Prevalence and Outcomes of Gastrointestinal Manifestations in an Australian Scleroderma Cohort.

Objective: The gastrointestinal tract (GIT) is the most commonly affected internal organ in systemic sclerosis (SSc). We sought to determine the prevalence and impact of GIT symptoms on survival and patient-reported outcomes.

Methods: A total of 907 consecutive patients from the Australian Scleroderma Cohort Study who had prospectively completed the University of California, Los Angeles, Scleroderma Clinical Trials Consortium Gastrointestinal Tract 2.

Inflammatory Arthritis in Systemic Sclerosis: Its Epidemiology, Associations, and Morbidity.

Objective: To describe the epidemiology, associations, and impact of inflammatory arthritis (IA) in systemic sclerosis (SSc).

Methods: Patients with SSc prospectively enrolled in the Australian Scleroderma Cohort Study were included. IA was defined clinically as the presence of synovitis on examination.

Randomised trial of genetic testing and targeted intervention to prevent the development and progression of Paget's disease of bone.

Introduction: Paget's disease of bone (PDB) frequently presents at an advanced stage with irreversible skeletal damage. Clinical outcomes might be improved by earlier diagnosis and prophylactic treatment.

Methods: We randomised 222 individuals at increased risk of PDB because of pathogenic variants to receive 5 mg zoledronic acid (ZA) or placebo.

Safer medicines To reduce falls and refractures for OsteoPorosis (#STOP): a study protocol for a randomised controlled trial of medical specialist-initiated pharmacist-led medication management reviews in primary care.

Introduction: Minimal trauma fractures (MTFs) often occur in older patients with osteoporosis and may be precipitated by falls risk-increasing drugs. One category of falls risk-increasing drugs of concern are those with sedative/anticholinergic properties. Collaborative medication management services such as Australia's Home Medicine Review (HMR) can reduce patients' intake of sedative/anticholinergics and improve continuity of care.

Outcome predictors of immune-mediated necrotizing myopathy-a retrospective, multicentre study.

Objectives: Evidence-based treatment protocols are currently lacking for immune-mediated necrotizing myopathy (IMNM). In this multicentre retrospective study, we examined baseline clinical characteristics and treatment variables that may predict short-term outcomes of patients with IMNM.

Methods: Muscle biopsies from the John Hunter Hospital and the Royal Adelaide Hospital obtained between 2012 and 2019 were reviewed at a single laboratory at South Australia Pathology.

Patient and parent perspectives on transition from paediatric to adult healthcare in rheumatic diseases: an interview study.

Objectives: To describe the experiences, priorities, and needs of patients with rheumatic disease and their parents during transition from paediatric to adult healthcare.

Setting: Face-to-face and telephone semistructured interviews were conducted from December 2018 to September 2019 recruited from five hospital centres in Australia.

Participants: Fourteen young people and 16 parents were interviewed.

Occupational silica exposure in an Australian systemic sclerosis cohort.

Objective: To determine the frequency of self-reported occupational exposure to silica in SSc patients enrolled in the Australian Scleroderma Cohort Study, and to compare the disease characteristics of the silica-exposed patients with those of the non-exposed patients.

Method: Data collected over a 12-year period from 1670 SSc patients were analysed. We compared the demographic and clinical characteristics of those who reported occupational silica exposure with those who did not.

Patients' attitudes and experiences of transition from paediatric to adult healthcare in rheumatology: a qualitative systematic review.

Objectives: We aimed to describe patients' attitudes and experiences of transition from paediatric to adult healthcare in rheumatology to inform patient-centred transitional care programmes.

Methods: We searched MEDLINE, EMBASE, PsycINFO and CINAHL to August 2019 and used thematic synthesis to analyse the findings.

Results: From 26 studies involving 451 people with juvenile-onset rheumatic conditions we identified six themes: a sense of belonging (comfort in familiarity, connectedness in shared experiences, reassurance in being with others of a similar age, desire for normality and acceptance); preparedness for sudden changes (confidence through guided introductions to the adult environment, rapport from continuity of care, security in a reliable point of contact, minimizing lifestyle disruptions); abandonment and fear of the unknown (abrupt and forced independence, ill-equipped to hand over medical information, shocked by meeting adults with visible damage and disability, vulnerability in the loss of privacy); anonymous and dismissed in adult care (deprived of human focus, sterile and uninviting environment, disregard of debilitating pain and fatigue); quest for autonomy (controlled and patronized in the paediatric environment, liberated from the authority of others, freedom to communicate openly); and tensions in parental involvement (overshadowed by parental presence, guilt of excluding parents, reluctant withdrawal of parental support).

Characteristics of Early Paget's Disease in SQSTM1 Mutation Carriers: Baseline Analysis of the ZiPP Study Cohort.

Mutations in SQSTM1 are strongly associated with Paget's disease of bone (PDB), but little is known about the clinical characteristics of those with early disease. Radionuclide bone scans, biochemical markers of bone turnover, and clinical characteristics were analyzed in SQSTM1 mutation carriers who took part in the Zoledronic acid in the Prevention of Paget's disease (ZiPP) study. We studied 222 individuals, of whom 54.

Clinical Features of Systemic Sclerosis-Mixed Connective Tissue Disease and Systemic Sclerosis Overlap Syndromes.

Objective: To describe the clinical characteristics and outcomes of systemic sclerosis-mixed connective tissue disease (SSc-MCTD) and SSc overlap syndrome.

Methods: We included patients from the Australian Scleroderma Cohort Study who met American College of Rheumatology/European Alliance of Associations for Rheumatology criteria for SSc. Three mutually exclusive groups were created: SSc-MCTD, SSc overlap, and SSc only.

External Validation and Evaluation of Adding MRI or Extended Myositis Antibody Panel to the 2017 EULAR/ACR Myositis Classification Criteria.

Objective: To externally validate the European League Against Rheumatism/American College of Rheumatism (EULAR/ACR) classification criteria for idiopathic inflammatory myositis (IIM) and determine the optimal cut points for Australian patients. To determine the level of agreement with traditional criteria and assess the effect of including magnetic resonance imaging (MRI) and an extended myositis antibody panel as well as extending histological criteria to include myofiber invasion.

Methods: Data were collected on adult patients referred for muscle biopsy to two Australian teaching hospitals.

Zoledronate in the prevention of Paget's (ZiPP): protocol for a randomised trial of genetic testing and targeted zoledronic acid therapy to prevent -mediated Paget's disease of bone.

Introduction: Paget's disease of bone (PDB) is characterised by increased and disorganised bone remodelling affecting one or more skeletal sites. Complications include bone pain, deformity, deafness and pathological fractures. Mutations in sequestosome-1 () are strongly associated with the development of PDB.

Can Patient-Reported Symptoms Be Used to Measure Disease Activity in Systemic Sclerosis?

Objective: To evaluate the association between patient-reported symptoms and changes in disease activity over time in systemic sclerosis (SSc).

Methods: Using data from 1,636 patients enrolled in the Australian Scleroderma Cohort Study, we used generalized estimating equations to determine the relationship between patient-reported worsening of Raynaud's phenomenon (RP), skin involvement, and breathlessness in the month preceding each study visit and features of disease activity in the corresponding organ systems. The associations between the following parameters were analyzed: patient-reported worsening RP and the presence of new-onset digital pitting and digital ulcers; patient-reported worsening skin involvement and increasing modified Rodnan skin thickness score (MRSS); new areas of skin involvement and new-onset joint contractures; patient-reported worsening breathlessness and deteriorating respiratory functions test (RFT) results, indicated by a 10% decrease in forced vital capacity (FVC) and a 15% decrease in diffusing capacity for carbon monoxide (DLco), new-onset interstitial lung disease (ILD), and new-onset pulmonary arterial hypertension (PAH).

Patient and Caregiver Priorities for Medication Adherence in Gout, Osteoporosis, and Rheumatoid Arthritis: Nominal Group Technique.

Objective: This study aimed to identify and prioritize factors important to patients and caregivers with regard to medication adherence in gout, osteoporosis (OP), and rheumatoid arthritis (RA) and to describe the reasons for their decisions.

Methods: Patients with gout, OP, and RA and their caregivers, purposively sampled from 5 rheumatology clinics in Australia, identified and ranked factors that they considered important for medication adherence using nominal group technique and discussed their decisions. An importance score (IS; scale 0-1) was calculated, and qualitative data were analyzed thematically.

Significance of anti-neutrophil cytoplasmic antibodies in systemic sclerosis.

Background: Up to 12% of patients with systemic sclerosis (SSc) have anti-neutrophil cytoplasmic antibodies (ANCA). However, the majority of these patients do not manifest ANCA-associated vasculitis (AAV) and the significance of ANCA in these patients is unclear. The aim of this study is to determine the prevalence of ANCA in a well-characterised SSc cohort and to examine the association between ANCA and SSc clinical characteristics, other autoantibodies, treatments and mortality.

The Costs of Confronting Osteoporosis: Cost Study of an Australian Fracture Liaison Service.

Fracture liaison services (FLS) are an accepted approach to lowering rates of osteoporotic refractures. However, resource allocations to FLS are open to challenge, as most relevant cost analyses are based on anticipated, rather than observed, benefits. To support informed decision making, we have estimated the cost of operating an FLS, from the perspective of the Australian health system, with real life costs.

The role of inflammatory markers in assessment of disease activity in systemic sclerosis.

Objectives: The role of the erythrocyte sedimentation rate (ESR) and C-reactive protein (CRP) in the assessment of disease activity in systemic sclerosis (SSc) remains controversial. We sought to evaluate the relationship between clinical features of SSc and raised inflammatory markers and to determine if changes in ESR and CRP reflect changes in other disease features over time.

Methods: One thousand, five hundred and forty-five patients enrolled in the Australian Scleroderma Cohort Study were observed over a mean 3.

Unleashing the tiger - iatrogenic autoimmunity from cancer immunotherapy drugs.

Immune checkpoint inhibitors can lead to the development of organ and non-organ specific immune related adverse events.

Australian and New Zealand recommendations for the diagnosis and management of gout: integrating systematic literature review and expert opinion in the 3e Initiative.

Aim: To develop evidence-based recommendations for the diagnosis and management of gout in Australia and New Zealand as part of the multi-national 3e Initiative.

Method: Using a formal voting process, a panel of 78 international rheumatologists selected 10 key clinical questions pertinent to the diagnosis and management of gout. An additional question was also developed by participating Australian and New Zealand rheumatologists.

Australian and New Zealand evidence-based recommendations for pain management by pharmacotherapy in adult patients with inflammatory arthritis.

Aim: To develop Australian and New Zealand evidence-based recommendations for pain management by pharmacotherapy in adult patients with optimally treated inflammatory arthritis (IA).

Methods: Four hundred and fifty-three rheumatologists from 17 countries including 46 rheumatologists from Australia and New Zealand participated in the 2010 3e (Evidence, Expertise, Exchange) Initiative. Using a formal voting process, rheumatologists from 15 national scientific committees selected 10 clinical questions regarding the use of pain medications in IA.