Enhancing translational research in paediatric rheumatology through standardization

Yeung, Rae S. M.; Albani, Salvatore; Feldman, Brian M.; Mellins, Elizabeth; Prakken, Berent J.; Wedderburn, Lucy R.

doi:10.1038/nrrheum.2016.156

Cited by 15 publications

(11 citation statements)

References 64 publications

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“…To make matters worse, there were no internationally-accepted standard protocols for the collection, shipping, processing, and storage of pediatric rheumatology biological samples until recently. This short-coming has been addressed by the international consortium UCAN (Understanding Childhood Arthritis Network) [ 36 , 59 ]. The PRCSG interacts with UCAN in supporting translational research by pediatric rheumatology investigators that make use of samples and data from drug trials.…”

Section: Other Activities Of the Prcsgmentioning

confidence: 99%

Pediatric Rheumatology Collaborative Study Group – over four decades of pivotal clinical drug research in pediatric rheumatology

et al. 2018

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ImportanceSpecialized research networks are essential to achieve drug approvals for rare pediatric diseases. Such networks help realize the potential of global legislation enacted upon the recognition that most children are treated with drugs whose most beneficial dose and regimen have not been established in pediatric patients. The Pediatric Rheumatology Collaborative Study Group (PRCSG) is a North American clinical trials network that is specialized in the performance of clinical trials of new therapies for pediatric populations with rheumatic diseases. This review provides an overview of the strategies employed by this research network to achieve drug and biologic approvals for children with pediatric rheumatic diseases, particularly juvenile idiopathic arthritis.ObservationsClinical trial conduct in rare pediatric diseases has required global recruitment. Supported or led by the PRCSG, highly responsive, validated, composite measures have been established to assess drug efficacy. For pediatric orphan diseases with high disease burdens, specialized investigative sites and study designs are needed to complete adequately powered trials at the high standard necessary to enable drug labeling by regulatory agencies. Novel trial designs have been utilized for more efficient testing of innovative drug candidates. All these have been developed or co-developed by the PRCSG research network.Conclusions and relevanceSpecialized research networks in pediatric rheumatology, such as the PRCSG, have changed the landscape of available therapies and improved overall disease outcomes for children with pediatric rheumatic diseases.

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Section: Other Activities Of the Prcsgmentioning

confidence: 99%

Pediatric Rheumatology Collaborative Study Group – over four decades of pivotal clinical drug research in pediatric rheumatology

et al. 2018

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“…Another challenge is the standardization of definitions and data collection (19). Uniform approaches are necessary for robust collaborative research, particularly involving rare diseases.…”

Section: Necessary Factors For Creating a Successful International Comentioning

confidence: 99%

“…Collaborative research involving multiple centers and groups requires critical procedures to be coordinated to facilitate accurate comparisons of data. The use of standard operating procedures for the collection and handling of samples and data is a critical first step in ensuring high-quality translational research (19). Disseminating such information among researchers requires a flexible and secure data-sharing infrastructure (19).…”

Section: Necessary Factors For Creating a Successful International Comentioning

confidence: 99%

Importance of an International Registry for and Collaborative Research on Esophageal Atresia

et al. 2017

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Esophageal atresia (EA) is a rare congenital defect. Data on EA prevalence, management, and long-term outcome are lacking because the available data come from small retrospective series from tertiary referral centers. An international multicenter registry would provide strong epidemiological data from large population-based cohorts on EA prevalence and incidence, treatment, long-term morbidity, and prognosis and would thus provide accurate data for evaluation of the current guidelines for EA management. The future challenge of the new international network on EA, which was created in 2013, is to promote the creation of a collaborative database and further studies.

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“…Obesity was defined as BMI !95 percentile in conformity with Centers for Disease Control and Prevention (CDC) (3). Persistant purpura was defined as skin involvement persisting for !30 days.…”

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Thu0523 obesity Is Associated With Severe Renal Involvement, Persistent Purpura and Longer Joint Symptoms in Children With Henoch Shönlein Purpura

Dündar

Pektanc

Bayram

et al. 2019

Poster Presentations

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BackgroundOver the last 20 years, the prevalence of obesity is increased in the developed and developing countries. Adipose tissue has an effect on inflammatory processes and immune system besides metabolic and appetite regulating mechanisms. On this basis, it is now of major interest to clarify the relationship between obesity and autoimmune/inflammatory diseases (1,2).ObjectivesWe aimed to evaluate the role of obesity on the clinical course and response to treatment in patients with Henoch Shönlein Purpura (HSP).MethodsData charts of children with HSP followed in Dokuz Eylül University Childrens’ Hospital were reviewed retrospectively. Obesity was defined as BMI ≥95 percentile in conformity with Centers for Disease Control and Prevention (CDC) (3). Persistant purpura was defined as skin involvement persisting for ≥30 days. Mild nephropathy was defined by the presence of microscopical hematuria and/or nonnephrotic proteinuria, and severe nephropathy by nephrotic syndrome and/or acute nephritic syndrome and/or renal insufficiency (4). Patients were grouped as obese and non-obese depending on BMI. Two groups were compared for demographic, clinical and laboratory parameters.ResultsThere were 199 patients [M/F:104/95; presenting age 7.1 years (range 5.0-9.2); follow-up period 17.5 months (range 3-50)]. Obesity was present in 35 (17.5%) of children. These patients were determined to have significantly higher rate of persistent purpura (45.7% vs 20.7%), severe renal involvement (57.7% vs 30.8%), hypertension (28.6% vs 9.1%) and increased erythrocyte sedimentation rate (79.2% vs 55.9%). Obese patients also showed delayed improvement of skin (25 vs 14 days), joint (12.5 vs 10.0 days) and renal (280 vs 57 days) symptoms. While the rate of children requiring steroid treatment was not different between the two groups, obese children used steroids for significantly longer time (236 vs 40 days). Furthermore, need for immunosuppressive medications (azathioprine and cyclophosphamide) were higher in obese patients (40.0% vs 8.6%).ConclusionObese children with HSP seem to have higher ESR, hypertension and severe renal involvement; show delayed improvement of skin, joint and renal findings; need more immunosuppressive medications and longer period of immunosuppressive treatment. These findings may be associated with the effect of adipose tissue on inflammation.References[1] Versini M, Jeandel P-Y, Rosenthal E, Shoenfeld Y. Obesity in autoimmune diseases: not a passive bystander. Autoimmun Rev. 2014;13:981–1000.[2] Zhao Y, Liu Z, Bai X et all. Obesity increases the risk of renal involvement in children with Henoch–Schönlein purpura. Eur J Pediatr (2015) 174:1357–1363 [3] Styne DM, Arslanian SA, Connor EL et all. Pediatric Obesity—Assessment, Treatment, and Prevention: An Endocrine Society Clinical Practice Guideline. The Journal of Clinical Endocrinology & Metabolism, Volume 102, Issue 3, 1 March 2017, Pages 709–757[4] Trapani S, Micheli A, Grisolia F et all. Henoch Schonlein Purpura in childhood: epidemiological and clinical analy...

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Enhancing translational research in paediatric rheumatology through standardization

Cited by 15 publications

References 64 publications

Pediatric Rheumatology Collaborative Study Group – over four decades of pivotal clinical drug research in pediatric rheumatology

Pediatric Rheumatology Collaborative Study Group – over four decades of pivotal clinical drug research in pediatric rheumatology

Importance of an International Registry for and Collaborative Research on Esophageal Atresia

Thu0523 obesity Is Associated With Severe Renal Involvement, Persistent Purpura and Longer Joint Symptoms in Children With Henoch Shönlein Purpura

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