Development of consensus treatment plans for juvenile localized scleroderma: A roadmap toward comparative effectiveness studies in juvenile localized scleroderma

Abstract

Objective To develop standardized treatment plans, clinical assessments, and response criteria for active, moderate to high severity juvenile localized scleroderma (jLS). Background jLS is a chronic inflammatory skin disorder associated with substantial morbidity and disability. Although a wide range of therapeutic strategies have been reported in the literature, a lack of agreement on treatment specifics and accepted methods for clinical assessment of have made it difficult to compare approaches and identify optimal therapy. Methods A core group of pediatric rheumatologists, dermatologists and a lay advisor was engaged by the Childhood Arthritis and Rheumatology Research Alliance (CARRA) to develop standardized treatment plans and assessment parameters for jLS using consensus methods/nominal group techniques. Recommendations were validated in two face-to-face conferences with a larger group of practitioners with expertise in jLS and with the full membership of CARRA, which encompasses the majority of pediatric rheumatologists in the U.S and Canada. Results Consensus was achieved on standardized treatment plans that reflect the prevailing treatment practices of CARRA members. Standardized clinical assessment methods and provisional treatment response criteria were also developed. Greater than 90% of pediatric rheumatologists responding to a survey (67% of CARRA membership) affirmed the final recommendations and agreed to utilize these consensus plans to treat patients with jLS. Conclusions Using consensus methodology, we have developed standardized treatment plans and assessment methods for jLS. The high level of support among pediatric rheumatologists will support future comparative effectiveness studies and enable the development of evidence-based guidelines for the treatment of jLS.

Localized scleroderma – classification and tools used for the evaluation of tissue damage and disease activity/severity

“…Pojawienie się nowych zmian lub powiększenie istniejących wykwitów w ciągu ostatniego miesiąca ocenia się na 3 pkt, a brak powyższych cech -0 pkt. Grupa Childhood Arthritis and Rheumatology Research Alliance (CARRA) stworzyła cludes features for which expert consensus has been reached regarding their significance, and level 2 comprises parameters for which partial expert consensus has been achieved (Table 6) [46]. For a patient to be considered to have active disease, a single level 1 parameter or two level 2 parameters had to be identified [46].…”

“…Grupa Childhood Arthritis and Rheumatology Research Alliance (CARRA) stworzyła cludes features for which expert consensus has been reached regarding their significance, and level 2 comprises parameters for which partial expert consensus has been achieved (Table 6) [46]. For a patient to be considered to have active disease, a single level 1 parameter or two level 2 parameters had to be identified [46]. Subjective physician assessment of disease activity (PGA-A) should be based on clinical variables classified into three groups depending on the consensus agreement (Table 7) [47].…”

“…Kategoria 1 zawiera cechy, które otrzymały zgodne oceny ekspertów w zakresie istotności, a kategoria 2 cechy, co do których eksperci nie są w pełni zgodni (tab. 6) [46]. Stwierdzenie aktywnego procesu chorobowego opierało się na wykazaniu obecności jednego parametru z kategorii 1 bądź dwóch parametrów z kategorii 2 [46].…”

“…6) [46]. Stwierdzenie aktywnego procesu chorobowego opierało się na wykazaniu obecności jednego parametru z kategorii 1 bądź dwóch parametrów z kategorii 2 [46]. Subiektywna ocena lekarska aktywności choroby (PGA-A) powinna bazować na cechach klinicznych, które zostały zakwalifikowane do trzech grup, w zależności od zgodności ocen (tab.…”

Вопросы Классификации, Клиническая Картина И Базисная Терапия Ювенильной Склеродермии

“…Нет единства во мнении о дозе ГК, разные специалисты используют до-зы от 0,5 до 1 мг/кг в сутки, чаще на 8-12 нед, с полной отменой через 1-6-12 мес. Имеется описание назначе-ния ГК в стартовой дозе 2 мг/кг в сутки на 2 нед, с после-дующим медленным снижением и отменой к 12 мес [28].…”