Research graph
References from Advancing evidence generation in pediatric rheumatology beyond traditional randomized clinical trials: the potential of innovative designs. Local targets link to admitted publications; unresolved targets remain external evidence.
Juvenile idiopathic arthritis
10.1038/s41572-021-00332-8 · 2022 · External reference
Clinical trial design in juvenile idiopathic arthritis
10.1007/s40272-017-0244-2 · 2017 · External reference
Transforming clinical trials in rheumatology: towards patient-centric precision medicine
10.1038/s41584-020-0491-4 · 2020 · External reference
Is it time to move to active comparator trials in juvenile idiopathic arthritis?: a review of current study designs
10.1002/art.27670 · 2010 · External reference
How can trial designs better serve the needs of children and young people with juvenile idiopathic arthritis?
10.1016/s2665-9913(24)00026-2 · 2024 · External reference
25 years of biologics for the treatment of pediatric rheumatic disease: advances in prognosis and ongoing challenges
10.1002/acr.25482 · 2025 · External reference
Master protocols to study multiple therapies, multiple diseases, or both
10.1056/nejmra1510062 · 2017 · External reference
Master protocol trials in oncology: review and new trial designs
10.1016/j.conctc.2018.08.009 · 2018 · External reference
The master protocol concept
10.1053/j.seminoncol.2015.07.009 · 2015 · External reference
Novel clinical trial design and analytic methods to tackle challenges in therapeutic development in rare diseases
10.21037/atm-21-5496 · 2022 · External reference
Type I error considerations in master protocols with common control in oncology trials: report of an American Statistical Association Biopharmaceutical Section Open Forum Discussion
10.1080/19466315.2021.1906743 · 2022 · External reference
Bayesian methods for information borrowing in basket trials: an overview
10.3390/cancers16020251 · 2024 · External reference
The universal effects of low-dose interleukin-2 across 13 autoimmune diseases in a basket clinical trial
10.1016/j.jaut.2024.103172 · 2024 · External reference
Multicentre, 26-week, open-label phase 2 trial of the JAK inhibitor filgotinib in Behçet’s disease, idiopathic inflammatory myopathies and IgG4-related disease: DRIMID study protocol
10.1136/bmjopen-2024-089827 · 2025 · External reference
CD19 CAR-T cells for treatment-refractory autoimmune diseases: the phase 1/2 CASTLE basket trial
10.1038/s41591-025-04185-6 · 2026 · External reference
Immune mediated inflammatory diseases: moving from targeted biologic therapy, stem cell therapy to targeted cell therapy
2025 · External reference
The challenges of new biologic therapies in pediatrics: an update on the limited therapeutic options for juvenile psoriatic arthritis
10.1016/j.rdc.2025.05.002 · 2025 · External reference
Long-term efficacy and safety of canakinumab in patients with mevalonate kinase deficiency: results from the randomised phase 3 CLUSTER trial
10.1093/rheumatology/keab696 · 2022 · External reference
Tocilizumab in patients with anti-TNF refractory juvenile idiopathic arthritis-associated uveitis (APTITUDE): a multicentre, single-arm, phase 2 trial
10.1016/s2665-9913(20)30008-4 · 2020 · External reference
Baricitinib in juvenile idiopathic arthritis: an international, phase 3, randomised, double-blind, placebo-controlled, withdrawal, efficacy, and safety trial
10.1016/s0140-6736(23)00921-2 · 2023 · External reference
Elicitation of expert prior opinion to design the BARJDM trial in juvenile dermatomyositis
10.1093/rheumatology/keae392 · 2024 · External reference
Bayesian trial of adalimumab versus secukinumab for children with juvenile idiopathic arthritis associated uveitis or chronic anterior uveitis
10.1186/s12969-025-01107-1 · 2025 · External reference
Application of Bayesian statistics to support approval of intravenous belimumab in children with systemic lupus erythematosus in the United States
10.1177/09612033251349930 · 2025 · External reference
The 4th NextGen therapies of SJIA and MAS, part 4: it is time for IL-18 based trials in systemic juvenile idiopathic arthritis?
10.1186/s12969-023-00867-y · 2024 · External reference
Systemic juvenile idiopathic arthritis and adult-onset Still’s disease are the same disease and should now be designated by the same unique name, Still’s disease
10.1016/j.jbspin.2025.105872 · 2025 · External reference
Emerging concepts and treatments in autoinflammatory interferonopathies and monogenic systemic lupus erythematosus
10.1038/s41584-024-01184-8 · 2025 · External reference
ANA-associated arthritis: clinical and biomarker characterization of a population for basket trials
10.1093/rheumatology/keae269 · 2024 · External reference
Basket trials: review of current practice and innovations for future trials
10.1200/jco.21.02285 · 2022 · External reference
Categories, components, and techniques in a modular construction of basket trials for application and further research
10.1002/bimj.202000314 · 2021 · External reference
Oral ritlecitinib and brepocitinib for moderate-to-severe ulcerative colitis: results from a randomized, phase 2b study
10.1016/j.cgh.2022.12.029 · 2023 · External reference
Design and analysis of umbrella trials: where do we stand?
10.3389/fmed.2022.1037439 · 2022 · External reference
Synovial tissue atlas in juvenile idiopathic arthritis reveals pathogenic niches associated with disease severity
10.1126/scitranslmed.adt6050 · 2025 · External reference
Comparison of multi-arm multi-stage design and adaptive randomization in platform clinical trials
10.1016/j.cct.2017.01.003 · 2017 · External reference
Current state-of-the-art and gaps in platform trials: 10 things you should know, insights from EU-PEARL
10.1016/j.eclinm.2023.102384 · 2024 · External reference
Adaptive seamless phase II/III designs: background, operational aspects, and examples
10.1177/216847900604000412 · 2006 · External reference
Platform trials: key features, when to use them and methodological challenges
10.5694/mja2.52711 · 2025 · External reference
Design and rationale of RECOVER-AUTONOMIC: a randomized platform trial evaluating interventions for Long COVID postural orthostatic tachycardia syndrome
10.1016/j.ahj.2026.107384 · 2026 · External reference
RECOVER-NEURO: study protocol for a multi-center, multi-arm, phase 2, randomized, active comparator trial evaluating three interventions for cognitive dysfunction in post-acute sequelae of SARS-CoV-2 infection (PASC)
10.1186/s13063-024-08156-z · 2024 · External reference
Developing an adaptive platform trial for evaluation of medical treatments for Crohn’s disease
10.1038/s41575-025-01052-0 · 2025 · External reference
The future of clinical trials in inflammatory bowel disease
10.1016/j.cgh.2024.06.036 · 2025 · External reference
Adaptive platform trials: definition, design, conduct and reporting considerations
10.1038/s41573-019-0034-3 · 2019 · External reference
Use of Bayesian techniques in clinical trials for rheumatoid arthritis and systemic sclerosis: a scoping review
10.1186/s41927-025-00563-2 · 2025 · External reference
Regulatory expectations for Bayesian methods in drug and biologic clinical trials: a practical perspective on FDA’s 2026 draft guidance
2026 · External reference
Response adaptive randomisation in clinical trials: current practice, gaps and future directions
10.1177/09622802251348183 · 2025 · External reference
Randomization in the age of platform trials: unexplored challenges and some potential solutions
10.1186/s12874-025-02693-0 · 2025 · External reference
Statistical modeling to adjust for time trends in adaptive platform trials utilizing non-concurrent controls
10.1002/bimj.70059 · 2025 · External reference
Rheumatoid Arthritis Prevention: catalysing PlatfORm Trial delivery (RAPPORT)
2025 · External reference
Clinical predictors of remission in systemic juvenile idiopathic arthritis/Still’s disease: insight from real-life data and the potential relevance of the “window of opportunity
2025 · External reference
Transcriptional profiling of tofacitinib treatment in juvenile idiopathic arthritis: implications for treatment response prediction
10.1002/acr.25459 · 2025 · External reference
Transcriptional profiles of JIA patient blood with subsequent poor response to methotrexate
10.1093/rheumatology/kex206 · 2017 · External reference
Dynamic treatment regimes
10.1146/annurev-statistics-022513-115553 · 2014 · External reference
Comparing dose levels to placebo using a continuous outcome in a small n, sequential, multiple assignment, randomized trial (snSMART)
10.1080/19466315.2022.2118162 · 2023 · External reference
Multisystem inflammatory syndrome therapies in children (MISTIC): a randomized trial
10.1016/j.conctc.2023.101060 · 2023 · External reference
Addressing sequential and concurrent treatment regimens in a small n sequential, multiple assignment, randomized trial (snSMART) in the MISTIC study
10.1080/10543406.2023.2292206 · 2025 · External reference
Treating juvenile idiopathic arthritis to target: recommendations of an international task force
10.1136/annrheumdis-2018-213030 · 2018 · External reference
Recent innovations in adaptive trial designs: a review of design opportunities in translational research
2023 · External reference
Adaptive platform trials: the impact of common controls on type one error and power
10.1080/10543406.2023.2275765 · 2024 · External reference
Evaluating the efficiency of platform trials relative to stand-alone trials: a simulation study for the evaluation of new antibacterials
10.1093/jac/dkag263 · 2026 · External reference
Economic evaluation of cost and time required for a platform trial vs conventional trials
10.1001/jamanetworkopen.2022.21140 · 2022 · External reference
Economic evaluation of cost and time required for a platform trial vs conventional trials
10.1001/jamanetworkopen.2022.21140 · ExternalCitation · doi-reference
Transcriptional profiling of tofacitinib treatment in juvenile idiopathic arthritis: implications for treatment response prediction
10.1002/acr.25459 · ExternalCitation · doi-reference
25 years of biologics for the treatment of pediatric rheumatic disease: advances in prognosis and ongoing challenges
10.1002/acr.25482 · ExternalCitation · doi-reference
Is it time to move to active comparator trials in juvenile idiopathic arthritis?: a review of current study designs
10.1002/art.27670 · ExternalCitation · doi-reference
Categories, components, and techniques in a modular construction of basket trials for application and further research
10.1002/bimj.202000314 · ExternalCitation · doi-reference
Statistical modeling to adjust for time trends in adaptive platform trials utilizing non-concurrent controls
10.1002/bimj.70059 · ExternalCitation · doi-reference
Clinical trial design in juvenile idiopathic arthritis
10.1007/s40272-017-0244-2 · ExternalCitation · doi-reference
Design and rationale of RECOVER-AUTONOMIC: a randomized platform trial evaluating interventions for Long COVID postural orthostatic tachycardia syndrome
10.1016/j.ahj.2026.107384 · ExternalCitation · doi-reference
Comparison of multi-arm multi-stage design and adaptive randomization in platform clinical trials
10.1016/j.cct.2017.01.003 · ExternalCitation · doi-reference
Oral ritlecitinib and brepocitinib for moderate-to-severe ulcerative colitis: results from a randomized, phase 2b study
10.1016/j.cgh.2022.12.029 · ExternalCitation · doi-reference
The future of clinical trials in inflammatory bowel disease
10.1016/j.cgh.2024.06.036 · ExternalCitation · doi-reference
Master protocol trials in oncology: review and new trial designs
10.1016/j.conctc.2018.08.009 · ExternalCitation · doi-reference
Multisystem inflammatory syndrome therapies in children (MISTIC): a randomized trial
10.1016/j.conctc.2023.101060 · ExternalCitation · doi-reference
Current state-of-the-art and gaps in platform trials: 10 things you should know, insights from EU-PEARL
10.1016/j.eclinm.2023.102384 · ExternalCitation · doi-reference
The universal effects of low-dose interleukin-2 across 13 autoimmune diseases in a basket clinical trial
10.1016/j.jaut.2024.103172 · ExternalCitation · doi-reference
Systemic juvenile idiopathic arthritis and adult-onset Still’s disease are the same disease and should now be designated by the same unique name, Still’s disease
10.1016/j.jbspin.2025.105872 · ExternalCitation · doi-reference
The challenges of new biologic therapies in pediatrics: an update on the limited therapeutic options for juvenile psoriatic arthritis
10.1016/j.rdc.2025.05.002 · ExternalCitation · doi-reference
Baricitinib in juvenile idiopathic arthritis: an international, phase 3, randomised, double-blind, placebo-controlled, withdrawal, efficacy, and safety trial
10.1016/s0140-6736(23)00921-2 · ExternalCitation · doi-reference
Tocilizumab in patients with anti-TNF refractory juvenile idiopathic arthritis-associated uveitis (APTITUDE): a multicentre, single-arm, phase 2 trial
10.1016/s2665-9913(20)30008-4 · ExternalCitation · doi-reference
How can trial designs better serve the needs of children and young people with juvenile idiopathic arthritis?
10.1016/s2665-9913(24)00026-2 · ExternalCitation · doi-reference
Juvenile idiopathic arthritis
10.1038/s41572-021-00332-8 · ExternalCitation · doi-reference
Adaptive platform trials: definition, design, conduct and reporting considerations
10.1038/s41573-019-0034-3 · ExternalCitation · doi-reference
Developing an adaptive platform trial for evaluation of medical treatments for Crohn’s disease
10.1038/s41575-025-01052-0 · ExternalCitation · doi-reference
Transforming clinical trials in rheumatology: towards patient-centric precision medicine
10.1038/s41584-020-0491-4 · ExternalCitation · doi-reference
Emerging concepts and treatments in autoinflammatory interferonopathies and monogenic systemic lupus erythematosus
10.1038/s41584-024-01184-8 · ExternalCitation · doi-reference
CD19 CAR-T cells for treatment-refractory autoimmune diseases: the phase 1/2 CASTLE basket trial
10.1038/s41591-025-04185-6 · ExternalCitation · doi-reference
The master protocol concept
10.1053/j.seminoncol.2015.07.009 · ExternalCitation · doi-reference
Master protocols to study multiple therapies, multiple diseases, or both
10.1056/nejmra1510062 · ExternalCitation · doi-reference
Adaptive platform trials: the impact of common controls on type one error and power
10.1080/10543406.2023.2275765 · ExternalCitation · doi-reference
Addressing sequential and concurrent treatment regimens in a small n sequential, multiple assignment, randomized trial (snSMART) in the MISTIC study
10.1080/10543406.2023.2292206 · ExternalCitation · doi-reference
Type I error considerations in master protocols with common control in oncology trials: report of an American Statistical Association Biopharmaceutical Section Open Forum Discussion
10.1080/19466315.2021.1906743 · ExternalCitation · doi-reference
Comparing dose levels to placebo using a continuous outcome in a small n, sequential, multiple assignment, randomized trial (snSMART)
10.1080/19466315.2022.2118162 · ExternalCitation · doi-reference
Evaluating the efficiency of platform trials relative to stand-alone trials: a simulation study for the evaluation of new antibacterials
10.1093/jac/dkag263 · ExternalCitation · doi-reference
Long-term efficacy and safety of canakinumab in patients with mevalonate kinase deficiency: results from the randomised phase 3 CLUSTER trial
10.1093/rheumatology/keab696 · ExternalCitation · doi-reference
ANA-associated arthritis: clinical and biomarker characterization of a population for basket trials
10.1093/rheumatology/keae269 · ExternalCitation · doi-reference
Elicitation of expert prior opinion to design the BARJDM trial in juvenile dermatomyositis
10.1093/rheumatology/keae392 · ExternalCitation · doi-reference
Transcriptional profiles of JIA patient blood with subsequent poor response to methotrexate
10.1093/rheumatology/kex206 · ExternalCitation · doi-reference
Synovial tissue atlas in juvenile idiopathic arthritis reveals pathogenic niches associated with disease severity
10.1126/scitranslmed.adt6050 · ExternalCitation · doi-reference
Treating juvenile idiopathic arthritis to target: recommendations of an international task force
10.1136/annrheumdis-2018-213030 · ExternalCitation · doi-reference
Multicentre, 26-week, open-label phase 2 trial of the JAK inhibitor filgotinib in Behçet’s disease, idiopathic inflammatory myopathies and IgG4-related disease: DRIMID study protocol
10.1136/bmjopen-2024-089827 · ExternalCitation · doi-reference
Dynamic treatment regimes
10.1146/annurev-statistics-022513-115553 · ExternalCitation · doi-reference
Application of Bayesian statistics to support approval of intravenous belimumab in children with systemic lupus erythematosus in the United States
10.1177/09612033251349930 · ExternalCitation · doi-reference
Response adaptive randomisation in clinical trials: current practice, gaps and future directions
10.1177/09622802251348183 · ExternalCitation · doi-reference
Adaptive seamless phase II/III designs: background, operational aspects, and examples
10.1177/216847900604000412 · ExternalCitation · doi-reference
Randomization in the age of platform trials: unexplored challenges and some potential solutions
10.1186/s12874-025-02693-0 · ExternalCitation · doi-reference
The 4th NextGen therapies of SJIA and MAS, part 4: it is time for IL-18 based trials in systemic juvenile idiopathic arthritis?
10.1186/s12969-023-00867-y · ExternalCitation · doi-reference
Bayesian trial of adalimumab versus secukinumab for children with juvenile idiopathic arthritis associated uveitis or chronic anterior uveitis
10.1186/s12969-025-01107-1 · ExternalCitation · doi-reference
RECOVER-NEURO: study protocol for a multi-center, multi-arm, phase 2, randomized, active comparator trial evaluating three interventions for cognitive dysfunction in post-acute sequelae of SARS-CoV-2 infection (PASC)
10.1186/s13063-024-08156-z · ExternalCitation · doi-reference
Use of Bayesian techniques in clinical trials for rheumatoid arthritis and systemic sclerosis: a scoping review
10.1186/s41927-025-00563-2 · ExternalCitation · doi-reference
Basket trials: review of current practice and innovations for future trials
10.1200/jco.21.02285 · ExternalCitation · doi-reference
Novel clinical trial design and analytic methods to tackle challenges in therapeutic development in rare diseases
10.21037/atm-21-5496 · ExternalCitation · doi-reference
Design and analysis of umbrella trials: where do we stand?
10.3389/fmed.2022.1037439 · ExternalCitation · doi-reference
Bayesian methods for information borrowing in basket trials: an overview
10.3390/cancers16020251 · ExternalCitation · doi-reference
Platform trials: key features, when to use them and methodological challenges
10.5694/mja2.52711 · ExternalCitation · doi-reference
