Longitudinal Course of Long Finger Flexor Shortening in Males with Duchenne Muscular Dystrophy: A Retrospective Review1.

BACKGROUND: Shortening of the long finger flexors (Flexor Digitorum Profundus, FDPs) in Duchenne Muscular Dystrophy (DMD) causes reduced hand function. Until now, longitudinal studies on the natural course of the shortening of the FDPs are lacking, which impedes recommendations on timing and evaluation of preventive measures. OBJECTIVE: To investigate the longitudinal course of the FDP length during different disease stages focusing on symmetry, timing, and decline of the FDP length. METHODS: A retrospective, longitudinal multicenter study was conducted in the Radboud university medical center and the Leiden university medical center. The FDP outcome was measured using goniometry and gross motor function was assessed using the Brooke score. Longitudinal mixed model analyses were used to describe the course of the FDP outcome, and to investigate symmetry in both hands. RESULTS: Data on 534 visits of 197 males (age ranged 4-48 years) showed that in the ambulatory stages the FDP outcome was within a normal range. The mean decline in FDP outcome is 3.5 degrees per year, the biggest decline was seen in Brooke 5 (>15 degrees per year). In Brooke 4, 41% of the FDP outcome was < 40 degrees. No significant differences were found between right and left. CONCLUSIONS: This study supports the consideration of preventive measures to delay shortening of the FDPs in DMD patients transitioning to a Brooke scale of 4 or higher. Besides, natural history of FDP outcome has been established, which provides a base to evaluate (preventive) interventions.

Differences in the perspectives of functioning and health in the ICF model between patients with brachial plexus birth injury and their parents versus healthcare professionals.

PURPOSE: To explore and compare the perspectives of patients and their parents (PPs) with a brachial plexus birth injury (BPBI) with those of health care professionals (HCPs). MATERIALS AND METHODS: We conducted a study using a questionnaire among PPs and HCPs. Importance of different outcome categories was scored on a Likert scale. Items were linked to corresponding categories of the International Classification of Functioning, Disability and Health. Means were compared using analysis of variance (ANOVA). RESULTS: Data were collected from 184 patients and 65 HCPs. We found a difference in 7/14 outcome categories between joint PP groups and HCPs. Parents scored outcome evaluation categories as more important than patients, categories filled out together by patient and parent scored in between (p < 0.05). The majority of PPs and HCPs rated outcome assessment as important in more domains than "Body functions" and "Body structures". The biggest difference was found in the importance of evaluation of pain. CONCLUSIONS: Outcome assessment in the domains "Activities and participation" and "Environmental factors" was rated as important by both PPs and HCPs. Evaluation of pain was more often scored as important by PPs. Different domains seem to be underestimated by HCPs and need more attention during consultation. Implications for rehabilitationThe importance of outcome evaluation concerning "Pain", "Interaction with peers", and "Interaction with medical and paramedical specialists" should be taken into account in the rehabilitation for this specific group of patients and their parents (PPs).Comparison of perspectives on functioning between brachial plexus birth injury (BPBI) PPs and health care professionals.Outcome assessment in domains "Activities and participation" and "Environmental factors" is important.Health care professionals undervalue the importance of pain evaluation in BPBI as compared with PPs.

The Relation Between Nutritional Intake and Body Weight in 4-18 Year Old Patients with DMD: What could possibly be done to prevent weight gain?

BACKGROUND: Overweight is a common problem in Duchenne muscular dystrophy (DMD) and is associated with reduced mobility and quality of life. The influence of nutritional intake on (over)weight is unclear. OBJECTIVE: To investigate weight and energy and macronutrients intake compared to age-specific requirements in DMD patients (4-18 years). METHODS: We assessed weight and body mass index (BMI) and the amount of energy (kcal/day) and macronutrients based on self-reported nutrition diaries. Nutritional intake was compared to requirements for 3 age-groups according to the Dutch Healthy Diet Guideline (4-8/9-13/14-18 years) using a student's t-test, and relations with age and BMI were investigated by means of Pearson's correlations. RESULTS: Forty-eight patients participated, 22 ambulatory, median age 10.8 years. The majority used corticosteroids (N = 41). Overweight (BMI z-score > 2.07) was present in 19 patients; 6% (4-8 years), 73% (9-13 years) and 47% (14-18 years). Overweight was more common in non-ambulatory (61.6%) than ambulatory patients (13.6%). Patients aged 4-8 received 290 kcal/day more than required (p < 0.001). Patients aged 9-13 received 349 kcal/day (p = 0.005) less than required. Overall, intake of fibre, nuts, meat/fish/eggs/legumes and dairy was lower than recommended (p < 0.05). The difference between energy intake versus requirement correlated moderately to age (r = -0.549, p < 0.001) and BMI (r = -0.562, p < 0.001). CONCLUSIONS: Overweight was found especially in patients aged 9-18 even though they received less energy than required. Younger patients (4-8) had good weight but consumed more energy than required. All patients did not consume enough fibre, nuts, meat/fish/eggs/legumes and dairy. Limiting energy and increasing fibre/protein intake at an early age may prevent overweight at a later age.

Fatigue in young patients with acquired brain injury in the rehabilitation setting: Categorizing and interpreting fatigue severity levels.

PURPOSE: Fatigue in patients with acquired brain injury (ABI) is common. However, to better target fatigue, clear ways to categorize/interpret fatigue-severity in individual patients are lacking. This study aims to determine/categorize fatigue severity among children, adolescents, and young adults with ABI. METHODS: This cross-sectional study included young patients admitted to outpatient rehabilitation and their parents. To determine fatigue, the PedsQL™Multidimensional-Fatigue-Scale was used (MFS, scores 0-100, lower scores = higher fatigue, patient-/parent-reported). Based on scores from a reference population, four categories were formed: "1 = no/little fatigued" to "4 = severely-more fatigued." RESULTS: All scores were lower than those from the reference population, with comparisons in the adolescent and young adult groups reaching statistical significance (p < .05). The proportions of patients in category 4 were: 9%/50%/58% among children/adolescents/young adults, showing that many patients were "severely-more fatigued"-than the reference population. CONCLUSIONS: Measuring fatigue and categorizing fatigue severity looks promising for clinical practice and could help to better target fatigue.

The Black Box of Technological Outcome Measures: An Example in Duchenne Muscular Dystrophy.

BACKGROUND: Outcome measures for non-ambulant Duchenne muscular dystrophy (DMD) patients are limited, with only the Performance of the Upper Limb (PUL) approved as endpoint for clinical trials. OBJECTIVE: We assessed four outcome measures based on devices developed for the gaming industry, aiming to overcome disadvantages of observer-dependency and motivation. METHODS: Twenty-two non-ambulant DMD patients (range 8.6-24.1 years) and 14 healthy controls (HC; range 9.5-25.4 years) were studied at baseline and 16 patients at 12 months using Leap Motion to quantify wrist/hand active range of motion (aROM) and a Kinect sensor for reached volume with Ability Captured Through Interactive Video Evaluation (ACTIVE), Functional Workspace (FWS) summed distance to seven upper extremity body points, and trunk compensation (KinectTC). PUL 2.0 was performed in patients only. A stepwise approach assessed quality control, construct validity, reliability, concurrent validity, longitudinal change and patient perception. RESULTS: Leap Motion aROM distinguished patients and HCs for supination, radial deviation and wrist flexion (range p = 0.006 to <0.001). Reliability was low and the manufacturer's hand model did not match the sensor's depth images. ACTIVE differed between patients and HCs (p < 0.001), correlated with PUL (rho = 0.76), and decreased over time (p = 0.030) with a standardized response mean (SRM) of -0.61. It was appraised as fun on a 10-point numeric rating scale (median 9/10). PUL decreased over time (p < 0.001) with an SRM of -1.28, and was appraised as fun (median 7/10). FWS summed distance distinguished patients and HCs (p < 0.001), but reliability in patients was insufficient. KinectTC differed between patients and HCs (p < 0.01), but correlated insufficiently with PUL (rho = -0.69). CONCLUSIONS: Only ACTIVE qualified as potential outcome measure in non-ambulant DMD patients, although the SRM was below the commonly used threshold of 0.8. Lack of insight in technological constraints due to intellectual property and software updates made the technology behind these outcome measures a kind of black box that could jeopardize long-term use in clinical development.

Measuring treatment outcome in children with developmental coordination disorder; responsiveness of six outcome measures.

PURPOSE: Although measuring outcome of rehabilitation in children with Developmental Coordination Disorder is considered important no consensus exists on which instruments to use. An important attribute of a measurement instrument would be that it is sensitive to clinical changes. The aim of this prospective, observational study was therefore to investigate the responsiveness of six potentially suitable instruments. METHODS: Forty-one children (34 boys, median age 7.8 years, Inter Quartile Range: 7.2-9.2) receiving multidisciplinary rehabilitation treatment for Developmental Coordination Disorder were included (mean treatment time: 32.8 h, Standard Deviation 7.3). The following instruments were applied before and after rehabilitation: Movement-Asessment-Battery-Children-2 (MABC-2), Canadian Occupational Performance Measure (COPM), Systematic detection writing problems (SOS-2-NL), DCD-daily, Behaviour Rating Inventory of Executive Function (BRIEF), and TNO-AZL children's Quality of Life questionnaire (TACQOL)). Change-scores (paired t-test/Wilcoxon-test) and responsiveness (Effect-sizes and Standardized-Response-Means) were calculated. RESULTS: Significant differences over time were found for the Canadian Occupational Performance Measure, DCDdaily and Movement-Asessment-Battery-Children-2 (p < 0.05). The responsiveness of these instruments was moderate-high (Canadian Occupational Performance Measure-performance Effect-Size:1.70/Standardized-Response-Mean:1.81, Canadian Occupational Performance Measure-satisfaction Effect-Size:1.65/Standardized-Response-Mean 1.53; DCDdaily-total-score Effect-Size:0.40/Standardized-Response-Mean:0.62, DCDdaily-Quality-score Effect-Size:0.74/Standardized-Response-Mean:0.89, DCDdaily-time-score Effect-Size:0.21/Standardized-Response-Mean:0.43; MABC-2-total-score Effect-Size:0.42/Standardized-Response-Mean:0.43, MABC-2-Ball-skills-score Effect-Size:0.33/Standardized-Response-Mean:0.36). Systematic detection of writing problems (SOS-2-NL), Behaviour Rating Inventory of Executive Function (BRIEF) and TNO-AZL children's Quality of Life questionnaire (TACQOL) were not responsive to change. CONCLUSION: Although the Movement-Asessment-Battery-Children-2 test is the most widely used instrument when measuring rehabilitation outcome in Developmental Coordination Disorder, the Canadian Occupational Performance Measure and DCDdaily seem to be more responsive and constitute a valuable addition.Implications for rehabilitationCurrently, there is no consensus on the preferred measurement instruments to evaluate the outcomes of rehabilitation in children with Developmental Coordination Disorder.The responsiveness of the Canadian Occupational Performance Measure (COPM) was found to be large, of the DCDdaily moderate to large, and of the Movement-Assessment-Battery-Children-2 (MABC-2) small, whereas the systematic detection of writing problems (SOS-2-NL; Systematisch Opsporing Schrijfproblemen-2-NL), Behavior Rating Inventory of Executive Functioning (BRIEF) and TNO-AZL Questionnaire for Children's Health-Related Quality of Life (TACQOL) were not responsive to change.Although the Movement-Assessment-Battery-Children-2 (MABC-2) is commonly used in Developmental Coordination Disorder rehabilitation, applying the Canadian Occupational Performance Measure (COPM) and DCDdaily should be considered, as these instruments were more sensitive to clinical changes.

Association of Elbow Flexor MRI Fat Fraction With Loss of Hand-to-Mouth Movement in Patients With Duchenne Muscular Dystrophy.

BACKGROUND AND OBJECTIVES: To study the potential of quantitative MRI (qMRI) fat fraction (FF) as a biomarker in nonambulant patients with Duchenne muscular dystrophy (DMD), we assessed the additive predictive value of elbow flexor FF to age at loss of hand-to-mouth movement. METHODS: Nonambulant patients with DMD (age ≥8 years) were included. Four-point Dixon MRI scans of the right upper arm were performed at baseline and at the 12-, 18-, or 24-month follow-up. Elbow flexor FFs were determined from 5 central slices. Loss of hand-to-mouth movement was determined at study visits and by phone calls every 4 months. FFs were fitted to a sigmoidal curve by use of a mixed model with random slope to predict individual trajectories. The added predictive value of elbow flexor FF to age at loss of hand-to-mouth movement was calculated from a Cox model with the predicted FF as a time-varying covariate, yielding a hazard ratio. RESULTS: Forty-eight MRIs of 20 patients with DMD were included. The hazard ratio of a percent-point increase in elbow flexor FF for the time to loss of hand-to-mouth movement was 1.12 (95% confidence interval 1.04-1.21; p = 0.002). This corresponded to a 3.13-fold increase in the instantaneous risk of loss of hand-to-mouth movement in patients with a 10-percent points higher elbow flexor FF at any age. DISCUSSION: In this prospective study, elbow flexor FF predicted loss of hand-to-mouth movement independently of age. qMRI-measured elbow flexor FF can be used as a surrogate endpoint or stratification tool for clinical trials in nonambulant patients with DMD. CLASSIFICATION OF EVIDENCE: This study provides Class II evidence that qMRI FF of elbow flexor muscles in patients with DMD predicts loss of hand-to-mouth movement independently of age.

Aging With Cerebral Palsy: A Photovoice Study Into Citizenship.

Background: Adults with cerebral palsy (CP) may experience an increasing impact of their disability on daily life and this may interfere with their citizenship. Citizenship is a layered construct. Next to formal and theoretical significations, and civil rights acts such as the UN Convention on the Rights for Persons with Disabilities (CRPD), the meaning of citizenship is formed by the person themselves. The present study aimed to gain insight into what citizenship means for adults with CP 40 years or older and what is needed to support and pursue their citizenship to improve person-centered rehabilitation which can facilitate this process. Methods: Adults with CP (>40 years) without intellectual disability were recruited from medical records of a large rehabilitation center to participate in a qualitative study using the photovoice method. Participants were asked to take photos of objects or life situations that constituted citizenship for them; these photos were then the prompts for the semi-structured interviews that were held face-to-face at their homes. Background and clinical characteristics were gathered using a short face-to-face questionnaire. Data were analyzed through inductive thematic analysis. Results: Nineteen adults participated [mean age (SD) 57.8 (9.4) years (range 44-79), six men]. From the analysis four themes emerged: (a) Meanings of citizenship; (b) Citizenship: Facilitator and barriers; (c) Paradoxes of support and participation; and (d) Future. Furthermore, next to the ability to participate in society without restrictions, sense of belonging was reported to be an important aspect of "meanings of citizenship." The physiotherapist was perceived as an important health professional to maintain physical activity and deal with the impact of aging with CP on daily activities. Complex healthcare and support services regulations and aging affected citizenship negatively. Conclusion: Middle-aged and older adults with CP view citizenship as the ability to participate and belong in society. To optimize their citizenship the challenges and individual needs must be seen and supported by person-centered rehabilitation and support services. Simplification of complex healthcare and services regulations can further improve citizenship.

Preserved thenar muscles in non-ambulant Duchenne muscular dystrophy patients.

BACKGROUND: Clinical trials in Duchenne muscular dystrophy (DMD) focus primarily on ambulant patients. Results cannot be extrapolated to later disease stages due to a decline in targeted muscle tissue. In non-ambulant DMD patients, hand function is relatively preserved and crucial for daily-life activities. We used quantitative MRI (qMRI) to establish whether the thenar muscles could be valuable to monitor treatment effects in non-ambulant DMD patients. METHODS: Seventeen non-ambulant DMD patients (range 10.2-24.1 years) and 13 healthy controls (range 9.5-25.4 years) underwent qMRI of the right hand at 3 T at baseline. Thenar fat fraction (FF), total volume (TV), and contractile volume (CV) were determined using 4-point Dixon, and T2water was determined using multiecho spin-echo. Clinical assessments at baseline (n = 17) and 12 months (n = 13) included pinch strength (kg), performance of the upper limb (PUL) 2.0, DMD upper limb patient reported outcome measure (PROM), and playing a video game for 10 min using a game controller. Group differences and correlations were assessed with non-parametric tests. RESULTS: Total volume was lower in patients compared with healthy controls (6.9 cm3 , 5.3-9.0 cm3 vs. 13.0 cm3 , 7.6-15.8 cm3 , P = 0.010). CV was also lower in patients (6.3 cm3 , 4.6-8.3 cm3 vs. 11.9 cm3 , 6.9-14.6 cm3 , P = 0.010). FF was slightly elevated (9.7%, 7.3-11.4% vs. 7.7%, 6.6-8.4%, P = 0.043), while T2water was higher (31.5 ms, 30.0-32.6 ms vs. 28.1 ms, 27.8-29.4 ms, P < 0.001). Pinch strength and PUL decreased over 12 months (2.857 kg, 2.137-4.010 to 2.243 kg, 1.930-3.339 kg, and 29 points, 20-36 to 23 points, 17-30, both P < 0.001), while PROM did not (49 points, 36-57 to 44 points, 30-54, P = 0.041). All patients were able to play for 10 min at baseline or follow-up, but some did not comply with the study procedures regarding this endpoint. Pinch strength correlated with TV and CV in patients (rho = 0.72 and rho = 0.68) and controls (both rho = 0.89). PUL correlated with TV, CV, and T2water (rho = 0.57, rho = 0.51, and rho = -0.59). CONCLUSIONS: Low thenar FF, increased T2water , correlation of muscle size with strength and function, and the decrease in strength and function over 1 year indicate that the thenar muscles are a valuable and quantifiable target for therapy in later stages of DMD. Further studies are needed to relate these data to the loss of a clinically meaningful milestone.

Participation Restrictions among Children and Young Adults with Acquired Brain Injury in a Pediatric Outpatient Rehabilitation Cohort: The Patients' and Parents' Perspective.

Improving participation is an important aim in outpatient rehabilitation treatment. Knowledge regarding participation restrictions in children and young adults with acquired brain injury (ABI) is scarce and little is known regarding the differences in perspectives between patients and parents in the outpatient rehabilitation setting. The aims are to describe participation restrictions among children/young adults (5-24 years) with ABI and investigating differences between patients' and parents' perspectives. At admission in 10 rehabilitation centers, patients and parents were asked to complete the Child and Adolescent Scale of Participation (CASP; score 0-100; lower score = more restrictions) and injury/patient/family-related questions. CASP scores were categorized (full/somewhat-limited/limited/very-limited participation). Patient/parent-reported outcomes were compared using the Wilcoxon signed-rank test. 223 patients and 245 parents participated (209 paired-samples). Median patients' age was 14 years (IQR; 11-16), 135 were female (52%), 195 had traumatic brain injury (75%). The median CASP score reported by patients was 82.5 (IQR: 67.5-90) and by parents 91.3 (IQR: 80.0-97.5) (difference = p < 0.05). The score of 58 patients (26%) and 25 parents (10%) was classified as 'very-limited'. Twenty-six percent of children and young adults referred for rehabilitation after ABI had "very-limited" participation. Overall, parents rated their child's participation better than patients themselves. Quantifying participation restrictions after ABI and considering both perspectives is important for outpatient rehabilitation treatment.

Health-care use and information needs of children with neonatal brachial plexus palsy: A cross-sectional survey among 465 Dutch patients.

To investigate health-care use (HCU) and information needs of children aged 0-18 years with neonatal brachial plexus palsy (NBPP), a cross-sectional study was performed. Patients and/or parents seen in our NBPP clinic were invited to complete a survey comprising questions on HCU due to NBPP and current information needs. Outcomes were described for three age-groups (0-1/2-9/10-18 years), based on follow-up status (early/late/no-discharge). Four hundred sixty-five parents/patients participated (59 in the 0-1, 226 in the 2-9, and 180 in the 10-18-year group). Two hundred ninety-three patients had C5-C6 lesions, 193 were discharged from follow-up, 83 of whom categorized as 'early discharged' (<1 year of age). Over the past year, 198 patients had contact with the expert team (49 in the 0-1, 81 in the 2-9, and 68 in the 10-18-year group) and 288 with at least one other health-care professional (53 in the 0-1, 133 in the 2-9, and 102 in the 10-18-year group). Of the 83 patients discharged early, 34 reported health-care use. Two hundred twenty-eight participants reported current information needs of whom 23 were discharged early. HCU and information needs of Dutch children with NBPP remains considerable even in children who were discharged. Stricter follow-up and information provision for these patients is needed.

Fatigue, Participation and Quality of Life in Adolescents and Young Adults with Acquired Brain Injury in an Outpatient Rehabilitation Cohort.

PURPOSE: To study the association between fatigue and participation and QoL after acquired brain injury (ABI) in adolescents and young adults (AYAs). MATERIALS & METHODS: Cross-sectional study with AYAs aged 14-25 years, diagnosed with ABI. The PedsQL™ Multidimensional Fatigue Scale, Child & Adolescent Scale of Participation, and PedsQL™4.0 Generic Core Scales were administered. RESULTS: Sixty-four AYAs participated in the study, 47 with traumatic brain injury (TBI). Median age at admission was 17.6 yrs, 0.8 yrs since injury. High levels of fatigue (median 44.4 (IQR 34.7, 59.7)), limited participation (median 82.5 (IQR 68.8, 92.3)), and diminished QoL (median 63.0 (IQR 47.8, 78.3)) were reported. More fatigue was significantly associated with more participation restrictions (ß 0.64, 95%CI 0.44, 0.85) and diminished QoL (ß 0.87, 95%CI 0.72, 1.02). CONCLUSIONS: AYAs with ABI reported high levels of fatigue, limited participation and diminished quality of life with a significant association between fatigue and both participation and QoL. Targeting fatigue in rehabilitation treatment could potentially improve participation and QoL.

Hand-Use-at-Home Questionnaire: validity and reliability in children with neonatal brachial plexus palsy or unilateral cerebral palsy.

OBJECTIVE: To investigate construct validity and test-retest reliability of the parent-rated Hand-Use-at-Home questionnaire (HUH) in children with neonatal brachial plexus palsy or unilateral cerebral palsy. DESIGN AND SUBJECTS: For this cross-sectional study, children with neonatal brachial plexus palsy or unilateral cerebral palsy, aged 3-10 years, were eligible. MAIN MEASURES: The HUH, Pediatric Outcome Data Collection Instrument Upper Extremity Scale (neonatal brachial plexus palsy only), and Children's Hand-Use Experience Questionnaire (unilateral cerebral palsy only) were completed. The HUH was completed twice in subgroups of both diagnoses. Lesion-extent (indication of involved nerve rootlets in neonatal brachial plexus palsy as confirmed during clinical observation and/or nerve surgery) and Manual Ability Classification System levels (unilateral cerebral palsy) were obtained from the medical records. Spearman correlation coefficients between the HUH and all clinical variables, agreement, standard error of measurement, smallest detectable change and intra-class correlation were calculated. RESULTS: A total of 260 patients participated (neonatal brachial plexus palsy: 181), of which 56 completed the second HUH (neonatal brachial plexus palsy: 16). Median age was 6.9 years for children with neonatal brachial plexus palsy, 116 had C5-C6 lesions. Median age for children with unilateral cerebral palsy was 6.4 years, 33 had Manual Ability Classification System Level II. The HUH correlated moderately with lesion-extent ( rs =-0.5), Pediatric Outcome Data Collection Instrument Upper Extremity Scale ( rs = 0.6) and Children's Hand-Use Experience Questionnaire ( rs = 0.5) but weakly with Manual Ability Classification System levels ( rs = -0.4). Test-retest reliability was excellent (intra-class correlation2,1 = 0.89, standard error of measurement = 0.599 and smallest detectable change = 1.66 logits) and agreement was good (mean difference HUH1 - HUH2 = 0.06 logits). CONCLUSION: The HUH showed good construct validity and test-retest reliability in children with neonatal brachial plexus palsy or unilateral cerebral palsy.

Participation restrictions among adolescents and adults with neonatal brachial plexus palsy: the patient perspective.

PURPOSE: To examine the impact of neonatal brachial plexus palsy (NBPP) on societal participation of adolescents and adults. METHODS: This cross-sectional study was conducted among patients with NBPP, aged ≥16 years, who had visited our NBPP clinic. Patients completed questions on the influence of NBPP on their choices regarding education/work and their work-performance, the Impact on Participation/Autonomy questionnaire and the Utrecht Scale for Evaluation of Rehabilitation-Participation (USER-P). In addition, health-related quality of life (HRQoL) was assessed. RESULTS: Seventy-five patients participated (median age 20, inter quartile range 17-27). Twenty were full-time students, 28 students with a job, 21 employed, two unemployed, and four work-disabled. Sixty-six patients had had a job at some stage. Patients' overall HRQoL was comparable to the general population. 27/75 patients reported that NBPP had affected their choices regarding education and 26/75 those regarding work. 33/66 reported impact on their work performance. On the Impact on Participation/Autonomy questionnaire, 80% (49/61) reported restrictions in the work-and-education domain, 74% in social-relations and 67% in autonomy-outdoors. 37/61 reported participation restrictions on the USER-P. CONCLUSIONS: Although their overall HRQoL was not impaired, a substantial proportion of adolescent/adult patients reported that NBPP had an impact on choices regarding education and profession, as well as on work-performance. Restrictions in participation, especially in work and education were also reported. Guiding patients in making choices on education and work at an early stage and providing tailored physical as well as psychosocial care may prevent or address restrictions, which may improve participation. Implications for Rehabilitation Adolescent and adult patients with neonatal brachial plexus palsy perceive restrictions in societal participation, especially regarding the work-and-education domain. All patients with neonatal brachial plexus palsy may perceive restrictions in societal participation regardless of lesion severity, treatment history and side of the lesion. Adolescents and adults with neonatal brachial plexus palsy report that their choices regarding education and work, as well as their work-performance are influenced by their neonatal brachial plexus palsy. Patients with neonatal brachial plexus palsy should be followed throughout their life in order to provide them with appropriate information and treatment when health- or participation-related issues arise. Rehabilitation treatment is the best option to address all of the aforementioned issues, as surgical options in adolescents and adults are limited.

Development and psychometric properties of the Hand-Use-at-Home questionnaire to assess amount of affected hand-use in children with unilateral paresis.

AIM: To describe the development of the parent-rated Hand-Use-at-Home questionnaire (HUH) assessing the amount of spontaneous use of the affected hand in children with unilateral paresis, and to test its internal structure, unidimensionality, and validity. METHOD: Parents of children with unilateral cerebral palsy (CP) and professionals participated in the development of the HUH. To examine internal validity, data of 322 children (158 males, 164 females; mean age 6y 7mo, standard deviation [SD] 2y 1mo) with unilateral CP (n=131) or neonatal brachial plexus palsy (NBPP) (n=191) were collected. Rasch analysis was used to examine discriminative capacity of the 5-category rating scale as well as unidimensionality and hierarchy of the item set. Additionally, data of 55 children with typical development (24 males, 31 females; 6y 9mo, SD 2y 5mo) were used to examine construct validity. RESULTS: The 5-category rating scale was disordered in all items and was collapsed to obtain the best discriminating sum score. Ten misfitting or redundant items were removed. Eighteen hierarchically ordered bimanual items fitted the unidimensional model within acceptable range. The HUH significantly discriminated between the three groups (children with typical development, NBPP, unilateral CP; H(2) =118.985, p<0.001), supporting its construct validity. INTERPRETATION: The HUH is a valid instrument to assess the amount of spontaneous use of the affected hand in children with unilateral upper-limb paresis.

Neonatal Brachial Plexus Palsy in Children Aged 0 to 2.5 Years; Parent-Perceived Family Impact, Quality of Life, and Upper Extremity Functioning.

BACKGROUND: To investigate whether parents perceive impact of neonatal brachial plexus palsy on family and quality of life and upper extremity functioning in children less than 2.5 years. METHODS: This cross-sectional study used the PedsQL Family Impact Module (36 items/one total/four scales/scores 0 to 100), TNO-AZL (Dutch Organisation of Applied Natural Science and Academic Hospital Leiden) Preschool Children Quality of Life (43 items/12 scales/scores 0 to 100) and 21 upper extremity functioning questions. Associations between neonatal brachial plexus palsy/patient characteristics and family impact, perceived quality of life, and upper extremity functioning were investigated using regression analysis. RESULTS: Parents of 59 children (median age, 18 months) participated, 49 with C5-C6/C5-C7 lesions. Median Family Impact Module and TNO-AZL Preschool Children Quality of Life scores were 81.3 to 100.0/100.0 and 78.6 to 100.0/100.0. TNO-AZL Preschool Children Quality of Life scores did not differ significantly to healthy references except for stomach, skin, communication, and motor functioning problems. Parents reported around three upper extremity functioning problems. Greater lesion extent, lower age, still being in follow-up, and right-sided lesions were associated with greater family impact (P < 0.01 to P < 0.1). No clinically relevant associations were found for perceived quality of life. Greater lesion extent and nerve surgery history were associated with more upper extremity functioning problems (P < 0.01). Problems were associated with parental worrying (P < 0.05). CONCLUSIONS: Parents perceive having a child with neonatal brachial plexus palsy as impacting on their family depending on the side and severity of the lesion, treatment history, still being in follow-up, and age. They perceive the child's quality of life as relatively normal and not significantly different to healthy peers. However, parents noticed upper extremity functioning problems which increased parental worrying. Health care specialists should take these findings into account to better inform or counsel parents in an early stage during treatment.

Outcome of secondary shoulder surgery in children with neonatal brachial plexus palsy with and without nerve surgery treatment history: A long-term follow-up study.

OBJECTIVE: Irrespective of treatment history, shoulder dysfunction may occur in children with neonatal brachial plexus palsy. Following internal contracture release and/or muscle tendon transfer (ICR/MTT) shoulder function gain is possible. This study describes the outcomes of ICR/MTT for children with neonatal brachial plexus palsy, with or without prior nerve surgery (a group with prior nerve surgery and a group without prior nerve surgery). PATIENTS AND METHODS: The study included children who underwent an ICR/MTT with a minimum follow-up of 6 months. Active/passive range of motion (aROM/pROM)/Mallet scores were recorded (pre-operatively, 6 months, and 1, 3, 5 and 10 years post-surgery). Changes over time within groups were analysed using a linear mixed model. RESULTS: A total of 115 children (60 boys) were included, 82 with nerve surgery history, mean age 4.7 years (standard deviation (SD) 3.3 years), mean follow-up 6 years (SD 3.2 years). Pre-operatively active external rotation, abduction and forward-flexion were worse in the group with prior nerve surgery. aROM, pROM and Mallet scores, improved at all time-points in both groups. The course and magnitude of these improvements were largely similar in both groups. In the long-term, the effects of ICR/MTT decrease, but remain significant. CONCLUSION: In children with neonatal brachial plexus palsy shoulder function improved after ICR/MTT, irrespective of treatment history. Pre-operative shoulder function was worse in the group with prior nerve surgery, resulting in less function in this group after ICR/MTT. Reporting on outcome after secondary shoulder surgery should be stratified into children with and without prior nerve surgery, in order to prevent over- or underestimation of results. LEVEL OF EVIDENCE: This study concerned a retrospective treatment case series study. LEVEL OF EVIDENCE: IV.

Evaluation of shoulder function after secondary surgery in children with Neonatal Brachial Plexus Palsy.

PURPOSE: Shoulder function in children with Neonatal Brachial Plexus Palsy (NBPP) can be impaired. Functional gain is possible by an internal contracture release and muscle tendon transfer (ICL+MTT) for external rotation. This study evaluates the functional results of this intervention. METHODS: Assessments were done pre-operatively and 3, 6 and 12 months thereafter and included joint-mobility (ROM), muscle strength, arm function (Assisting Hand Assessment (AHA) and Mallet-score), Quality of Life (QoL) (Pediatric Outcome Data Collecting Instrument (PODCI)) and parental satisfaction. Changes were examined using Wilcoxon's Signed-Rank test and Cohen's effect size. RESULTS: Ten children (5 boys) aged 3-10 years who underwent a combined ICL+MTT (mm. Latissimus Dorsi/Teres Major) were included.Active and passive external rotation ROM and muscle strength improved (p < 0.05). Arm function improved according to the Mallet-score (Hand-to-Head, Hand-to-Mouth, External-Rotation) (p < 0.05) and the arm use and pace scales of the AHA (p < 0.05). The PODCI Upper Extremity/Physical Functioning and Global Functioning subscales also showed improvements (p < 0.05). Parents were highly satisfied concerning daily life activities and sports. CONCLUSION: ICL+MTT leads to improvement of ROM, strength, arm function, QoL and high parental satisfaction in this studies' patients and is therefore a good intervention to consider in children with NBPP with limited shoulder function.

Translation and adaptation of the Pediatric Outcome Data Collecting Instrument (PODCI) into the Dutch language and preliminary validation in children with Neonatal Brachial Plexus Palsy.

PURPOSE: This study aimed to translate and cross-culturally adapt the Pediatric Outcome Data Collecting Instrument (PODCI) into the Dutch language and evaluate its measurement properties among children (age 3-10) with Neonatal Brachial Plexus Palsy (NBPP). METHODS: The PODCI was translated and adapted according to international guidelines and administered to 10 children with NBPP before and after surgery and thereafter twice again. Subsequently, the Mallet-score, Assisting Hand Assessment and active Range of Motion (aROM) were recorded. Cronbach's-α and correlations between the PODCI and other outcome measures were determined, as well as Intraclass Correlation Coefficients (ICC). In addition, effect sizes (ES), Standard Response Means (SRM) and change scores with the 95% Confidence Interval (95% CI) were calculated. RESULTS: The final Dutch PODCI `Upper Extremity and Physical Function' subscale and total score `Global Functioning' showed good internal consistency (Cronbach's-α 0.695/0.781) and reliability (ICC 0.97/0.80) and were significantly associated with aROM and the Mallet-score. After surgery a significant change of the total score (ES 0.57, SRM 1.23, change 4.22 points, 95% CI 1.04-7.4) was seen. CONCLUSION: The final Dutch PODCI had good measurement properties and appears useful in evaluating quality of life and functioning in children with NBPP.

Concepts of functioning and health important to children with an obstetric brachial plexus injury: a qualitative study using focus groups.

AIMS: The aims of this study were to explore and understand the perspectives of children with an obstetric brachial plexus injury (OBPI) regarding functioning and health, and to create an overview of problems and difficulties that patients encounter in daily life. METHOD: We conducted a focus group study with 48 children (25 male, 23 female), aged 8 to 18 years, with an OBPI. Eleven open-ended questions regarding problems or difficulties in daily life were asked in group sessions with 4 to 7 children within the same age range. These group sessions were tape-recorded and transcribed verbatim. All problems and difficulties mentioned in each focus group were linked to corresponding categories of the International Classification of Functioning, Disability and Health - Children & Youth Version (ICF-CY). RESULTS: Eight focus groups were conducted. A total of 143 unique ICF-CY categories were identified. Of these categories, 61 (43%) were related to the ICF-CY component 'activities and participation', 31 (22%) were related to 'body functions', 29 (20%) were related to 'environmental factors', and 22 (15%) were related to 'body structures'. INTERPRETATION: This study shows that children with OBPI experience difficulties in all areas of functioning, as well as in both environmental and personal factors.