Characteristics of patients who experienced treatment failure (TF).
| Characteristics | No TF | TF | p | |
|---|---|---|---|---|
| No. patients | 33 | 11 | ||
| CTP A | 8 (24) | 4 (36) | NS | |
| CTP B | 17 (51.5) | 6 (55) | NS | |
| CTP C | 8 (24) | 1 (9) | NS | |
| Age at enrollment, yrs, median (IQR) | 13 (9.4–14.1) | 12.1 (9.8–15.1) | NS | |
| Age of onset, yrs, median (IQR) | 9.03 (5.7–10.6) | 10.1 (9.2–13.5) | 0.076 | |
| Disease duration at enrollment, mos, median (IQR) | 24 (6.50–76.0) | 10 (6–13.0) | NS | |
| Female | 23 (70) | 8 (73) | NS | |
| Race | 0.038 | |||
| White | 32 (97) | 9 (82) | ||
| Asian | 0 | 2 (18) | ||
| Mixed | 1 (3) | 0 | ||
| Hispanic | 7 (21) | 2 (18) | NS | |
| Subtype | 0.010 | |||
| Circumscribed superficial | 3 (9) | 0 | ||
| Circumscribed deep | 1 (3) | 1 (9) | ||
| Linear scleroderma | 25 (76) | 3 (27) | ||
| Generalized morphea | 0 | 1 (9) | ||
| Pansclerotic morphea | 0 | 1 (9) | ||
| Mixed morphea | 4 (12) | 5 (45.5) | ||
| Head involvement | 15 (45.5) | 2 (18) | NS | |
| Trunk involvement | 12 (36) | 8 (73) | 0.036 | |
| Limb involvement | 16 (48.5) | 9 (82) | 0.053 | |
| Affected anatomic sites, median (IQR) | 2 (1 3) | 4 (2 8) | 0.096 | |
| Extracutaneous involvement | 22 (67) | 11 (100) | 0.027 | |
| Growth disturbance | 14 (42.4) | 7 (63.6) | NS | |
| Joint involvement | 10 (30) | 10 (91) | 0.005 | |
| PGA-A | Baseline, median (IQR) | 4 (3–6) | 5 (4, 7) | NS |
| Last, median (IQR) | 0 (0–1)** | 2 (1, 3)** | 0.011 | |
| mLoSSI | Baseline, median (IQR) | 8 (4–10) | 5 (4, 9) | NS |
| Last, median (IQR) | 0 (0–1)** | 2 (1, 4)** | 0.003 | |
| LSCAM | Baseline, median (IQR) | 7 (4–12) | 8 (6, 23) | NS |
| Last, median (IQR) | 1 (0–2)** | 4 (3, 10)** | < 0.001 | |
| LoSDI | Baseline, median (IQR) | 7 (3–13) | 14 (2, 23) | NS |
| Last, median (IQR | 7 (4–11) | 12 (4, 26) | NS | |
| LSDam | Baseline, median (IQR) | 9 (5–16) | 16 (4, 34) | NS |
| Last, median (IQR) | 8 (5–11) | 12 (6, 35)* | NS | |
| PGA-D | Baseline, median (IQR) | 4 (3–5) | 5 (4–7) | NS |
| Last, median (IQR) | 3 (2–4) | 5 (3–7) | 0.003 | |
| ANA positivity | 14 (52) | 9 (82) | 0.087 | |
| Family history of rheumatic disease | 11 (34) | 7 (64) | 0.090 | |
Values are n (%) unless otherwise specified. P values for differences between patients who experienced TF versus those who did not are shown in last column. Significant p values are in bold face; values that approached significance are in italics. Significant changes in scores from baseline to last visit are indicated in “No TF” and “TF” columns:
↵* p < 0.05;
↵** p < 0.001.
The 11 patients who experienced TF were compared to the 33 other patients who remained on their initial CTP treatment and did not require additional treatment. Head, trunk, limb involvement refers to the no. patients who had an LS lesion in that anatomic site. The no. anatomic sites is based upon the divisions used for the LS Cutaneous Activity measure; the body is divided into 19 sites. The p values on the Race and Subtype rows refer to the significant difference found in the distribution of all of the listed races or subtypes, respectively. Also found significant were frequency of linear scleroderma (p = 0.0089), mixed morphea (p = 0.0305), and mixed, generalized, and pansclerotic morphea together (p = 0.002); generalized and pansclerotic morphea together approached significance (p = 0.058). ANA: antinuclear antibody; CTP: consensus treatment plan; IQR: interquartile range; LoSDI: Localized Scleroderma Damage Index; LSCAM: localized scleroderma cutaneous activity measure; LSDam: localized scleroderma cutaneous damage measure; mLoSSI: modified Localized Scleroderma Severity Index; NS: nonsignificant; PGA-A: physician’s global assessment of disease activity; PGA-D: PGA of disease damage.