdens on families and health care systems without affecting patient outcomes.
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Fracturas del Húmero , Radiografía , Humanos , Fracturas del Húmero/cirugía , Fracturas del Húmero/diagnóstico por imagen , Estudios Retrospectivos , Niño , Masculino , Femenino , Preescolar , Clavos Ortopédicos , Reducción Cerrada/métodos , Estudios Prospectivos , Cuidados Posoperatorios/métodosRESUMEN
AIM: To assess the association between epilepsy characteristics and proxy-reported health-related quality of life (HRQoL) in children and young people with non-ambulatory cerebral palsy (CP) and seizures. METHOD: This was a cross-sectional study of 164 children and young people (74 females, 90 males; mean age 10 years 6 months, range 2-21 years, SD 5 years 5 months). Caregivers completed the Child Health Index of Life with Disabilities (CPCHILD) in an outpatient setting. We utilized univariable linear regression and multivariable modeling to study relationships between variables and CPCHILD scores. RESULTS: Gross Motor Function Classification System levels were 37% IV and 63% V. Sociodemographic factors included the Child Opportunity Index (median 51, interquartile range [IQR] 25-80). A median of 2 (IQR 1-3) antiseizure medications (ASMs) were used, and days with seizures ranged from 0 (30%) to 28 (20%) days in the previous 4 weeks. Total CPCHILD scores decreased 2.3 points for each ASM (95% confidence interval [CI] -4.1 to -0.42). Compared to persons with focal epilepsy, those with generalized epilepsy had lower total CPCHILD scores (-5.7; 95% CI -11 to -0.55). Number of days with seizures was not associated with total CPCHILD scores. INTERPRETATION: Proxy-reported HRQoL was affected by epilepsy-specific features in children and young people with severe CP. WHAT THIS PAPER ADDS: Health-related quality of life (HRQoL) was lower with increasing numbers of antiseizure medications. Overall quality of life (QoL) scores were lower by a similar amount, independent of seizure frequency. HRQoL was lower in persons with recent hospital admissions for epilepsy.
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Parálisis Cerebral , Epilepsia , Masculino , Femenino , Niño , Humanos , Adolescente , Lactante , Calidad de Vida , Estudios Transversales , Encuestas y Cuestionarios , Epilepsia/epidemiología , Epilepsia/complicacionesRESUMEN
The unique four-level photocycle characteristics of excited-state intramolecular proton transfer (ESIPT) materials enable population inversion and large spectral separation between absorption and emission through their respective enol and keto forms. This leads to minimal or no self-absorption losses, a favorable feature in acting as an optical gain medium. While conventional ESIPT materials with an enol-keto tautomerism process are widely known, zwitterionic ESIPT materials, particularly those with high photoluminescence, are scarce. Facilitated by the synthesis and characterization of a new family of 2-hydroxyphenyl benzothiazole (HBT) with fluorene substituents, HBT-Fl1 and HBT-Fl2, we herein report the first efficient zwitterionic ESIPT lasing material (HBT-Fl2). The zwitterionic ESIPT HBT-Fl2 not only shows a remarkably low solid-state amplified spontaneous emission (ASE) threshold of 5.3 µJ/cm2 with an ASE peak at 609 nm but also exhibits high ASE photostability. Coupled with its substantially large Stokes shift (≈236 nm ≈10,390 cm-1) and an extremely small overlap of excited-state absorption with ASE emission, comprehensive density functional theory (DFT) and time-dependent DFT studies reveal the zwitterionic characteristics of HBT-Fl2. In opposition to conventional ESIPT with π-delocalized tautomerism as observed in analogue HBT-Fl1 and parent HBT, HBT-Fl2 instead shows charge redistribution in the proton transfer through the fluorene conjugation. This structural motif provides a design tactic in the innovation of new zwitterionic ESIPT materials for efficient light amplification in red and longer-wavelength emission.
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Fluorenos , ProtonesRESUMEN
The Gait Outcome Assessment List (GOAL) is a patient or caregiver-reported assessment of gait-related function across different domains of the International Classification of Functioning, Disability, and Health (ICF) developed for ambulant children with cerebral palsy (CP). So far, the questionnaire is only available in English. The aim of this study was to translate the GOAL into German and to evaluate its reliability and validity by studying the association between GOAL scores and gross motor function as categorized by the gross motor function classification system (GMFCS) in children with cerebral palsy (CP). The GOAL was administered to primary caregivers of n = 91 children and adolescents with CP (n = 32, GMFCS levels I; n = 27, GMFCS level II; and n = 32, GMFCS level III) and n = 15 patients were capable of independently completing the whole questionnaire (GMFCS level I). For assessing test-retest reliability, the questionnaire was completed for a second time 2 weeks after the first by the caregivers of n = 36 patients. Mean total GOAL scores decreased significantly with increasing GMFCS levels with scores of 71 (95% confidence interval [CI]: 66.90-74.77) for GMFCS level I, 56 (95% CI: 50.98-61.86) for GMFCS level II, and 45 (95% CI: 40.58-48.48) for GMFCS level III, respectively. In three out of seven domains, caregivers rated their children significantly lower than children rated themselves. The test-retest reliability was excellent as was internal consistency given the GOAL total score. The German GOAL may serve as a much needed patient-reported outcome measure of gait-related function in ambulant children and adolescents with CP.
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Parálisis Cerebral , Adolescente , Parálisis Cerebral/diagnóstico , Niño , Marcha , Objetivos , Humanos , Evaluación de Resultado en la Atención de Salud , Reproducibilidad de los Resultados , Encuestas y CuestionariosRESUMEN
BACKGROUND: To develop a priority-based patient/parent reported outcome measure for children with lower-limb differences (LD) by adapting the Gait Outcomes Assessment List (GOAL) questionnaire. METHODS: Guided by a conceptual framework of patient priorities, the GOAL questionnaire was iteratively modified and its sensibility evaluated by field-testing it on children with LD, and their parents. Cognitive interviews were conducted with a subgroup of these children, and an e-survey administered to a multidisciplinary group of health care professionals with expertise in paediatric LD. Findings were integrated to create the final version of the GOAL-LD. RESULTS: Twenty-five children (9-18 years), 20 parents, and 31 healthcare professionals evaluated the content and sensibility of the GOAL, with an emphasis on the relevance and importance of the items to patients' health related quality of life (HRQL). This resulted in the retention of 26 of the original 50 items, elimination of 12, modification of 12, and addition of seven new items. The new 45-item GOAL-LD questionnaire was shown to be sensible, and its content deemed important. CONCLUSIONS: The GOAL-LD questionnaire has a high level of face and content validity, and sensibility. It comprehensively captures the HRQL goals and outcomes that matter to children with LD and their parents. Following further psychometric evaluation, the GOAL-LD may serve as a much needed patient and parent reported outcome measure for this population.
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Marcha/fisiología , Personal de Salud/psicología , Extremidad Inferior/fisiología , Padres/psicología , Calidad de Vida/psicología , Encuestas y Cuestionarios/normas , Evaluación de Síntomas/normas , Adolescente , Adulto , Niño , Femenino , Personal de Salud/estadística & datos numéricos , Humanos , Masculino , Persona de Mediana Edad , Medición de Resultados Informados por el Paciente , Psicometría/normas , Psicometría/estadística & datos numéricos , Encuestas y Cuestionarios/estadística & datos numéricos , Evaluación de Síntomas/estadística & datos numéricos , Adulto JovenRESUMEN
BACKGROUND: Despite being a common procedure, there are no standard protocols for postoperative immobilization and rehabilitation following reconstructive hip surgery in children with cerebral palsy (CP). The purpose of this study was to investigate variability in postoperative management and physical therapy (PT) recommendations among orthopaedic surgeons treating hip displacement in children with CP. METHODS: An invitation to participate in an anonymous, online survey was sent to 44 pediatric orthopaedic surgeons. The case of a child undergoing bilateral femoral varus derotation osteotomies and adductor tenotomies was presented. Surgeons were asked to consider their typical practice and the case scenario when answering questions related to immobilization, weight-bearing, and rehabilitation. Recommendations with increasingly complex surgical interventions and different age or level of motor function were also assessed. RESULTS: Twenty-eight orthopaedic surgeons from 9 countries with a mean 21.3 years (range: 5 to 40 y) of experience completed the survey. Postoperative immobilization was recommended by 86% (24/28) of respondents with 7 different methods of immobilization identified. All but 1 (23/24) reported immobilizing full time. Most (20/23) reported using immobilization for 4 to 6 weeks. Return to weight-bearing varied from 0 to 6 weeks for partial weight-bearing and 0 to 12 weeks for full weight-bearing. PT in the first 1 to 2 weeks postoperatively was reported as unnecessary by 29% (8/28) of surgeons. PT for range of motion, strengthening, and return to function was recommended by 96% (27/28) of surgeons, starting at a mean of 2.6 weeks postoperatively (range: 0 to 16 wk). Only 48% (13/27) reported all of their patients would receive PT for these goals in their practice setting. Inpatient rehabilitation was available for 75% (21/28) but most surgeons (17/21) reported this was accessed by 20% or fewer of their patients. CONCLUSIONS: Postoperative immobilization and PT recommendations were highly variable among surgeons. This variability may influence surgical outcomes and complication rates and should be considered when evaluating procedures. Further study into the impact of postoperative immobilization and rehabilitation is warranted.
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BACKGROUND: The Gait Outcomes Assessment List for children with Lower Limb Difference (GOAL-LD) is a patient and parent-reported outcome measure that incorporates the framework of the International Classification of Functioning, Disability, and Health. This prospective multicenter cohort study evaluates the validity and reliability of the GOAL-LD and the differences between parent and adolescent report. METHOD: One hundred thirty-seven pediatric patients aged over 5 years attending limb reconstruction clinics at the participating sites were assessed at baseline, and a self-selected cohort also completed an assessment 2 to 6 weeks later. Construct and criterion validity were assessed by comparing GOAL-LD scores with a measure of limb deformity complexity (LLRS-AIM) and the Pediatric Outcomes Data Collection Instrument, using Spearman correlation coefficients. Face and content validity were determined through ratings of item importance. Test-retest reliability was reported as an intraclass correlation coefficient and internal consistency using Cronbach α. Adolescent reports were compared with their parents using paired t tests. RESULTS: The GOAL-LD demonstrated a moderate negative correlation with the LLRS-AIM (r=-0.40, P<0.001) and was able to discriminate between deformity complexity groups as defined by the LLRS-AIM (χ2=11.43, P=0.022). Internal consistency was high across all domains (α≥0.68 to 0.97). Like domains of the Pediatric Outcomes Data Collection Instrument and the GOAL-LD were well correlated. Parents reported a lower total GOAL-LD score when compared with adolescents (mean difference 3.04; SE 1.06; 95% confidence interval, 0.92-5.16; P<0.01); however this difference was only significant for body image and self-esteem (Domain F) and gait appearance (Domain D). Test-retest reliability remained high over the study period (intraclass correlation coefficient 0.85; SE 0.03; 95% confidence interval, 0.77-0.91). CONCLUSIONS: The GOAL-LD is a valid and reliable self and parent-reported outcome measure for children with lower limb difference. Parents report a lower level of function and attribute a higher importance to items when compared with their children. The GOAL-LD helps to communicate parent and child perspectives on their function and priorities and therefore has the capacity to facilitate family centered treatment planning and care. LEVEL OF EVIDENCE: Level II-diagnostic. Prospective cross-sectional and a longitudinal cohort design.
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BACKGROUND: The purpose of this study was to identify differences in caregiver responses to Korean-language and English-language versions of the Caregiver Priorities & Child Health Index of Life with Disabilities (CPCHILD) questionnaire. METHODS: Patient data were acquired from the Cerebral Palsy Hip Outcomes Project database, which was established to run a large international multicenter prospective cohort study of the outcomes of hip interventions in cerebral palsy. Thirty-three children whose caregivers had completed the Korean version of CPCHILD were matched by propensity scoring with 33 children whose parents completed the English version. Matching was performed on the basis of 12 covariates: age, gender, gross motor function classification system level, migration percentage of right and hip, seizure status, feeding method, tracheostomy status, pelvic obliquity, spinal deformity, parental report of hip pain and contracture interfering with care. RESULTS: There were no significant differences in CPCHILD scores for section 4 (Communication and Social Interaction), and section 5 (Health) between two groups. Korean-language CPCHILD scores were significantly lower than English-language CPCHILD scores for section 1 (Personal Care/Activities of Daily Living), section 2 (Positioning, Transferring and Mobility), section 3 (Comfort and Emotions) and section 6 (Overall Quality of Life) as well as in terms of total score. CONCLUSIONS: Cultural influences, and the community or social environment may impact the caregivers' perception of the health-related quality of life of their children. Therefore, physicians should consider these differences when interpreting the study outcomes across different countries.
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Cuidadores/psicología , Parálisis Cerebral/psicología , Padres/psicología , Encuestas y Cuestionarios/normas , Adolescente , Adulto , Niño , Preescolar , Comparación Transcultural , Personas con Discapacidad/psicología , Femenino , Humanos , Lenguaje , Masculino , Persona de Mediana Edad , Estudios Prospectivos , Calidad de Vida , República de CoreaRESUMEN
AIM: To identify and assess the quality of decision aids that align the decision, values and information provided for parents making end-of-life or palliative care decisions for children with life-threatening conditions. METHODS: Six databases and the grey literature were searched in December 2018. Two reviewers independently reviewed database citations, and one reviewed grey literature citations. Citation chaining via Scopus was conducted. Quality was assessed using IPDAS Collaboration Criteria. RESULTS: After reviewing 18 671 database citations and 10 988 grey literature citations, 18 citations describing 11 decision aids remained. Decision aids targeted premature infants, children requiring airway management, children with cancer and children with scoliosis. Three aids underwent testing beyond initial development. Quality scores averaged 27 of 50 points. CONCLUSIONS: There are few high-quality decision aids available for use and a lack evidence of widespread clinical use. Additional research is needed to support systematic development and the use of decision aids with families.
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Cuidados Paliativos , Padres , Niño , Muerte , Toma de Decisiones , Técnicas de Apoyo para la Decisión , Humanos , Lactante , Recién Nacido , Participación del PacienteRESUMEN
INTRODUCTION: It is unclear what factors influence health-related quality of life (HRQOL) in neuromuscular scoliosis. The aim of this study was to evaluate which factors are associated with an improvement in an HRQOL after spinal fusion surgery for nonambulatory patients with cerebral palsy (CP). METHODS: A total of 157 patients with nonambulatory CP (Gross Motor Function Classification System IV and V) with a minimum of 2-year follow-up after PSF were identified from a prospective multicenter registry. Radiographs and quality of life were evaluated preoperatively and 2 years postoperatively. Quality of life was evaluated using the validated Caregiver Priorities and Child Health Index of Life with Disabilities (CPCHILD) questionnaire. Patients who had an increase of 10 points or greater from baseline CPCHILD scores were considered to have meaningful improvement at 2 years postoperatively. 10 points was chosen as a threshold for meaningful improvement based on differences between Gross Motor Function Classification System IV and V patients reported during the development of the CPCHILD. Perioperative demographic, clinical, and radiographic variables were analyzed to determine predicators for meaningful improvement by univariate and multivariate regression analysis. RESULTS: A total of 36.3% (57/157) of the patients reported meaningful improvement in CPCHILD scores at 2 years postoperatively. Preoperative radiographic parameters, postoperative radiographic parameters, and deformity correction did not differ significantly between groups. Patients who experienced meaningful improvement from surgery had significantly lower preoperative total CHPILD scores (43.8 vs. 55.2, P<0.001). On backwards conditional binary logistic regression, only the preoperative comfort, emotions, and behavior domain of the CPCHILD was predictive of meaningful improvement after surgery (P≤0.001). CONCLUSION: Analysis of 157 CP patients revealed a meaningful improvement in an HRQOL in 36.3% of the patients. These patients tended to have lower preoperative HRQOL, suggesting more "room for improvement" from surgery. A lower score within the comfort, emotions, and behavior domain of the CPCHILD was predictive of meaningful improvement after surgery. Radiographic parameters of deformity or curve correction were not associated with meaningful improvement after surgery. LEVEL OF EVIDENCE: Level II-retrospective review of prospectively collected data.
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Parálisis Cerebral/complicaciones , Calidad de Vida , Escoliosis , Fusión Vertebral , Niño , Femenino , Humanos , Masculino , Satisfacción del Paciente , Periodo Preoperatorio , Radiografía/métodos , Estudios Retrospectivos , Escoliosis/diagnóstico , Escoliosis/psicología , Escoliosis/cirugía , Índice de Severidad de la Enfermedad , Fusión Vertebral/métodos , Fusión Vertebral/psicología , Resultado del TratamientoRESUMEN
AIM: To develop a patient-reported outcome measure that comprehensively captures the health-related priorities of children with Duchenne muscular dystrophy (DMD). METHOD: Children with DMD and their parents completed the iteratively revised versions of the Caregiver Priorities and Child Health Index of Life with Disabilities (CPCHILD), followed by a cognitive interview to develop a pilot version of a new measure. Multidisciplinary health care professionals completed an item-by-item analysis of the measure and a 14-item sensibility questionnaire. Minimum content validity ratio for each item of the new measure and the mean score (0-7) for the items of the sensibility questionnaire were calculated. RESULTS: The CPCHILD underwent changes over 19 interviews with children and their parents, resulting in the pilot Muscular Dystrophy Child Health Index of Life with Disabilities (MDCHILD). The content validity ratio of each MDCHILD item ranged from 0.85 to 1 based on health care professionals' ratings. The mean score exceeded the threshold of four for all items of the sensibility questionnaire. Based on child, parent, and health care professional recommendations, 16 items were added, six eliminated, and 15 items modified from the original CPCHILD. The MDCHILD consists of 47 items over seven domains. INTERPRETATION: The MDCHILD met all sensibility criteria by children with DMD, their parents, and health care professionals, and is ready for psychometric evaluation. WHAT THIS PAPER ADDS: The Muscular Dystrophy Child Health Index of Life with Disabilities (MDCHILD) is a new patient-reported outcome measure for Duchenne muscular dystrophy (DMD). The Priority Framework of Outcomes underpins the content for the MDCHILD. The MDCHILD incorporates the health-related priorities of males with DMD and their parents. The MDCHILD was deemed sensible by children, their parents, and health care professionals.
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Niños con Discapacidad/psicología , Distrofia Muscular de Duchenne/psicología , Medición de Resultados Informados por el Paciente , Encuestas y Cuestionarios , Adolescente , Cuidadores , Niño , Preescolar , Niños con Discapacidad/rehabilitación , Personal de Salud , Humanos , Entrevistas como Asunto , Masculino , Distrofia Muscular de Duchenne/diagnóstico , Distrofia Muscular de Duchenne/terapia , PadresRESUMEN
BACKGROUND: Implants are commonly used to stabilize proximal femoral osteotomies in children with cerebral palsy (CP). Removal of implants is common practice and believed to avoid infection, fracture, or pain that might be associated with retained hardware. There is little evidence to support a prophylactic strategy over a reactive approach based on symptoms. The aim of this study was to compare the outcomes of prophylactic and reactive approaches to removal of proximal femoral implants in children with CP. METHODS: An intention-to-treat model was used to compare 2 institutions that followed a prophylactic (within â¼1 y) and reactive (following complication/symptoms) approach to hardware removal, respectively. Patients with CP who had femoral implants placed at or before age 16, and had ≥2-year postsurgical follow-up were included. Demographics, surgical details, reasons for removal, and complications were recorded. χ and t tests were used. RESULTS: Six hundred twenty-one patients (prophylactic=302, reactive=319) were followed for an average of 6 years (range, 2 to 17 y). Two hundred eighty-seven (95%) implants were removed in the prophylactic group at 1.2 years. In the reactive group, 64 (20%) implants were removed at an average of 4.2 years. Reasons for removal included pain; infection; fracture; or for repeat reconstruction. The rate of unplanned removals due to fracture or infection was higher in the reactive group (4.7% vs. 0.7%, P=0.002), but there was no difference in the rate of complications during/after removal between the 2 groups (1.7% vs. 3.1%; P=0.616). No specific risk factor associated with unplanned removal could be identified; but children under 8 years old seemed more likely to undergo later removal (odds ratio 1.98; 95% confidence interval, 0.99-3.99). CONCLUSIONS: Eighty percent of patients in the reactive removal strategy avoided surgery. This group did have a 4% higher rate of fracture or infection necessitating unplanned removal but these were successfully treated at time of removal with no difference in complication rates associated with removal between both groups. One would need to remove implants from 25 patients to avoid 1 additional complication, providing some support for a reactive approach to removal of proximal femoral implants in this population. LEVEL OF EVIDENCE: Level III-therapeutic.
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Parálisis Cerebral/cirugía , Remoción de Dispositivos/métodos , Fémur/cirugía , Osteotomía , Complicaciones Posoperatorias , Procedimientos Quirúrgicos Profilácticos/métodos , Adolescente , Niño , Femenino , Humanos , Masculino , Osteotomía/efectos adversos , Osteotomía/métodos , Evaluación de Procesos y Resultados en Atención de Salud , Complicaciones Posoperatorias/etiología , Complicaciones Posoperatorias/cirugía , Prótesis e Implantes/efectos adversos , Reoperación/métodos , Reoperación/estadística & datos numéricos , Factores de RiesgoRESUMEN
INTRODUCTION: Children with cerebral palsy are at risk for progressive hip displacement. Since surveillance for hip displacement uses specific radiographic measurements to guide decision making, it is important to establish the reliability of these measurements, which include Reimer's migration percentage (MP), acetabular index or acetabular angle (AI or AA), and pelvic obliquity (PO). The purpose of this study was to determine the intraobserver and interobserver reliability of these radiographic measures among an international group of pediatric orthopaedic surgeons participating in the prospective international multicenter Cerebral Palsy Hip Outcomes Project (CHOP) currently underway to evaluate the outcomes of hip interventions in cerebral palsy. METHODS: Two compact discs (CDs) containing the same 25 anteroposterior pelvis radiographs in Digital Imaging and Communications in Medicine (DICOM) format were provided to participating surgeons at least 2 weeks apart. To reduce the likelihood of recall or any effects of learning or fatigue, the order of the radiographs varied on the 2 CD versions, and participating surgeons received the 2 CDs in random order. The intraclass correlation coefficients (ICCs) were calculated to assess interobserver and intraobserver reliability. Mean absolute differences of hip measurements obtained at 2 time points were also calculated. RESULTS: The MP had the highest reliability followed by PO, AI, and AA with a mean intrarater ICC (SD; range) of 0.95 (0.04; 0.84 to 0.98); 0.92 (0.03; 0.85 to 0.97); 0.84 (0.05; 0.75 to 0.92); and 0.82 (0.14; 0.51 to 0.98); respectively. The mean interrater ICC (SD; range) for MP, PO, AI, and AA were 0.94 (0.05; 0.78 to 0.99); 0.90 (0.04; 0.76 to 0.99); 0.79 (0.08; 0.52 to 0.93); and 0.69 (0.23; 0.42 to 0.98) for MP, PO, AI, and AA, respectively. The mean (SD; 95% confidence interval) for the absolute difference between the 2 measurements for the raters was 4.9% (2.9%; 3.4%-6.4%); 3. 8 degrees (1.2 degrees; 3.1-4.5 degrees); 2.6 degrees (1.5 degrees; 1.7-3.5 degrees); and 1.3 degrees (0.3 degrees; 1.29-1.31 degrees) for MP, AI, AA, and PO, respectively. CONCLUSIONS: MP is a reproducible measure with excellent intrarater and interrater reliability. However, differences in MP of <7% should be treated with caution as these might be a consequence of measurement error. Although we found a high level of intrarater and interrater reliability of the AI, AA, and PO, these measurements are more variable and not ideal for use as discrete outcome measures. Instead, these parameters might be useful for prognostication and decision making when consistent trends are observed longitudinally over time which might be better indications of true change.
Asunto(s)
Parálisis Cerebral , Luxación de la Cadera , Pelvis/diagnóstico por imagen , Radiografía/métodos , Parálisis Cerebral/complicaciones , Parálisis Cerebral/diagnóstico , Niño , Europa (Continente) , Luxación de la Cadera/diagnóstico , Luxación de la Cadera/etiología , Humanos , Variaciones Dependientes del Observador , Ortopedia/métodos , Evaluación de Resultado en la Atención de Salud , Pediatría/métodos , Pronóstico , Estudios Prospectivos , Reproducibilidad de los ResultadosRESUMEN
Demonstration of monoclonal immunoglobulin molecule in serum forms the mainstay in the diagnosis of monoclonal gammopathies. The major tests that help in this regard are serum protein electrophoresis (SPEP), serum immunofixation electrophoresis (sIFE) and serum free light chain assay (sFLC). Our objectives were to study the accuracy of sFLC and sIFE in the diagnosis of monoclonal gammopathies and also to study the role of combination of SPEP + sIFE + sFLC in the diagnosis of the same. 46 patients who attended the hemato-oncology clinic with signs and symptoms suggestive of monoclonal gammopathy were enrolled in this study. SPEP, sIFE, sFLC and pre-treatment serum beta-2 microglobulin levels were analysed among the study population. Both SPEP and sIFE were performed in the Interlab Genios fully automated machine. Serum beta-2 microglobulin and sFLC were estimated by immunoturbidimetry in Beckman Coulter AU 2700 analyzer. The accuracy of sIFE came to be 80% with respect to sFLC assay. Sensitivity, specificity, positive and negative predictive value of sIFE with respect to sFLC were 81.3, 78.6, 89.7 and 64.7% respectively. It was observed that a combination panel of SPEP + sIFE + sFLC could detect all the cases of myeloma included in this study. Further testing in large samples is required for generalising the findings of this study. The pre-treatment beta-2 microglobulin levels were significantly higher in the group which was positive for myeloma. A combination panel of SPEP + sIFE + sFLC prove to be more useful than individual tests for the detection of myeloma.
RESUMEN
AIM: We investigated the validity of the Gait Outcomes Assessment List (GOAL), as an assessment of gait function in children with cerebral palsy (CP). METHOD: We studied a prospective cohort of 105 children with CP (Gross Motor Function Classification System [GMFCS] levels I-III; 65 males, 40 females; mean [SD] age 11y 11mo [3y 5mo], range 6-20y), who attended gait assessment over a 10-month period. Parents completed the GOAL, Functional Mobility Scale (FMS), and Functional Assessment Questionnaire (FAQ) during their child's gait evaluation. Ninety children completed instrumented gait analysis (IGA). Total GOAL and domain scores, Gait Profile Score (GPS), and Gait Variable Scores were calculated. RESULTS: The total GOAL discriminated between GMFCS levels (mean [SD] GMFCS level I, 72.5 [12.7]; GMFCS level II, 61.4 [13.0]; GMFCS level III, 38.8 [10.6]; [F2,97 =42.4, p<0.001]). Moderate correlations were found between total GOAL and FMS (5m and 50m r=0.59; 500m r=0.66) and FAQ walking (r=0.77) and activities list (r=0.75, p<0.01). There was a moderate negative correlation between total GOAL and GPS (r=-0.59) and gait appearance domain and GPS (r=-0.52, p<0.01). INTERPRETATION: The GOAL is a valid assessment of gait function in ambulant children with CP. It has the potential to improve understanding of the child's and parents' priorities and thus, in conjunction with IGA, provide a more balanced assessment across the domains of the World Health Organization's International Classification of Functioning, Disability and Health. WHAT THIS PAPER ADDS: The Gait Outcomes Assessment List (GOAL) can discriminate between Gross Motor Function Classification System levels. The GOAL correlates with standard functional assessments and gait analysis. Used with gait analysis, the GOAL provides comprehensive assessment across all International Classification of Functioning, Disability and Health domains.