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AB0958 Proposal for a juvenile systemic sclerosis response index (JSSCRI): result of the consensus meeting in hamburg. germany 11th of december 2016
  1. I Foeldvari1,
  2. DF Furst2,
  3. J Anton3,
  4. E Baildem4,
  5. M Blakley5,
  6. T Constantin6,
  7. P Costa Reis7,
  8. M Curran8,
  9. M Cutolo9,
  10. C Denton10,
  11. K Fligelstone11,
  12. B Hinrichs12,
  13. F Ingegnoli13,
  14. A Kienast1,
  15. D Němcova14,
  16. C Pain4,
  17. C Pilkington15,
  18. V Smith16,
  19. D Khanna17
  1. 1Hamburg Center for Pediatric and Adolescent Rheumatology, Am Schön Klinik Eilbek, Hamburg, Germany
  2. 2UCLA, Los Angeles, United States
  3. 3Sant Joan de Déu Hospital, Barcelona, Spain
  4. 4Alder Hey Children's Foundation NHS Trust, Liverpool, United Kingdom
  5. 5Indiana University, Indianapolis, United States
  6. 6Semmelweis University, Budapest, Hungary
  7. 7Hospital de Santa Maria, Lisbon, Portugal
  8. 8Northwestern University, Chicago, United States
  9. 9University of Genova, Genova, Italy
  10. 10Royal Free Hospital
  11. 11FESCA, London, United Kingdom
  12. 12Practice Buchholz, AMS, Hamburg, Germany
  13. 13University of Milan, Milano, Italy
  14. 14Department of Pediatrics and Adolescent Medicine Charles University, Prague, Czech Republic
  15. 15GOSH, London, United Kingdom
  16. 16GU(H), Ghent, Belgium
  17. 17Univ Michigan, Michigan, United States


Background Juvenile Systemic Sclerosis (jSSc) is an orphan disease. There is increasing interest in testing novel therapies in the management of fibrotic diseases. Therefore, it is very important to develop a Response Index for jSSc (JSScRI) to distinguish effective therapies from placebo. In 2014 at the 1st JSScRI Consensus Meeting in Hamburg, following two rounds of a Delphi process, a proposal for domains and items for a JSScRI were made. In 2016, the 2nd JSScRI Consensus Meeting was held in Hamburg, Germany.

Objectives To conduct a Nominal Group Technique (NGT) and select potential core data set items that could change as outcome measures) that will be incorporated in the development of a JSScRI.

Methods Before the 2nd JSScRI Consensus Meeting, the items from the 1st JSScRI Consensus Meeting (2014) were scored via Email, in a Delphi by the participants of the current meeting. Participants included 14 experts in adult and juvenile SSc and a patient partner. During the subsequent face to face NGT meeting, moderated by DEF, and the items were scored anonymously by the participants after a nominal group discussion. The domains and items were scored regarding their importance for 1 year clinical trial from 1 (not relevant at all) to 9 (most relevant). A priori, it was agreed by the participants that the goal of the NGT was to exclude items that: 1. Are not feasible and 2. do not represent a changeable outcome measure even though they may represent the impact of disease on quality of life, vocational or recreational activity. Items with a median score of <4 in which greater than 1/3 of participants scored 1–3 [despite the item having a median score >3] were excluded.

Results Seventy-one items in 13 domains were scored. Six items were not scored as they were felt to not represent an outcome measure or were non-feasible and six items received a median score less then 4.

Table 1 provides an example of the musculoskeletal domain with the median scores for different outcome measures.

Table 1.

Assessment of the Activity of the Musculoskeletal domain

Conclusions In a rigorous, NGT consensus meeting, some item reduction for the JSScRI was achieved. Items will be tested in a prospective way in the patients of the inception cohort of juvenile SSc (

Disclosure of Interest None declared

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