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AimsTo analyze needs and requirements of Pediatric Physical Therapists (PPTs), parents, children and adolescents with and without developmental disabilities in the future use of an activity monitor prototype (AM-p) in everyday clinical practice.MethodsQualitative exploratory study with a thematic analysis approach, based on Braun and Clarke's six steps. Codes derived from the analysis and central themes were collated, based on Fleuren et al.'s groupings of determinants.ResultsWe interviewed 25 PPTs, 12 parents, and 12 children and adolescents. Within four groupings of determinants, we found nine themes: 1) development of information materials; 2) application: output visualization and ease of use; 3) design; 4) relevance and acceptance; 5) shared decision-making; 6) compatibility in daily living; 7) finances, 8) time, and 9) legislation and regulations.ConclusionsEnd-users have similar basic needs, with individual fine-tuning to be addressed during further development of the AM-p. A child-friendly design, information material, and an easy-to-use application to read and interpret results, need to be developed. Efficient training for PPTs is important for the use of the AM-p and analysis of results. Communication between PPTs and children as well as parents enhances shared decision-making. We recommend involving diverse end-users to enable maximum customization of the AM-p.

Objective: To develop consensus among experts to guide physical rehabilitation in children and adolescents with acquired brain injury during the subacute phase. Design: International Delphi study. Methods: A 3-round online Delphi study was conducted with 11 international experts in rehabilitation for children and adolescents with acquired brain injury. The first round consisted of open-ended questions; the second and third round consisted of ranking 139 statements on a 5-point Likert scale. Results: The panel reached consensus on 116/139 statements. Consensus was reached on the importance of age, pre-injury developmental stage and the clinical presentation of the child when determining content and focus of physical rehabilitation. In addition, consensus was reached on the importance of participation-focused interventions, and involvement of family members in goal-setting and therapeutic activities. Although dosage was deemed very important, no consensus was reached for determination of dose-response variables to suit and influence the child’s needs. Conclusion: This study provides a framework for clinicians to design physical rehabilitation interventions in children with acquired brain injury in the sub-acute recovery phase. The promotion of physical activity in meaningful contexts and involvement of family members are considered as important components to optimize recovery.

For older adults, acute hospitalization is a high-risk event with poor health outcomes, including functional decline. In absence of practical guidelines and high quality randomized controlled trials, this Delphi study was conducted. The aim of this study was to obtain consensus on an exercise intervention program, a core outcome set (COS) and handover information to prevent functional decline or restore physical function in acutely hospitalized older patients transitioning from hospital to home. An internal panel of experts in the field of exercise interventions for acutely hospitalized older adults were invited to join the study. In the Delphi study, relevant topics were recognized, statements were formulated and ranked on a 9-point Likert scale in two additional rounds. To reaching consensus, a score of 7-9 was classified as essential. Results were expressed as median and semi-interquartile range (SIQR), and consensus threshold was set at SIQR≤0.5. Fifteen international experts from eight countries participated in the panel. The response rate was 93%, 93% and 80% for the three rounds respectively. After three rounds, consensus was reached on 167 of the 185 (90.3%) statements, of which ninety-five (51.4%) were ranked as essential (median Likert-score ≥7.0, SIQR ≤0.5). This Delphi study provides starting points for developing an exercise intervention, a COS and handover information. The results of this Delphi study can assist physical therapists to provide a tailored exercise intervention for older patients with complex care needs after hospital discharge, to prevent functional decline and/or restore physical function.

Abstract Background Survivors of critical illness experience long-term functional challenges, which are complex, heterogeneous, and multifactorial in nature. Although the importance of rehabilitation interventions after intensive care unit (ICU) discharge is universally recognized, evidence on feasibility and effectiveness of home-based rehabilitation programs is scarce and ambiguous. This study investigates the feasibility of an interdisciplinary rehabilitation program designed for patients with Post-Intensive Care Syndrome (PICS) who are discharged home. Methods A mixed method, non-randomized, prospective pilot feasibility study was performed with a 6-month follow-up, comparing the intervention (REACH) with usual care. REACH was provided by trained professionals and included a patient-centered, interdisciplinary approach starting directly after hospital discharge. Primary outcomes were patient safety, satisfaction, adherence, referral need and health care usage. Secondary outcomes, measured at 3 timepoints, were functional exercise capacity, self-perceived health status, health-related quality of life (HRQoL), return to work and psychotrauma. Risk of undernutrition was assessed at baseline. Results 43 patients with a median mechanical ventilation duration of 8 (IQR:10) days, were included in the study and 79.1% completed 6-month follow-up. 19 patients received the intervention, 23 received usual care. Groups were similar for gender distribution and ICU length of stay. No adverse events occurred. REACH participants showed higher satisfaction with treatment and reported more allied health professional visits, while the usual care group reported more visits to medical specialists. Qualitative analysis identified positive experiences among REACH-professionals related to providing state-of-the-art interventions and sharing knowledge and expertise within an interprofessional network. Similar recovery was seen between groups on all secondary outcomes, but neither group reached reference values for HRQoL at 6 months. Larger return to work rates were seen in the REACH group. Prevalence of undernutrition at hospital discharge was high in both groups (> 80%), warranting the need for careful tuning of physical therapy and nutritional interventions. Conclusions This study shows that providing early, home-based rehabilitation interventions for patients with PICS-related symptoms is feasible and perceived positively by patients and professionals. When provided in an interdisciplinary collaborative network state of the art, person-centered interventions can be tailored to individual needs potentially increasing patient satisfaction, adherence, and efficacy. Registered in the Dutch Trial register: NL7792: https://www.trialregister.nl/trial/7792 , registered 7-06-2019.

BackgroundThe diagnosis of sarcopenia is essential for early treatment of sarcopenia in older adults, for which assessment of appendicular lean mass (ALM) is needed. Multi-frequency bio-electrical impedance analysis (MF-BIA) may be a valid assessment tool to assess ALM in older adults, but the evidences are limited. Therefore, we validated the BIA to diagnose low ALM in older adults.MethodsALM was assessed by a standing-posture 8 electrode MF-BIA (Tanita MC-780) in 202 community-dwelling older adults (age ≥ 55 years), and compared with dual-energy X-ray absorptiometry (DXA) (Hologic Inc., Marlborough, MA, United States; DXA). The validity for assessing the absolute values of ALM was evaluated by: (1) bias (mean difference), (2) percentage of accurate predictions (within 5% of DXA values), (3) the mean absolute error (MAE), and (4) limits of agreement (Bland–Altman analysis). The lowest quintile of ALM by DXA was used as proxy for low ALM (< 22.8 kg for men, < 16.1 kg for women). Sensitivity and specificity of diagnosing low ALM by BIA were assessed.ResultsThe mean age of the subjects was 72.1 ± 6.4 years, with a BMI of 25.4 ± 3.6 kg/m2, and 71% were women. BIA slightly underestimated ALM compared to DXA with a mean bias of −0.6 ± 1.2 kg. The percentage of accurate predictions was 54% with a MAE of 1.1 kg, and limits of agreement were −3.0 to + 1.8 kg. The sensitivity for ALM was 80%, indicating that 80% of subjects who were diagnosed as low ALM according to DXA were also diagnosed low ALM by BIA. The specificity was 90%, indicating that 90% of subjects who were diagnosed as normal ALM by DXA were also diagnosed as normal ALM by the BIA.ConclusionThis comparison showed a poor validity of MF-BIA to assess the absolute values of ALM, but a reasonable sensitivity and specificity to recognize the community-dwelling older adults with the lowest muscle mass.

Abstract Background Early mobilization has been proven effective for patients in intensive care units (ICUs) to improve functional recovery. However, early mobilization of critically ill, often mechanically ventilated, patients is cumbersome because of the attachment to tubes, drains, monitoring devices and muscle weakness. A mobile treadmill with bodyweight support may help to initiate mobilization earlier and more effectively. The aim of this study is to assess the effectiveness of weight-supported treadmill training in critically ill patients during and after ICU stay on time to independent functional ambulation. Methods In this randomized controlled trial, a custom-built bedside body weight-supported treadmill will be used and evaluated. Patients are included if they have been mechanically ventilated for at least 48 hours, are able to follow instructions, have quadriceps muscle strength of Medical Research Council sum-score 2 (MRC 2) or higher, can sit unsupported and meet the safety criteria for physical exercise. Exclusion criteria are language barriers, no prior walking ability, contraindications for physiotherapy or a neurological condition as reason for ICU admission. We aim to include 88 patients and randomize them into either the intervention or the control group. The intervention group will receive usual care plus bodyweight-supported treadmill training (BWSTT) daily. The BWSSTT consists of walking on a mobile treadmill while supported by a harness. The control group will receive usual care physiotherapy treatment daily consisting of progressive activities such as bed-cycling and active functional training exercises. In both groups, we will aim for a total of 40 minutes of physiotherapy treatment time every day in one or two sessions, as tolerated by the patient. The primary outcome is time to functional ambulation as measured in days, secondary outcomes include walking distance, muscle strength, status of functional mobility and symptoms of post-traumatic stress. All measurements will be done by assessors who are blinded to the intervention on the regular wards until hospital discharge. Discussion This will be the first study comparing the effects of BWSTT and conventional physiotherapy for critically ill patients during and after ICU stay. The results of this study contribute to a better understanding of the effectiveness of early physiotherapy interventions for critically ill patients. Trial registration Dutch Trial Register (NTR) ID: NL6766 . Registered at 1 December 2017.

Abstract Background Intensive Care Units (ICUs) are daunting environments for physiotherapy (PT) students performing clinical rotations. To prepare students for this environment, a newly developed, evidence-based e-learning module was designed and implemented in the undergraduate curriculum. The aim of this study was to investigate whether e-learning is a feasible method in preparing PT students for clinical work in complex ICU environments, as perceived by students and experts. Methods A mixed methods proof of concept study was undertaken. Participants were final-year students of an international curriculum, and experts from didactic and clinical fields. An e-learning module consisting of 7 separate chapters based on the latest scientific evidence and clinical expertise was developed, piloted and incorporated into the undergraduate curriculum as a compulsory course to be completed prior to clinical ICU rotations. Data were collected through 3 focus group meetings and 5 semi-structured interviews; these meetings and interviews were audio recorded, transcribed verbatim and analyzed. Results The study sample comprised of 14 students and 5 experts. Thematic analysis revealed three themes: expected competencies of PT students in ICU, feeling prepared for ICU clinical work and dealing with local variety. The e-learning module enabled students to anticipate clinical situations and PT tasks in the ICU. Higher level clinical reasoning skills, handling of lines and wires and dealing with out-of-textbook situations could not be achieved with the e-learning module alone. Conclusions An e-learning module can sufficiently prepare PT students for their clinical tasks in the ICU, as long as it is integrated with, or closely connected to, the students’ clinical placement.

Abstract Background Marfan syndrome (MFS) is a heritable connective tissue disease caused by a defect in FBN1. The diagnosis is based on the revised Ghent criteria. The main features involve the cardiovascular, musculoskeletal, ophthalmic, pulmonary systems and facial features. Although the clinical manifestations of MFS in children are thoroughly addressed in several studies, literature on the impact of MFS on daily functioning is restricted to pediatric advice on sports and leisure participation. Therefore, the full impact of MFS on daily functioning remains unclear. The aim of this qualitative study was to explore parents’ perspectives on the impact of MFS on daily functioning of children with MFS aged 4–12 years, themselves and family regarding functional performance, activities, participation, personal and environmental factors, and disease burden. Methods In this qualitative study parents participated in individual semi-structured interviews (n = 10) and 3 focus groups (n = 5, n = 5 and n = 6). Meetings were transcribed, and data were analyzed using thematic analysis. Meaningful concepts were coded, and concepts concerning children with MFS were linked to the International Classification of Functioning, Disability and Health for Children and Youth. Thereafter themes were identified and interpreted. Results Parents reported their children could not keep up with peers because of fatigue, pain and physical impairments. Children experienced participation restrictions in school, sports, play and other leisure activities. Parents reported their child as being different due to physical appearance, which provoked unsupportive attitudes. Parental burden was caused by high care needs, lack of support, a limited social life, and concerns about the child’s development. Family burden was caused by adjusted and complex family schedules, other family members with MFS, and reproductive planning decision-making, whereas family cohesiveness and caring were positively perceived factors. Conclusions Parents perceived a large impact of MFS on daily functioning of their children with MFS, themselves and their family. More awareness among all professionals involved in the care of children with MFS and their families is needed so that professionals can address their support needs and provide tailored interventions, rehabilitation and/or educational programs to empower and improve daily functioning of the children, parents and family.

Objectives:. To conduct a scoping review to 1) describe findings and determinants of physical functioning in children during and/or after PICU stay, 2) identify which domains of physical functioning are measured, 3) and synthesize the clinical and research knowledge gaps. Data Sources:. A systematic search was conducted in PubMed, Embase, Cumulative Index to Nursing and Allied Health Literature, and Cochrane Library databases following the Preferred Reporting Items for Systematic Reviews and Meta-analyses extension for Scoping Reviews guidelines. Study Selection:. Two investigators independently screened and included studies against predetermined criteria. Data Extraction:. One investigator extracted data with review by a second investigator. A narrative analyses approach was used. Data Synthesis:. A total of 2,610 articles were identified, leaving 68 studies for inclusion. Post-PICU/hospital discharge scores show that PICU survivors report difficulties in physical functioning during and years after PICU stay. Although sustained improvements in the long-term have been reported, most of the reported levels were lower compared with the reference and baseline values. Decreased physical functioning was associated with longer hospital stay and presence of comorbidities. A diversity of instruments was used in which mobility and self-care were mostly addressed. CONCLUSIONS:. The results show that children perceive moderate to severe difficulties in physical functioning during and years after PICU stay. Longitudinal assessments during and after PICU stay should be incorporated, especially for children with a higher risk for poor functional outcomes. There is need for consensus on the most suitable methods to assess physical functioning in children admitted to the PICU.

Abstract Background Increased physical activity and dietary protein intake are promising interventions to prevent or treat the age-related decline in physical performance in older adults. There are well-controlled exercise as well as dietary intervention studies that show beneficial effects on physical performance in older adults. In practice, however, weekly group based exercise or nutritional programs may not be as effective. To optimise these exercise programs for community dwelling older adults, a digitally supported and personalised home-based exercise training program has been designed aiming to improve physical performance in older adults. In addition, a protein intervention in combination with the training program may further improve physical performance in older adults. Methods The VITAMIN study will be a cluster randomised controlled trial with three parallel arms. In total, 240 community dwelling older adults (≥ 55 years) participating in weekly group exercise are randomly allocated into: 1) regular weekly exercise program (Control group, n = 80), 2) digitally supported personalised home-based exercise training program group (VITA group, n = 80) and 3) digitally supported personalised home-based exercise training program group plus dietary protein counselling (VITA-Pro group, n = 80). The VITAMIN study aims to evaluate effectiveness of the digitally supported personalised home-based exercise training program as well as the additional value of dietary protein on physical performance after 6 months. In addition, a 12 month follow-up measurement will assess the retaining effect of the interventions. Primary outcome is physical performance measured by the Modified Physical Performance Test (M-PPT) and relevant secondary and observational outcomes include habitual physical activity and dietary intake, body composition, cognitive performance, quality of life, compliance and tablet usage. Data will be analysed by Linear Mixed Models. Discussion To our knowledge, the VITAMIN study is the first study that investigates the impact of home-based exercise, protein intake as well as use of persuasive technology in the population of community dwelling older adults. Trial registration NL56094.029.16 / NTR (TC = 5888; registered 03–06-2016).

Migraine, tension-type headache (TTH) and headaches attributed to temporomandibular disorders (TMD) are prevalent in patients with TMD-pain. The objective was to describe the course of headache complaints as compared to the course of TMD complaints in TMD-pain patients with headache during usual care multidisciplinary treatment for TMD. This was a 12-week longitudinal observational study following adults with TMD-pain and headache during a usual-care multidisciplinary TMD-treatment. The Graded Chronic Pain Scale was used for both TMD and headache to measure pain-related disability (primary outcome measure), pain intensity, days with pain and days experiencing disability (secondary outcome measures). Stratified for the headache type, general linear modelling for repeated measures was used to analyze changes over time in the TMD complaints and the headache complaints. TMD-pain patients with migraine (n = 22) showed significant decrease of pain-related disability for both TMD and headache complaints over time. No difference in the effect over time was found between the two complaints. Patients with TMD-pain and TTH (n = 21) or headache attributed to TMD (n = 17) did not improve in disability over time. For the secondary outcome measures, the results were equivocal. In conclusion, TMD-pain patients with migraine, improvement in TMD-related disability was comparable to headache-related disability for TMD-pain patients with TTH or with headache attributed to TMD, no improvements in disability were found.

Background30 to 60% of the acute hospitalized older adults experience functional decline after hospitalization. The first signs of functional decline after discharge can often be observed in the inability to perform mobility tasks, such as raising from a chair or walking. Information how mobility develops over time is scarce. Insight in the course of mobility is needed to prevent and decrease mobility limitations.ObjectivesThe objectives of this study were to determine (i) the course of mobility of acute hospitalized older adults and (ii) the association between muscle strength and the course of mobility over time controlled for influencing factors.MethodsIn a multicenter, prospective, observational cohort study, measurements were taken at admission, discharge, one- and three months post-discharge. Mobility was assessed by the De Morton Mobility Index (DEMMI) and muscle strength by the JAMAR. The longitudinal association between muscle strength and mobility was analysed with a Linear Mixed Model and controlled for potential confounders.Results391 older adults were included in the analytic sample with a mean (SD) age of 79.6 (6.7) years. Mobility improved significantly from admission up to three months post-discharge but did not reach normative levels. Muscle strength was associated with the course of mobility (beta = 0.64; pConclusionMuscle strength is longitudinally associated with mobility. Interventions to improve mobility including muscle strength are warranted, in acute hospitalized older adults.

Purpose: Physical interventions during subacute rehabilitation have potential to improve functional recovery. This study explored the perspectives of children and adolescents with acquired brain injury (ABI) and their parents with respect to physical rehabilitation during the subacute phase. Methods: Thirteen children and adolescents with ABI and their parents were included and interviewed using semi-structured interviews. Interview transcripts were analysed using inductive thematic analysis approach. Results: Six themes were identified: 1) beliefs of physical rehabilitation, 2) content of physical rehabilitation, 3) tailored care, 4) impact of context, 5) communication and 6) transition. The importance of intensive physical practice was widely supported. The positive can-do mentality was emphasised to create an atmosphere of hope, meaning that every effort would be made to achieve maximum recovery. Intensive involvement of parents is considered essential during subacute rehabilitation including an open and mutual dialogue about the focus of rehabilitation, therapy goals and future participation in their own environment. Conclusions: Our findings highlight the need for an intensive rehabilitation approach, tailored to the individual's needs. The perspectives of children and adolescents and their parents in our study contribute to a better understanding of factors that are important for optimal recovery through physical rehabilitation during the subacute phase. IMPLICATIONS FOR REHABILITATION: Children with acquired brain injury and their parents indicate the potential and thus the importance of intensive physical practice to enhance optimal recovery. Involvement of parents and the potential of their continuous presence during subacute rehabilitation may have a positive impact on the effect of rehabilitation efforts. The positive can-do mentality of rehabilitation professionals creates an atmosphere of hope and is an important requisite to achieve maximum recovery. Open dialogue between clinicians and the family is warranted about the focus of interventions. [ABSTRACT FROM AUTHOR]

Aims: To analyze needs and requirements of Pediatric Physical Therapists (PPTs), parents, children and adolescents with and without developmental disabilities in the future use of an activity monitor prototype (AM-p) in everyday clinical practice. Methods: Qualitative exploratory study with a thematic analysis approach, based on Braun and Clarke's six steps. Codes derived from the analysis and central themes were collated, based on Fleuren et al.'s groupings of determinants. Results: We interviewed 25 PPTs, 12 parents, and 12 children and adolescents. Within four groupings of determinants, we found nine themes: 1) development of information materials; 2) application: output visualization and ease of use; 3) design; 4) relevance and acceptance; 5) shared decision-making; 6) compatibility in daily living; 7) finances, 8) time, and 9) legislation and regulations. Conclusions: End-users have similar basic needs, with individual fine-tuning to be addressed during further development of the AM-p. A child-friendly design, information material, and an easy-to-use application to read and interpret results, need to be developed. Efficient training for PPTs is important for the use of the AM-p and analysis of results. Communication between PPTs and children as well as parents enhances shared decision-making. We recommend involving diverse end-users to enable maximum customization of the AM-p. [ABSTRACT FROM AUTHOR]

To assess the experience and perceived added value of an e-Health application during the physical therapy treatment of patients with temporomandibular disorders (TMD). A mixed-methods study including semi-structured interviews was performed with orofacial physical therapists (OPTs) and with TMD patients regarding their experience using an e-Health application, Physitrack. The modified telemedicine satisfaction and usefulness questionnaire and pain intensity score before and after treatment were collected from the patients. Ten OPTs, of which nine actively used Physitrack, described that the e-Health application can help to provide personalised care to patients with TMD, due to the satisfying content, user-friendliness, accessibility, efficiency, and ability to motivate patients. Ten patients, of which nine ended up using Physitrack, felt that shared decision-making was very important. These patients were positive towards the application as it was clear, convenient, and efficient, it helped with reassurance and adherence to the exercises and overall increased self-efficacy. This was mostly built on their experience with exercise videos, as this feature was most used. None of the OPTs or patients used all features of Physitrack. The overall satisfaction of Physitrack based on the telemedicine satisfaction and usefulness questionnaire (TSUQ) was 20.5 ± 4.0 and all patients (100%) showed a clinically relevant reduction of TMD pain (more than 2 points and minimally 30% difference). OPTs and patients with TMD shared the idea that exercise videos are of added value on top of usual physical therapy care for TMD complaints, which could be delivered through e-Health. Physical therapists and patients with temporomandibular disorders do not use all features of the e-Health application Physitrack in a clinical setting. Exercise videos were the most often used feature and seen as most valuable by physical therapists and patients. Based on a small number of participants, e-Health applications such as Physitrack may be perceived as a valuable addition to the usual care, though this would need verification by a study designed to evaluate the therapeutic effect (e.g., a randomised clinical trial). [ABSTRACT FROM AUTHOR]

Abstract Introduction Real-world data from multinational observational studies are required to better understand the role and performance of isavuconazole in real-world practice in Europe. Methods A retrospective medical record review was conducted at 16 sites in Europe (France, Germany, Italy, Spain, and the United Kingdom). Eligible records were from patients aged ≥ 18 years at the time of isavuconazole initiation and received at least one dose of isavuconazole for suspected or confirmed invasive aspergillosis (IA) or invasive mucormycosis (IM) during the eligibility period (October 15, 2015 to June 30, 2019). Data were descriptively analysed. Success rates, overall survival, and times to these events were descriptively analysed. Results Data were abstracted from 218 patients (201, IA; 17, IM) who received isavuconazole as monotherapy (initiated as infusion, 52%; oral, 46%). Isavuconazole was initiated as primary therapy in 92 patients (42.2%) and salvage therapy in 121 patients (55.5%) (unknown for five patients). Mean (standard deviation) age was 56.8 (15.6) years, 66% were men and 62% had at least three comorbidities, most frequently haematologic malignancy (62%). Estimated clinical response rate at week 24 was 54.5% (95% confidence interval [CI], 38.2–66.5%) for primary treatment and 73.5% (95% CI, 62.7–81.1%) for salvage therapy. Overall, 45 patients (21%) experienced at least one adverse event (AE). Serious AEs were experienced by 37 patients (17%), with seven related to isavuconazole; five patients (2.3%) discontinued isavuconazole monotherapy due to the serious AE. A total of 137 patients (63%) died, with 17 deaths (12.4%) related to their invasive fungal infection, 11 of whom initiated isavuconazole as salvage therapy. Conclusions This study adds to the growing body of evidence that whether used as first-line therapy or after the failure of other antifungal therapies, isavuconazole appears to have a promising clinical response and a good safety profile as an antifungal agent in patients with varied underlying conditions.

Abstract Background Oral function tests have been shown to reliably detect impaired bulbar function in adults with spinal muscular atrophy (SMA). Although not routinely recorded, it is known that persons with SMA are affected to varying degrees. Detecting differences in bite and tongue force, endurance, and maximum mouth opening has become particularly promising since the introduction of causal therapy for SMA. This study aimed to compare oral function among adult persons with SMA with different SMA types, walking abilities, and treatment status to a healthy control group. Methods Data from oral function tests conducted on 58 persons with SMA and 45 healthy individuals were analyzed. Differences in oral function between SMA subgroups were pairwise tested and compared to the healthy control group using Wilcoxon rank sum tests. Results In an overall comparison, three out of five oral function tests revealed lower values for the SMA group compared to the control group. Subgroup analyses indicated lower scores for most oral function tests in non-ambulatory, untreated patients with SMA type 2 compared to controls. Ambulatory, treated patients with SMA type 3 achieved strength and endurance values comparable to those of healthy individuals. Conclusions The impairment of oral function varies across persons with SMA. Routine measurement of oral function is warranted to determine individual bulbar involvement stages. Further evaluation should be scheduled if indicators such as restricted maximum mouth opening arise. Trial registration DRKS, DRKS00015842. Registered 30 July 2019, https://drks.de/register/de/trial/DRKS00015842/preview .

The aim of the present study was to investigate the nature and prevalence of nonspecific somatic symptoms, pain and catastrophizing in children with Heritable Connective Tissue Disorders (HCTD), and to determine their association with disability. This observational, multicenter study included 127 children, aged 4–18 years, with Marfan syndrome (MFS) (59%), Loeys‐Dietz syndrome (LDS) (8%), Ehlers‐Danlos syndromes (EDS) (12%) and hypermobile Ehlers‐Danlos syndrome (hEDS) (23%). The assessments included the Children's Somatization Inventory or parent proxy (CSI, PCSI), pain visual‐analogue scale (VAS), SUPERKIDZ body diagram, Pain Catastrophizing Scale Child or parent proxy (PCS‐C, PCS‐P) and Childhood Health Assessment Questionnaire (CHAQ‐30). Data from children aged ≥8 years were compared to normative data. In children ≥ 8 years (n = 90), pain was present in 59%, with a median of 4 (IQR = 3–9) pain areas. Compared to normative data, the HCTD group reported significantly higher on the CSI (p ≤ 0.001, d = 0.85), VAS pain intensity (p ≤ 0.001, d = 1.22) and CHAQ‐30 (p ≤ 0.001, d = 1.16) and lower on the PCS‐C (p = 0.017, d = −0.82) and PCS‐P (p ≤ 0.001, d = −0.49). The intensity of nonspecific somatic symptoms and pain explained 45% of the variance in disability (r2 = 0.45 F(2,48) = 19.70, p ≤ 0.001). In children ≤ 7 years (n = 37), pain was present in 35% with a median of 5(IQR = 1–13) pain areas. The mean(SD) VAS scores for pain intensity was 1.5(2.9). Functional disability was moderately correlated to the number of pain areas (r = 0.56, p ≤ 0.001), intensity of nonspecific somatic symptoms (r = 0.63, p ≤ 0.001) and pain (r = 0.83, p ≤ 0.001). In conclusion, this study supports the need for comprehensive assessment of nonspecific somatic symptoms, pain, and disability in children with HCTD to allow tailored treatment. [ABSTRACT FROM AUTHOR]