Adult Rheumatology Research Articles

A meta-analysis and systematic review of the use of transition tools for patients transitioning from paediatric to adult rheumatology services

Abstract Objectives Transition from paediatric to adult care can be challenging, but transition tools are designed to increase successful rates of transfer. We aimed to conduct a meta-analysis and systematic review into the use of transition tools on transfer of care from paediatric to adult services for rheumatology patients. Methods An extensive literature search was conducted using Medline, PubMed and Embase. 12 papers were included in a systematic review and 8 papers in the meta-analysis looking at use of transition tools on successful follow up in adult clinics. Results The meta-analysis showed 86.6% (95% CI: 53.3–80.6%) of patients who used a transition tool attended an adult outpatient clinic within 1 year of their last paediatric appointment, compared with 67.7% (95% CI: 56.7–99.7%) of patients who successfully transferred without a transition tool. Conclusions The systematic review demonstrated several unique transition tools with local success but no dominant method. This paper demonstrated a general positive influence of transition tools on successful transfer, but more research is needed to strengthen the association.

Childhood-onset versus adult-onset Takayasu arteritis: A study of 239 patients from China

Objective: We aimed to compare clinical features, vascular involvement, disease activity, and management between Chinese patients with childhood-onset Takayasu arteritis (cTAK) and adult-onset Takayasu arteritis (aTAK).Methods: In total, 239 patients with TAK from the pediatric and adult rheumatology departments of two tertiary care centers were retrospectively evaluated. Data on demographic characteristics, laboratory results, disease activity, vascular involvement, and management regimens were collected and compared between the cTAK and aTAK patients.Results: In total, 85 cTAK and 154 aTAK patients were enrolled. There was a significant difference in the female/male ratio between the cTAK and aTAK groups (2.3:1 vs. 10.8:1, P < 0.001). Fever, hypertension, arrhythmia, and heart failure were more frequent in the cTAK group. The median Indian Takayasu Clinical Activity Score-scores were significantly higher in the cTAK group compared to the aTAK group (5 vs. 3, P = 0.004). The prevalence of supra-aortic artery involvement was significantly higher in the aTAK group than in the cTAK group (P < 0.05). The rate of involvement of coronary and renal arteries was significantly lower in the aTAK group than in the cTAK group (P < 0.05). Compared to the aTAK patients, the cTAK patients were more likely to be treated with biological agents (76.5% vs. 9.1%, P < 0.001).Conclusion: cTAK patients had more severe disease and inflammation. Coronary and renal artery involvement was more common among cTAK patients, whereas supra-aortic artery involvement was more common in aTAK patients increasing their risk of cerebral infarction. Biologics were used more frequently in cTAK patients.

Transition readiness assessment in Portuguese adolescents and young adults with pediatric-onset rheumatic diseases: a single-center study.

In this study, we aimed to assess the transition readiness levels amongst patients with childhood-onset rheumatic diseases. Additionally, we sought to identify and analyze predictive factors associated with better transition readiness skills in adolescent and young adult (AYAs) patients. This is a monocentric cross-sectional study that includes patients between 14 and 26 years of age who attended outpatient pediatric and young adult rheumatology appointments between October and December of 2023 and that were diagnosed with an immune-mediated rheumatic disease before reaching 18 years of age, with at least 1 year of disease duration. Patients were presented with a questionnaire that contained demographic and clinical questions, TRACS (Questionário de Preparação da Transição para a Autonomia nos Cuidados de Saúde) questionnaire - a validated Portuguese version of the Transition Readiness Assessment Questionnaire (TRAQ), and Hospital Anxiety and Depression Scale (HADS) questionnaire. Data was analyzed to assess the significant associations between the different variables and transition readiness outcome measured by the TRACS. Descriptive statistics, statistical comparisons and logistic regression analysis were performed. A total of 69 patients with a median age of 20 [17.5-22.5] were included in this study. The median TRACS score was 4.41 [4.09-4.74]. Significantly higher TRACS scores were observed in patients who were female, 18 years of age or older, had a higher level of education, were employed, had active disease or that belonged to middle-class (when compared to patients belonging to upper- middle class). The logistic regression analysis demonstrated that being a female or having an educational status equal to 12th grade or superior emerged as predictors of higher transition readiness levels. Our study identified female sex and higher level of education as predictors of increased transition readiness levels. Therefore, healthcare providers should consider these variables when assessing patients for transition readiness and focus on improving transition process, especially in male and less educated AYAs.

Relationship between ethnicity and multidisciplinary intervention for young people with rheumatic and musculoskeletal diseases

There is growing recognition of the impact of societal factors on health throughout a patient's lifespan. The COVID-19 pandemic has exposed the impact of racial disparity on health outcomes. We aimed to investigate the association between ethnicity and the multidisciplinary team (MDT) interventions for young people (YP) with complex care needs. This retrospective, single-centre, cross-sectional study was conducted within the department of adolescent and young adult rheumatology at University College Hospital, London, between August 2019 and August 2021. We extracted demographic, clinical and laboratory data. The index of multiple deprivation was extracted from the Office for National Statistics database. R software was used for analysis. We identified 310 YP referred to the MDT with a median age of 18 years (interquartile range 17-19). The female patient to male patient ratio was 2.4. Over a third of our cohort were from deprived areas. Comparison between Black, Asian and minority ethnic (BAME) and White ethnic groups revealed significant differences in terms of referral for pain optimisation (p=0.006), social support (p<0.00001), and adherence and non-clinic attendance (p=0.0004). Our findings reveal the importance of quality data for early identification and support of vulnerable YP, particularly those from BAME communities.

Measuring Transition Readiness of Patients After Transfer from Pediatric to Adult Care in Rheumatology.

Transitional care is essential to maintain the continuity of care in younger patients with rheumatic diseases. In this study, we aimed to assess the transition readiness of rheumatology patients who had already transferred from pediatric to adult care using a questionnaire. We included young adult rheumatology patients who had already transferred to adult rheumatology care. The Transition Readiness Assessment Questionnaire (TRAQ) was used in the adult rheumatology clinic to assess the patients' readiness; a retrospective chart review was conducted to include diagnosis, age at diagnosis, age at transfer, and current age. Three hundred and ten patients (184 female and 126 male) participated in this study. The mean age at diagnosis, the mean age at transfer, and the mean age at the time of the study were 10.7 ± 4.29, 21.1 ± 1.69, and 24.0 ± 2.26 years, respectively. Most of the patients had familial Mediterranean fever, followed by arthritis, connective tissue disorders, and other diseases. Tracking health issues was the lowest-scored domain. Females scored significantly higher than males in the tracking health issue domain (P = .006) and managing health issue domain (P = .028) but not in the overall TRAQ score (P = .053). Patients in different diagnosis and transfer age groups scored similarly across the domains. In this study, females performed better than males in 2 domains of the TRAQ questionnaire. Diagnoses or transfer age groups were not associated with TRAQ outcomes.

Transition to Adulthood through Coaching and Empowerment in Rheumatology (TRACER): A feasibility study protocol.

The transition from pediatric to adult care for patients with chronic disease is a vulnerable period, with risks of disengagement from care and subsequent complications of inadequately managed disease. This period comes at a time when there are many other transitions occurring in the young person's life, including changes to vocation, social supports, and to their physiology. The aim of the TRACER study is to assess the feasibility of conducting a multi-center, randomized-controlled trial of a virtual Transition Coach Intervention in youth transferring from pediatric to adult rheumatology care. Patients are being recruited at their last pediatric rheumatology visit from McMaster Children's Hospital and Children's Hospital, London Health Sciences Centre in Ontario, Canada. Participants are then randomized to standard of care or to eight transition coaching sessions, covering topics around health management, future planning, and self-advocacy. The primary outcomes of the study are to demonstrate protocol feasibility, including optimal recruitment and consent rates, ≥ 90% coaching session completion, and complete data collection with ≤ 5% missing data. Baseline demographics, transition readiness, global functional assessment, disease activity, and self-efficacy will be collected to characterize the study population. Recruitment has begun and is estimated to last 19 months. This study will inform the design of a robust, multi-centered, randomized-controlled study to investigate the impact of a virtual transition coaching program in supporting the physical, mental, and social well-being of youth with rheumatic disease transitioning into adult care. Clinical trial registration: ClinicalTrials.Gov protocol ID: 14499.

Assessing Competencies, Needs, and Satisfaction With the Transition From Pediatric to Adult Health Care in Rheumatology: Development and Validation of the Transition-KompAZ.

The aim of this study was to develop and psychometrically validate a self-report instrument to assess (1) competencies, (2) needs, and (3) satisfaction among youth transitioning from pediatric to adult rheumatology. The Transition-KompAZ was developed in several steps with conceptual and psychometric analyses. To test its psychometric properties, the instrument was administered to adolescents and young adults (AYAs; 16-25 years) with inflammatory rheumatic diseases before (group 1) or after (group 2) transfer to adult rheumatology. A 2-factor, higher-order confirmatory factor analysis (CFA) model was applied to test the hypothesized factor structure. Internal consistency was estimated using the approach of Raykov with the factor loadings and error variances estimated in the CFA. Spearman rank correlation coefficients were used to assess construct validity. The Transition-KompAZ includes the following modules: (1) competencies in transition (knowledge, self-management), (2) needs (healthcare services, information), and (3) satisfaction (general, transitional care). A total of 173 AYAs (group 1: n = 86; group 2: n = 87) from 12 rheumatology sites completed the Transition-KompAZ. It showed good model fit (comparative fit index > 0.9; Tucker-Lewis index > 0.9; weighted root mean square residual < 0.9) with good internal consistency. The instrument demonstrated moderate-to-good construct validity and good test-retest reliability. The Transition-KompAZ appears to be a reliable tool for assessing important dimensions of transition. It may support a structured and individualized transition, as well as the evaluation of transition services. However, further studies are required to assess its predictive value in terms of transfer readiness and successful transition.

Longitudinal program evaluation of an inter-institutional mentorship network for pediatric rheumatology using a quality improvement framework

BackgroundThe American College of Rheumatology (ACR)/Childhood Arthritis and Rheumatology Research Alliance (CARRA) Mentoring Interest Group (AMIGO) is an inter-institutional mentorship program launched to target mentorship gaps within pediatric rheumatology. Initial program evaluation indicated increased mentorship access. Given the small size of the pediatric rheumatology workforce, maintaining a consistent supply of mentors was a potential threat to the longevity of the network. Our aims were to: (i) describe the sustainability of AMIGO over the period 2011–2018, (ii) highlight ongoing benefits to participants, and (iii) describe challenges in the maintenance of a mentorship network.MethodsA mixed-methods approach centered on a quality improvement framework was used to report on process and outcomes measures associated with AMIGO annual cycles.ResultsUS and Canada Pediatric rheumatology workforce surveys identified 504 possible participants during the time period. As of fall 2018, 331 unique individuals had participated in AMIGO as a mentee, mentor or both for a program response rate of 66% (331/504). Survey of mentees indicated high satisfaction with impact on general career development, research/scholarship and work-life balance. Mentors indicated increased sense of connection to the community and satisfaction with helping mentees despite limited perceived benefit to their academic portfolios. Based on AMIGO’s success, a counterpart program for adult rheumatology, Creating Adult Rheumatology Mentorship in Academia (CARMA), was launched in 2018.ConclusionsDespite the challenges of a limited workforce, AMIGO continues to provide consistent access to mentorship opportunities for the pediatric rheumatology community. This experience can inform approaches to mentorship gaps in other academic subspecialties.

Ready to leave? – Adolescents’ and parents’ perceptions of transition from paediatric to adult rheumatology care

BackgroundIn Sweden, approximately 2000 children live with Juvenile Idiopathic Arthritis (JIA). About half of them continue to have an active disease and need to transfer to adult rheumatology care. This study aimed to investigate Swedish adolescents’ and parents´ perceptions of readiness for transition from pediatric to adult rheumatology care.MethodsThe study was a cross-sectional quantitative study. Patients at the pediatric rheumatology clinic at a university hospital in Sweden and members of The Swedish National Organization for Young Rheumatics aged 14–18 and their parents were invited to participate in the study. Data was collected with the Readiness for Transition Questionnaire (RTQ) focusing on adolescents' transition readiness, adolescents' healthcare behaviors and responsibility, and parental involvement. Data were analyzed with descriptive statistics. Comparative analyses were made using non-parametric tests with significance levels of 0.05 as well as factor analyses and logistic regression.ResultsThere were 106 adolescents (85 girls, 20 boys) and 96 parents answering the RTQ. The analysis revealed that many adolescents and parents experienced that the adolescents were ill-prepared to take over responsibility for several healthcare behaviors, such as booking specialty care appointments, calling to renew prescriptions and communicating with medical staff on phone and to transfer to adult care. Parents and adolescents alike stated that it was especially difficult for the adolescents to take responsibility for healthcare behaviors meaning that the adolescents had to have direct interaction with the healthcare professionals (HCPs) at the paediatric rheumatology clinic, for example to renew prescriptions. It was evident that the adolescents who perceived they were ready to take responsibility for the aspects related to direct interaction with HCPs were more overall ready to be transferred to adult care.ConclusionAdolescents need more support to feel prepared to transfer to adult care. With the results from this study, we can develop, customize, and optimize transitional care programs in Sweden for adolescents.

Autoinflammatory Diseases: A Review.

Autoinflammatory disease (AID) is a vast spectrum of disorders characterized by recurrent attacks of sterile inflammation. Since the first cloning of the familial Mediterranean fever gene in 1997, there has been a rapid rate of discovery of new AIDs. As of 2022, there have been 485 inborn errors of immunity documented by the International Union of Immunological Societies, for which many display aspects of autoinflammation. The pathophysiology of AIDs is complex. Although many are caused by rare mutations in genes that govern innate immunity, others are polygenic, where disease expression is thought to be triggered by environmental factors in genetically predisposed hosts. AIDs range in prevalence from common entities like gout to ultrarare monogenic diseases. Whereas AIDs were initially studied in pediatric populations, it is now apparent that they can present in adulthood and even in the elderly. AIDs can be clinically challenging given their rarity, as well as the heterogeneity in presentation and underlying etiology. Although the care of AIDs can span medical disciplines, the rheumatologist often plays a central role given the inflammatory nature of these illnesses. In this review, we explore the current understanding of the pathophysiology of these complex conditions and propose a classification system for AIDs. We place an emphasis on AIDs that present to the adult rheumatologist and discuss important AIDs that can mimic more classic rheumatic diseases such as systemic lupus erythematosus and inflammatory arthritis. Finally, we offer an approach to the clinical assessment, diagnosis, and management of AIDs.

Implementing a Staff-Led Smoking Cessation Intervention in a Diverse Safety-Net Rheumatology Clinic: A Pre-Post Scalability Study in a Low-Resource Setting.

Quit Connect (QC), our specialty clinic smoking cessation intervention, supports clinic staff to check, advise, and connect willing patients to a state quit line or class. QC improved tobacco screening and quit line referrals 26-fold in a predominantly White academic health care system population. Implementing QC includes education, electronic health record (EHR) reminders, and periodic audit feedback. This study tested QC's feasibility and impact in a safety-net rheumatology clinic with a predominantly Black population. In this pre-post study, adult rheumatology visits were analyzed 12 months before through 18 months after QC intervention (November 2019 through November 2021, omitting COVID-19 peak April through November 2020). EHR data compared process and clinical outcomes, including offers, referrals to resources, completed referrals, and documented cessation. Clinic staff engaged in pre-post focus groups and questionnaires regarding intervention feasibility and acceptability. Cost-effectiveness was also assessed. Visit-level patients who smoked were 89.8% Black and 69.5% women (n = 550). Before intervention, clinic staff rarely asked patients about readiness to cut back smoking (<10% assessment). After QC intervention, staff assessed quit readiness in 31.8% of visits with patients who smoked (vs 8.1% before); 58.9% of these patients endorsed readiness to cut back or quit. Of 102 accepting cessation services, 37% (n = 17) of those reached set a quit date. Staff found the intervention feasible and acceptable. Each quit attempt cost approximately $4 to $10. In a safety-net rheumatology clinic with a predominantly Black population, QC improved tobacco screening, readiness-to-quit assessment, and referrals and was also feasible and cost-effective.

Barriers and facilitators to medical care retention for pediatric systemic lupus erythematosus in South Africa: a qualitative study

BackgroundSystemic lupus erythematosus (SLE) is a life-threatening, chronic, autoimmune disease requiring long term subspecialty care due to its complex and chronic nature. Childhood-onset SLE (cSLE) is more severe than adult-onset, and the cSLE population in South Africa has been reported to have an even higher risk than patients elsewhere. Therefore, it is critical to promptly diagnose, treat, and manage cSLE. In this paper, we aim to describe and evaluate barriers and enablers of appropriate long-term care of cSLE South Africa from the perspective of caregivers (parents or family members).MethodsCaregivers (n = 22) were recruited through pediatric and adult rheumatology clinics. Individuals were eligible if they cared for youth (≤ 19 years) who were diagnosed with cSLE and satisfied at least four of the eleven ACR SLE classification criteria. Individual in-depth, semi-structured interviews were conducted between January 2014 and December 2014, and explored barriers to and facilitators of ongoing chronic care for cSLE. Data were analyzed using applied thematic analysis.ResultsFour barriers to chronic care engagement and retention were identified: knowledge gap, financial burdens, social stigma of SLE, and complexity of the South African medical system. Additionally, we found three facilitators: patient and caregiver education, robust support system for the caregiver, and financial support for the caregiver and patient.ConclusionThese findings highlight multiple, intersecting barriers to routine longitudinal care for cSLE in South Africa and suggest there might be a group of diagnosed children who don’t receive follow-up care and are subject to loss to follow-up. cSLE requires ongoing treatment and care; thus, the different barriers may interact and compound over time with each follow-up visit. South African cSLE patients are at high risk for poor outcomes. South African care teams should work to overcome these barriers and place attention on the facilitators to improve care retention for these patients and create a model for other less resourced settings.

The transition process for paediatric rheumatology clinic patients at a single tertiary paediatric rheumatology centre in Australia.

This study aimed to examine the transition process of paediatric rheumatology patients from the Monash Children's Hospital (MCH) in Melbourne in order to identify areas that could be improved. Retrospective review of clinical data from the rheumatology database of paediatric rheumatology patients eligible for transition between January 2015 and September 2020. One hundred and sixty-five patients were included; 57 patients were transitioned. Of patients transitioned to an adult service, 38 (88%) were on medication and 14 (33%) had active disease. All patients transitioned to the general practitioner (GP) had inactive disease off medication. Juvenile idiopathic arthritis (JIA) (non-systemic) was the most common diagnosis in patients transitioned. The mean age at which transition was first discussed was 18.0 years; the first referral was made at a mean of 18.3 years. The mean age at the first adult appointment was 18.5 years. Thirty-nine (91%) patients had a referral completed and 8 (19%) had a transfer letter. Thirteen (93%) patients transferred to the GP had a transfer letter. Transfer documents to an adult public rheumatology service rated 4.3 for quality, compared to 5.5 to the GP. Transfer of care was confirmed in 40 (93%) patients transitioned to an adult service; however, correspondence was available for only 3 (7%). Although the transition process at MCH was adequate, it could be improved through earlier discussion of the process and improved referrals and documentation. A readiness-to-transfer checklist and a young adult clinic have the potential to improve the process of transition to adult rheumatology care.

Needs Assessment of Musculoskeletal Ultrasound Training in North American Pediatric Rheumatology Fellowship Programs.

Musculoskeletal ultrasound (MSUS) is widely used in adult rheumatology practice for diagnosis of arthritis and procedural guidance; however, it is not yet common practice in pediatric rheumatology. MSUS is advantageous to the pediatric population because it lacks radiation and eliminates need for sedation. This study aims to assess interest in, access to, and barriers to MSUS training in pediatric rheumatology fellowship programs in North America. A survey was developed by pediatric rheumatology providers with experience in medical and/or MSUS education and distributed via REDCap anonymously in March 2022 (Supplementary Material). Eligible participants included current and recently graduated (<1 year) pediatric rheumatology fellows at a North American program. Descriptive statistics and bivariate analyses using design-based Pearson chi-squared tests were performed. Overall response rate was 78% (88/113), and 75% reported some form of MSUS training during fellowship. Only 36% indicated their program had a formal MSUS curriculum. Of those with MSUS training, 23% reported adult-only MSUS education. Eighty-four percent felt MSUS would be beneficial to their career. Major barriers to MSUS training included lack of MSUS-trained faculty, lack of time, and lack of hands-on MSUS sessions. Those who had access to MSUS training were significantly more interested in MSUS than those without (P = 0.0036). This study demonstrates that North American pediatric rheumatology fellows have a strong interest in learning MSUS, but they face significant challenges in accessing MSUS training (lack of MSUS-trained faculty, time, and access to hands-on training). MSUS should be incorporated into fellowship curriculum; however, implementation remains a challenge.

P007 Successful use of patient initiated follow-up in a tertiary paediatric rheumatology service

Abstract Background/Aims Patient initiated follow-up (PIFU) is a national strategy enabling patients or carers to initiate contact with a service rather than a traditional model of routine scheduled appointments. PIFU enables patient empowerment and elective recovery after the Covid-19 pandemic. NHSE identified Paediatrics as appropriate for PIFU, but specific measures are needed to ensure safeguarding of paediatric patients is maintained. Methods With NHSE &amp; I support and extrapolating guidance implementing PIFU in adult rheumatology services, we developed a clinical pathway that included safeguarding/governance structures within Paediatric Rheumatology at Alder Hey Children’s Hospital. Two strategies were developed - PIFU culminating with either a timed return (PEND) or discharge (DISCHARGE) model. A PIFU feasibility study in paediatric rheumatology initially targeted three index conditions (arthritis, mechanical pain and primary Raynaud’s) in March 2022. This was extended within a Plan-Do-Study-Act model of Quality Improvement. Results We placed 536 patients within PIFU pathways (PEND = 297, DISCHARGE = 239). The monthly usage of PIFU increased from &amp;lt;10 patients per month in March 2022 to &amp;gt; 40 patients per month in August 2023 with expansion of PIFU conditions. Within this time frame the number of patients put through the DISCHARGE model increased steadily to 60% of the monthly proportion as clinician confidence in PIFU increased. We demonstrate an 11% (total Rheumatology patients) PIFU rate over the study time frame, with a 2.7% (15/536) reactivation rate. Where calculable, timescale for review following activation was &amp;lt;48 hours. In our experience, patients/families are comfortable with PIFU and feel reassured by the safety-net provided. Decision for PIFU PEND/DISCHARGE is made in partnership with patients/families. As well as increased use within Rheumatology, PIFU use has extended to 80% of clinical teams across Alder Hey. The Trust-wide PIFU rate of 10% in September 2023 is double the original NHS England guidance target of 5%. Conclusion We advocate safety of PIFU in the correct population and its ability to empower patients. Table 1 shows the significant impact that correct use of PIFU can have on increasing capacity for seeing new patients, decreasing time to review and matching the number of patient reviews to referrals. Disclosure W. Calvert: None. J. Spinty: None. L.J. McCann: None. G. Cleary: None.

Teleteaching in paediatric rheumatology: an Asia Pacific League of Associations for Rheumatology experience

Aim: To evaluate the reach and potential effectiveness of teleteaching of health providers in paediatric rheumatology by observing the pattern of sessions, attendance, and attendees’ feedback in paediatric rheumatology teleteaching sessions across the Asia Pacific region. These were conducted by the Asia Pacific League of Associations for Rheumatology (APLAR) Paediatric Rheumatology Special Interest Group (Paeds Rheum SIG) as an APLAR academy activity. Methods: A retrospective, descriptive analysis of electronic records of synchronous telehealth sessions held between July 2021 to July 2023. These followed three formats: case-based webinars, single topic modular online course, and a hybrid one-day short course. The topics of sessions, attendees’ registration details, specifically primary area of practice/specialty, geographic location, and their satisfaction as per feedback forms were noted. Results: Case-based webinars had an average attendance of 200 per webinar: majority adult rheumatologists, paediatric rheumatologists (PRs), or paediatricians. The modular course consisted of nine sessions with an average of 63 participants per session. Specialties comprised PRs, adult rheumatologists with immunologists, and general paediatricians. The one-day hybrid course had over 600 attendees. Attendees were adult rheumatologists, general physicians, paediatricians, and PRs. Although the majority (79.3%) of attendees from these sessions were from APLAR member nation organisations (MNOs), a significant percentage (20.7%) were from non-APLAR MNOs and even non-Asia Pacific regions. The attendees’ feedback for all three formats showed a high level of satisfaction with case-based webinars being most favoured. Conclusions: Teleteaching in paediatric rheumatology has the potential to fulfill the dire need for improved expertise of health professionals managing children with rheumatic diseases. Case-based, interactive sessions of shorter duration and a blended hybrid format may garner the most attendance and best learning outcomes.

Changes in the Workforce Characteristics of Providers Who Care for Adult Patients With Rheumatologic and Musculoskeletal Disease in the United States.

The aim of this study was to describe the adult rheumatology workforce in the United States, assess change in rheumatology providers over time, and identify variation in rheumatology practice characteristics. Using national Medicare claims data from 2006 to 2020, clinically active rheumatology physicians and advanced practice providers (APPs) were identified. Each calendar year was used for inclusion, exclusion, and analysis, and providers were determined to be entering, exiting, or stable based upon presence or absence in the prior or subsequent years of data. Characteristics (age, gender, practice type, rural, and region) of rheumatologists were determined for 2019 and in mutually exclusive study periods from 2009 to 2011, 2012 to 2015, and 2016 to 2019. The location of rheumatology practice was determined by billing tax identification and mapped. Demographics of physicians exiting or entering the rheumatology workforce were compared separately to those stable by logistic regression. The clinically active adult rheumatology workforce identified in US Medicare in 2019 was 5,667 rheumatologists and 379 APPs. From 2009 to 2020, the number of rheumatologists increased 23% and the number of APPs increased 141%. There was an increase in female rheumatologists over time, rising to 43% in 2019. Women and those employed by a health care system were more likely to exit, and those in a small practice or in the South were less likely to exit. The overall number of clinically active rheumatology providers grew more than 20% over the last decade to a high of 6,036 in 2020, although this rate of growth appears to be flattening off in later years.

90 Novel approach for childhood Sjögren’s Disease therapies: multistakeholder design of a series of N-of-1 trials

OBJECTIVES/GOALS: Childhood Sjögren’s disease (cSD) is a rare autoimmune disease. Despite the profound impact on children and their families, pediatric-specific clinical trials to inform therapeutic strategies in cSD are lacking. In 2022 we participated in the Trial Innovation Network (TIN) Design Lab with the purpose of designing a series of N-of-1 trials for cSD. METHODS/STUDY POPULATION: New medications have the potential to be safe/effective treatments for cSD but must be evaluated in randomized trials. To overcome limitations of traditional parallel-group designs given the rarity of cSD, we developed an N-of-1 trial approach. Our proposal was selected by the Tufts TIN Design Lab. The Design Lab multi-stakeholder process involved parents of and patients with cSD, pediatric and adult rheumatologists, and experts in clinical trial design and outcomes. We engaged all stakeholders in protocol development to maximize the impact of the proposed approach on clinical care, ensure a successful recruitment plan, and inform the choice of endpoints as there are no widely accepted cSD outcome measures to determine treatment efficacy. RESULTS/ANTICIPATED RESULTS: Using the Design Lab methodology, we clarified the N-of-1 study goals and engaged in an iterative process to develop a “briefing book” that ensured a sound premise for our study. We reviewed and accumulated published literature to support our focus on mucosal/glandular manifestations, identified potential interventions to be used in the N-of-1 trials, and enumerated possible outcomes, including outcomes important to patient/parents. This work culminated in a full-day Design Lab event that included multiple stakeholders who provided expertise from different perspectives on the full drug development pathway. Study design feedback focused on three specific areas. 1) Inclusion and exclusion criteria; 2) Identification of outcome measures; 3) Treatment and washout periods. DISCUSSION/SIGNIFICANCE: To address the critical need and move treatment of cSD forward, we are designing a prototype N-of-1 trial in children with rheumatic disease. We will continue to engage stakeholders by using a series of Delphi surveys and an in-person meeting to create composite outcome measures to test cSD therapies in personalized trials.

The First Nations experience of accessing rheumatology services in a metropolitan hospital: A qualitative study.

First Nations Australians experience a higher burden and severity of Rheumatic Disease with poorer outcomes than the general population. Despite a widely acknowledged need to improve health outcomes, there has been minimal research assessing existing models of care from a First Nations perspective in Australia. The objective of this study was to describe First Nations experiences and barriers and enablers to accessing a hospital-based adult Rheumatology service in Sydney. A qualitative study using semi-structured interviews was undertaken. Patients who self-identified as First Nations attending the Prince of Wales Hospital Rheumatology Clinic in 2021 were invited to participate. Interviews were conducted face-to-face or by telephone using culturally-appropriate Yarning methods with an Aboriginal Health Worker (AHW) at the request of participants. Thematic analysis was done in consultation with an Aboriginal Reference Group (ARG). Four categories, which encapsulated 11 themes were identified. Participants reported barriers to care such as logistics of the referral process, not feeling culturally safe because of uncomfortable clinic environments and health worker behaviours, inadequate cultural support and community perceptions of the specialty. Enabling factors included family member involvement, AHW support and telehealth consultation. The current model of care perpetuates access challenges for First Nations Australians within rheumatology. Barriers to care include the delayed referral process, limited cultural responsivity in the clinic environment and poor cross-cultural communication. There is a need for models of care that are co-designed with First Nations Peoples to address these barriers. Participants were First Nations Australians with lived experience attending the rheumatology clinic. All interviewees were offered the opportunity to review their transcripts to ensure trustworthiness of the data. Preliminary thematic analysis was conducted in partnership with the AHW who has over 20 years experience. Following preliminary coding, a list of themes were presented to the ARG for iterative discussion and refinement. The ARG provided community representation and ensured that First Nations voices were privileged in the analysis. It's intended that the findings of this study will support the upcoming co-design of a First Nations health service for Rheumatology patients.

Transition to Adult Rheumatology Care: A Disease-Specific Guide.

Young adults with childhood-onset rheumatic diseases are more frequently establishing and continuing care with adult rheumatologists. The transfer of care can be challenging for both the young adult patients and their adult rheumatologists, in large part due to differences between pediatric-onset rheumatic diseases and their adult-onset counterparts, or due to the rarity of some pediatric-onset rheumatic conditions. Other challenges are due to cultural differences between pediatric and adult medical care and to the young adult needing to increasingly perform self-management skills that were previously managed by parents or other caregivers. In this review, we will provide a summary of strategies for working effectively with young adults as they transition to adult care. We will then discuss a subset of childhood-onset rheumatic diseases-including juvenile idiopathic arthritis, localized scleroderma, autoinflammatory diseases, pediatric-onset systemic lupus erythematosus, juvenile-onset dermatomyositis, and autoimmune encephalitis-for which clinical manifestations, management, and prognosis frequently differ between pediatric onset and adult onset. Our aim is to highlight differences that make caring for this population of transitioning young adults unique, providing tools and knowledge to empower the adult rheumatologist to care for these young adults in ways that are evidence-based, effective, efficient, and rewarding.