Maria F. Munns-Craig

Description:Objective: To examine the overall incidence rate and trends in emergency department (ED) presentations related to asthma and allergic diseases in regional Australia with a particular focus on First Nations Australians. Methods: A retrospective analysis of data from the Emergency Department Information System. Methods: This study used data from 12 public hospitals in Central Queensland, Australia, a region encompassing regional, rural and remote outback areas. Methods: A total of 813 112 ED presentations between 2018 and 2023. Methods: Asthma and allergic diseases were identified using the International Classification of Diseases-Tenth Revision-Australian Modification codes. Results: There were 13 273 asthma and allergic disease-related ED presentations, with an overall prevalence of 1.6% (95% CI 1.6, 1.7). There was a significantly higher incidence rate of asthma and allergic disease-related ED presentations among First Nations Australians at 177.5 per 10 000 person-years (95% CI 169.3, 186.0) compared with 98.9 per 10 000 person-years (95% CI 97.2, 100.8) among Australians of other descents. The incidence rates, with corresponding 95% CIs, of the four most common cases among First Nations Australians and Australians of other descents, respectively, were as follows: asthma (87.8 (82.0, 93.8) and 40.2 (39.0, 41.3)), unspecified allergy (55.3 (50.8, 60.2) and 36.0 (34.9, 37.1)), atopic/allergic contact dermatitis (17.1 (14.6, 19.9) and 10.6 (10.0, 11.2)) and anaphylaxis (7.2 (5.6, 9.1) and 6.2 (5.7, 6.6)). Conclusions: Our findings highlight a significantly higher rate of asthma and allergic disease-related ED presentations among First Nations Australians compared with Australians of other descents. This underscores the urgent need for targeted healthcare interventions integrating culturally appropriate approaches, alongside additional research to understand causality.

Description:Background: Cerebral palsy (CP) is the most common physical disability of childhood, affecting movement and posture, resulting from a neurological insult during pregnancy or the neonatal period. While the brain lesion is static, the musculoskeletal sequelae in CP are often progressive and lifelong, associated with pain and can impact the lives of children with CP, their families and the healthcare system. The Australasian Cerebral Palsy Musculoskeletal Health Network (AusCP MSK) study will conduct comprehensive, population-based surveillance of children with moderate to severe functional mobility limitations (Gross Motor Function Classification System (GMFCS) levels III-V) to explore the early biomarkers of, and interactions between, musculoskeletal complications related to CP, including hip displacement, scoliosis and skeletal fragility. Methods: The AusCP MSK study involves three cohorts of children. Cohort A (n=500) is a multicentre retrospective (3 years) and prospective (4 years) cohort study in children aged 4-9 years that will be implemented at five sites across Australia and New Zealand. Retrospective data will include clinical history, information on CP diagnosis and other investigations (previous X-rays and biochemistry). Primary prospective outcomes will involve measures of hip displacement (migration percentage, acetabular index, femoral head orientation, Hilgenreiner's epiphyseal angle), scoliosis (Anteroposterior/Posteroanterior and lateral spine X-ray), skeletal fragility (Dual Energy X-ray Absorptiometry, peripheral quantitative computed tomography), motor function (GMFCS, Manual Ability Classification System (MACS) and Communication Function Classification System (CFCS)) and range of movement (lower limb and spine). Cohort B (n=4000) is a retrospective analysis of data to evaluate fractures in children up to 18 years of age with CP (GMFCS I-V) from the New South Wales (NSW)/Australian Capital Territory CP Registers linked with corresponding records from NSW administrative health data (n=3000), and a New Zealand cohort of linked data from the New Zealand Cerebral Palsy Register to the Accident Compensation Corporation data for fracture claims (n=1000). Cohort C (n=30) will cross-sectionally examine bone quality through a transiliac bone biopsy in children undergoing scheduled hip surgery. Relationships between early biomarkers, early brain structure and musculoskeletal complications will be explored using multilevel mixed-effect models. Background: Ethical approval for this study was granted by Children's Health Queensland Hospital and Health Service Human Research Ethics Committee, The University of Queensland Human Research Ethics Committee and the New Zealand Health and Disability Ethics Committee.Research outcomes will be disseminated via scientific conferences and publications in peer-reviewed journals; to the National Bodies and Clinicians; and to people with CP and their families. Background: Australian New Zealand Clinical Trials Registry number: ACTRN12622000788774p.

Description:Achondroplasia is the most common form of skeletal dysplasia. Over the last 24 months, a new treatment (vosoritide) is now available to promote endochondral growth through a daily injection. While research has identified increased annualized growth velocity in children with achondroplasia receiving vosoritide injections, there is currently little information on functional change and independence in self-care following this treatment. Here, we present a 5-year-old boy with achondroplasia who received vosoritide injections for 12 months. His functional performance was measured using the Functional Independence Measure for Children (WeeFIM). He was found to have a slightly higher level of independence when compared to age-matched peers with achondroplasia, and his rate of improvement in independence skills was twice that recorded in a previous study monitoring independence in children with achondroplasia following 12 months on vosoritide. When compared with WeeFIM normative data from average-statured children across the 12 months from 4 to 5 years, our child demonstrated almost three times the change for total WeeFIM and Self-Care domain scores and nine times the change across the Mobility domain scores, indicating a rapid increase in independence development. Our findings suggest that vosoritide may support the improvement of functional independence in children with achondroplasia receiving vosoritide to increase bone growth.

Description:Objective: To identify the types of conditions reported in peer-reviewed literature that result in chronic musculoskeletal lower limb pain in children and adolescents and explore the alignment of these conditions with the chronic pain reporting codes indexed in the International Classification of Diseases 11th Revision (ICD-11). Methods: This scoping review follows the Preferred Reporting Items for Systematic Reviews and Meta-Analyses guidelines. Methods: Five electronic databases were searched (Medline, EMBASE, PsycINFO, CINAHL and the Cochrane Library). Methods: Articles involving children and adolescents under 18 years and reporting on chronic musculoskeletal pain of the lower limb were included. Methods: We assigned an ICD-11 code to each condition based on details reported in the study. We recorded whether any of the presenting conditions were linked to an ICD-11 chronic pain manifestation code. Results: From 12 343 records, 418 papers were included. There were 124 unique conditions associated with chronic lower limb pain, the most commonly reported being chronic widespread musculoskeletal pain (24 studies) and juvenile idiopathic arthritis (26 studies). Only 11.1% of presenting conditions were linked to an ICD-11 chronic pain manifestation code. Conclusions: Most presenting conditions associated with chronic pain in the lower limb do not have a chronic pain manifestation code in the new global standard for recording health information. This means chronic pain associated with common lower limb conditions may remain invisible in global statistics.

Description:X linked hypophosphataemia (XLH) is a systemic, chronic condition that significantly impairs quality of life. In XLH, a phosphate regulating endopeptidase homologue X-linked (PHEX) gene mutation leads to excess fibroblast growth factor 23 (FGF23), causing hypophosphataemia and subsequent rickets, lower limb deformity, pain and other sequelae, however there are likely other non-FGF23 mediated mechanisms contributing to disease. Burosumab is an FGF23 inhibiting monoclonal antibody that has been shown to be significantly more effective in treating X linked hypophosphataemia than previously available treatment ("conventional therapy" with oral phosphate and active vitamin D). Clinical trials and real-world studies have shown that burosumab can improve lower limb deformity, growth, pain, exercise capacity, biochemistry, rickets, and quality of life. However, the full effect of burosumab on the lives of individuals with X linked hypophosphataemia is yet to be determined. How burosumab may impact some of the lesser understood clinical features, including dental abscesses, craniosynostosis, enthesopathy, and osteoarthritis, is unclear. Whether burosumab mitigates the risk of complications associated with conventional therapy (nephrocalcinosis and hyperparathyroidism) has also not been established. There are conflicting recommendations on who should receive burosumab, when they should start it, and for how long they should continue taking it. This review summarises what is known, and more importantly what is unknown, about burosumab use in X linked hypophosphataemia. We highlight important areas for future research to better understand the impact of burosumab in XLH, improve management of XLH, assess cost benefit of, and advocate for fair and equitable access to burosumab.