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To elicit adolescents' and young adults' (AYAs') perspectives about how to implement the Six Core Elements of Healthcare TransitionAYAs (16-28 years old) with self-reported rheumatic conditions were recruited through patient organizations and social media. In Phase One (QUAL), 90-minute focus groups were facilitated to elicit AYAs' reactions to Six Core Elements content. In Phase Two (QUAN), a national survey was conducted to determine generalizability of recommendations extracted from Phase One. Mixed methods analyses were conducted by a multi-disciplinary team of social science researchers, pediatric rheumatologists, and patients.While focus group participants (n=39) were previously unfamiliar with the Six Core Elements, they reacted favorably to its format and content. Participants provided suggestions for how to logistically execute each component in clinic. Additionally, three overarching recommendations emerged that focused on motivating AYAs to engage: 1. Frame healthcare transition as an opportunity for empowerment, 2. Implement a structured education plan, and 3. Consider the role of parents. In line with qualitative findings, survey participants (n=137) reported that they would prefer to learn most transitional skills from and discuss developmentally specific topics with their rheumatology team. Participants reported they would likely complete programs to learn transitional skills from allied professionals, via patient portals, or in group settings.Incorporating patient perspectives into research and clinical practice is an opportunity to strengthen educational programs. AYAs emphasized the importance of gaining independence and becoming empowered through the healthcare transition process with structured support from their rheumatology teams.

Background: Juvenile localized scleroderma (LS) and systemic sclerosis (SSc) are rare pediatric conditions often associated with severe morbidities. Delays in diagnosis are common, increasing the risk for permanent damage and worse outcomes. This study explored caregiver perspectives on barriers they encountered while navigating diagnosis and care for their child’s scleroderma. Methods: In this cross-sectional study, caregivers of juvenile LS or SSc patients were recruited from a virtual family scleroderma educational conference and a juvenile scleroderma online interest group. The survey queried respondents about their child’s condition and factors affecting diagnosis and treatment. Results: The response rate was 61% (73/120), with 38 parents of LS patients and 31 parents of SSc patients. Most patients were female (80%) and over half were non-Hispanic white (55%). Most families had at least one person with a college education or higher (87%), traveled < 2 hours to see their doctor (83%), and had private insurance (75%). Almost half had an annual household income > $100,000 (46%). Families identified the following factors as barriers to care: lack of knowledge about scleroderma in the medical community, finding reliable information about pediatric scleroderma, long wait times for a rheumatology/specialist appointment, balance of school/work and child’s healthcare needs, medication side effects, and identifying effective medications. The barrier most identified as a major problem was the lack of knowledge about juvenile scleroderma in the medical community. Diagnosis and systemic treatment initiation occurred at greater than one year from initial presentation for approximately 28% and 36% of patients, respectively. Conclusion: Caregivers of children with LS or SSc reported numerous common barriers to the diagnosis, treatment, and ongoing care of juvenile scleroderma. The major problem highlighted was the lack of knowledge of scleroderma within the general medical community. Given that most of the caregiver respondents to the survey had relatively high socioeconomic status, additional studies are needed to reach a broader audience, including caregivers with limited English proficiency, geographical limitations, and financial constraints, to determine if the identified problems are generalizable. Identifying key care barriers will help direct efforts to address needs, reduce disparities in care, and improve patient outcomes.

Objective To assess the attitudes and common practices of adult rheumatologists in the US regarding health care transition (HCT) for young adults with rheumatic diseases. Methods An anonymous online survey was sent to US adult rheumatologist members of the American College of Rheumatology to collect demographic data and information on attitudes and common practices regarding the transition process. Results Of 4,064 contacted rheumatologists, 203 (5%) completed the survey. Almost half of respondents (45.1%) were never trained in transition practices, and 74.7% were not familiar with the American Academy of Pediatrics/American Academy of Family Physicians/American College of Physicians Consensus Statement About Transitions for Youth with Special Healthcare Needs. Only 56.2% felt comfortable caring for former pediatric patients. The vast majority of respondents (90.7%) did not have a multidisciplinary transition team, and 37% did not have a plan for transitioning pediatric patients into their practice. Most adult rheumatologists were unsatisfied with the current transition process (92.9%), due to insufficient resources, personnel (91.1%), and time in clinic (86.9%). They also were unsatisfied with referral data received concerning previous treatments (48.9%), hospitalization history (48%), disease activity index (45.1%), medical history summary (43.9%), comorbidities (36.4%), medication list (34.1%), and disease classification (32.6%). Three major barriers to HCT were lack of insurance reimbursement (33.7%), knowledge about community resources (30.8%), and lapses in care between primary provider and specialist (27.8%). Conclusion This survey identified substantial gaps in knowledge and resources regarding HCT for young adults with rheumatic diseases. These may be best addressed by further training, research, dedicated resources, adequate payment, and practice guidelines.

ObjectiveSince 2010, the rheumatology community has developed guidelines and tools to improve healthcare transition. In this study, we aimed to compare current transition practices and beliefs among Childhood Arthritis and Rheumatology Research Alliance (CARRA) rheumatology providers with transition practices from a provider survey published in 2010.MethodsIn 2018, CARRA members completed a 25-item online survey about healthcare transition. Got Transition’s Current Assessment of Health Care Transition Activities was used to measure clinical transition processes on a scale of 1 (basic) to 4 (comprehensive). Bivariate analyses were used to compare 2010 and 2018 survey findings.ResultsOver half of CARRA members completed the survey (202/396), including pediatric rheumatologists, adult- and pediatric-trained rheumatologists, pediatric rheumatology fellows, and advanced practice providers. The most common target age to begin transition planning was 15–17 years (49%). Most providers transferred patients prior to age 21 years (75%). Few providers used the American College of Rheumatology transition tools (31%) or have a dedicated transition clinic (23%). Only 17% had a transition policy in place, and 63% did not consistently address healthcare transition with patients. When compared to the 2010 survey, improvement was noted in 3 of 12 transition barriers: availability of adult primary care providers, availability of adult rheumatologists, and pediatric staff transition knowledge and skills (P < 0.001 for each). Nevertheless, the mean current assessment score was < 2 for each measurement.ConclusionThis study demonstrates improvement in certain transition barriers and practices since 2010, although implementation of structured transition processes remains inconsistent.

Objective To determine the efficacy in improving pain and health-related quality of life (HRQOL) of an online self-management program for adolescents with juvenile idiopathic arthritis (JIA). Methods Youth ages 12-18 years with JIA were recruited from 10 rheumatology clinics across the United States and randomized to complete an online self-management program (n = 144) or an online disease education program (n = 145). Participants in the self-management group worked through multimedia-based modules comprising psychoeducation, training in cognitive-behavioral coping skills and stress management, and other self-management topics over a 12-week period. Participants in the control group viewed a series of preselected quality educational websites about JIA over the same interval. Online content for both groups was made available in English and Spanish to facilitate inclusion of Hispanic participants. Blinded assessment of main outcomes (pain intensity, pain interference, and HRQOL) and process outcomes (disease knowledge, self-efficacy, pain coping, and emotional adjustment) occurred at baseline, posttreatment, and at 6- and 12-month postrandomization follow-up visits. Results Participants on average demonstrated significant improvements over the study period in the main outcomes, with no significant group differences in the degree of improvement. Effect sizes for these improvements were small. The amount of improvement in self-efficacy, emotional avoidance coping, disease knowledge, and emotional functioning in part predicted improvement in pain and HRQOL outcomes. Conclusions Primarily self-directed online self-management training and online disease education comparably and modestly improve pain and HRQOL in youth with JIA.

Objective To assess perception and behavior after reproductive health counseling among adolescent patients in a tertiary care–based pediatric rheumatology clinic. Methods Adolescent females seen at a Stanford pediatric rheumatology clinic were prospectively enrolled during routine visits. At the study start, standard clinic procedures were reviewed with providers for the following: 1) the home, education, activities, drugs, sexual activity, and suicide/depression (HEADSS) assessment; 2) reproductive health counseling; and 3) medical record documentation. Patients were enrolled if providers indicated that they performed the HEADSS assessment and reproductive health counseling. At enrollment, patients completed a survey to assess their perceptions of reproductive health counseling. Chart reviews confirmed documented discussions. A followup survey performed 3–5 months after enrollment tracked reproductive health information–seeking behavior. Results In total, 90 females (mean ± SD age 17 ± 2 years) participated. Almost all patients (99%) agreed that reproductive health was discussed; 71% reported that pregnancy risks were discussed, 42% had recent concerns about reproductive health, and 33% reported their provider recommended that they seek further reproductive health care. Eighty-four patients completed followup phone surveys, with 25% reporting seeking further information on reproductive health concerns, but with only 9.5% actually seeking further care. Only 18% of patients reported having ever asked their rheumatology provider for guidance regarding reproductive health care concerns. Conclusion Routine reproductive health discussion and counseling are necessary in a rheumatology clinic because, in our experience, a substantial number of adolescents have concerns and actively seek reproductive health information. Despite these discussions, adolescents rarely pursue further reproductive health care. Further work to bridge this gap is needed.

Objective.To assess North American pediatric rheumatology providers’ perspectives on practices, barriers, and opportunities concerning the transition from pediatric-centered to adult-centered care.Methods.Childhood Arthritis and Rheumatology Research Alliance (CARRA) members completed a 25-item survey assessing current transition practices, transition policy awareness, and transitional care barriers and needs. Results were compared to the American Academy of Pediatrics (AAP) 2008 survey on transitional care.Results.Over half (158/288, 55%) of CARRA members completed the survey. Fewer than 10% are very familiar with AAP guidelines about transition care for youth with special healthcare needs. Eight percent have a formal written transition policy, but 42% use an informal approach. Patient request (75%) most frequently initiates transfer to adult care. Two major barriers to transition are fragmented adult medical care and lack of sufficient time to provide services. Compared with AAP survey participants, pediatric rheumatology providers are significantly more likely to help youth find an adult specialist (63% vs 45%) and discuss confidentiality and consent before age 18 (45% vs 33%), but are less likely to help with medical summary creation (16% vs 27%) or find a primary care provider (25% vs 47%). Outcomes ranked as “very important” in defining a successful transition are survival (76%), seeing an adult rheumatologist within 6 months of final pediatric rheumatology visit (66%), and maintaining insurance coverage (57%).Conclusion.This comprehensive survey of North American pediatric rheumatology providers regarding transitional care practices demonstrates deficiencies in education, resources, and a formalized process. Respondents support development of standardized rheumatology-specific transition practices.

Objective.Granulomatosis with polyangiitis (Wegener’s; GPA) and other antineutrophil cytoplasmic antibody (ANCA)-associated vasculitides (AAV) are rare in childhood and are sometimes difficult to discriminate. We compared use of adult-derived classification schemes for GPA against validated pediatric criteria in the ARChiVe (A Registry for Childhood Vasculitis e-entry) cohort, a Childhood Arthritis and Rheumatology Research Alliance initiative.Methods.Time-of-diagnosis data for children with physician (MD) diagnosis of AAV and unclassified vasculitis (UCV) from 33 US/Canadian centers were analyzed. The European Medicines Agency (EMA) classification algorithm and European League Against Rheumatism/Paediatric Rheumatology International Trials Organisation/Paediatric Rheumatology European Society (EULAR/PRINTO/PRES) and American College of Rheumatology (ACR) criteria for GPA were applied to all patients. Sensitivity and specificity were calculated (MD-diagnosis as reference).Results.MD-diagnoses for 155 children were 100 GPA, 25 microscopic polyangiitis (MPA), 6 ANCA-positive pauciimmune glomerulonephritis, 3 Churg-Strauss syndrome, and 21 UCV. Of these, 114 had GPA as defined by EMA, 98 by EULAR/PRINTO/PRES, and 87 by ACR. Fourteen patients were identified as GPA by EULAR/PRINTO/PRES but not by ACR; 3 were identified as GPA by ACR but not EULAR/PRINTO/PRES. Using the EMA algorithm, 135 (87%) children were classifiable. The sensitivity of the EMA algorithm, the EULAR/PRINTO/PRES, and ACR criteria for classifying GPA was 90%, 77%, and 69%, respectively, with specificities of 56%, 62%, and 67%. The relatively poor sensitivity of the 2 criteria related to their inability to discriminate patients with MPA.Conclusion.EULAR/PRINTO/PRES was more sensitive than ACR criteria in classifying pediatric GPA. Neither classification system has criteria for MPA; therefore usefulness in discriminating patients in ARChiVe was limited. Even when using the most sensitive EMA algorithm, many children remained unclassified.

Objective.There are no validated tools for measuring disease activity in pediatric vasculitis. The Birmingham Vasculitis Activity Score (BVAS) is a valid disease activity tool in adult vasculitis. Version 3 (BVAS v.3) correlates well with physician’s global assessment (PGA), treatment decision, and C-reactive protein in adults. The utility of BVAS v.3 in pediatric vasculitis is not known. We assessed the association of BVAS v.3 scores with PGA, treatment decision, and erythrocyte sedimentation rate (ESR) at diagnosis in pediatric antineutrophil cytoplasmic antibody-associated vasculitis (AAV).Methods.Children with AAV diagnosed between 2004 and 2010 at all ARChiVe centers were eligible. BVAS v.3 scores were calculated with a standardized online tool (www.vasculitis.org). Spearman’s rank correlation coefficient (rs) was used to test the strength of association between BVAS v.3 and PGA, treatment decision, and ESR.Results.A total of 152 patients were included. The physician diagnosis of these patients was predominantly granulomatosis with polyangiitis (n = 99). The median BVAS v.3 score was 18.0 (range 0–40). The BVAS v.3 correlations were rs = 0.379 (95% CI 0.233 to 0.509) with PGA, rs = 0.521 (95% CI 0.393 to 0.629) with treatment decision, and rs = 0.403 (95% CI 0.253 to 0.533) with ESR.Conclusion.Applied to children with AAV, BVAS v.3 had a weak correlation with PGA and moderate correlation with both ESR and treatment decision. Prospective evaluation of BVAS v.3 and/or pediatric-specific modifications to BVAS v.3 may be required before it can be formalized as a disease activity assessment tool in pediatric AAV.

Living Profiles is a health media platform in development that aggregates multiple data flows to help teens with special healthcare needs (SHCN), particularly with regard to self-management and independence. A teen-oriented personal health record (PHR) incorporates typical teen behaviors and attitudes about health and wellness, encompasses how teens perceive and convey quality of life, and aligns with data related to their chronic medical condition. We have conceived a secure personalized user interface called the Quality of Life Timeline, which will assist with the transition from pediatric care to an adult provider through modules that include a mood meter, reminder device, and teleport medicine. With this personalized PHR, teens with SHCN can better understand their condition and its effects on daily activities and life goals and vice versa; additionally, use of this PHR allows for better information sharing and communication between providers and patients. The use of a teen-oriented tool such as Living Profiles can impact teens’ overall quality of life and disease self-management, important attributes for a successful transition program.

Objective To compare the criteria for Wegener's granulomatosis (WG) of the American College of Rheumatology (ACR) with those of the European League Against Rheumatism/Pediatric Rheumatology European Society (EULAR/PRES) in a cohort of children with WG and other antineutrophil cytoplasmic antibody (ANCA)–associated vasculitides (AAVs), and to describe the interval to diagnosis, presenting features, and initial treatment for WG. Methods Eligible patients had been diagnosed by site rheumatologists (termed the “MD diagnosis”) since 2004. This diagnosis was used as a reference standard for sensitivity and specificity testing of the 2 WG classification criteria. Descriptive analyses were confined to ACR-classified WG patients. Results MD diagnoses of 117 patients (82 of whom were female) were WG (n = 76), microscopic polyangiitis (n = 17), ANCA-positive pauci-immune glomerulonephritis (n = 5), Churg-Strauss syndrome (n = 2), and unclassified vasculitis (n = 17). The sensitivities of the ACR and EULAR/PRES classification criteria for WG among the spectrum of AAVs were 68.4% and 73.6%, respectively, and the specificities were 68.3% and 73.2%, respectively. Two more children were identified as having WG by the EULAR/PRES criteria than by the ACR criteria. For the 65 ACR-classified WG patients, the median age at diagnosis was 14.2 years (range 4–17 years), and the median interval from symptom onset to diagnosis was 2.7 months (range 0–49 months). The most frequent presenting features by organ system were constitutional (89.2%), pulmonary (80.0%), ear, nose, and throat (80.0%), and renal (75.4%). Fifty-four patients (83.1%) commenced treatment with the combination of corticosteroids and cyclophosphamide, with widely varying regimens; the remainder received methotrexate alone (n = 1), corticosteroids alone (n = 4), or a combination (n = 6). Conclusion The EULAR/PRES criteria minimally improved diagnostic sensitivity and specificity for WG among a narrow spectrum of children with AAVs. Diagnostic delays may result from poor characterization of childhood WG. Initial therapy varied considerably among participating centers.

Objective.To determine the elapsed time while receiving aggressive therapy to the first observation of clinically inactive disease (CID), total duration of CID and potential predictors of this response in a cohort of children with recent onset of polyarticular juvenile idiopathic arthritis (poly-JIA).Methods.Eighty-five children were randomized blindly to methotrexate (MTX), etanercept, and rapidly tapered prednisolone (MEP) or MTX monotherapy and assessed for CID over 1 year of treatment. Patients who failed to achieve intermediary endpoints were switched to open-label MEP treatment.Results.Fifty-eight (68.2%) of the 85 patients achieved CID at 1 or more visits including 18 who received blinded MEP, 11 while receiving MTX monotherapy, and 29 while receiving open-label MEP. Patients starting on MEP achieved CID earlier and had more study days in CID compared to those starting MTX, but the differences were not significantly different. Patients given MEP (more aggressive therapy) earlier in the disease course were statistically more likely to have a higher proportion of followup visits in CID than those with longer disease course at baseline. Those who achieved American College of Rheumatology Pediatric 70 response at 4 months had a significantly greater proportion of followup visits in CID, compared to those who failed to achieve this improvement (p < 0.0001). Of the 32 patients who met criteria for CID and then lost CID status, only 3 fulfilled the definition of disease flare.Conclusion.Shorter disease duration prior to treatment, a robust response at 4 months, and more aggressive therapy result in a higher likelihood and longer duration of CID in patients with poly-JIA. The original trial from which data for this analysis were obtained is registered on www.clinicaltrials.gov NCT 00443430.

Objective: To assess perception and behavior after reproductive health counseling among adolescent patients in a tertiary care-based pediatric rheumatology clinic. Methods: Adolescent females seen at Stanford pediatric rheumatology clinic were prospectively enrolled during routine visits. At study start, standard clinic procedures for the following were reviewed with providers: 1) HEADSS (home, education, activities, drugs, sexual activity, and suicide/depression) assessment; 2) reproductive health counseling; and 3) medical record documentation. Patients were enrolled if providers indicated that they performed HEADSS assessment and reproductive health counseling. At enrollment, patients completed a survey to assess perceptions of reproductive health counseling. Chart review confirmed documented discussions. Follow-up survey 3-5 months after enrollment tracked reproductive health information seeking behavior. Results: Ninety females (ages 17 ± 2 years old) participated. Almost all patients (99%) agreed that reproductive health was discussed. Seventy-one percent reported that pregnancy risks were discussed, 42% had recent concerns about reproductive health, and 33% reported their provider recommended that they seek further reproductive health care. Eighty-four patients completed follow-up phone surveys, with 25% reporting seeking further information on reproductive health concerns but merely 9.5% actually sought further care. Only 18% reported having ever asked their rheumatology provider for guidance regarding reproductive health care concerns. Conclusion: Routine reproductive health discussion and counseling are necessary in a rheumatology clinic; as in our experience, a substantial number of adolescents have concerns and actively seek reproductive health information. Despite these discussions, teens rarely pursued further reproductive health care. Further work to bridge this gap is needed.

Objective.To determine whether adult disease severity subclassification systems for antineutrophil cytoplasmic antibody-associated vasculitis (AAV) are concordant with the decision to treat pediatric patients with cyclophosphamide (CYC).Methods.We applied the European Vasculitis Study (EUVAS) and Wegener’s Granulomatosis Etanercept Trial (WGET) disease severity subclassification systems to pediatric patients with AAV in A Registry for Childhood Vasculitis (ARChiVe). Modifications were made to the EUVAS and WGET systems to enable their application to this cohort of children. Treatment was categorized into 2 groups, “cyclophosphamide” and “no cyclophosphamide.” Pearson’s chi-square and Kendall’s rank correlation coefficient statistical analyses were used to determine the relationship between disease severity subgroup and treatment at the time of diagnosis.Results.In total, 125 children with AAV were studied. Severity subgroup was associated with treatment group in both the EUVAS (chi-square 45.14, p < 0.001, Kendall’s tau-b 0.601, p < 0.001) and WGET (chi-square 59.33, p < 0.001, Kendall’s tau-b 0.689, p < 0.001) systems; however, 7 children classified by both systems as having less severe disease received CYC, and 6 children classified as having severe disease by both systems did not receive CYC.Conclusion.In this pediatric AAV cohort, the EUVAS and WGET adult severity subclassification systems had strong correlation with physician choice of treatment. However, a proportion of patients received treatment that was not concordant with their assigned severity subclass.

Juvenile idiopathic arthritis (JIA) encompasses a group of diseases of unknown etiology, defined by the International League of Associations for Rheumatology as having in common arthritis in one or more joints that persists for at least 6 weeks and beginning before 16 years of age with other conditions excluded (1). With a prevalence of approximately one per thousand children in the US, JIA is the most common pediatric rheumatic illness and cause of acquired childhood disability (2, 3). During the last 20 years the advent of a host of immune response modifiers (biologics) that directly inhibit the action of pro-inflammatory mediators has revolutionized the treatment and expected outcome of JIA (4-7) such that extended periods of clinically quiescent disease may now be induced. Newly published guidelines from the American College of Rheumatology (ACR) provide some guidance for the initiation and safety monitoring of drugs commonly used in JIA, including biologics (8). However, it remains unclear as to exactly when in the course of the disease and in what combination these treatments should be started to produce optimal outcomes. At present, it cannot be predicted with confidence which children with JIA have a less favorable outcome, although some risk factors have been identified (8-10). The polyarticular (both rheumatoid factor positive and negative) categories comprise approximately 30% of all patients with JIA, and the majority of these children remain on combinations of multiple medications for many years (11, 12); disease free periods off medication greater than 1 year are uncommon (13). Investigations in adult rheumatoid arthritis (RA) have demonstrated improved outcomes, including less radiographic progression of joint damage, when aggressive treatment is started early in the disease course (14-17). Thus, many rheumatologists now believe there is a “window of opportunity” early in the disease during which aggressive therapy has a profound long term effect (17-19). To date, there have not been any double-blind, randomized placebo controlled trials of biological agents in children with recent onset JIA in which the primary endpoint is clinical inactive disease (CID) (20). The trial described here was designed to determine if two aggressive treatment regimens started early in the course of polyarticular JIA results in CID within 6 months of initiation. An exploratory phase investigated the potential of the treatments to induce clinical remission on medication (CRM: 6 continuous months of CID while on treatment) within 12 months of initiation.

To the Editor: We appreciate the contribution of Dr. Chanchlani, et al regarding their retrospective assessment of transfer of adolescent rheumatology patients in the United Kingdom because it demonstrated the real-world challenges of implementing a successful transition program1. The UK has always been a leader in rheumatology adolescent transitional care as noted by this group’s previous work2. While their recent data showed favorable results in actual transfers, as noted by adult rheumatology referral notes being sent to the pediatric rheumatology consultant (86%), concerns were raised with regard to … Address correspondence to Dr. P. Chira, 3020 Children’s Way, Mail Code 5113, San Diego, California 92123, USA. E-mail: pchira{at}ucsd.edu

Purpose of review Better understanding of the etiology of autoimmune diseases and their progression has brought about numerous novel therapies used in the treatment of pediatric rheumatic diseases. The introduction of biologic agents such as tumor necrosis factor inhibitors has changed how we approach and manage autoimmune diseases. This has led to a proliferation of other therapies targeting specific inflammatory processes evident in many rheumatic illnesses, with hopes of improving efficacy and decreasing adverse effects from treatment. Recent findings Clinical studies demonstrate safety and efficacy of these newer medications in both adults and children. Although most of the novel therapies have been studied primarily in the adult rheumatic population, many are being evaluated in children in randomized controlled and open label trials as well. Long-term results are being collected regarding these newer regimens in both adults and children. Summary This review looks at the risks and benefits of the variety of novel therapies including the new biologics, immunosuppressives, and stem cell transplantation currently being used in rheumatic conditions. Using these new therapies along with traditional antirheumatic medications, pediatric rheumatologists intervene to control disease early and more effectively to prevent long-term damage and complications.