Severe Diffuse Takayasu Arteritis Manifestation in Childhood.

Background: Takayasu arteritis (TA) is a large-vessel vasculitis that primarily affects the aorta and the pulmonary and coronary arteries.

Case Summary: A 4-year-old boy was diagnosed in 2022 with pyoderma gangrenosum (PG) and required prolonged multidisciplinary care, including aggressive medical immunosuppression for severe skin lesions. In October 2024, after a period of clinical stability, he presented with a seizure and right-sided weakness.

The interplay between pain and disease activity: personal models of pain beliefs and emotional representations in children and young people with juvenile idiopathic arthritis in a UK nationwide prospective inception cohort.

Objectives: Juvenile idiopathic arthritis (JIA) is a group of childhood-onset inflammatory rheumatic conditions characterized by pain as one of the most common and distressing symptoms. This cross-sectional study aimed to investigate whether relationships between reported pain and disease activity in JIA affected beliefs about pain, known as "personal models."

Methods: 187 out of a possible 363 participants with JIA who completed questionnaires about function and pain perception were recruited through the Childhood Arthritis Prospective Study (CAPS).

Virtually the same, but remotely different: health professionals, parents and children's experiences of remote out-patient consultations.

Objective: To explore experiences, benefits and concerns associated with remote (telephone/video) consultations from the perspectives of children and young people with juvenile idiopathic arthritis (JIA), their parents and health professionals who were members of a multidisciplinary team in a paediatric rheumatology setting.

Methods: Qualitative design (Interpretive Description) utilizing observation of remote (telephone/video) consultations and remote follow-up interviews with children and young people (7-18 years) with JIA, their parents and health professionals. The setting was a tertiary paediatric rheumatology clinic in a hospital in Northwest England.

Long-term efficacy and safety of subcutaneous tocilizumab in clinical trials of polyarticular or systemic juvenile idiopathic arthritis.

Objective: To investigate the safety and efficacy of subcutaneous tocilizumab (SC-TCZ) treatment in a long-term extension (LTE) of clinical trials in polyarticular or systemic juvenile idiopathic arthritis (pJIA or sJIA).

Methods: Patients with pJIA or sJIA from two open-label, 52-week phase 1b core trials of SC-TCZ who had adequate response per investigator assessment entered the LTE and continued SC-TCZ treatment according to body weight-based dosing regimens until commercial availability or up to 5 years. Pharmacokinetics, pharmacodynamics, and efficacy were assessed for up to 3 years, and safety for up to 5 years in the LTE.

Overlap of International League of Associations for Rheumatology and Preliminary Pediatric Rheumatology International Trials Organization Classification Criteria for Nonsystemic Juvenile Idiopathic Arthritis in an Established UK Multicentre Inception Cohort.

Objective: The goal was to assess the degree of overlap between existing International League of Associations for Rheumatology (ILAR) and preliminary Paediatric Rheumatology International Trials Organisation (PRINTO) classification criteria for juvenile idiopathic arthritis (JIA).

Methods: Participants from the Childhood Arthritis Prospective Study, a multicenter UK JIA inception cohort, were classified using the PRINTO and ILAR classification criteria into distinct categories. Systemic JIA was excluded because several classification items were not collected in this cohort.

Quality improvement in juvenile idiopathic arthritis: a mixed-methods implementation pilot of the CAPTURE-JIA dataset.

Background: A significant proportion of children and young people with juvenile idiopathic arthritis (JIA) do not achieve inactive disease during the first two years following diagnosis. Refinements to clinical care pathways have the potential to improve clinical outcomes but a lack of consistent and contemporaneous clinical data presently precludes standard setting and implementation of meaningful quality improvement programmes. This study was the first to pilot clinical data collection and analysis using the CAPTURE-JIA dataset, and to explore patient and clinician-reported feasibility and acceptability data.

A "cat"-astrophic case of Bartonella infective endocarditis causing secondary cryoglobulinemia: a case report.

Background: Culture-negative infective endocarditis (IE) constitutes approximately 10% of all cases of IE. Bartonella endocarditis is a common cause of culture-negative endocarditis and is associated with a high mortality rate. To date, no cases of Bartonella IE has been reported in association with cryoglobulinemia in the UK.

Evaluation of Serious Infection in Pediatric Patients with Low Immunoglobulin Levels Receiving Rituximab for Granulomatosis with Polyangiitis or Microscopic Polyangiitis.

Introduction: The aim of this work was to assess the impact of prolonged low immunoglobulin (IgG or IgM) serum concentrations on the potential cumulative serious infection (SI) risk in pediatric patients following rituximab treatment for granulomatosis with polyangiitis or microscopic polyangiitis (GPA/MPA) in PePRS.

Methods: Patients aged ≥ 2 to < 18 years received four weekly intravenous rituximab infusions of 375 mg/m and concomitant glucocorticoid taper. After 6 months, patients could receive further rituximab and/or other immunosuppressants per investigator discretion.

Telemedicine: improving clinical care and medical education in paediatrics.

The COVID-19 pandemic has led to a rapid increase in the use of telemedicine. This is likely to continue when the social distancing restrictions have been eased. There have been a number of technological advances that have contributed to the roll-out and improved quality of telemedicine consultations.

Impact of COVID-19 on training: a single-centre survey of trainees.

To review the impact of COVID-19 on postgraduate paediatric training, a 10-question online survey was designed to evaluate trainees' training opportunities. 56 trainees working at a single centre, Alder Hey Children's Hospital, completed the survey. The majority of trainees felt that COVID-19 had affected their training.

Phase IIa Global Study Evaluating Rituximab for the Treatment of Pediatric Patients With Granulomatosis With Polyangiitis or Microscopic Polyangiitis.

Objective: To assess the safety, tolerability, pharmacokinetics, and efficacy of rituximab (RTX) in pediatric patients with granulomatosis with polyangiitis (GPA) or microscopic polyangiitis (MPA).

Methods: The Pediatric Polyangiitis Rituximab Study was a phase IIa, international, open-label, single-arm study. During the initial 6-month remission-induction phase, patients received intravenous infusions of RTX (375 mg/m body surface area) and glucocorticoids once per week for 4 weeks.

Mycophenolate Mofetil Versus Cyclophosphamide for Remission Induction in Childhood Polyarteritis Nodosa: An Open-Label, Randomized, Bayesian Noninferiority Trial.

Objective: Cyclophosphamide (CYC) is used in clinical practice off-label for the induction of remission in childhood polyarteritis nodosa (PAN). Mycophenolate mofetil (MMF) might offer a less toxic alternative. This study was undertaken to explore the relative effectiveness of CYC and MMF treatment in a randomized controlled trial (RCT).

Validation of novel patient-centred juvenile idiopathic arthritis-specific patient-reported outcome and experience measures (PROMs/PREMs).

Background: Measuring the outcomes that matter to children and young people (CYP) with juvenile idiopathic arthritis (JIA), is a necessary precursor to patient-centred improvements in quality of clinical care. We present a two-centre validation of novel JIA patient-reported outcome and experience measures (PROM and PREM) developed as part of the CAPTURE-JIA project.

Methods: CYP with JIA were recruited from paediatric rheumatology clinics, completing the CAPTURE-JIA PROM and PREM, CHAQ and CHU 9D.

Short-term outcome of surgical arthrodiastasis of the ankle with Ilizarov frame in a cohort of children and young people with juvenile idiopathic arthritis.

Objectives: Despite medical advances, life-changing articular damage may still occur in patients with JIA. We report a cohort with destructive arthropathy of the ankle treated by surgical arthrodiastasis.

Methods: Eight patients (nine ankles) received arthrodiastasis by means of an Ilizarov frame between 2009 and 2013.

CAPTURE-JIA: a consensus-derived core dataset to improve clinical care for children and young people with juvenile idiopathic arthritis.

Objectives: Data collected during routine clinic visits are key to driving successful quality improvement in clinical services and enabling integration of research into routine care. The purpose of this study was to develop a standardized core dataset for juvenile idiopathic arthritis (JIA) (termed CAPTURE-JIA), enabling routine clinical collection of research-quality patient data useful to all relevant stakeholder groups (clinicians, service-providers, researchers, health service planners and patients/families) and including outcomes of relevance to patients/families.

Methods: Collaborative consensus-based approaches (including Delphi and World Café methodologies) were employed.

Effect of socioeconomic conditions on frequent complaints of pain in children: findings from the UK Millennium Cohort Study.

Background: Frequent complaints of pain (FCP) are common in high-income countries, affecting about 25% of children, and may have significant adverse consequences including prolonged school absence and disability. Most FCP are unexplained, and the aetiology is poorly understood. This study aimed to identify risk factors for FCP and explore how risk factors explain variation in pain reporting by childhood socioeconomic conditions (SECs).

Hypercalcaemic Pancreatitis, Adrenal Insufficiency, Autoimmune Thyroiditis and Diabetes Mellitus in a girl with Probable Sarcoidosis.

Introduction: Sarcoidosis is a multisystemic granulomatous disease with diverse and often non-specific symptoms during childhood. The clinical manifestations sometimes include endocrinopathies related to sarcoid infiltration of various endocrine organs, but more commonly due to the associated autoimmune endocrine disorders. There are only a few reports of multiple autoimmune and non-autoimmune endocrine problems occurring simultaneously in patients with sarcoidosis.

Development of a national audit tool for juvenile idiopathic arthritis: a BSPAR project funded by the Health Care Quality Improvement Partnership.

Objective: Timely access to holistic multidisciplinary care is the core principle underpinning management of juvenile idiopathic arthritis (JIA). Data collected in national clinical audit programmes fundamentally aim to improve health outcomes of disease, ensuring clinical care is equitable, safe and patient-centred. The aim of this study was to develop a tool for national audit of JIA in the UK.