gms | German Medical Science

47. Kongress der Deutschen Gesellschaft für Rheumatologie (DGRh), 33. Jahrestagung der Deutschen Gesellschaft für Orthopädische Rheumatologie (DGORh), 29. Jahrestagung der Gesellschaft für Kinder- und Jugendrheumatologie (GKJR)

04.09. - 07.09.2019, Dresden

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Proposal of outcome measures to be used on a 12-month open label drug trial in Juvenile Systemic Sclerosis. Results of the 3rd Consensus Meeting in Hamburg December 2018

Meeting Abstract

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  • Ivan Foeldvari - Hamburger Zentrum für Kinder- und Jugendrheumatologie, Hamburg
  • Kathryn Torok - University Childrens Hospital, Pittsburgh, United States of America
  • Lusine Ambartsumyan - Seattle Children's, Seattle, USA
  • Jordi Anton - University Children’s Hospital, Pediatric Rheumatology, Barcelona, Spain
  • Christian Beyer - Schwerpunktpraxis für Kinder- und Jugendkardiologie, Hamburg
  • Michael Blakley - Indianapolis University, Indianapolis, USA
  • Tamas Constantin - Semmelweis University, Faculty of Medicine, Budapest, Hungary
  • Patrícia Costa Reis - Hospital de Santa Maria, Lisbon, Portugal
  • Megan Curran - University of Colorado, School of Medicine, Chicago, USA
  • Maurizio Cutolo - Universita degli Studi di Genova, Genova, Italy
  • Francesco del Gado - Faculty of Medicine and Health, Leeds, UK
  • Christopher P. Denton - Royal Free and University College, Centre for Rheumatology, London, United Kingdom
  • Kim Fligelstone - FESCA, London, UK
  • Bernd Hinrichs - Practice Buchholz, AMS, Hamburg
  • Antonia Höger - Hamburger Zentrum für Kinder- und Jugendrheumatologie, Kinder- und Jugendrheumatologie, Hamburg
  • Francesca Ingegnoli - University of Milan, Milan, Italy
  • Ozgur Kasapcopur - Department of Pediatric Nephrology, Cerrahpasa Faculty of Medicine, Istanbul University, Istanbul
  • Suzanne Li - Hackensack University Medical Center, Hackensack, United States of America
  • Dana Nemcova - University Childrens Hospital, Pediatric Rheumatology, Prague, Czech
  • Catherine Orteu - Royal Free London, London, UK
  • Clarissa Pilkington - Great Ormond Street Hospital, London, Vereinigtes Königreich
  • Vanessa Smith - Gent University Hospital, Rheumatology, Gent, Belgium
  • Ann Stevens - Seattle Children's, Seattle, USA
  • Brandi Stevens - Indiana University School of Medicine, Indianapolis, USA
  • Allison Zhen - Chinese Organization for Scleroderma, Chengdu, China
  • D. Khanna - University of Michigan, Ann Arbor, United States of America
  • D. Furst - University of California, Los Angeles, United States of America

Deutsche Gesellschaft für Rheumatologie. Deutsche Gesellschaft für Orthopädische Rheumatologie. Gesellschaft für Kinder- und Jugendrheumatologie. 47. Kongress der Deutschen Gesellschaft für Rheumatologie (DGRh), 33. Jahrestagung der Deutschen Gesellschaft für Orthopädische Rheumatologie (DGORh), 29. Jahrestagung der Gesellschaft für Kinder- und Jugendrheumatologie (GKJR). Dresden, 04.-07.09.2019. Düsseldorf: German Medical Science GMS Publishing House; 2019. DocKR.09

doi: 10.3205/19dgrh170, urn:nbn:de:0183-19dgrh1701

Published: October 8, 2019

© 2019 Foeldvari et al.
This is an Open Access article distributed under the terms of the Creative Commons Attribution 4.0 License. See license information at http://creativecommons.org/licenses/by/4.0/.

Outline

Text

Background: Juvenile systemic sclerosis (jSSc) is an orphan disease, associated with high morbidity and mortality. New treatment strategies are much needed. To develop an open label drug trial for the treatment of jSSc patients, it is necessary to clearly define how to evaluate outcomes in this disease, which are currently not existing. A group of experts in jSSc has met annually and worked to develop an index to evaluate outcomes in this disease.

Methods: In the consensus meeting 26 jSSc international experts with various specialties participated (22 voted). In a nominal group technic, moderated by DEF, was used to develop the outcome measures. Agreement was defined if 80% or more of the participants approved an item.

Results: Domains and items suggested in the 2017 consensus meeting were reconsidered and selected or rejected during the 2018 meeting, as were additional domains/items (Table 1).

Domain

  • Item - voted for: yes/nominator

Global Disease Activity

  • Physician global of disease activity - 22/22
  • Change in HAQ/CHAQ- DI - 22/22
  • Scleroderma HAQ - 22/22

Skin

  • Change in Modified Rodnan Skin Score - 22/22

Raynaud Phenomenon

  • Scleroderma HAQ question regarding Raynaud - 22/22

Digital ulcerations

  • DUCAS Score - 22/22

Musculoskeletal System

  • Juvenile Idiopathic Arthritis Definition of active joint - 22/22

Cardiac Involvement

  • Left ventricular ejection fraction - 22/22
  • Development of clinically significant arrhythmia as a sign of non-response - 22/22

Pulmonary Involvement

  • FVC - 22/22
  • age- defined DLCO in all trials - 20/22

Renal

  • New occurrence of renal crisis - 22/22

Gastrointestinal Involvement

  • Body Mass Index - 22/22
  • Scleroderma HAQ- Gastrointestinal section - 22/22

Global health/Health related Quality of Life (QOL)

  • QOL instrument should be used - 18/18

Conclusion: We reached consensus on domains and items which should be assessed in an open label 1 year clinical jSSc trial. We also listed research items which should be assessed but should not currently be included as an outcome in such a trial.