Individuals and Families Affected by RYR1-Related Diseases: The Patient/Caregiver Perspective.

Background and objective: Pathogenic variants of RYR1, the gene encoding the principal sarcoplasmic reticulum calcium release channel (RyR1) with a crucial role in excitation-contraction coupling, are among the most common genetic causes of non-dystrophic neuromuscular disorders. We recently conducted a questionnaire study focusing on functional impairments, fatigue, and quality of life (QoL) in patients with RYR1-related diseases (RYR1-RD) throughout the recognized disease spectrum. In this previous questionnaire study the medical perspective was taken, reflective of a study protocol designed by neurologists and psychologists. With this present study we wanted to specifically address the patient perspective. Methods: Together with affected individuals, family members, and advocates concerned with RYR1-RD, we developed an online patient survey that was completed by 227 patients or their parents/other caretakers (143 females and 84 males, 0-85 years). We invited 12 individuals, representing most of the patient group based on age, sex, race, and type and severity of diagnosis, to share their personal experiences on living with a RYR1-RD during an international workshop in July 2022. Data were analyzed through a mixed-methods approach, employing both a quantitative analysis of the survey results and a qualitative analysis of the testimonials. Results: Data obtained from the combined quantitative and qualitative analyses provide important insights on six topics: 1) Diagnosis; 2) Symptoms and impact of the condition; 3) Physical activity; 4) Treatment; 5) Clinical research and studies; and 6) Expectations. Conclusions: Together, this study provides a unique patient perspective on the RYR1-RD spectrum, associated disease impact, suitable physical activities and expectations of future treatments and trials, and thus, offers an essential contribution to future research.

Experiences of health services for adults with cerebral palsy, their support people, and service providers.

AIM: To explore the experiences of health services among adults with cerebral palsy (CP) in Ireland, from the perspectives of adults with CP, their support people, and service providers. METHOD: A qualitative descriptive study design was used. In-depth semi-structured interviews were conducted between March and August 2021 with adults with CP, people who supported them, and health professionals. Thematic analysis was used to evaluate the data. RESULTS: Twenty-one adults with CP, seven support people (family carer[s], spouse or partner, or friend), and 15 service providers participated in the study. Adults had a mean age of 38 years 5 months (range 22-58 years) and were classified in Gross Motor Function Classification System levels I to V. Five themes were identified from the data: (1) access challenges in adult services; (2) knowledge and understanding of CP; (3) support people's role and care burden; (4) communication and interaction in adult services; and (5) health system challenges. CONCLUSION: Adults with CP in Ireland face multiple challenges accessing the health services they need. Services were reported to be inadequate, with limited resources and understanding of CP. Participants highlighted a need for system-level interventions, including enhanced training for health professionals to effectively meet the needs of adults with CP.

Aging Well With a Lifelong Disability: A Scoping Review.

BACKGROUND AND OBJECTIVES: Existing literature highlights notable health and social inequalities for people aging with a lifelong disability and the need for research to better understand how we can support this group to age well. This scoping review mapped existing literature related to "aging well" in people with lifelong disabilities. RESEARCH DESIGN AND METHODS: Five scientific databases and gray literature sources were searched for studies related to "aging well" and "lifelong disability" (defined as a disability that a person had lived with since birth or early childhood). RESULTS: We identified 81 studies that discussed aging well with a lifelong disability, with most (70%) focusing on intellectual disabilities. Two themes captured existing research on aging well with a lifelong disability: (1) framing aging well with a lifelong disability, which included the ways that people with lifelong disability, their supporters, and existing research frame aging well for this group and (2) supporting people to age well with a lifelong disability, which involves the micro-, meso-, and macro-level factors where research suggests interventions to facilitate aging well could be situated. DISCUSSION AND IMPLICATIONS: This synthesis highlights how aging well is currently framed in the literature and where interventions to improve aging well in this group could be situated. Literature highlights the importance of considering multilevel interventions to improve aging well. Evidence gaps include the lack of research conducted with groups other than those with intellectual disabilities and the need for more research examining aging well interventions.

Spatial updating of gaze position in younger and older adults - A path integration-like process in eye movements.

Path integration (PI) is a navigation process that allows an organism to update its current location in reference to a starting point. PI can involve updating self-position continuously with respect to the starting point (continuous updating) or creating a map representation of the route which is then used to compute the homing vector (configural updating). One of the brain areas involved in PI, the entorhinal cortex, is modulated similarly by whole-body and eye movements, suggesting that if PI updates self-position, an analogous process may be used to update gaze position, and may undergo age-related changes. Here, we created an eyetracking version of a PI task in which younger and older participants followed routes with their eyes as guided by visual onsets; at the end of each route, participants were cued to return to the starting point or another enroute location. When only memory for the starting location was required for successful task performance, younger and older adults were generally not influenced by the number of locations, indicative of continuous updating. However, when participants could be cued to any enroute location, thereby requiring memory for the entire route, processing times increased, accuracy decreased, and overt revisits to enroute locations increased with the number of locations in a route, indicative of configural updating. Older participants showed evidence for similar updating strategies as younger participants, but they were less accurate and made more overt revisits to mid-route locations. These findings suggest that spatial updating mechanisms are generalizable across effector systems.

Integrity of autobiographical memory and episodic future thinking in older adults varies with cognitive functioning.

Research has documented changes in autobiographical memory and episodic future thinking in mild cognitive impairment (MCI) and Alzheimer's disease (AD). However, cognitive decline occurs gradually and recent findings suggest that subtle alterations in autobiographical cognition may be evident earlier in the trajectory towards dementia, before AD-related symptoms emerge or a clinical diagnosis has been given. The current study used the Autobiographical Interview to examine the episodic and semantic content of autobiographical past and future events generated by older adults (N = 38) of varying cognitive functioning who were grouped into High (N = 20) and Low Cognition (N = 18) groups based on their Montreal Cognitive Assessment (MoCA) scores. Participants described 12 past and 12 future autobiographical events, and transcripts were scored to quantify the numbers of internal (episodic) or external (non-episodic, including semantic) details. Although the Low Cognition group exhibited a differential reduction for internal details comprising both past and future events, they did not show the expected overproduction of external details relative to the High Cognition group. Multilevel modelling demonstrated that on trials lower in episodic content, semantic content was significantly increased in both groups. Although suggestive of a compensatory mechanism, the magnitude of this inverse relationship did not differ across groups or interact with MoCA scores. This finding indicates that external detail production may be underpinned by mechanisms not affected by cognitive decline, such as narrative style and the ability to contextualize one's past and future events in relation to broader autobiographical knowledge.

Identifying the top 10 priorities of adolescents with a physical disability regarding participation in physical activity: A Delphi study.

AIM: To establish consensus among adolescents with a physical disability regarding their priorities for enhancing participation in physical activity and help inform the design of future interventions for participation in physical activity. METHOD: We conducted a national multi-round Delphi study involving adolescents with a physical disability aged 13 to 17 years. Round 1 of the initial survey consisted of open-ended questions. Free-text responses were then analysed thematically, creating items categorized according to the family of participation-related constructs (fPRC). In round 2, participants rated the perceived importance of these items using a 5-point Likert scale. The top 10 priorities were constructed from the highest-ranked items. RESULTS: One hundred and sixteen participants (mean age = 14 years 7 months, range = 13-17 years; 66 males; 58 with cerebral palsy; 43 wheelchair users) completed round 1; 108 items were included in round 2. Fifty-eight items were rated as either 'important' or 'really important' by 70% of participants. The top 10 priorities were rated as important or really important by 82% to 94% of participants with a mean Likert score of 4.40 (range = 4.25-4.63). Seven of the top 10 priorities were related to the environmental context of the fPRC. The other three were related to involvement and the related concept of preference. INTERPRETATION: The priorities identified will help inform future physical activity interventions for adolescents with a physical disability.

The importance of male underwear in cases of alleged sexual assault.

The potential evidential value of male underwear in cases of alleged sexual assault is often overlooked. Male underwear can be a critical item in the investigation of alleged sexual assaults. Body fluids/DNA, which may transfer to the penis during sexual contact, may in turn transfer to the inside front of the underwear, and persist for months or years, provided the underwear are not washed. Here, we demonstrate how the case circumstances drive the sampling strategy of male underwear, in order to maximize the effectiveness of the forensic analysis. Sampling considerations including recovery methods and sampling sequence are discussed, and a methodical examination strategy of male underwear is proposed. To highlight the pertinence of male underwear to the investigation of alleged sexual assaults, three real-life cases are discussed, in which male underwear were examined for multiple body fluids/DNA, and the findings obtained proved evidentially significant. The different cases demonstrate the versatility of male underwear examination in situations, where different body fluids and DNA may transfer based on the specific allegation, and emphasize how targeted sampling can allow the scientist to assess the probability of the findings based on two competing propositions. Accurate sampling strategies are imperative for robust probability assignment in evaluative reporting of scientific findings.

Pupil Size and Eye Movements Differently Index Effort in Both Younger and Older Adults.

The assessment of mental effort is increasingly relevant in neurocognitive and life span domains. Pupillometry, the measure of the pupil size, is often used to assess effort but has disadvantages. Analysis of eye movements may provide an alternative, but research has been limited to easy and difficult task demands in younger adults. An effort measure must be sensitive to the whole effort profile, including "giving up" effort investment, and capture effort in different age groups. The current study comprised three experiments in which younger (n = 66) and older (n = 44) adults listened to speech masked by background babble at different signal-to-noise ratios associated with easy, difficult, and impossible speech comprehension. We expected individuals to invest little effort for easy and impossible speech (giving up) but to exert effort for difficult speech. Indeed, pupil size was largest for difficult but lower for easy and impossible speech. In contrast, gaze dispersion decreased with increasing speech masking in both age groups. Critically, gaze dispersion during difficult speech returned to levels similar to easy speech after sentence offset, when acoustic stimulation was similar across conditions, whereas gaze dispersion during impossible speech continued to be reduced. These findings show that a reduction in eye movements is not a byproduct of acoustic factors, but instead suggest that neurocognitive processes, different from arousal-related systems regulating the pupil size, drive reduced eye movements during high task demands. The current data thus show that effort in one sensory domain (audition) differentially impacts distinct functional properties in another sensory domain (vision).

Understanding the use of digital technologies to provide disability services remotely during the COVID-19 pandemic; a multiple case study design.

BACKGROUND: Using digital technologies to provide services and supports remotely may improve efficiency and accessibility of healthcare, and support people with disabilities to live independently. This study aimed to explore the experience of using digital technologies to access and provide disability services and supports during the Covid-19 pandemic, from the perspective of people with disabilities, families and service providers. METHODS: Using a multiple case study design, we purposively sampled three cases based on service user characteristics and geographical reach of the service. We conducted semi-structured interviews with 40 service users and service providers. Topic guides and analysis were informed by the Consolidated Framework for Implementation Research (CFIR). Analysis followed a largely deductive approach, using the CFIR constructs as a coding framework. A summary memo was developed for each case. Influence and strength of each construct was rated to identify constructs that influenced implementation of digital technologies. Ratings were compared across services to identify facilitators and barriers to implementation. RESULTS: Service users and providers were positive about using digital technologies to access and provide disability services and supports remotely. Advantages over in-person delivery included reduced travel time, increased opportunity for peer support and peer learning, more choice and opportunity to participate in activities, and an enhanced sense of self while accessing services from the secure environment of their home. The urgency to identify new modes of service delivery to meet the needs of service users during Covid-19 was a strong facilitator but did not necessarily result in successful implementation. Other factors that were strong facilitators were the use of adaptations to enable service users to access the online service, service users' willingness to try the online service, service users' persistence when they encountered challenges, and the significant time and effort that service providers made to support service users to participate in the online service. Barriers to implementation included the complexity of accessing online platforms, poor design quality of online platforms, and organisations prioritising in-person delivery over online services. CONCLUSIONS: These findings may allow service providers to leverage facilitators that support implementation of online disability services and supports.

Effects of extended practice and unitization on relational memory in older adults and neuropsychological lesion cases.

Unitization - the fusion of objects into a single unit through an action/consequence sequence - can mitigate relational memory impairments, but the circumstances under which unitization is effective are unclear. Using transverse patterning (TP), we compared unitization (and its component processes of fusion, motion, and action/consequence) with extended practice on relational learning and transfer in older adults and neuropsychological cases with lesions (to varying extents) in the medial prefrontal cortex (mPFC) or hippocampus/medial temporal lobe (HC/MTL). The latter included a person with bilateral HC lesions primarily within the dentate gyrus. For older adults, TP accuracy increased, and transfer benefits were observed, with extended practice and unitization. Broadly, the lesion cases did not benefit from either extended practice or unitization, suggesting the mPFC and dentate gyrus play important roles in relational memory and in unitization. The results suggest that personalized strategy interventions must align with the cognitive and neural profiles of the user.

Optimizing an exercise training program in pediatric brain tumour survivors: Does timing postradiotherapy matter?

Background: While exercise training (ET) programs show positive outcomes in cognition, motor function, and physical fitness in pediatric brain tumor (PBT) survivors, little is known about the optimal timing of intervention. The aim of this work was to explore the feasibility and benefits of ET based on its timing after radiotherapy. Methods: This retrospective analysis (ClinicalTrials.gov, NCT01944761) analyzed data based on the timing of PBT survivors' participation in an ET program relative to their completion of radiotherapy: <2 years (n = 9), 2-5 years (n = 10), and > 5 years (n = 13). We used repeated measures analysis of variance to compare feasibility and efficacy indicators among groups, as well as correlation analysis between ET program timing postradiotherapy and preliminary treatment effects on cognition, motor function and physical fitness outcomes. Results: Two to five years postradiotherapy was the optimal time period in terms of adherence (88.5%), retention (100%), and satisfaction (more fun, more enjoyable and recommend it more to other children). However, the benefits of ET program on memory recognition (r = -0.379, P = .047) and accuracy (r = -0.430, P = .032) decreased with increased time postradiotherapy. Motor function improved in all groups, with greater improvements in bilateral coordination (P = .043) earlier postradiotherapy, and in running (P = .043) later postradiotherapy. The greatest improvement in pro-rated work rate occurred in the < 2-year group (P = .008). Conclusion: Participation in an ET program should be offered as part of routine postradiotherapy care in the first 1-2 years and strongly encouraged in the first 5 years.

Rehabilitation via HOMe-Based gaming exercise for the Upper limb post Stroke (RHOMBUS): a qualitative analysis of participants' experience.

OBJECTIVE: To report participants' experiences of trial processes and use of the Neurofenix platform for home-based rehabilitation following stroke. The platform, consisting of the NeuroBall device and Neurofenix app, is a non-immersive virtual reality tool to facilitate upper limb rehabilitation following stroke. The platform has recently been evaluated and demonstrated to be safe and effective through a non-randomised feasibility trial (RHOMBUS). DESIGN: Qualitative approach using semistructured interviews. Interviews were audio recorded, transcribed verbatim and analysed using the framework method. SETTING: Participants' homes, South-East England. PARTICIPANTS: Purposeful sample of 18 adults (≥18 years), minimum 12 weeks following stroke, not receiving upper limb rehabilitation prior to the RHOMBUS trial, scoring 9-25 on the Motricity Index (elbow and shoulder), with sufficient cognitive and communicative abilities to participate. RESULTS: Five themes were developed which explored both trial processes and experiences of using the platform. Factors that influenced participant's decision to take part in the trial, their perceptions of support provided during the trial and communication with the research team were found to be important contextual factors effecting participants' overall experience. Specific themes around usability and comfort of the NeuroBall device, factors motivating persistence and perceived effectiveness of the intervention were highlighted as being central to the usability and acceptability of the platform. CONCLUSION: This study demonstrated the overall acceptability of the platform and identified areas for enhancement which have since been implemented by Neurofenix. The findings add to the developing literature on the interface between virtual reality systems and user experience. TRIAL REGISTRATION NUMBER: ISRCTN60291412.

Impaired perceptual discrimination of complex objects in older adults at risk for dementia.

Tau pathology accumulates in the perirhinal cortex (PRC) of the medial temporal lobe (MTL) during the earliest stages of the Alzheimer's disease (AD), appearing decades before clinical diagnosis. Here, we leveraged perceptual discrimination tasks that target PRC function to detect subtle cognitive impairment even in nominally healthy older adults. Older adults who did not have a clinical diagnosis or subjective memory complaints were categorized into "at-risk" (score <26; n = 15) and "healthy" (score ≥26; n = 23) groups based on their performance on the Montreal Cognitive Assessment. The task included two conditions known to recruit the PRC: faces and complex objects (greebles). A scene condition, known to recruit the hippocampus, and a size control condition that does not rely on the MTL were also included. Individuals in the at-risk group were less accurate than those in the healthy group for discriminating greebles. Performance on either the face or size control condition did not predict group status above and beyond that of the greeble condition. Visual discrimination tasks that are sensitive to PRC function may detect early cognitive decline associated with AD.

Adjuvant chemotherapy improves survival amongst octogenarians following resection of colorectal adenocarcinoma.

BACKGROUND: The Australian population is ageing, with an increasing number of patients diagnosed with colorectal cancer in their eighth decade. This group represents a challenging cohort to deliver multidisciplinary care, with increased morbidity and mortality related to surgical and oncological therapies. This project aimed to compare surgical and oncological outcomes in octogenarian and non-octogenarian patients with colorectal cancer. METHODS: A matched case-control retrospective review of a prospectively maintained institutional database of colorectal cancer patients treated between 2005 and 2021 was conducted. Patient demographics, treatment details, peri-operative complications, and long-term survival were reported. The primary outcome, cancer-specific survival, was investigated using time-to-event analysis and interrogated with multivariate regression. RESULTS: There were 747 patients included in the study. There was no difference in five-year cancer-specific survival and recurrence-free survival between cohorts. Multivariate analysis for octogenarians confirmed a significant hazard signal associated with worsening cancer stage and a survival benefit associated with the administration of adjuvant therapy. However, we noted that stage-matched octogenarian patients received less adjuvant treatment compared to non-octogenarians. CONCLUSION: Octogenarians undergoing treatment for colorectal cancer have similar cancer-specific survival and recurrence-free survival to non-octogenarians. Our study demonstrates a survival benefit when adjuvant therapy is provided to octogenarians, despite only half of this cohort receiving therapy compared to their younger peers. Further research into adjuvant therapy with surgical resection for CRC in older Australians is required.

Transition from child to adult services for young people with cerebral palsy in Ireland: Influencing factors at multiple ecological levels.

AIM: To explore the factors that influence the process of transitioning from child to adult services in Ireland among young people with cerebral palsy, their parents, and service providers. METHOD: This study followed a qualitative descriptive approach. Semi-structured interviews were conducted with 54 participants, including young people with cerebral palsy aged 16 to 22 years (n = 13), their parents (n = 14), and service providers (n = 27). Data were analysed using the Framework Method. Findings were categorized using an ecological model across four levels: individual, microsystem, mesosystem, and exosystem. RESULTS: Limited awareness, preparation, and access to information hindered successful transition. Microsystem factors such as family knowledge, readiness, resilience, and health professional expertise influenced transition experience. Mesosystem factors encompassed provider-family interaction, interprofessional partnerships, and interagency collaboration between child and adult services. Exosystem factors included inadequate availability and distribution of adult services, limited referral options, coordination challenges, absence of transition policies, staffing issues, and funding allocation challenges. INTERPRETATION: Transition is influenced by diverse factors at multiple ecological levels, including interactions within families, between health professionals, and larger systemic factors. Given the complexity of transition, a comprehensive multi-level response is required, taking into account the interactions among individuals, services, and systems.

Stathmin-2 loss leads to neurofilament-dependent axonal collapse driving motor and sensory denervation.

The mRNA transcript of the human STMN2 gene, encoding for stathmin-2 protein (also called SCG10), is profoundly impacted by TAR DNA-binding protein 43 (TDP-43) loss of function. The latter is a hallmark of several neurodegenerative diseases, including amyotrophic lateral sclerosis (ALS). Using a combination of approaches, including transient antisense oligonucleotide-mediated suppression, sustained shRNA-induced depletion in aging mice, and germline deletion, we show that stathmin-2 has an important role in the establishment and maintenance of neurofilament-dependent axoplasmic organization that is critical for preserving the caliber and conduction velocity of myelinated large-diameter axons. Persistent stathmin-2 loss in adult mice results in pathologies found in ALS, including reduced interneurofilament spacing, axonal caliber collapse that drives tearing within outer myelin layers, diminished conduction velocity, progressive motor and sensory deficits, and muscle denervation. These findings reinforce restoration of stathmin-2 as an attractive therapeutic approach for ALS and other TDP-43-dependent neurodegenerative diseases.

Clinicians' perspectives on motor learning strategy selection and application in occupational therapy and speech-language pathology interventions for children with acquired brain injury.

PURPOSE: Explore occupational therapists' (OTs) and speech language pathologists' (SLPs) process of selecting and applying motor learning strategies (MLS) in their interventions for children with acquired brain injury (ABI), and identify similarities and differences between OTs and SLPs in MLS selection and application. METHODS: This qualitative descriptive study involved individual semi-structured interviews with OTs and SLPs from the ABI program at Holland Bloorview Kids Rehabilitation Hospital (Toronto, Canada). Interviews were analyzed using thematic analysis. A modified constant comparison method permitted comparison within and between professions. RESULTS: Four OTs and three SLPs were interviewed. Four themes were developed: aligning MLS application with the child's cognitive ability, using MLS to promote success within a single session, adjusting MLS across treatment sessions, and promoting generalization and transfer of motor skills beyond the therapy session. MLS application was predominately based on child-specific factors with task-specific considerations. OTs and SLPs used similar clinical reasoning processes for selecting and applying MLS. CONCLUSIONS: This study provides a greater understanding of OTs' and SLPs' clinical reasoning process when applying MLS in pediatric ABI interventions. The similarities in MLS selection and application between disciplines suggest that an interprofessional approach to MLS is suitable for pediatric ABI rehabilitation.

Pediatric occupational therapists and speech language pathologists use a similar process for selecting and applying motor learning strategies (MLS) in their interventionsClinicians continually reflect upon and adapt their use of MLS based on each child's performance, progress, and goalsAdopting an interprofessional approach to MLS selection and application, including the use of a common MLS language, has the potential to optimize motor outcomes in pediatric acquired brain injury rehabilitation.

Interventions for improving adherence to airway clearance treatment and exercise in people with cystic fibrosis.

BACKGROUND: Cystic fibrosis (CF) is a life-limiting genetic disorder predominantly affecting the lungs and pancreas. Airway clearance techniques (ACTs) and exercise therapy are key components of physiotherapy, which is considered integral in managing CF; however, low adherence is well-documented. Poor physiotherapy adherence may lead to repeated respiratory infections, reduced exercise tolerance, breathlessness, reduced quality of life, malaise and reduced life expectancy, as well as increased use of pharmacology, healthcare access and hospital admission. Therefore, evidence-based strategies to inform clinical practice and improve adherence to physiotherapy may improve quality of life and reduce treatment burden. OBJECTIVES: To assess the effects of interventions to enhance adherence to airway clearance treatment and exercise therapy in people with CF and their effects on health outcomes, such as pulmonary exacerbations, exercise capacity and health-related quality of life. SEARCH METHODS: We searched the Cochrane Cystic Fibrosis Trials Register, compiled from electronic database searches and handsearching of journals and conference abstract books. Date of last search: 1 March 2023. We also searched online trials registries and the reference lists of relevant articles and reviews. Date of last search: 28 March 2023. SELECTION CRITERIA: We included randomised controlled trials (RCTs) and quasi-RCTs of parallel design assessing any intervention aimed at enhancing adherence to physiotherapy in people with CF versus no intervention, another intervention or usual care. DATA COLLECTION AND ANALYSIS: Two review authors independently checked search results for eligible studies and independently extracted data. We used standard procedures recommended by Cochrane and assessed the certainty of evidence using the GRADE system. MAIN RESULTS: Two RCTs (77 participants with CF; age range 2 to 20 years; 44 (57%) males) met the inclusion criteria of this review. One study employed an intervention to improve adherence to exercise and the second an intervention to improve adherence to ACT. Both studies measured outcomes at baseline and at three months, but neither study formally assessed our primary outcome of adherence in terms of our planned outcome measures, and results were dependent on self-reported data. Adherence to ACTs One RCT (43 participants) assessed using specifically-composed music alongside ACTs compared to self-selected or no music (usual care). The ACT process consisted of nebuliser inhalation treatment, ACTs and relaxation or antibiotic nebuliser treatment. We graded all evidence as very low certainty. This study reported adherence to ACTs using the Morisky-Green questionnaire and also participants' perception of treatment time and enjoyment, which may influence adherence (outcome not reported specifically in this review). We are uncertain whether participants who received specifically-composed music may be more likely to adhere at six and 12 weeks compared to those who received usual care, risk ratio (RR) 1.75 (95% confidence interval (CI) 1.07 to 2.86) and RR 1.56 (95% CI 1.01 to 2.40) respectively. There may not be any difference in adherence when comparing specifically-composed music to self-selected music at six weeks, RR 1.21 (95% CI 0.87 to 1.68) or 12 weeks, RR 1.52 (95% CI 0.97 to 2.38); or self-selected music to usual care at six weeks, RR 1.44 (95% CI 0.82 to 2.52) or 12 weeks, RR 1.03 (95% CI 0.57 to 1.86). The music study also reported the number of respiratory infections requiring hospitalisation at 12 weeks, with no difference seen in the risk of hospitalisation between all groups. Adherence to exercise One RCT (24 participants) compared the provision of a manual of aerobic exercises, recommended exercise prescription plus two-weekly follow-up phone calls to reinforce exercise practice over a period of three months to verbal instructions for aerobic exercise according to the CF centre's protocol. We graded all evidence as very low certainty. We are uncertain whether an educational intervention leads to more participants in the intervention group undertaking increased regular physical activity at three months (self-report), RR 3.67 (95% CI 1.24 to 10.85), and there was no reported difference between groups in the number undertaking physical activity three times per week or undertaking at least 40 minutes of physical activity. No effect was seen on secondary outcome measures of spirometry, exercise capacity or any CF quality of life domains. This study did not report on the frequency of respiratory infections (hospitalised or not) or adverse events. AUTHORS' CONCLUSIONS: We are uncertain whether a music-based motivational intervention may increase adherence to ACTs or affect the risk of hospitalisation for a respiratory infection. We are also uncertain whether an educational intervention increases adherence to exercise or reduces the frequency of respiratory infection-related hospital admission. However, these results are largely based on self-reported data and the impact of strategies to improve adherence to ACT and exercise in children and adolescents with stable CF remains inconclusive. Given that adherence to ACT and exercise therapy are fundamental to the clinical management of people with CF, there is an urgent need for well-designed, large-scale clinical trials in this area, which should conform to the CONSORT statement for standards of reporting and use appropriate, validated outcome measures. Studies should also ensure full disclosure of data for all important clinical outcomes.

Primary care-based models of care for osteoarthritis; a scoping review.

OBJECTIVE: To identify and describe the extent, nature, characteristics, and impact of primary care-based models of care (MoCs) for osteoarthritis (OA) that have been developed and/or evaluated. DESIGN: Six electronic databases were searched from 2010 to May 2022. Relevant data were extracted and collated for narrative synthesis. RESULTS: Sixty-three studies pertaining to 37 discrete MoCs from 13 countries were included, of which 23 (62%) could be classified as OA management programmes (OAMPs) comprising a self-management intervention to be delivered as a discrete package. Four models (11%) focussed on enhancing the initial consultation between a patient presenting with OA at the first point of contact into a local health system and the clinician. Emphasis was placed on educational training for general practitioners (GPs) and allied healthcare professionals delivering this initial consultation. The remaining 10 MoCs (27%) detailed integrated care pathways of onward referral to specialist secondary orthopaedic and rheumatology care within local healthcare systems. The majority (35/37; 95%) were developed in high-income countries and 32/37 (87%) targeted hip/and or knee OA. Frequently identified model components included GP-led care, referral to primary care services and multidisciplinary care. The models were predominantly 'one-size fits all' and lacked individualised care approaches. A minority of MoCs, 5/37 (14%) were developed using underlying frameworks, three (8%) of which incorporated behaviour change theories, while 13/37 (35%) incorporated provider training. Thirty-four of the 37 models (92%) were evaluated. Outcome domains most frequently reported included clinical outcomes, followed by system- and provider-level outcomes. While there was evidence of improved quality of OA care associated with the models, effects on clinical outcomes were mixed. CONCLUSION: There are emerging efforts internationally to develop evidence-based models focused on non-surgical primary care OA management. Notwithstanding variations in healthcare systems and resources, future research should focus on model development alignment with implementation science frameworks and theories, key stakeholder involvement including patient and public representation, provision of training and education for providers, treatment individualisation, integration and coordination of services across the care continuum and incorporation of behaviour change strategies to foster long-term adherence and self-management.