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Background <p>Biologic and targeted synthetic disease-modifying anti-rheumatic drugs (b/tsDMARDs) have transformed outcomes for juvenile idiopathic arthritis (JIA). However, contemporary understanding of real-world treatment selection, and access across the United Kingdom (UK), remains limited. Commissioning pathways, service configuration, and age- and specialty-based prescribing frameworks may contribute to variations in care. We describe current perspectives of paediatric and adolescent rheumatology multidisciplinary teams (MDTs) on the selection and access to b/tsDMARDs for JIA across the four UK nations.</p> Methods <p>A 26-question survey was distributed to MDTs across UK paediatric and adolescent rheumatology centres via national networks. Data on MDT composition, b/tsDMARD choice, commissioned pathways, dosing constraints and barriers to access were analysed by JIA subtype, UK region, age, and specialty pathway. Heatmaps and non-parametric permutation testing were used to quantify treatment patterns. Free-text responses underwent qualitative thematic analysis.</p> Results <p>Responses were received from 13 of 17 (76%) tertiary and quaternary centres across the four UK nations, representing 123 MDT professionals. Adalimumab was most commonly used first-line for all JIA subtypes except systemic JIA (sJIA)/Still’s disease. Tocilizumab was frequently used second-line across multiple subtypes, including polyarticular disease and uveitis, and was widely used first- and second-line in sJIA/Still’s. Interleukin-1 inhibition with anakinra was commonly used in sJIA, particularly in macrophage activation syndrome (MAS). In England and Wales, access to canakinumab for sJIA was unavailable despite supportive trial evidence, and adalimumab was the only commissioned therapy for JIA-associated uveitis. Paediatric access to anti–IL-17 agents for enthesitis-related arthritis (ERA) was limited. Broader therapeutic choice and higher biologic dosing were more accessible via dermatology or gastroenterology pathways, for example, for psoriatic JIA and inflammatory bowel disease (IBD)-associated arthritis. Adult/adolescent rheumatologists reported wider biologic access, though reclassification to adult diagnoses was sometimes required. Biosimilars were universally adopted, but specialist pharmacy support for implementation was inconsistent. External advice was sought for refractory disease, including consideration of haematopoietic stem cell transplantation and combination biologic therapy.</p> Conclusions <p>Selection and access to effective b/tsDMARD therapies for JIA varies considerably across the UK, shaped by commissioning structures, age thresholds and specialty pathways rather than clinical need. National alignment of services is urgently required to ensure equitable, timely access to evidence-based therapies for children and young people with JIA.</p>

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Variation in the access and use of biologic and targeted synthetic DMARD in juvenile idiopathic arthritis: insights from a national UK survey

  • 期刊论文

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  • 发表日期：

    2026年7月6日

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  • Samantha Cooray,
  • Kalindi Rajani,
  • Sandrine Compeyrot-Lacassagne,
  • Ethan S. Sen,
  • Catriona Anderson,
  • Andrew Fell,
  • Dearbhla McKenna,
  • Clara Danielsen,
  • Mario Cortina-Borja,
  • Coziana Ciurtin,
  • Ameenat Lola Solebo,
  • Paul Brogan

摘要

Background

Biologic and targeted synthetic disease-modifying anti-rheumatic drugs (b/tsDMARDs) have transformed outcomes for juvenile idiopathic arthritis (JIA). However, contemporary understanding of real-world treatment selection, and access across the United Kingdom (UK), remains limited. Commissioning pathways, service configuration, and age- and specialty-based prescribing frameworks may contribute to variations in care. We describe current perspectives of paediatric and adolescent rheumatology multidisciplinary teams (MDTs) on the selection and access to b/tsDMARDs for JIA across the four UK nations.

Methods

A 26-question survey was distributed to MDTs across UK paediatric and adolescent rheumatology centres via national networks. Data on MDT composition, b/tsDMARD choice, commissioned pathways, dosing constraints and barriers to access were analysed by JIA subtype, UK region, age, and specialty pathway. Heatmaps and non-parametric permutation testing were used to quantify treatment patterns. Free-text responses underwent qualitative thematic analysis.

Results

Responses were received from 13 of 17 (76%) tertiary and quaternary centres across the four UK nations, representing 123 MDT professionals. Adalimumab was most commonly used first-line for all JIA subtypes except systemic JIA (sJIA)/Still’s disease. Tocilizumab was frequently used second-line across multiple subtypes, including polyarticular disease and uveitis, and was widely used first- and second-line in sJIA/Still’s. Interleukin-1 inhibition with anakinra was commonly used in sJIA, particularly in macrophage activation syndrome (MAS). In England and Wales, access to canakinumab for sJIA was unavailable despite supportive trial evidence, and adalimumab was the only commissioned therapy for JIA-associated uveitis. Paediatric access to anti–IL-17 agents for enthesitis-related arthritis (ERA) was limited. Broader therapeutic choice and higher biologic dosing were more accessible via dermatology or gastroenterology pathways, for example, for psoriatic JIA and inflammatory bowel disease (IBD)-associated arthritis. Adult/adolescent rheumatologists reported wider biologic access, though reclassification to adult diagnoses was sometimes required. Biosimilars were universally adopted, but specialist pharmacy support for implementation was inconsistent. External advice was sought for refractory disease, including consideration of haematopoietic stem cell transplantation and combination biologic therapy.

Conclusions

Selection and access to effective b/tsDMARD therapies for JIA varies considerably across the UK, shaped by commissioning structures, age thresholds and specialty pathways rather than clinical need. National alignment of services is urgently required to ensure equitable, timely access to evidence-based therapies for children and young people with JIA.