Session Information
Date: Sunday, November 8, 2015
Title: Systemic Sclerosis, Fibrosing Syndromes, and Raynaud's - Clinical Aspects and Therapeutics I
Session Type: ACR Concurrent Abstract Session
Session Time: 4:30PM-6:00PM
Background/Purpose: There is increasing interest in cohort
enrichment for clinical trials of early diffuse SSc (dcSSc). Recent
EUSTAR database analysis (Maurer et al. 2015) suggests that patients with a low
modified Rodnan skin score (mRss)<
22 and short disease duration (< 15 months from first non-Raynaud symptom)
have the highest probability of worsening (defined as progressive skin
involvement) at one year. The autoantibody profile in the US dcSSc population is different than Europe, with greater
percentages of patients with anti-RNA polymerase III (RNAP) antibody in the US. Anti-RNAP positive patients often present
with higher skin scores. If an upper threshold skin score is used as an
inclusion criterion thenmany RNAP positive patients could be excluded. The objective of this study was to examine
the effect of restricting mRss in early diffuse SSc clinical trials with respect to: 1)the percentage of
patients experiencing a significant change in mRss
and 2) recruitment in a US population.
Methods: We used a single-center cohort of
prospectively followed early dcSSc patients seen for
an initial visit between Jan. 1, 1980 and Dec. 31, 2013 at a US Scleroderma
Center. Early was defined as < 18
months from the first non-Raynaud symptom.
Patients had to have at least two mRss within
one year of the first SScCenter visit. After descriptive baseline statistics, time
to peak skin score, and the percentage of patients who had improvement or
worsening of mRss over one year were calculated. Data is presented by skin score at
presentation.
Results: Among 304 eligible patients, mean age at
the first SSc Center visit was 51.8 ±13.5
years. The cohort was 76% female and 93%Caucasian. Overall patients were 58% RNAP positive, 21%
anti-Scl70 positive, 11% other SSc-autoantibody
positive and 10% unknown. The percentage
of patients with improving or worsening mRss by ≥5
points,≥5
points and 25% change, and the times to peak skin score are
shown in Table 1. Including patients
with higher baseline skin score did not change the overall % of patients
improving or worsening their mRss within one year. The median time to peak skin score was nearly
identical in the patient groups. By
restricting our inclusion criterion to mRss< 25
points, 27-40% of patients with worsening mRss would
have been excluded with different progression.
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Table 1: Skin score change at one year by baseline mRss at presentation
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mRss at first SSc Center visit
|
N
|
mRssworsened by ≥5 points
|
mRss improved by ≥5 points (%)
|
< 5 point change in mRss
|
Median (IQR) time to peak mRss from first visit
|
|
|
10-25 |
176 |
87 (49%) |
47 (27%) |
42 (24%) |
0.45 (0.00, 0.64) |
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|
10-30 |
217 |
110 (51%) |
55 (25%) |
52 (24%) |
0.46 (0.16,0.67) |
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10-35 |
253 |
127 (50%) |
69 (27%) |
57 (23%) |
0.45 (0.15, 0.66) |
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|
10-40 |
284 |
142 (50%) |
82 (29%) |
60 (21%) |
0.42 (0.07, 0.66) |
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|
10-45 |
304 |
143 (47%) |
90 (30%) |
71 (23%) |
0.40 (0.00, 0.63) |
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N
|
mRssworse ≥5 points and 25%
|
mRss improved by ≥5 points and 25%
|
< 5 point and 25% change in mRss
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|
10-25 |
176 |
84 (47%) |
44 (25%) |
58 (33%) |
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|
10-30 |
217 |
97 (45%) |
49 (22%) |
71 (33%) |
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|
|
10-35 |
253 |
108 (43%) |
61 (24%) |
84 (33%) |
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|
10-40 |
284 |
115 (41%) |
69 (24%) |
100 (35%) |
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10-45 |
304 |
115 (38%) |
71 (23%) |
118 (36%) |
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Conclusion: In one
US SSc Center population expanding the allowable mRss from ≥22 to ≤45 did not decrease the percentof
patients changing their mRss. Restricting mRss at≤
22 may significantly limit our potential to recruit patients in the US. Further study of this issue should be
undertaken with additional modeling and consideration to different autoantibody
frequencies in geographic regions.
Limitations of our data include that it is single center
population.
References: Maurer et
al., Annals Rheum Dis. 2015; 74: 1124.
To cite this abstract in AMA style:
Domsic RT, Khanna D, Lucas M, Steen VD, Furst DE, Lafyatis R, Medsger TA Jr.. The Potential Effect on Recruitment of Restricting Skin Scores Eligibility Criteria in Early Diffuse Scleroderma Trials [abstract]. Arthritis Rheumatol. 2015; 67 (suppl 10). https://acrabstracts.org/abstract/the-potential-effect-on-recruitment-of-restricting-skin-scores-eligibility-criteria-in-early-diffuse-scleroderma-trials/. Accessed .« Back to 2015 ACR/ARHP Annual Meeting
ACR Meeting Abstracts - https://acrabstracts.org/abstract/the-potential-effect-on-recruitment-of-restricting-skin-scores-eligibility-criteria-in-early-diffuse-scleroderma-trials/