Beyond pGALS: the need for a multifaceted musculoskeletal decision-making tool ('pGALSplus') in community-based clinical practice.

Musculoskeletal (MSK) problems in children are common, and health-care professionals must identify those requiring onward referral. Paediatric gait, arms, legs and spine (pGALS) is an MSK assessment to discern abnormal joints. We aimed to identify MSK assessments to add to pGALS (pGALSplus) to facilitate decision-making in the context of exemplar conditions representing a spectrum of MSK presentations, namely JIA, mucopolysaccharidoses, muscular dystrophy and developmental co-ordination disorder. A literature review identified 35 relevant articles that focused on clinical assessments [including questionnaire(s), physical examination and functional tests] used by health-care professionals in the context of the exemplar conditions. We provide a description of these assessments and the rationale regarding how they, or components of such tools, might be useful within pGALSplus. This process provides a foundation for further work to develop and validate pGALSplus.

Bridging gaps: a qualitative inquiry on improving paediatric rheumatology care among healthcare workers in Kenya.

BACKGROUND: Due to the paucity of paediatric rheumatologists in Kenya, it is paramount that we explore strategies to bridge clinical care gaps for paediatric rheumatology patients in order to promote early diagnosis, prompt referral, and optimal management. PURPOSE: To identify proposed interventions which can improve the ability of non-specialist healthcare workers to care for paediatric rheumatology patients across Kenya. METHODS: We conducted 12 focus group discussions with clinical officers (community physician assistants), nurses, general practitioners and paediatricians across six regions in Kenya. Interviews were conducted, audio-recorded, transcribed verbatim, and analysed using MAXQDA 2022.2 software. RESULTS: A total of 68 individuals participated in the study; 11 clinical officers, 12 nurses, 10 general practitioners, 27 paediatricians and eight other healthcare workers. Proposed patient interventions included patient education and psychosocial support. Community interventions were outreach awareness campaigns, mobilising financial support for patients' care, mobilising patients to access diagnostic and therapeutic interventions. Healthcare worker interventions include diagnostic, management, and referral guidelines, as well as research and educational interventions related to symptom identification, therapeutic strategies, and effective patient communication skills. In addition, it was highlighted that healthcare systems should be bolstered to improve insurance coverage and access to integrated multi-disciplinary clinical care. CONCLUSIONS: Study participants were able to identify potential initiatives to improve paediatric rheumatology care in Kenya. Additional efforts are underway to design, implement and monitor the impact of some of these potential interventions.

Screening utility and acceptability of the Kiswahili-pGALS (paediatric Gait, Arms, Legs, Spine) at a tertiary referral hospital in Kenya-A diagnostic accuracy study.

BACKGROUND: Paediatric rheumatic diseases cause considerable disease burden to children and their families (Moorthy LN, Peterson MGE, Hassett AL, et al, Pediatric Rheumatology 8:20, 2010). Delayed diagnosis is a significant determinant of severity and mortality attributed to these conditions (Foster HE, Eltringham MS, Kay LJ, et al, Arthritis Care Res 57(6):921-7, 2007). pGALS is a simple clinical tool used to assess joints and identify musculoskeletal (MSK) conditions in school-going children to enable early referral to paediatric rheumatologists. OBJECTIVES: This study aimed to translate and determine the diagnostic accuracy and acceptability of a Kiswahili version of the pGALS screening tool among Kiswahili-speaking children. METHODS: The pGALS screening questions were translated into Kiswahili according to the World Health Organisation (WHO) standard for translation of a tool. The validity of the Kiswahili PGALS was ascertained and acceptability rated (time taken, discomfort). Using systematic random sampling, we enrolled children aged 5-16 years presenting at the Aga Khan University Hospital's (AKUH) emergency department in Kenya, who spoke Kiswahili and had symptoms suggestive of an MSK condition. Those already under follow-up at the paediatric rheumatology service at AKUH were excluded. MSK assessment was undertaken by two resident doctors using the newly translated Kiswahili-pGALS and findings were compared with a paediatric rheumatologist examination ('gold-standard') on the same day, and who was blinded to the pGALS findings. We analysed demographic details of the participants and determined the diagnostic accuracy by cross tabulation of the index test results by the results of the reference standard. RESULTS: One hundred children with a median age of nine years (IQR 7-11) were enrolled. The sensitivity and specificity of the Kiswahili-pGALS screening tool were 76.8% (95%CI 63.6-87.0%) and 40.0% (95%CI 23.9-57.9%), respectively. The diagnostic accuracy was 62.7% (95%CI 52.1-72.1%), area under the ROC was 0.58 (95%CI 0.48-0.68). The median time to perform the Kiswahili-pGALS was 5.0 min (IQR 3.5-6.0 min). Ninety percent of the guardians found the practice of Kiswahili-pGALS to have none, or only some discomfort. CONCLUSIONS: The Kiswahili-pGALS's was found to be a useful screening tool to aid early identification of MSK conditions in Kiswahili-speaking settings. However, the low specificity implies that relatively large number of false positives would still need to be reviewed by a rheumatologist if the tool is adapted for use.

An iceberg I can't handle: a qualitative inquiry on perceptions towards paediatric rheumatology among healthcare workers in Kenya.

BACKGROUND: Delay in diagnosis and access to specialist care is a major problem for many children and young people with rheumatic disease in sub-Saharan Africa. Most children with symptoms of rheumatic disease present to non-specialists for care. There is an urgent need to understand and scale-up paediatric rheumatology knowledge and skills amongst non-specialist healthcare workers to promote early diagnosis, prompt referral, and management. PURPOSE: We evaluated the knowledge, attitudes and practices towards diagnosis and care of paediatric rheumatology patients among health care workers in Kenya. METHODS: We conducted 12 focus group discussions with clinical officers (third-tier community health workers) nurses, general practitioners and paediatricians across 6 regions in Kenya. Interviews were conducted on zoom, audio-recorded, transcribed, and analysed using NVIVO software. RESULTS: A total of 68 individuals participated; 11 clinical officers, 12 nurses, 10 general practitioners, 27 paediatricians and 7 others. Most (n = 53) were female, and the median age was 36 years (range 31-40 years). Fifty per cent of the participants (34 of 68) worked in public health facilities. Our study revealed gaps in knowledge of paediatric rheumatology amongst healthcare workers which contributes to delayed diagnosis and poor management. Healthcare workers reported both positive and negative attitudes towards diagnosis and care of paediatric rheumatology patients. Perceived complexity and lack of knowledge in diagnosis, management and lack of health system clinical pathways made all cadres of healthcare workers feel helpless, frustrated, inadequate and incompetent to manage paediatric rheumatology patients. Positive attitudes arose from a perceived feeling that paediatric rheumatology patients pose unique challenges and learning opportunities. CONCLUSION: There is an urgent need to educate healthcare workers and improve health systems to optimize clinical care for paediatric rheumatology patients.

The molecular structure of IFT-A and IFT-B in anterograde intraflagellar transport trains.

Anterograde intraflagellar transport (IFT) trains are essential for cilia assembly and maintenance. These trains are formed of 22 IFT-A and IFT-B proteins that link structural and signaling cargos to microtubule motors for import into cilia. It remains unknown how the IFT-A/-B proteins are arranged into complexes and how these complexes polymerize into functional trains. Here we use in situ cryo-electron tomography of Chlamydomonas reinhardtii cilia and AlphaFold2 protein structure predictions to generate a molecular model of the entire anterograde train. We show how the conformations of both IFT-A and IFT-B are dependent on lateral interactions with neighboring repeats, suggesting that polymerization is required to cooperatively stabilize the complexes. Following three-dimensional classification, we reveal how IFT-B extends two flexible tethers to maintain a connection with IFT-A that can withstand the mechanical stresses present in actively beating cilia. Overall, our findings provide a framework for understanding the fundamental processes that govern cilia assembly.

Context and priorities for health systems strengthening for pain and disability in low- and middle-income countries: a secondary qualitative study and content analysis of health policies.

Musculoskeletal (MSK) health impairments contribute substantially to the pain and disability burden in low- and middle-income countries (LMICs), yet health systems strengthening (HSS) responses are nascent in these settings. We aimed to explore the contemporary context, framed as challenges and opportunities, for improving population-level prevention and management of MSK health in LMICs using secondary qualitative data from a previous study exploring HSS priorities for MSK health globally and (2) to contextualize these findings through a primary analysis of health policies for integrated management of non-communicable diseases (NCDs) in select LMICs. Part 1: 12 transcripts of interviews with LMIC-based key informants (KIs) were inductively analysed. Part 2: systematic content analysis of health policies for integrated care of NCDs where KIs were resident (Argentina, Bangladesh, Brazil, Ethiopia, India, Kenya, Malaysia, Philippines and South Africa). A thematic framework of LMIC-relevant challenges and opportunities was empirically derived and organized around five meta-themes: (1) MSK health is a low priority; (2) social determinants adversely affect MSK health; (3) healthcare system issues de-prioritize MSK health; (4) economic constraints restrict system capacity to direct and mobilize resources to MSK health; and (5) build research capacity. Twelve policy documents were included, describing explicit foci on cardiovascular disease (100%), diabetes (100%), respiratory conditions (100%) and cancer (89%); none explicitly focused on MSK health. Policy strategies were coded into three categories: (1) general principles for people-centred NCD care, (2) service delivery and (3) system strengthening. Four policies described strategies to address MSK health in some way, mostly related to injury care. Priorities and opportunities for HSS for MSK health identified by KIs aligned with broader strategies targeting NCDs identified in the policies. MSK health is not currently prioritized in NCD health policies among selected LMICs. However, opportunities to address the MSK-attributed disability burden exist through integrating MSK-specific HSS initiatives with initiatives targeting NCDs generally and injury and trauma care.

EULAR/PRES recommendations for vaccination of paediatric patients with autoimmune inflammatory rheumatic diseases: update 2021.

OBJECTIVES: Recent insights supporting the safety of live-attenuated vaccines and novel studies on the immunogenicity of vaccinations in the era of biological disease-modifying antirheumatic drugs in paediatric patients with autoimmune/inflammatory rheumatic diseases (pedAIIRD) necessitated updating the EULAR recommendations. METHODS: Recommendations were developed using the EULAR standard operating procedures. Two international expert committees were formed to update the vaccination recommendations for both paediatric and adult patients with AIIRD. After a systematic literature review, separate recommendations were formulated for paediatric and adult patients. For pedAIIRD, six overarching principles and seven recommendations were formulated and provided with the level of evidence, strength of recommendation and Task Force level of agreement. RESULTS: In general, the National Immunisation Programmes (NIP) should be followed and assessed yearly by the treating specialist. If possible, vaccinations should be administered prior to immunosuppressive drugs, but necessary treatment should never be postponed. Non-live vaccines can be safely given to immunosuppressed pedAIIRD patients. Mainly, seroprotection is preserved in patients receiving vaccinations on immunosuppression, except for high-dose glucocorticoids and B-cell depleting therapies. Live-attenuated vaccines should be avoided in immunosuppressed patients. However, it is safe to administer the measles-mumps-rubella booster and varicella zoster virus vaccine to immunosuppressed patients under specific conditions. In addition to the NIP, the non-live seasonal influenza vaccination should be strongly considered for immunosuppressed pedAIIRD patients. CONCLUSIONS: These recommendations are intended for paediatricians, paediatric rheumatologists, national immunisation agencies, general practitioners, patients and national rheumatology societies to attain safe and effective vaccination and optimal infection prevention in immunocompromised pedAIIRD patients.

Successful stopping of biologic therapy for remission in children and young people with juvenile idiopathic arthritis.

OBJECTIVES: Clinicians concerned about long-term safety of biologics in JIA may consider tapering or stopping treatment once remission is achieved despite uncertainty in maintaining drug-free remission. This analysis aims to (i) calculate how many patients with JIA stop biologics for remission, (ii) calculate how many later re-start therapy and after how long, and (iii) identify factors associated with re-starting biologics. METHODS: Patients starting biologics between 1 January 2010 and 7 September 2021 in the UK JIA Biologics Register were included. Patients stopping biologics for physician-reported remission, those re-starting biologics and factors associated with re-starting, were identified. Multiple imputation accounted for missing data. RESULTS: Of 1451 patients with median follow-up of 2.7 years (IQR 1.4, 4.0), 269 (19%) stopped biologics for remission after a median of 2.2 years (IQR 1.7, 3.0). Of those with follow-up data (N = 220), 118 (54%) later re-started therapy after a median of 4.7 months, with 84% re-starting the same biologic. Patients on any-line tocilizumab (prior to stopping) were less likely to re-start biologics (vs etanercept; odds ratio [OR] 0.3; 95% CI: 0.2, 0.7), while those with a longer disease duration prior to biologics (OR 1.1 per year increase; 95% CI: 1.0, 1.2) or prior uveitis were more likely to re-start biologics (OR 2.5; 95% CI: 1.3, 4.9). CONCLUSIONS: This analysis identified factors associated with successful cessation of biologics for remission in JIA as absence of uveitis, prior treatment with tocilizumab and starting biologics earlier in the disease course. Further research is needed to guide clinical recommendations.

Developing new portals to safety for domestic abuse survivors in the context of the pandemic.

This study examined the emergence and implementation of community touchpoints established in the UK during the COVID-19 pandemic for victims/survivors of domestic abuse (DA). Community touchpoints are designated places, both online and in accessible settings such as pharmacies and banks, where victims/survivors can seek confidential advice and be directed to expert DA services. The research adopted a case study approach and explored a range of perspectives through expert interviews, document analysis, consultation with survivors and stakeholders and a survey of DA co-ordinators. Four national community touchpoint schemes were identified and, of these, three were implemented rapidly and were available in 2020-2021 when the UK experienced lockdowns. Partnerships between Government/voluntary organisations and commercial businesses-assisted design and implementation. Some stakeholders considered that the schemes lacked responsivity to the local context and noted challenges in providing a confidential service in rural areas. Whilst pharmacies, banks and online spaces were identified as non-stigmatised and trusted places to seek advice, community touchpoints were judged less accessible for some groups including those experiencing digital poverty and victims whose movements were heavily scrutinised. Most of the touchpoint schemes targeted adults only. There were also concerns about whether frontline staff in commercial businesses received sufficient training. Whilst robust evidence of outcomes was limited, there were indications that the schemes had achieved good reach with some early evidence of take-up. Testimonials indicated that victims/survivors were using the touchpoints in flexible ways which met their needs. Moreover, the wide reach and visibility of these initiatives delivered in non-stigmatised settings may have served to raise public awareness of DA, reducing the silence that has traditionally surrounded it. Further research into the use and impact of these initiatives is required and there may be future potential to extend community touchpoints to include children and young people experiencing DA.

Efficacy, Immunogenicity and Safety of Vaccination in Pediatric Patients With Autoimmune Inflammatory Rheumatic Diseases (pedAIIRD): A Systematic Literature Review for the 2021 Update of the EULAR/PRES Recommendations.

Background: In 2011, the first European League Against Rheumatism (EULAR) vaccination recommendations for pediatric patients with autoimmune inflammatory rheumatic diseases (pedAIIRD) were published. The past decade numerous new studies were performed to assess the safety, efficacy and immunogenicity of vaccinations in pedAIIRD. A systematic literature review (SLR) was therefore performed to serve as the basis for the updated 2021 EULAR/PRES recommendations. Methods: An SLR was performed according to the standard operating procedures for EULAR-endorsed recommendations. Primary outcomes were efficacy, immunogenicity and safety of vaccination in pedAIIRD. The search was performed in Medline, Embase and the Cochrane Library and included studies published from November 2010 until July 2020. Results: The SLR yielded 57 studies which were included for critical appraisal and data extraction. Only 8 studies described the occurrence of vaccine-preventable infections after vaccination (efficacy), none of these studies were powered to assess efficacy. The majority of studies assessed (humoral) immune responses as surrogate endpoint for vaccine efficacy. Studies on non-live vaccines showed that these were safe and in general immunogenic. Biologic disease-modifying antirheumatic drugs (bDMARDs) in general did not significantly reduce seroprotection rates, except for B-cell depleting therapies which severely hampered humoral responses. Four new studies on human papilloma virus vaccination showed that this vaccine was safe and immunogenic in pedAIIRD. Regarding live-attenuated vaccinations, level 1 evidence of the measles mumps rubella (MMR) booster vaccination became available which showed the safety of this booster for patients treated with methotrexate. In addition, level 3 evidence became available that suggested that the MMR and varicella zoster virus (VZV) vaccination for patients on low dose glucocorticosteroids and bDMARDs might be safe as well. Conclusions: The past decade, knowledge on the safety and immunogenicity of (live-attenuated) vaccines in pedAIIRD significantly increased. Data on efficacy (infection prevention) remains scarce. The results from this SLR are the basis for the updated EULAR/PRES vaccination recommendations in pedAIIRD.

A Mixed Method Study: Defining the Core Learning Needs of Nurses Delivering Care to Children and Young People with Rheumatic Disease to Inform Paediatric Musculoskeletal Matters, a Free Online Educational Resource.

Children and young people with rheumatic diseases and their families are often supported by nurses who may not have had specialist training in paediatric rheumatology. The purpose of our study was to establish the core learning needs of all nurses who may encounter these children and young people in their clinical practice and use this information to inform the content and format of Paediatric Musculoskeletal Matters Nursing (PMM-Nursing) Engagement with nurses working in different roles and with various levels of experience in musculoskeletal medicine informed these learning needs and PMM-Nursing content. Mixed methods ascertained learning needs under the following themes: (1) Need for increased awareness about rheumatic disease; (2) Impact of experience and nursing role; (3) Need for increased knowledge about rheumatic disease and management. In addition, our methods informed design components for an impactful learning and information resource. Representatives from stakeholder nursing groups, social sciences, and web development used this information to create a suitable framework for PMM-Nursing. The content of PMM-Nursing is now live and freely available.

Bimodal endocytic probe for three-dimensional correlative light and electron microscopy.

We present a bimodal endocytic tracer, fluorescent BSA-gold (fBSA-Au), as a fiducial marker for 2D and 3D correlative light and electron microscopy (CLEM) applications. fBSA-Au consists of colloidal gold (Au) particles stabilized with fluorescent BSA. The conjugate is efficiently endocytosed and distributed throughout the 3D endolysosomal network of cells and has an excellent visibility in both fluorescence microscopy (FM) and electron microscopy (EM). We demonstrate that fBSA-Au facilitates rapid registration in several 2D and 3D CLEM applications using Tokuyasu cryosections, resin-embedded material, and cryoelectron microscopy (cryo-EM). Endocytosed fBSA-Au benefits from a homogeneous 3D distribution throughout the endosomal system within the cell, does not obscure any cellular ultrastructure, and enables accurate (50-150 nm) correlation of fluorescence to EM data. The broad applicability and visibility in both modalities makes fBSA-Au an excellent endocytic fiducial marker for 2D and 3D (cryo)CLEM applications.

Repo-Man/protein phosphatase 1 SUMOylation mediates binding to lamin A and serine 22 dephosphorylation.

Lamin A phosphorylation/de-phosphorylation is an important process during cells division as it allows for nuclear envelope (NE) disassembly at mitotic entry and its re-assembly during mitotic exit. Several kinases have been identified as responsible for these phosphorylations, but no protein phosphatase has been implicated in their reversal. One of the mitotic phosphosites in lamin A responsible for its dynamic behaviour is serine 22 (S22) which is de-phosphorylated during mitotic exit. Recent evidence has also linked the nuclear pool of lamin A S22ph in interphase to gene expression regulation. Previous work suggested that the phosphatase responsible for lamin A S22 de-phosphorylation is chromatin bound and interacts with lamin A via SUMO-SIM motives. We have previously reported that Repo-Man/protein phosphatase 1 (PP1) is a chromatin-associated phosphatase that regulates NE reformation. Here we propose that Repo-Man/PP1 phosphatase mediates lamin A S22 de-phosphorylation. We indeed show that depletion of Repo-Man leads to NE defects, causes hyperphosphorylation of lamin A S22 that can be rescued by a wild-type but not a SUMOylation-deficient mutant. Lamin A and Repo-Man interact in vivo and in vitro, and the interaction is mediated by SUMOylation. Moreover, the localization of Repo-Man/PP1 to the chromatin is essential for lamin A S22 de-phosphorylation.

A Comparison of Different Approaches for Characterizing Microplastics in Selected Personal Care Products.

Any uncertainty in determining numbers of microplastics in the environment may be a barrier to assessing their impact and may stem from various aspects of methodologies used to quantify them. We undertook a comparison of approaches to quantify and characterize microplastics in 4 personal care products. The aim was not only to determine how many particles were present but to assess any differences due to the methods used. Counting of extracted microplastics was undertaken using particle size analysis, light microscopy, and imaging flow cytometry. Micro-Fourier transform infrared spectroscopy (µ-FTIR) was used to characterize the particles in each product. The mean size distribution of microplastics differed depending on the method employed, and it was apparent that imaging flow cytometry was affected by high background noise that may require staining of plastics to overcome. The application of µ-FTIR confirmed polyethylene as the microplastic in each product. Methodological challenges encountered in the study and the literature have highlighted the need for standardization of methods for determining microplastics. Environ Toxicol Chem 2022;41:880-887. © 2021 The Authors. Environmental Toxicology and Chemistry published by Wiley Periodicals LLC on behalf of SETAC.

A cryo-ET survey of microtubules and intracellular compartments in mammalian axons.

The neuronal axon is packed with cytoskeletal filaments, membranes, and organelles, many of which move between the cell body and axon tip. Here, we used cryo-electron tomography to survey the internal components of mammalian sensory axons. We determined the polarity of the axonal microtubules (MTs) by combining subtomogram classification and visual inspection, finding MT plus and minus ends are structurally similar. Subtomogram averaging of globular densities in the MT lumen suggests they have a defined structure, which is surprising given they likely contain the disordered protein MAP6. We found the endoplasmic reticulum in axons is tethered to MTs through multiple short linkers. We surveyed membrane-bound cargos and describe unexpected internal features such as granules and broken membranes. In addition, we detected proteinaceous compartments, including numerous virus-like capsid particles. Our observations outline novel features of axonal cargos and MTs, providing a platform for identification of their constituents.

Microgravity and space radiation inhibit autophagy in human capillary endothelial cells, through either opposite or synergistic effects on specific molecular pathways.

Microgravity and space radiation (SR) are two highly influential factors affecting humans in space flight (SF). Many health problems reported by astronauts derive from endothelial dysfunction and impaired homeostasis. Here, we describe the adaptive response of human, capillary endothelial cells to SF. Reference samples on the ground and at 1g onboard permitted discrimination between the contribution of microgravity and SR within the combined responses to SF. Cell softening and reduced motility occurred in SF cells, with a loss of actin stress fibers and a broader distribution of microtubules and intermediate filaments within the cytoplasm than in control cells. Furthermore, in space the number of primary cilia per cell increased and DNA repair mechanisms were found to be activated. Transcriptomics revealed the opposing effects of microgravity from SR for specific molecular pathways: SR, unlike microgravity, stimulated pathways for endothelial activation, such as hypoxia and inflammation, DNA repair and apoptosis, inhibiting autophagic flux and promoting an aged-like phenotype. Conversely, microgravity, unlike SR, activated pathways for metabolism and a pro-proliferative phenotype. Therefore, we suggest microgravity and SR should be considered separately to tailor effective countermeasures to protect astronauts' health.

Developmentally appropriate transitional care during the Covid-19 pandemic for young people with juvenile-onset rheumatic and musculoskeletal diseases: the rationale for a position statement.

BACKGROUND: The importance of developmentally appropriate transitional care in young people with juvenile-onset rheumatic and musculoskeletal disease is well recognised. The Paediatric Rheumatology European Society (PReS) / European League Against Rheumatism (EULAR) Taskforce has developed international recommendations and standards for transitional care and a growing evidence base supports the positive benefits of such care. However, there is also evidence that universal implementation has yet to be realised. In 2020, against this background the COVID-19 pandemic arrived with significant impact on all our lives, young and old, patient, public and professional alike. The unfortunate reality of the pandemic with potential for unfavourable outcomes on healthcare provision during transition was acknowledged by the PReS working groups in a position statement to support healthcare professionals, young people and their caregivers. AIM: The aim of this review is to present the literature which provides the rationale for the recommendations in the PReS Position Statement. The following areas are specifically addressed: the prime importance of care coordination; the impact of the pandemic on the various aspects of the transition process; the importance of ensuring continuity of medication supply; the pros and cons of telemedicine with young people; ensuring meaningful involvement of young people in service development and the importance of core adolescent health practices such as routine developmental assessment psychosocial screening and appropriate parental involvement during transitional care.

A mixed methods evaluation of the Paediatric Musculoskeletal Matters (PMM) online portfolio.

BACKGROUND: The PMM Portfolio is comprised of the Paediatric Musculoskeletal Matters (PMM) website, the paediatric Gait, Arms, Legs and Spine (pGALS) app and e-learning modules (ELM). The target audiences are non-specialists in paediatric musculoskeletal medicine. Our study aimed to evaluate impact on learning and clinical practice. METHODS: Mixed methods (analytics, online survey, interviews) were used with PMM and ELM registered users and purposive sampling of users using international contacts within paediatrics and paediatric rheumatology. Data was analysed using descriptive statistics and qualitative techniques. A Paired T-Test compared self-rated confidence before and after use of the PMM Portfolio. RESULTS: There has been wide reach for all the e-resources; PMM website (662,827 hits, 262,476 users, 214 countries, data 31st July 2020); pGALS app (12,670 downloads, 70 countries, data 31st July 2020); ELM (150 users, 30 countries, data 30th May 2019). There were 164 responses (students, trainees and health care professionals) to the survey from 25 countries. Most responders deemed the PMM Portfolio useful / very useful for their learning with significantly increased self-rated confidence in their clinical examination and reasoning skills following access to the PMM website, p = < 0.01, pGALS app, p = < 0.01 and ELM, p = < 0.01. The most popular PMM website pages related to clinical assessment techniques (especially pGALS). There was high uptake of the pGALS app and pGALS ELM especially from trainees and allied health professionals. Many clinicians reported the PMM Portfolio to be useful when used to teach others. User feedback reported that easy navigation, open access, clinical images and cases were the most valued features. User feedback highlighted need to increase awareness of the e-resources through training programmes. CONCLUSIONS: The PMM Portfolio was developed to aid learning for clinicians who are not specialists in paediatric MSK medicine. Our evaluation demonstrates wide international reach and positive feedback on learning. The PMM Portfolio is a highly useful e-resource for paediatric rheumatologists in their teaching of others to raise awareness, facilitate early diagnosis and referral of children with suspected disease. The wide user engagement informed future PMM Portfolio development and the mixed method of evaluation is transferable to other e-resources.

'Snakes & Ladders': factors influencing access to appropriate care for children and young people with suspected juvenile idiopathic arthritis - a qualitative study.

BACKGROUND: Many children and young people with juvenile idiopathic arthritis (JIA) experience delay in diagnosis and access to right care. The reasons for delay are multi-factorial and influenced by patient and family, clinician and organisational factors. Our aim was to explore the experiences of care, from initial symptoms to initial referral to paediatric rheumatology. METHODS: We analysed one-to-one and joint qualitative interviews with families of children with JIA (n = 36) presenting to a regional paediatric rheumatology service in the UK. We interviewed 51 family members (including mothers, fathers, patients, grandmothers and an aunt) and 10 health professionals (including orthopaedic surgeons, paediatricians, paediatric immunologist, General Practitioner and nurse) and a teacher involved in the care pathway of these JIA patients. Interviews were audio-recorded and analysed according to the standard procedures of rigorous qualitative analysis - coding, constant comparison, memoing and deviant case analysis. RESULTS: The median age of the children was 6 years old (range 1-17), with a spread of JIA subtypes. The median reported time to first PRh MDT visit from symptom onset was 22 weeks (range 4-364 weeks). Three key factors emerged in the pathways to appropriate care: (i) the persistence of symptoms (e.g. 'change' such as limp or avoidance of previously enjoyed activities); (ii) the persistence of parents help-seeking actions (e.g. repeat visits to primary and hospital care with concern that their child is not 'normal'; iii) the experience and skills of health professionals resulting in different trajectories (e.g. no-real-concern-at-this-point or further-investigation-is-required). JIA was more likely to be considered amongst health practitioner if they had prior experiences of a child with JIA (moreso with a 'protracted pathway') or exposure to paediatric rheumatology in their training. Conversely JIA was more likely to be overlooked if the child had comorbidity such as learning disability or a chronic illness. CONCLUSIONS: Care pathways are often 'turbulent' prior to a diagnosis of JIA with physical and emotional distress for families. There is need for greater awareness about JIA amongst health care professionals and observations of change (from family and non-health care professionals such as teachers) are key to trigger referral for paediatric rheumatology opinion.