Proposed Response Parameters for Twelve-Month Drug Trial in Juvenile Systemic Sclerosis: Results of the Hamburg International Consensus Meetings

Abstract

Objective Juvenile systemic sclerosis (SSc) is an orphan disease, associated with high morbidity and mortality. New treatment strategies are much needed, but clearly defining appropriate outcomes is necessary if successful therapies are to be developed. Our objective here was to propose such outcomes.

Methods This proposal is the result of 4 face-to-face consensus meetings with a 27-member multidisciplinary team of pediatric rheumatologists, adult rheumatologists, dermatologists, pediatric cardiologists, pulmonologists, gastroenterologists, a statistician, and patients. Throughout the process, we reviewed the existing adult data in this field, the more limited pediatric literature for juvenile SSc outcomes, and data from 2 juvenile SSc patient cohorts to assist in making informed, data-driven decisions. The use of items for each domain as an outcome measure in an open label 12-month clinical trial of juvenile SSc was voted and agreed upon using a nominal group technique.

Results After voting, the domains agreed on were global disease activity, skin, Raynaud's phenomenon, digital ulcers, musculoskeletal, cardiac, pulmonary, renal, and gastrointestinal involvement, and quality of life. Fourteen outcome measures had 100% agreement, 1 item had 91% agreement, and 1 item had 86% agreement. The domains of biomarkers and growth/development were moved to the research agenda.

Conclusion We reached consensus on multiple domains and items that should be assessed in an open label, 12-month clinical juvenile SSc trial as well as a research agenda for future development.

Metadata

Item Type:	Article
Authors/Creators:	Foeldvari, I. https://orcid.org/0000-0003-0659-5298 Torok, K.S. Anton, J. https://orcid.org/0000-0002-8792-4219 Blakley, M Constantin, T Curran, M Cutolo, M. https://orcid.org/0000-0002-5396-0932 Denton, C. https://orcid.org/0000-0003-3975-8938 Fligelstone, K Ingegnoli, F Li, S.C. Němcová, D Orteu, C Pilkington, C. Smith, V. Stevens, A Klotsche, J. https://orcid.org/0000-0002-2954-5755 Khanna, D. https://orcid.org/0000-0003-1412-4453 Costa-Reis, P Del Gado, F. https://orcid.org/0000-0002-8528-2283 Hinrichs, B. Kasapcopur, O. https://orcid.org/0000-0002-1125-7720 Pain, C. Ruperto, N. https://orcid.org/0000-0001-8407-7782 Zheng, A Furst, D.E. https://orcid.org/0000-0001-7857-2373
Copyright, Publisher and Additional Information:	© 2023 American College of Rheumatology. This is the peer reviewed version of the following article: Foeldvari, I., Torok, K.S., Anton, J., Blakley, M., Constantin, T., Curran, M., Cutolo, M., Denton, C., Fligelstone, K., Ingegnoli, F., Li, S.C., Němcová, D., Orteu, C., Pilkington, C., Smith, V., Stevens, A., Klotsche, J., Khanna, D., Costa-Reis, P., Del Galdo, F., Hinrichs, B., Kasapcopur, O., Pain, C., Ruperto, N., Zheng, A. and Furst, D.E. (2023), Proposed Response Parameters for Twelve-Month Drug Trial in Juvenile Systemic Sclerosis: Results of the Hamburg International Consensus Meetings. Arthritis Care Res, which has been published in final form at https://doi.org/10.1002/acr.25171. This article may be used for non-commercial purposes in accordance with Wiley Terms and Conditions for Use of Self-Archived Versions. This article may not be enhanced, enriched or otherwise transformed into a derivative work, without express permission from Wiley or by statutory rights under applicable legislation. Copyright notices must not be removed, obscured or modified. The article must be linked to Wiley’s version of record on Wiley Online Library and any embedding, framing or otherwise making available the article or pages thereof by third parties from platforms, services and websites other than Wiley Online Library must be prohibited.
Dates:	Published: 28 November 2023 Published (online): 18 June 2023 Accepted: 8 June 2023
Institution:	The University of Leeds
Academic Units:	The University of Leeds > Faculty of Medicine and Health (Leeds) > School of Medicine (Leeds) > Institute of Rheumatology & Musculoskeletal Medicine (LIRMM) (Leeds) > Clinical Musculoskeletal Medicine (LIRMM) (Leeds)
Depositing User:	Symplectic Publications
Date Deposited:	20 Sep 2023 09:30
Last Modified:	18 Jun 2024 00:13
Status:	Published
Publisher:	Wiley
Identification Number:	10.1002/acr.25171
Related URLs:	PubMed URL
Open Archives Initiative ID (OAI ID):	oai:eprints.whiterose.ac.uk:203539

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Proposed Response Parameters for Twelve-Month Drug Trial in Juvenile Systemic Sclerosis: Results of the Hamburg International Consensus Meetings

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