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: Systemic
sclerosis (SSc) is a rare multisystem autoimmune disease characterized by
vasculopathy and organ fibrosis. We present baseline data on the North American
observational juvenile systemic sclerosis (jSSc) cohort from the CARRA registry.
Methods: Descriptive
statistics were used for demographic, clinical, and laboratory features.
Results: For the 64
children with SSc in the database, baseline CARRA visit occurred a median of
3.6 yrs after disease onset. The median age of onset was 10.3 yr. Median age at
first pediatric rheumatology (PRH) evaluation was 11.8 yr, with 23% having a
≥ 2 yr delay to PRH. Demographics and clinical manifestations are
detailed in Table 1. Overlap with juvenile dermatomyositis was identified in 3
individuals, with mixed connective tissue disease in 1. Of those with
documented testing for specific antibodies, 80% were ANA positive, 46% anti-Scl70
and 15% ACA. Three of 15 patients tested for anti-PM-Scl antibodies were
positive.
The most prevalent organ manifestations
were dermatologic (93%) and vascular (92%), with Raynaud phenomenon as the most
common feature. Multiple organ systems were affected in 93%. Reported prior or
current medications included use of DMARDs in 87%, steroids in 53%, and
biologics in 10%.
While the median Physician Global
Disease Activity (PGA) score was 2, patient reported measures reflected the
presence of functional impairment and perceived deficiencies in overall well-being
(Table 2). ACR functional class was > class I for 36% of individuals at
baseline and for 74% individuals at worst function.
Conclusion: Within the
CARRA registry, most jSSc patients were female and Caucasian with a median age
of onset of 10.3 years. From limited comparison, baseline clinical data from this
cohort are similar to the jSSc cohort described by Martini et al in 2006 except
the inclusion of overlap patients. While ANA positivity is similar or lower
than other cohorts, ACA positivity is higher which may reflect factors aside
from disease, such as changes in commercially available ANA testing. Overlap
syndrome, present in 29-37% of patients in 2 other cohorts, may be under
recognized. This identifies a potential gap in serologic testing, such as
anti-PM-Scl, and identification of jSSc subtypes.
Significant morbidity is seen in
this cohort, with the majority of individuals having multisystem involvement. Even
with low disease activity scores, patients and providers reported impacts on
function, pain, and quality of life. This identifies the need for development
of accurate serologic identification and clinical measurement indices of
damage, activity, and severity for jSSc.
|
Table 1. Patient Characteristics |
|
|
|
|
|
Demographics |
n (%) |
|
Median yrs (IQR) |
|
|
Gender |
|
|
|
|
|
Female |
54 (84%) |
|
Age at Disease Onset 10.3 (6.3-13.1) |
|
|
|
|
|
Time from Onset to PRH 0.8 (0.14-1.9) |
|
|
Race, Ethnicity |
|
|
Time from Onset to CARRA Baseline 3.6 (1.8-6.3) |
|
|
Caucasian |
50 (78%) |
|
|
|
|
African American |
12 (19%) |
|
|
|
|
Asian |
2 (3%) |
|
|
|
|
Non-Hispanic |
55 (86%) |
|
|
|
|
|
|
|
|
|
|
Clinical Manifestations |
|
|
|
|
|
Percentages are given for those with no missing data for the characteristic |
|
|||
|
Manifestation |
Affected (%) |
|
Manifestation |
Affected (%) |
|
Dermatologic |
54 (93%) |
|
Pulmonary |
20 (34%) |
|
Skin Thickening/Induration |
37 (64%) |
|
Restrictive Lung Disease |
13 (22%) |
|
Face |
27 (47%) |
|
Decreased DLCO/Hypoxemia |
11 (19%) |
|
Proximal to MCPs |
28 (48%) |
|
Radiologic or Pathologic Fibrosis |
6 (10%) |
|
Proximal to Elbow |
9 (16%) |
|
Parenchymal Pulmonary Disease |
4 (7%) |
|
Trunk |
4 (7%) |
|
Dyspnea |
4 (7%) |
|
Sclerodactyly |
36 (62%) |
|
Pulmonary Hypertension |
1 (2%) |
|
Telangiectasia |
21 (36%) |
|
Other |
2 (3%) |
|
Calcinosis |
6 (10%) |
|
|
|
|
Other Dermatologic |
5 (9%) |
|
Renal |
2 (3%) |
|
|
|
|
Renovascular Hypertension |
1 (2%) |
|
Vascular |
54 (92%) |
|
Other |
1 (2%) |
|
Raynaud’s |
43 (73%) |
|
|
|
|
Nailbed Capillary Abnormalities |
41 (70%) |
|
Cardiac |
1 (2%) |
|
Digital Ulceration/Gangrene |
27 (46%) |
|
Pericardial effusion |
1 (2%) |
|
|
|
|
|
|
|
Musculoskeletal |
27 (46%) |
|
Multiple Organ Involvement |
54 (93%) |
|
Contractures |
20 (34%) |
|
Number of organs affected |
|
|
Arthritis |
11 (19%) |
|
None |
1 (2%) |
|
Myositis |
7 (12%) |
|
One |
3 (5%) |
|
Tendinopathy |
3 (5%) |
|
Two |
14 (24%) |
|
|
|
|
Three |
18 (31%) |
|
GI |
25 (42%) |
|
Four or More |
22 (38%) |
|
GI Dysmotility |
12 (20%) |
|
Total organs affected, median(IQR) |
3 (2-4) |
|
Documented GERD |
11 (19%) |
|
|
|
|
Dysphagia |
10 (17%) |
|
|
|
|
Esophagitis |
2 (3%) |
|
|
|
|
Malabsorption |
1 (2%) |
|
|
|
|
Other |
4 (7%) |
|
|
|
|
Table 2. Physician and Patient Based Assessments. Percentages are given for those with no missing data for the characteristic. |
|
|
|
|
|
Physician Assessments |
|
|
|
Median (IQR) |
|
Physician Global Disease Activity*, n=56 |
2 (1-3) |
|
|
|
|
ACR Functional Class |
n (%) |
|
Current at Baseline, n=61 |
|
|
Class I |
39 (64%) |
|
Class II |
15 (25%) |
|
Class III |
4 (7%) |
|
Class IV |
3 (5%) |
|
|
|
|
Worst Ever, n=53 |
|
|
Class I |
14 (26%) |
|
Class II |
26 (49%) |
|
Class III |
8 (15%) |
|
Class IV |
5 (9%) |
|
|
|
|
Patient/Parent Reported Global Function and Quality of Life Metrics, n=64 |
|
|
|
Median (IQR) |
|
Childhood Health Assessment Questionnaire |
0.13 (0.0-0.63) |
|
Pain Scale* |
1 (0-4) |
|
Global Well Being Scale* |
3 (1-5) |
|
|
|
|
Health Related Quality of Life |
n (%) |
|
Excellent |
9 (14%) |
|
Very good |
22 (35%) |
|
Good |
28 (44%) |
|
Poor |
4 (6%) |
|
Very Poor |
0 (0%) |
|
|
|
|
*Physician global assessment of disease activity, patient reported global wellbeing, and patient reported pain were measured on a 0-10 numeric rating scale, 10 indicating worst disease, poorest wellbeing, or worst pain, respectively. |
|
To cite this abstract in AMA style:
Stevens BE, Torok KS, Li SC, Hershey N, Curran M, Higgins GC, Moore K, Rabinovich CE, Stevens AM. Characteristics of the Juvenile Systemic Sclerosis Cohort within the Childhood Arthritis and Rheumatology Research Alliance (CARRA) Registry [abstract]. Arthritis Rheumatol. 2015; 67 (suppl 10). https://acrabstracts.org/abstract/characteristics-of-the-juvenile-systemic-sclerosis-cohort-within-the-childhood-arthritis-and-rheumatology-research-alliance-carra-registry/. Accessed .« Back to 2015 ACR/ARHP Annual Meeting
ACR Meeting Abstracts - https://acrabstracts.org/abstract/characteristics-of-the-juvenile-systemic-sclerosis-cohort-within-the-childhood-arthritis-and-rheumatology-research-alliance-carra-registry/