Change measures between enrollment and 1 year, according to CR or PR vs SD or PD among patients with failure-free survival
| . | Overall CR or PR (n = 39) . | Overall SD or PD (n = 59) . | . | ||
|---|---|---|---|---|---|
| Assessment . | N . | Mean (SD) . | N . | Mean (SD) . | P* . |
| Provider grading of specific measures | |||||
| NIH Skin Score (0-3) | 39 | −1.05 (1.12) | 59 | −0.29 (1.07) | .001 |
| NIH Eye Score (0-3) | 39 | −0.18 (0.51) | 59 | 0.10 (0.69) | .02 |
| Modified Oral Mucosa Rating Scale (0-12) | 39 | −2.21 (2.62) | 59 | −0.51 (2.78) | .003 |
| Total of serum bilirubin | 39 | −0.59 (2.61) | 57 | −0.14 (0.42) | .29 |
| Alanine aminotransferase | 39 | −78.8 (144.2) | 57 | −75.0 (196.4) | .91 |
| Alkaline phosphatase | 39 | −43.3 (107.3) | 57 | −24.2 (158.1) | .48 |
| Percentage of predicted FEV1 | 17 | 1.76 (8.79) | 20 | −4.10 (16.1) | .17 |
| NIH Joint and Fascia Score (0-3) | 39 | −0.18 (0.45) | 59 | 0.12 (0.77) | .02 |
| Photographic range of motion (4-25) | 22 | 0.32 (1.49) | 39 | −0.49 (1.17) | .04 |
| Provider grading of specific symptoms | |||||
| NIH Lung Symptom Score (0-3) | 39 | −0.21 (0.52) | 59 | 0.19 (0.75) | .003 |
| Upper GI Score (0-3) | 39 | −0.38 (0.88) | 59 | −0.05 (0.63) | .04 |
| Lower GI Score (0-3) | 39 | −0.28 (0.72) | 59 | 0.07 (0.55) | .01 |
| Esophagus Score (0-3) | 39 | −0.26 (0.72) | 59 | 0.00 (0.32) | .04 |
| Patient grading of specific symptoms | |||||
| Skin itching (0-10) | 25 | −0.80 (2.20) | 38 | −1.00 (3.05) | .76 |
| Oral sensitivity (0-10) | 25 | −1.60 (2.16) | 40 | −0.63 (3.51) | .17 |
| Chief eye complaint (0-10) | 25 | −0.24 (2.45) | 39 | 0.21 (3.67) | .56 |
| Global rating scales | |||||
| Provider 0-3 | 39 | −0.87 (0.80) | 59 | −0.22 (0.74) | .0001 |
| Provider 0-10 | 39 | −3.03 (1.99) | 59 | −1.02 (1.91) | <.0001 |
| Patient 0-3 | 26 | −0.62 (0.75) | 37 | −0.05 (0.62) | .003 |
| Patient 0-10 | 23 | −2.83 (2.19) | 37 | −0.43 (2.22) | .0002 |
| Lee Symptom Scale (0-100)† | 27 | −5.51 (7.41) | 39 | −1.59 (12.3) | .11 |
| FACT-BMT | 25 | 11.9 (14.7) | 38 | 1.20 (14.4) | .007 |
| . | Overall CR or PR (n = 39) . | Overall SD or PD (n = 59) . | . | ||
|---|---|---|---|---|---|
| Assessment . | N . | Mean (SD) . | N . | Mean (SD) . | P* . |
| Provider grading of specific measures | |||||
| NIH Skin Score (0-3) | 39 | −1.05 (1.12) | 59 | −0.29 (1.07) | .001 |
| NIH Eye Score (0-3) | 39 | −0.18 (0.51) | 59 | 0.10 (0.69) | .02 |
| Modified Oral Mucosa Rating Scale (0-12) | 39 | −2.21 (2.62) | 59 | −0.51 (2.78) | .003 |
| Total of serum bilirubin | 39 | −0.59 (2.61) | 57 | −0.14 (0.42) | .29 |
| Alanine aminotransferase | 39 | −78.8 (144.2) | 57 | −75.0 (196.4) | .91 |
| Alkaline phosphatase | 39 | −43.3 (107.3) | 57 | −24.2 (158.1) | .48 |
| Percentage of predicted FEV1 | 17 | 1.76 (8.79) | 20 | −4.10 (16.1) | .17 |
| NIH Joint and Fascia Score (0-3) | 39 | −0.18 (0.45) | 59 | 0.12 (0.77) | .02 |
| Photographic range of motion (4-25) | 22 | 0.32 (1.49) | 39 | −0.49 (1.17) | .04 |
| Provider grading of specific symptoms | |||||
| NIH Lung Symptom Score (0-3) | 39 | −0.21 (0.52) | 59 | 0.19 (0.75) | .003 |
| Upper GI Score (0-3) | 39 | −0.38 (0.88) | 59 | −0.05 (0.63) | .04 |
| Lower GI Score (0-3) | 39 | −0.28 (0.72) | 59 | 0.07 (0.55) | .01 |
| Esophagus Score (0-3) | 39 | −0.26 (0.72) | 59 | 0.00 (0.32) | .04 |
| Patient grading of specific symptoms | |||||
| Skin itching (0-10) | 25 | −0.80 (2.20) | 38 | −1.00 (3.05) | .76 |
| Oral sensitivity (0-10) | 25 | −1.60 (2.16) | 40 | −0.63 (3.51) | .17 |
| Chief eye complaint (0-10) | 25 | −0.24 (2.45) | 39 | 0.21 (3.67) | .56 |
| Global rating scales | |||||
| Provider 0-3 | 39 | −0.87 (0.80) | 59 | −0.22 (0.74) | .0001 |
| Provider 0-10 | 39 | −3.03 (1.99) | 59 | −1.02 (1.91) | <.0001 |
| Patient 0-3 | 26 | −0.62 (0.75) | 37 | −0.05 (0.62) | .003 |
| Patient 0-10 | 23 | −2.83 (2.19) | 37 | −0.43 (2.22) | .0002 |
| Lee Symptom Scale (0-100)† | 27 | −5.51 (7.41) | 39 | −1.59 (12.3) | .11 |
| FACT-BMT | 25 | 11.9 (14.7) | 38 | 1.20 (14.4) | .007 |
Two-sample Student t test with Satterthwaite correction comparing mean change in measure according to CR or PR vs SD or PD by NIH criteria. Patients unaffected by specific manifestations are included. Some patient-reported data are missing.
None of the subscales showed statistically significant differences in mean change between patients with overall CR or PR in comparison with those who had SD or PD.