Proportions of patients with clinically significant improvement, according to CR or PR vs SD or PD with failure-free survival at 1 year after enrollment
| . | Overall CR or PR (n = 39) . | Overall SD or PD (n = 59) . | . | ||
|---|---|---|---|---|---|
| Assessment . | N . | Number improved (%) . | N . | Number improved (%) . | P* . |
| Provider grading of specific measures | |||||
| NIH Skin Score (0-3) | 27 | 24 (89) | 37 | 20 (54) | .003 |
| NIH Eye Score (0-3) | 17 | 7 (41) | 41 | 10 (24) | .22 |
| Modified Oral Mucosa Rating Scale (0-12) | 29 | 20 (69) | 48 | 16 (33) | .004 |
| Total serum bilirubin | 5 | 5 (100) | 5 | 5 (100) | NA |
| Alanine aminotransferase | 22 | 15 (68) | 27 | 25 (85) | .19 |
| Alkaline phosphatase | 18 | 11 (61) | 27 | 17 (63) | >.99 |
| Percentage of predicted FEV1 | 0 | NA | 4 | 1 (25) | NA |
| NIH Joint and Fascia Score (0-3) | 9 | 6 (67) | 26 | 5 (19) | .01 |
| Photographic range of motion (4-25) | 4 | 1 (25) | 24 | 4 (17) | >.99 |
| Provider grading of specific symptoms | |||||
| NIH Lung Symptom Score (0-3) | 6 | 6 (100) | 26 | 8 (31) | .003 |
| Upper GI Score (0-3) | 8 | 8 (100) | 13 | 8 (62) | .11 |
| Lower GI Score (0-3) | 9 | 8 (89) | 8 | 2 (25) | .02 |
| Esophagus Score (0-3) | 6 | 5 (83) | 8 | 3 (38) | .14 |
| Patient grading of specific symptoms | |||||
| Skin itching (0-10) | 21 | 8 (38) | 30 | 13 (43) | .78 |
| Oral sensitivity (0-10) | 18 | 10 (56) | 30 | 13 (43) | .55 |
| Chief eye complaint (0-10) | 15 | 5 (33) | 37 | 13 (35) | >.99 |
| Global rating scales | |||||
| Provider 0-3 | 39 | 25 (64) | 59 | 18 (31) | .002 |
| Provider 0-10 | 39 | 29 (74) | 59 | 23 (39) | .0009 |
| Patient 0-3 | 26 | 12 (46) | 37 | 8 (22) | .06 |
| Patient 0-10 | 22 | 17 (77) | 36 | 13 (36) | .003 |
| Lee Symptom Scale (0-100) | 27 | 11 (41) | 39 | 9 (23) | .17 |
| FACT-BMT | 25 | 14 (56) | 38 | 9 (24) | .02 |
| . | Overall CR or PR (n = 39) . | Overall SD or PD (n = 59) . | . | ||
|---|---|---|---|---|---|
| Assessment . | N . | Number improved (%) . | N . | Number improved (%) . | P* . |
| Provider grading of specific measures | |||||
| NIH Skin Score (0-3) | 27 | 24 (89) | 37 | 20 (54) | .003 |
| NIH Eye Score (0-3) | 17 | 7 (41) | 41 | 10 (24) | .22 |
| Modified Oral Mucosa Rating Scale (0-12) | 29 | 20 (69) | 48 | 16 (33) | .004 |
| Total serum bilirubin | 5 | 5 (100) | 5 | 5 (100) | NA |
| Alanine aminotransferase | 22 | 15 (68) | 27 | 25 (85) | .19 |
| Alkaline phosphatase | 18 | 11 (61) | 27 | 17 (63) | >.99 |
| Percentage of predicted FEV1 | 0 | NA | 4 | 1 (25) | NA |
| NIH Joint and Fascia Score (0-3) | 9 | 6 (67) | 26 | 5 (19) | .01 |
| Photographic range of motion (4-25) | 4 | 1 (25) | 24 | 4 (17) | >.99 |
| Provider grading of specific symptoms | |||||
| NIH Lung Symptom Score (0-3) | 6 | 6 (100) | 26 | 8 (31) | .003 |
| Upper GI Score (0-3) | 8 | 8 (100) | 13 | 8 (62) | .11 |
| Lower GI Score (0-3) | 9 | 8 (89) | 8 | 2 (25) | .02 |
| Esophagus Score (0-3) | 6 | 5 (83) | 8 | 3 (38) | .14 |
| Patient grading of specific symptoms | |||||
| Skin itching (0-10) | 21 | 8 (38) | 30 | 13 (43) | .78 |
| Oral sensitivity (0-10) | 18 | 10 (56) | 30 | 13 (43) | .55 |
| Chief eye complaint (0-10) | 15 | 5 (33) | 37 | 13 (35) | >.99 |
| Global rating scales | |||||
| Provider 0-3 | 39 | 25 (64) | 59 | 18 (31) | .002 |
| Provider 0-10 | 39 | 29 (74) | 59 | 23 (39) | .0009 |
| Patient 0-3 | 26 | 12 (46) | 37 | 8 (22) | .06 |
| Patient 0-10 | 22 | 17 (77) | 36 | 13 (36) | .003 |
| Lee Symptom Scale (0-100) | 27 | 11 (41) | 39 | 9 (23) | .17 |
| FACT-BMT | 25 | 14 (56) | 38 | 9 (24) | .02 |
Fisher's exact test comparing the proportions of improved patients according to CR or PR vs SD or PD by NIH criteria. Patients unaffected by specific manifestations are not included. Some patient-reported data are missing. See supplemental Table 2 for definitions of clinically significant improvement.
NA, not applicable.