bluuebunny/crossref_metadata_2025_split huggingface dataset:
P133 Chronic recurrent multifocal osteomyelitis (CRMO) associated with polymyositis and polyarthritis in a boy
Authors: Pandya Devang B 
Published: April 2020

Abstract

Background
CRMO is an autoinflammatory bone disease mainly affecting the paediatric age group. It may have overlapping features with other rheumatological diseases. I report a case of a 7-year-old boy who presented with fever, persistent limp and pain at multiple sites involving the extremities.

Methods
His complaint started with pain in the gluteal region with an accompanying limp. Two months later, he started having fever, pain and localised swelling around multiple sites - right knee, right ankle and left wrist associated with intermittent night pain and morning stiffness. MSK examination revealed localised peri-articular swelling and restriction of movements involving bilateral knees, ankles, hips and wrists. Lab reports showed low hemoglobin, neutrophilic leucocytosis, elevated platelets, CRP and ESR. Muscle enzymes (CPK, SGPT, SGOT, LDH), bone profile, ferritin, RF, ASO titer, ANA and ANA profile were normal. Whole body MRI (WBM) revealed multifocal bone marrow edema predominantly in metaphyseal regions involving multiple bones. Polymyositis and mild effusions were noted in bilateral hips and knees. He was put on oral naproxen, prednisolone, methotrexate and pamindronate infusion for two days. He responded dramatically and continues to remain well at two weeks' follow up.

Results
CRMO is mainly associated with severe acne, palmoplantar pustulosis, generalised pustular psoriasis, pyoderma gangrenosum, ankylosing spondylitis and inflammatory bowel disease. There are only 2-3 reported cases of CRMO associated myositis in adolescents to the best of my knowledge. This child will be the youngest diagnosed CRMO with polymyositis.

Conclusion
When any child presents with fever, recurrent or chronic night pain and/or swelling at multiple sites with moderate elevation of inflammatory markers, we should suspect CRMO and our first choice of investigation should be whole body MRI to reveal associated extra-osseous lesions.

Disclosures
D.B. Pandya None.
P138 Are adolescents prepared for transition? A survey of practice in Northern Ireland
Authors: Masih Claire , McKenna Dearbhla , McHenry Michelle , Rooney Madeline 
Published: April 2020

Abstract

Background
In Northern Ireland we have a single paediatric rheumatology service in which patients often stay until the end of education, before transferring to adult care. We lack a formal transition clinic or uniform process for transition. We wished to investigate whether adolescents transitioning to adult rheumatology care are sufficiently prepared for the process.

Methods
We surveyed the charts of patients within the paediatric rheumatology service aged over 16 years. Excluding patients who had were not under active follow up and those who had completed transition we studied 86 patients in a retrospective chart review. We studied the seven areas of readiness for transfer as recommended in the 'Ready Steady Go' programme.

Results
We had a population eligible for transition of 57 female and 29 male patients between the ages of 16 and 26. Around fifty percent had JIA, most commonly oligoarticular and polyarticular with other diagnoses including Raynauds, JIO, mechanical and pain conditions. Fifteen of these had been referred to the appropriate geographical area for transfer to adult service and 71 were yet to be referred. Readiness for transfer was poorly documented. Lifestyle issues (diet, exercise) and vocational (school, hobbies) were the best addressed with 41 and 56 patients respectively having at least one discussion on these aspects. Knowledge, self advocacy, ADLs, psychosocial and transition aspects were poorly documented.

Conclusion
We acknowledged that time pressures may have restricted what was recorded following appointments with focus on clinical information. However, we are aware of the lack of a formal transition programme within Northern Ireland and we feel this may contribute to patients spending a relatively prolonged time within the paediatric service. We have implemented quality improvement by using the 'Ready Steady Go' questionnaires within the paediatric service to improve preparedness for adult transfer and are reviewing our service of paediatric transfer. Introduction of the ‘Hello to adult services’ questionnaire by adult rheumatologists may help to achieve a more successful transition process by highlighting areas in which the young person may benefit from extra care by their adult team.

Disclosures
C. Masih None. D. McKenna None. M. McHenry None. M. Rooney None.
P139 What impact does JIA have on health-related quality of life over time and by gender?
Authors: Smith Andrew , Saqib Bishma , Lee Rebecca , Thomson Wendy , Cordingley Lis 
Published: April 2020

Abstract

Background
Juvenile idiopathic arthritis (JIA) is a heterogeneous group of arthritic conditions presenting in children and young people, in which physical limitations and associated complications can have detrimental effects on physical and psychosocial wellbeing. This study aims to investigate the impact of living with JIA on different aspects of health-related quality of life (HRQoL) and to explore how this changes over time, using data from the Childhood Arthritis Prospective Study (CAPS).

Methods
Longitudinal data collected as part of CAPS were analysed. HRQoL was assessed at baseline, 1 year and 3 years’ post-diagnosis using the Child Health Questionnaire (CHQ), a parent-completed form for children from 5 years of age. The CHQ measures physical, emotional and social components of child health status. Raw domain scores were transformed via algorithm into values ranging from 0-100, with higher scores indicating better health status. Mean (standard deviation) and median (interquartile range) for each domain were determined, both for the full cohort and by gender. Differences between median scores at baseline and 3 years were assessed using the Wilcoxon signed-rank test. Mean scores of each domain were visually compared with a reference population sample of healthy children from the United States.

Results
184 participants completed the questionnaire at all 3 time points. At baseline, compared to the reference population, children with JIA scored lower in every domain although scores were closer between the 2 groups at 3 years. Median scores improved over time, the exception being the general health perceptions domain which decreased after baseline. Domains with the greatest improvement were physical functioning,“bodily pain and social-physical. The largest changes occurred from baseline to 1 year. Statistically significant differences between baseline and 3-year scores were found for all domains. Domain scores for male and female participants were very similar at baseline, though scores for male participants indicated slightly better health at 1 and 3 years for both physical and psychosocial domains.

Conclusion
JIA has significant impact on HRQoL, which improves within 3 years of diagnosis with the greatest improvement occurring within the first year. Physical health domains show greater improvement over time than psychosocial domains, although psychosocial scores were generally higher throughout the study. Male participants tend to score slightly higher than female participants in both physical and psychosocial domains after baseline. Further research should explore measurable patient, age or disease-related drivers of HRQoL.

Disclosures
A. Smith None. B. Saqib None. R. Lee None. W. Thomson None. L. Cordingley None.
P140 Validation of EULAR/ACR 2019 classification criteria for SLE in a south Indian juvenile SLE cohort
Authors: Nair Harikrishnan Bhaskaran , Balan Suma 
Published: April 2020

Abstract

Background
To date there is no specific classification criteria for juvenile systemic lupus erythematosus (jSLE). Our aim is to compare the real world performance of EULAR/ACR 2019 criteria when applied to a known cohort of jSLE cases in Kerala, India.

Methods
We retrospectively reviewed the electronic medical record of all patients (n = 735), admitted under the Pediatric Rheumatology Department, Amrita Institute of Medical Sciences, Kochi, a tertiary care center in Kerala, India, during the period January 2012 to June 2019. 107 patients were diagnosed with jSLE by a qualified experienced pediatric rheumatologist and were independently assessed by an adult rheumatologist. Every sixth patient of the remaining 628 patients were selected as the control (n = 105). Patients were excluded if the diagnosis was uncertain or, if there was an overlap with jSLE. Each patient was evaluated to see if he or she satisfied the ACR-1997, SLICC-2012, or EULAR/ACR- 2019 criteria.

Results
Female: male ratio in cases and controls was 4.1:1 and 2.4 :1 respectively. Mean age for cases and controls was 12.6 +/-2.9 and 11.8 +/-3.5 respectively. The comparison of performance of each criterion is shown in table 1. Table 1: Validation of the EULAR/ACR 2019 SLE classification criteria using clinically diagnosed 107 jSLE cases.
ACR- American College of Rheumatology; EULAR- European League Against Rheumatism; SLICC-Systemic Lupus International Collaborating Clinics; PPV- positive predictive value; NPV- negative predictive value

Conclusion
The new EULAR/ACR 2019 criteria achieved sensitivity, specificity, PPV, NPV and accuracy greater than SLICC 2012 criteria. It also achieved sensitivity, NPV and accuracy greater than 1997 ACR criteria.

Disclosures
H. Bhaskaran Nair None. S. Balan None.
P149 Haematopoietic stem cell transplantation in systemic sclerosis: Sheffield Teaching Hospitals’ experience
Authors: Smith Ruth , Snowden John , Akil Mohammed 
Published: April 2020

Abstract

Background
Haematopoietic stem cell transplantation (HSCT) was first used as a treatment for systemic sclerosis (SSc) in the 1990s. By April 2018 the European Society for Blood and Bone Marrow Transplantation had 533 cases on its registry. Sheffield is the main UK centre for HSCT for SSc with 6 people having undergone the procedure since 2007. HSCT works by rebooting the immune system. Autoreactive T cells are suppressed by proliferation of regulatory T cells and improvement in T cell receptor diversity. Autologous HSCT has been shown to be an effective treatment for SSc, and has been compared to monthly pulsed intravenous cyclophosphamide in the ASSIST, ASTIS and SCOT trials. HSCT showed greater improvement in respiratory and cutaneous disease (ASSIST) and produced a superior global rank composite score based on a hierarchy of disease features (SCOT) compared with cyclophosphamide. However, although there was longer term survival benefit compared with cyclophosphamide, treatment related mortality (TRM) was high at 10% (ASTIS). TRM was attributed to SSc-related cardio-pulmonary dysfunction, and therefore patient selection for HSCT is important and all must undergo a thorough cardio-pulmonary evaluation to mitigate risk.

Methods
We performed a retrospective review of the characteristics and outcomes of patients who have undergone HSCT for SSc in Sheffield.

Results
Legend Symptom

s
R Raynaud’s; DU digital ulcers; dSSc diffuse systemic sclerosis; ILD interstitial lung disease; P pericarditis; M myositis; GAVE gastric antral vascular ectasia; SRC scleroderma renal crisis. Previous treatments: MTX methotrexate; MMF mycophenolate; CYA cyclosporine; CYCLO cyclophosphamide; AZA azathioprine; HCQ hydroxychloroquine mRSS modified Rodnan skin score

Conclusion
Patient numbers are small and follow-up is limited to 12 years, but the procedure has been well tolerated and improved patients’ skin score, with the exception of patient 6. His pre-procedure screening tests showed no evidence of interstitial lung disease, pulmonary artery hypertension or left ventricular dysfunction. The cause of his acute deterioration is unclear but acute campylobacter myocarditis or cyclophosphamide cardiac toxicity have been postulated. Sheffield’s experience has shown HSCT to be an effective treatment for SSc, but one that is not without risk.

Disclosures
R. Smith None. J. Snowden Other; Speaker fees at educational events supported by Janssen, Jazz, Mallinckrodt & Gilead. Member of a trial IDMC for Kiadis Pharma. Chairs NHS England Clinical Reference Group for Blood & Marrow Transplan. M. Akil Honoraria; AbbVie, Actelion, Celgene, Astra Zenica, Lilly and UCB.
P156 Understanding the associations and impact of severe gastrointestinal involvement in systemic sclerosis: a structured approach
Authors: Ahmed Fiza , Nihtyanova Svetlana I , Chatzinikolaou Stamatia , Ong Voon H , Murray Charles D , Denton Christopher P 
Published: April 2020

Abstract

Background
Gastrointestinal (GI) involvement in systemic sclerosis (SSc) has a profound negative impact on patients’ quality of life. However, due to the diversity in manifestations of SSc-associated GI disease, this impact has proven difficult to quantify and to associate with other clinical and demographic features, to identify those most at risk of severe disease.

Methods
Between September and December 2018, consecutive SSc patients attending a specialist scleroderma clinic completed the UCLA Scleroderma Clinical Trial Consortium Gastrointestinal Tract 2.0 (UCLA SCTC GIT 2.0) questionnaire. Total score and separate domain scores (reflux, distention/bloating, diarrhoea, soilage, constipation, social functioning and emotional wellbeing) were calculated. Domain scores range between 0 and 3, with higher scores indicating greater severity. Associations between mean scores and demographic or clinical characteristics were analysed using Wilcoxon rank-sum test.

Results
In total, 224 SSc patients fully completed the UCLA SCTC GIT 2.0. Eighty-three percent were female, mean age was 58.2 years and mean disease duration was 14.3 years. Diffuse cutaneous systemic sclerosis (dcSSc) was present in 37.1%, and 22.3% had an overlap syndrome. The most common autoantibodies were anti-centromere (ACA) in 38.0%, anti-topoisomerase I (ATA) in 20.1%, anti-RNA polymerase in 11.2% and anti-U3RNP in 4.5% of patients. For the cohort, the total GIT score was 0.62±0.57 (mean±SD), correlating strongly with visual analogue scale self-reported impact of GI disease (Pearson’s r = 0.72, p &lt; 0.001). Distribution of total and domain scores is summarised in Table 1. We found no association between demographic or clinical characteristics and total GIT score, reflux, distention/bloating, diarrhoea and social functioning. Constipation was most common among ATA + (score 0.69, p = 0.080 v ATA-) and least common among ARA+ subjects (score 0.25, p = 0.014 v ARA-). Soilage was more common in limited cutaneous SSc (score 0.56 v 0.29 in dcSSc, p = 0.048) and ACA+ subjects (score 0.67 v 0.33 in ACA-, p = 0.008). ACA+ patients also had worse emotional wellbeing (score 0.70 v 0.52 in ACA-, p = 0.014). Emotional wellbeing was more severely affected in females (score 0.62 v 0.43 in males, p = 0.078).

Conclusion
We found that moderate to severe GI disease affects nearly half of all subjects with SSc, which significantly contributes to disease-related morbidity.

Disclosures
F. Ahmed None. S.I. Nihtyanova None. S. Chatzinikolaou None. V.H. Ong None. C.D. Murray None. C.P. Denton None.
P170 Predictors of renal survival in a cohort of patients with lupus nephritis with more than 30 years of follow-up
Authors: Farinha Filipa , Pepper Ruth J , Oliveira Daniel G , McDonnell Thomas , Isenberg David A , Rahman Anisur 
Published: April 2020

Abstract

Background
Despite the improvement in survival of patients with lupus nephritis (LN) over the last decades, LN is associated with progression to end-stage renal disease (ESRD) in a significant proportion of patients. We aimed to investigate the factors influencing renal survival in patients with LN.

Methods
Single-centre retrospective observational study. Patients with biopsy-proven proliferative, membranous or mixed LN were included. Individual clinical files were reviewed to obtain demographic, clinical, laboratory and pathological data. Cox regression analysis was performed to investigate predictors of progression to ESRD and Kaplan-Meier curves were obtained.

Results
We studied 187 LN patients (135 proliferative, 38 membranous and 14 mixed LN), followed for up to 42 years (mean 13±9 years). Cumulative renal survival rates at 5, 10, 15 and 20 years were 93%, 85%, 78% and 70%, respectively. In univariable analysis, urinary protein/creatinine ratio (uPCR) above 42 mg/mmol or estimated glomerular filtration rate (eGFR) below 76 mL/min/1.73m2, one year after the diagnosis of LN, were the strongest predictors of progression to ESRD, with hazard ratios (HR) of 8.081 [95%CI:1.856-35.179] and 4.985 [95%CI:1.964-12.651], respectively. HR for uPCR and eGFR at the time of diagnosis were considerably smaller (2.508 [95%CI:1.062-5.922] and 2.833 [95%CI:1.156-6.945] respectively). Other factors associated with increased risk of ESRD were Afro-Caribbean ethnicity (HR = 3.861 [95%CI:1.817-8.206]), proliferative LN (HR = 3.423 [95%CI:1.049-11.173]), not having taken antimalarials (HR = 2.180 [95%CI:1.089-4.363]) and poorly controlled diastolic blood pressure (HR = 1.016 [95%CI:1.001-1.030]). The effect of uPCR and eGFR at one year remained significant after adjusting for ethnicity, histological class, uPCR and eGFR at the time of diagnosis, use of antimalarials and diastolic blood pressure (Table 1).

Conclusion
uPCR above 42 mg/mmol and eGFR below 76 mL/min/1.73m2, one year after the diagnosis of LN, were the strongest predictors of progression to ESRD.

Disclosures
F. Farinha None. R.J. Pepper None. D.G. Oliveira None. T. McDonnell None. D.A. Isenberg None. A. Rahman None.
P175 MASTERPLANS: prediction of response to rituximab in SLE using a validated two-score system for interferon
Authors: Alase Ade , Wigston Zoe , Burska Agata , Yusof Yuzaiful Md , Reynolds John , Consortium The MASTERPLANS , Wittmann Miriam , Bruce Ian , Vital Edward M 
Published: April 2020

Abstract

Background
Rituximab is used for resistant SLE, but clinical response varies. We previously validated two interferon-stimulated-gene expression scores (IFN-Score-A and IFN-Score-B) that improved prediction of clinical outcomes in SLE. IFN-Score-A included most reported ISGs and predicted flares and glucocorticoid requirements. IFN-Score-B included ISGs that respond to multiple IFN subtypes and predicted development of SLE in At-Risk individuals. Diagnosis of SLE was associated with both scores, while only IFN-Score-B was elevated in RA. The British Society for Rheumatology Biologics Registry (BILAG-BR) collects samples for RTX-treated patients in the UK. MASTERPLANS ISA consortium to identify predictors of drug response.

Methods
Patients were recruited if they were starting a first cycle of RTX for active SLE (BILAG A or 2xBILAG B) despite previous cyclophosphamide or mycophenolate mofetil. Disease activity was measured using BILAG-2004. Clinical response was defined as improvement by &gt; =1 grade in active BILAG-2004 systems with no worsening in other systems. Whole blood was collected into TEMPUS tubes and RNA extracted. IFN-Scores were measured using a custom Taqman array as previously described [El Sherbiny et al., 2018]. Multivariate logistic regression was used to test IFN-Scores and baseline clinical covariates as predictors of BILAG response at 6 months.

Results
Samples were available from 147 patients, of whom 84 had complete baseline and 6-month clinical data available and were included in this analysis. 40/84 (47.6%) patients had BILAG response at 6 months. In univariate and multivariate analysis, high IFN-Score-B expression was significantly associated with clinical response (see Table 1).

Conclusion
This preliminary analysis suggests that assessment of IFN activity has a role in predicting response to RTX. A novel IFN score (Score B) was more predictive than classic ISGs (Score A). These results add to a body of work showing that IFN-Score-B predicts clinically significant outcomes independently of overall IFN activity. Future work will analyse this biomarker in a larger cohort of patients and integrate with other putative clinical and biological predictors of response.

Disclosures
A. Alase None. Z. Wigston None. A. Burska None. M. Md Yusof None. J. Reynolds None. M. Wittmann None. I. Bruce None. E.M. Vital None.
P179 Glucocorticoid exposure and link to serious adverse events in patients with ANCA-associated vasculitis
Authors: Spearpoint Philip , Sammon Cormac , de Antonio Ramirez , Serna Arellano , Rutherford Peter 
Published: April 2020

Abstract

Background
Remission induction in ANCA-associated vasculitis (AAV) is with high dose glucocorticoids (GC) and immunosuppressants. Patients are exposed to high GC dose and/or prolonged low dose. EULAR/EDTA guidelines target 7.5-10mg at 3 months but acknowledge this is often achieved later. This study used UK real world practice data to examine the scale of GC exposure and associated clinical risks in AAV.

Methods
The study utilised the Clinical Practice Research Datalink (CPRD) - Hospital Episode Statistics (HES) linked database. AAV patients were identified using specific READ and ICD codes and followed between 01/01/1997 and 01/01/2018. GP prescriptions were used to describe periods of continuous GC use, stop and restart and when high dose (&gt; 30mg/day) and low dose (&lt;30mg/day) was prescribed. Diagnostic codes indicative of infections and adverse events linked to GCs were used to estimate rates in the AAV population using a generalized linear model with a Poisson distribution.

Results
450 AAV patients with at least one GC prescription were analysed. The median dose decreased to 9.3 mg (IQR 5.0 - 17.0) at 6 months and 5.1 mg (0.00 - 10.0) at 12 months,50% patients were taking &gt; 10mg at 5 months and 25% were still &gt; 10mg at 12 months. However, within 6 months of achieving 10mg/day, 50% relapse to needing dose &gt;10mg, 75% within 2 years and 90% within 6 years. In adjusted Poisson model (age, gender, year of diagnosis before/after 2013) the rate of infection in AAV patients taking high dose was 2.59 times (CI95 1.95, 3.45) that of those on low dose and lower in those not taking GCs (IRR 0.27 (0.22-0.34)). Increased risk of new onset cardiovascular disease (IRR 2.55 (0.92, 7.04)) and new onset renal disease (IRR 3.4 (1.29-8.96)) were higher in patients receiving high dose.

Conclusion
AAV patients have significant exposure to high dose GCs and in real world practice, GC dose remains higher than recommended in current clinical guidelines. High dose GCs are associated with high risk of infection and new cardiovascular disease and renal disease. This creates a significant patient burden and has implications for healthcare resource use.

Disclosures
P. Spearpoint: Corporate appointments; Employee of Vifor Pharma. C. Sammon: Corporate appointments; Employee of PHMR. A. Ramirez de Arellano Serna: Corporate appointments; Employee of Vifor Pharma. P. Rutherford: Corporate appointments; Employee of Vifor Pharma. Shareholder/stock ownership; Vifor Pharma.