Multi-centre national audit of juvenile localised scleroderma: describing current UK practice in disease assessment and management

Lythgoe, Hanna; Almeida, Beverley; Bennett, Joshua; Bhat, Chandrika; Bilkhu, Amarpal; Brennan, Mary; Deepak, Samundeeswari; Dawson, Pamela; Eleftheriou, Despina; Harrison, Kathryn; Hawley, Daniel; Heaf, Eleanor; Leone, Valentina; Long, E; Maltby, Sarah; McErlane, Flora; Rafiq, Nadia; Ramanan, Athimalaipet V; Riley, Phil; Rangaraj, Satyapal; Varnier, Giulia; Wilkinson, Nick; Pain, Clare

doi:10.1186/s12969-018-0295-0

Cited by 22 publications

(10 citation statements)

References 19 publications

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“…Although the evidence is weak, some authors consider mycophenolate mofetil (MMF) to be the second-line agent after MTX-based regimens. 33 In a retrospective study, MMF produced improvement in clinical score and thermal imaging findings when used in paediatric morphoea not responding to MTX and corticosteroids. 34 The CARRA guidelines did not recommend MMF in paediatric morphoea owing to lack of evidence despite small reports of some efficacy, and left the decision to the treating physician's discretion.…”

Section: Mycophenolate Mofetilmentioning

confidence: 98%

Paediatric morphoea: a holistic review. Part 2: diagnosis, measures of disease activity, management and natural history

Kaushik

Mahajan

et al. 2020

Clin Exp Dermatol

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Paediatric morphoea is a debilitating fibrosing disorder of uncertain aetiology, affecting the skin and subcutaneous tissues. Defining optimum management strategies in paediatric morphoea remains an ongoing challenge, owing to the varied presentations and a relative paucity of paediatric-specific studies. We performed a literature search on PubMed, MEDLINE and Google Scholar, using keywords such as 'pediatric morphea', 'juvenile localised scleroderma' and 'juvenile systemic sclerosis'. Relevant studies, including randomized trials, reviews of standard current guidelines and original research articles, were selected and results analysed before summarizing them. In Part 1 of this review, we described the epidemiology, aetiopathogenesis and clinical classification; in this part, we discuss the diagnosis, markers of disease activity, management and natural history in paediatric morphoea. ImagingInfrared thermography can be used to detect activity in lesions of morphoea, with active lesions showing raised temperature. One study reported thermography to have 92% sensitivity and 68% specificity in identifying active

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Section: Mycophenolate Mofetilmentioning

confidence: 98%

Paediatric morphoea: a holistic review. Part 2: diagnosis, measures of disease activity, management and natural history

Kaushik

Mahajan

et al. 2020

Clin Exp Dermatol

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“…[3], всем пациентам с выраженными косметическими и/или функциональными нарушениями может быть рекомендована терапия метотрексатом из расчета 15 мг/м 2 подкожно или перорально. В 2/3 случаев терапия метотрексатом позволяет достичь неактивной фазы болезни [33]. Результаты длительного (в среднем 30 лет) наблюдения за пациентами с локализованной склеродермией демонстрируют, что при ювенильном дебюте болезни признаки активного заболевания во взрослом возрасте имеют около 90% пациентов, необратимые осложнения -более половины [18,34].…”

Section: метотрексатunclassified

“…Согласно результатам британского национального аудита по оценке и лечению локализованной склеродермии, 95,5% из 149 пациентов получали метотрексат как первую линию терапии. Каждый третий пациент (34%) получал два болезньмодифицирующих лекарственных препарата или более, т. е. не достиг ремиссии на фоне лечения метотрексатом, что потребовало усиления терапии [33].…”

Section: терапия при неэффективности метотрексатаunclassified

“…При неэффективности метотрексата, при рецидивировании болезни после клинической ремиссии (про-/ 2020 / ТОМ 19 / № 3 CURRENT PEDIATRICS / 2020 / V. 19 / № 3 грессирование кожных очагов, тяжелые внекожные проявления) или в случае непереносимости метотрексата может быть использован микофенолата мофетил (MMФ) в дозе 500-1000 мг/м 2 [3] как в режиме монотерапии, так и в комбинации с метотрексатом [40]. MMФ является наиболее часто используемым препаратом второй линии терапии (у 89,5% больных) и обладает такой же эффективностью в отношении кожного статуса, как и монотерапия метотрексатом, но оказывает меньшее влияние на артрит по сравнению с метотрексатом [33,41].…”

Section: терапия при неэффективности метотрексатаunclassified

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Juvenile Localized Scleroderma. Questions of Treatment

Raupov

Костик

2020

Vopr. sovr. pediatr.

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Juvenile localized scleroderma (JLS) is a group of childhood diseases with the main symptom — skin and subcutaneous structures lesions, without any organ involvement. There is active (inflammatory) and fibrotic phase in development of JLS. The JLS treatment during active phase (when skin lesions are reversible) is the most effective. The management is determined by the area and depth of skin lesions, appearance and spread of new lesions, presence of extracutaneous signs of the disease. Topical and systemic immunosuppressants are the basic therapy for JLS. The use of antibiotics is not suggested. Clinical scores (LoSCAT), ultrasound, thermography and magnetic resonance imaging are recommended to estimate the treatment efficacy.

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“…A UK wide study showed that children discontinuing MTX for JLS, reported medication side-effects as the primary reason 9 .…”

Section: Introductionmentioning

confidence: 99%

Prior elicitation of the efficacy and tolerability of Methotrexate and Mycophenolate Mofetil in Juvenile Localised Scleroderma

et al. 2021

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Background Evidence is lacking for safe and effective treatments for juvenile localised scleroderma (JLS). Methotrexate (MTX) is commonly used first line and mycophenolate mofetil (MMF) second line, despite a limited evidence base. A head to head trial of these two medications would provide data on relative efficacy and tolerability. However, a frequentist approach is difficult to deliver in JLS, because of the numbers needed to sufficiently power a trial. A Bayesian approach could be considered. Methods An international consensus meeting was convened including an elicitation exercise where opinion was sought on the relative efficacy and tolerability of MTX compared to MMF to produce prior distributions for a future Bayesian trial. Secondary aims were to achieve consensus agreement on critical aspects of a future trial. Results An international group of 12 clinical experts participated. Opinion suggested superior efficacy and tolerability of MMF compared to MTX; where most likely value of efficacy of MMF was 0.70 (95% confidence interval (CI) 0.34-0.90) and of MTX was 0.68 (95% CI 0.41-0.8). The most likely value of tolerability of MMF was 0.77 (95% CI 0.3-0.94) and of MTX was 0.62 (95% CI 0.32-0.84). The wider CI for MMF highlights that experts were less sure about relative efficacy and tolerability of MMF compared to MTX. Despite using a Bayesian approach, power calculations still produced a total sample size of 240 participants, reflecting the uncertainty amongst experts about the performance of MMF. Conclusions Key factors have been defined regarding the design of a future Bayesian approach clinical trial including elicitation of prior opinion of the efficacy and tolerability of MTX and MMF in JLS. Combining further efficacy data on MTX and MMF with prior opinion could potentially reduce the pre-trial uncertainty so that, when combined with smaller trial sample sizes a compelling evidence base is available.

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Multi-centre national audit of juvenile localised scleroderma: describing current UK practice in disease assessment and management

Cited by 22 publications

References 19 publications

Paediatric morphoea: a holistic review. Part 2: diagnosis, measures of disease activity, management and natural history

Paediatric morphoea: a holistic review. Part 2: diagnosis, measures of disease activity, management and natural history

Juvenile Localized Scleroderma. Questions of Treatment

Prior elicitation of the efficacy and tolerability of Methotrexate and Mycophenolate Mofetil in Juvenile Localised Scleroderma

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