In 2012, a European initiative called Single Hub and Access point for paediatric Rheumatology in Europe (SHARE) was launched to optimise and disseminate diagnostic and management regimens in Europe for children and young adults with rheumatic diseases. Juvenile localised scleroderma (JLS) is a rare disease within the group of paediatric rheumatic diseases (PRD) and can lead to significant morbidity. Evidence-based guidelines are sparse and management is mostly based on physicians’ experience. This study aims to provide recommendations for assessment and treatment of JLS. Recommendations were developed by an evidence-informed consensus process using the European League Against Rheumatism standard operating procedures. A committee was formed, mainly from Europe, and consisted of 15 experienced paediatric rheumatologists and two young fellows. Recommendations derived from a validated systematic literature review were evaluated by an online survey and subsequently discussed at two consensus meetings using a nominal group technique. Recommendations were accepted if ≥80% agreement was reached. In total, 1 overarching principle, 10 recommendations on assessment and 6 recommendations on therapy were accepted with ≥80% agreement among experts. Topics covered include assessment of skin and extracutaneous involvement and suggested treatment pathways. The SHARE initiative aims to identify best practices for treatment of patients suffering from PRDs. Within this remit, recommendations for the assessment and treatment of JLS have been formulated by an evidence-informed consensus process to produce a standard of care for patients with JLS throughout Europe.
Objectives To investigate safety and efficacy of MMF in patients with severe or MTX-refractory juvenile localized scleroderma. Methods Consecutive juvenile localized scleroderma patients undergoing systemic treatment were included in a retrospective longitudinal study. Patients treated with MMF because they were refractory or intolerant to MTX (MMF-group) were compared with responders to MTX (MTX-group). Disease activity was assessed by Localized Scleroderma Cutaneous Assessment Tool and thermography. Disease course was established on the number of relapses and treatment changes. Relapse-free survival was examined by Kaplan–Meier analysis. Results MMF and MTX groups included 22 and 47 patients, respectively. No significant difference in demographics, follow-up duration and treatment before diagnosis was observed between groups. The most represented clinical subtypes in the MMF-group were pansclerotic morphea and mixed subtype (P = 0.008 and P = 0.029, respectively), and linear scleroderma of the face in the MTX-group (P = 0.048). MMF was started because of MTX resistance (18 patients), relapse during MTX tapering/withdrawal (3 patients) and anaphylaxis to MTX (1 patient). After mean 9.4 years of follow-up, 90.9% of patients on MMF and 100% of those on MTX had inactive disease. No significant difference in relapse-free survival between the groups was found (P = 0.066, log-rank test), although MMF likely induced more persistent remission. MMF was well tolerated and combination of MMF and MTX did not increase its efficacy. Conclusion The present study adds strong evidence on the efficacy and tolerance of MMF in severe and/or MTX-refractory juvenile localized scleroderma. Further controlled studies are needed to prove its efficacy as first line treatment.
Juvenile systemic sclerosis (JSSc) is a rare disease of childhood and currently no international consensus exists with regard to its assessment and treatment. This SHARE (Single Hub and Access point for paediatric Rheumatology in Europe) initiative, based on expert opinion informed by the best available evidence, provides recommendations for the assessment and treatment of patients with JSSc with a view to improving their outcome. Experts focused attention not only on the skin assessment but also on the early signs of internal organ involvement whose proper treatment can significantly affect the long-term outcome. A score for disease severity is proposed in order to perform a structured assessment of outcome over time but a validation in a wider patient population is recommended. Finally, a stepwise treatment approach is proposed in order to unify the standard of care throughout Europe with the aim to reduce morbidity and mortality in this disease.
Background Infrared Thermography (IRT) has been used for over 30 years in the assessment of Raynaud Phenomenon (RP) and other peripheral microvascular dysfunctions in adults but, to date, very little experience is available on its use in children for this purpose. The first aim of the study was to assess reproducibility of thermographic examination after cold exposure by comparing inter-observer agreement in thermal imaging interpretation. The secondary aim was to evaluate whether IRT is reliable to diagnose and differentiate peripheral circulation disturbances in children. Methods Children with clinical diagnosis of primary Raynaud’s phenomenon (PRP), secondary RP (SRP), acrocyanosis (AC) and age-matched controls underwent sequential measurements of skin temperature at distal interphalangeal (DIP) and metacarpophalangeal (MCP) joints with IRT at baseline and for 10 min after cold challenge test. Intraclass correlation coefficient (ICC) was calculated for inter-rater reliability in IRT interpretation, then temperature variations at MCP and DIP joints and the distal-dorsal difference (DDD) were analysed. Results Fourteen PRP, 16 SRP, 14 AC and 15 controls entered the study. ICC showed excellent agreement (> 0.93) for DIPs and MCPs in 192 measures for each subject. Patients with PRP, SRP and acrocyanosis showed significantly slower recovery at MCPs (p < 0.05) and at DIPs (p < 0.001) than controls. At baseline, higher temperature at DIPs and lower at MCPs was observed in PRP compared with SRP with significantly lower DDD (p < 0.001). Differently from AC, both PRP and SRP showed gain of temperature at DIPs and less at MCPs after cold challenge. PRP but not SRP patients returned to DIPs basal temperature by the end of re-warming time. Analysis of DDD confirmed that controls and PRP, SRP and AC patients significantly differed in fingers recovery pattern (p < 0.05). Conclusion IRT appears reliable and reproducible in identifying children with peripheral microvascular disturbances. Our results show that IRT examination pointed out that PRP, SRP and AC patients present significant differences in basal extremities temperature and in re-warming pattern after cold challenge therefore IRT can be suggested as an objective tool for diagnosis and monitoring of disease.
BackgroundRaynaud's phenomenon (RP) is a disorder characterized by paroxystic episodes of vasospasm of digital arteries following cold exposure or emotional distress. Infrared thermography detects temperature distribution of the skin and gives an indirect measure of microvascular flow. To date there is no evidence of the application of this method in pediatric age for evaluation of RP and differentiation from other conditions, such as acrocyanosis.Objectives1. To determine the reliability and reproducibility of thermography by comparing inter-observer concordance in thermal imaging interpretation; 2. To evaluate the diagnostic value of thermographic detection of extremities re-warming following cold challenge test by comparison between healthy controls and children with microvascular alterations (primary RP, secondary RP and acrocyanosis).Methods41 children were included in the study: 20 with RP (including primary and secondary RP), 8 with acrocyanosis and 13 healthy controls. All assessed subjects underwent the same cold challenge protocol under standardised conditions: baseline images of both hands were taken following acclimatisation at 23 °C for 15 min and then, patients submerged their gloved hands in a water bath at 15 °C (±1 °C) for 1 minute. Repeated thermal images were obtained in the 10 minutes following cold challenge (at 1 minute interval each). The temperature in the dorsal aspect of metacarpophalangeal joint and of the distal interphalangeal joint of each finger excluding the thumb was measured for 12 times respectively (T basal, T0-10). Two clinicians reviewed the thermal images and measured the temperature values independently. Interclass correlation coefficient (ICC) was calculated in each sites (metacarpophalangeal and interphalangeal joints) of each hand for all times in order to determine the inter-observer agreement. The modality of the rewarming in each group was evaluated considering the mean value between the two different operators' measurements and calculating the average temperature among different time intervals (ΔT). The average temperature variation (Δ°C) was analysed as a function of the site (right and left hand; metacarpophalangeal and interphalangeal joint). We accomplished our analysis considering two different time intervals: Tn-Tn-1 and Tn-T pre (where n designates the minutes of observation and pre the baseline). The differences in re-warming curves between RP and acrocyanosis were performed by calculating the mean longitudinal thermal gradient (Δ MCF-IFD) in each finger and time of observation.ResultsThe ICC showed a full inter-observer agreement between the two clinicians (range 0.93-0.96). The temperature variation in the 10 minutes following cold challenge compared with the baseline temperature showed statistically significant differences between children with microvascular alterations (RP and acrocyanosis) and healthy controls (p<0.001).The analysis of the longitudinal thermal gradient (Δ MCF-IFD) showed statistically significant differences in the comparison of patients ...
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