Session Information
Date: Sunday, November 8, 2015
Title: Pediatric Rheumatology – Clinical and Therapeutic Aspects Poster I: Lupus, Scleroderma, JDMS
Session Type: ACR Poster Session A
Session Time: 9:00AM-11:00AM
Background/Purpose:
Juvenile systemic sclerosis (jSSc) is an orphan disease. We have currently new promising effective medication to treat systemic sclerosis, but no valid outcome measures to assess the activity of the disease. In the frame of the juvenile scleroderma working group of Paediatric Rheumatology European Society (PRES JSWG), we developed a provisional response index for jSSc.
Methods:
The facilitating committee designed a 3-round Delphi-exercise to obtain a core set of disease activity measures in jSSc. The members of the international paediatric rheumatology email board, the PRES juvenile scleroderma working group and the active participants of the juvenile scleroderma inception cohort project were invited to participate in our online surveys. In Round 1, they were asked to add unique items in 21 preleminary domains. Round 2 judged the items’ applicability and importance using a 1-10 scale. Previous respondents were asked to re-rate uncertain (score 4-7) items in Round 3. The aforementioned were followed by a face-to-face Nominal Group discussion to decide final items and domain structure.
Results:
52 participants of Round 1 suggested a total of 131 items for the 21 domains. In round 2, 56 responders rated the significance of the items. Most of the participating experts took part in the 3rd Round by assessing the uncertain items. The Nominal Group accepted 11 main domains for further validation (global disease activity, biomarkers, Rayneud phenomenon, digital ulcers, skin, pulmonary, cardiac, gastrointestinal and renal involvement, ‘health related qualaity of life and function’) containing 41 items (see table 1). We have also felt essential to collect data regarding the development of the children.
table 1:
| Global disease activity | |||
| Global disease activity according to physician by visual analogue scale (VAS) | |||
| Global disease activity according to patient / parent by VAS | |||
| Scleroderma-related condition rating by physician as in the last 3 months | |||
| Scleroderma-related condition rating by patient / parent as in the last 3 months | |||
| Biomarkers | |||
| ESR | |||
| CRP | |||
| Hemoglobin | |||
| Raynaud phenomenon | |||
| Raynaud activity score | |||
| Raynaud severity due to patient by VAS | |||
| Digital ulcers | |||
| Number of digital ulcers | |||
| Newly occured digital ulcers | |||
| Digital ulcer severity due to patient by VAS | |||
| Skin | |||
| Modified Rodnan skin score (mRSS) | |||
| Newly occured calcinosis | |||
| Newly occured teleangiectasia | |||
| Presence of scleroderma-related edema | |||
| Pulmonary | |||
| Forced vital capacity (FVC) | |||
| Diffusion capacity (DLCO) | |||
| 6 minute walk test | |||
| Borg index | |||
| Cardiac | |||
| Left Ventricular ejection fraction (EF) by echocardiography | |||
| Right Ventricular ejection fraction (EF) by echocardiography | |||
| Newly occured carditis | |||
| Newly occured arrythmia | |||
| Pro-BNP level | |||
| New onset or worsening of pulmory arterial hypertension (PAH) | |||
| Gastrointestinal | |||
| New onset of swallowing difficulties | |||
| Change in stool frequency | |||
| New sign of reflux | |||
| Renal | |||
| Newly occured hypertension | |||
| Newly occured renal crisis | |||
| Proteinuria (by spot urine) | |||
| Creatinine clearence (in severe muscle loss, EDTA claerance should be measured) | |||
| Change in glomerular filtration rate (GFR) | |||
| Health related quality of life and function | |||
| PEDSQL between 8-16 years or SF-36 over 16 years of age | |||
| Childhood health assessment questionnaire (CHAQ) | |||
| Pain assessment by VAS as in CHAQ | |||
| Fatigue assessment by VAS | |||
| (Essential paediatric data to collect) | |||
| Weight | |||
| Height | |||
| Growth velocity | |||
| Tanner stage | |||
Conclusion:
We reached consensus and developed a Response Index, which should help to conduct clinical trials in jSSc. The validation of the JSSRI is planned on the patients of the Juvenile Scleroderma Inception Cohort.
To cite this abstract in AMA style:
Foeldvari I, Zoltan G, Baildam E, Batu ED, Blakley M, Cakan M, Curran M, Kienast A, Fligelstone K, Nemkova D, Pilkington C, Ruperto N, Tsinti M, Tsitsami E, Urbanvica K, Constantin T. Development of a Juvenile Systemic Sclerosis Response Index (JSSRI) [abstract]. Arthritis Rheumatol. 2015; 67 (suppl 10). https://acrabstracts.org/abstract/development-of-a-juvenile-systemic-sclerosis-response-index-jssri/. Accessed .« Back to 2015 ACR/ARHP Annual Meeting
ACR Meeting Abstracts - https://acrabstracts.org/abstract/development-of-a-juvenile-systemic-sclerosis-response-index-jssri/