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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
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Veröffentlicht: | 8. Oktober 2019 |
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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.