Baseline patient demographics and clinical characteristics between clinical mouth responders and nonresponders
| Characteristic . | Mouth responders (n = 14) . | Mouth nonresponders (n = 6) . |
|---|---|---|
| Age, median (range), y | 49.5 (18-70) | 51.5 (27-71) |
| Sex, female, n (%) | 7 (50.0) | 2 (33.3) |
| Race/ethnicity, n (%) | ||
| White | 13 (92.9) | 5 (83.3) |
| Black | — | 1 (16.7) |
| Other | 1 (7.1) | — |
| HSCT indication, n (%) | ||
| AML | 6 (42.9) | 3 (50.0) |
| ALL | 1 (7.1) | 2 (33.3) |
| MDS | 2 (14.3) | — |
| CML | 1 (7.1) | — |
| Myelofibrosis | 1 (7.1) | — |
| CLL | 1 (7.1) | — |
| NHL | 1 (7.1) | — |
| Other | 1 (7.1) | 1 (16.7) |
| Conditioning, n (%) | ||
| Myeloablative | 11 (78.6) | 6 (100) |
| Nonmyeloablative | 2 (14.3) | — |
| TBI | — | — |
| Unknown | 1 (7.1) | — |
| HSCT graft source, peripheral blood, n (%) | 14 (100) | 6 (100) |
| HLA matching of donor/recipient, n (%) | ||
| Matched (related) | 6 (42.9) | 2 (33.3) |
| Matched (unrelated) | 7 (50.0) | 4 (66.7) |
| Partially matched (related) | 1 (7.1) | — |
| CMV serostatus (donor/recipient), n (%) | ||
| Positive/positive | — | 3 (50.0) |
| Positive/negative | 4 (28.6) | 1 (16.7) |
| Negative/positive | 3 (21.4) | 2 (33.3) |
| Negative/negative | 5 (35.7) | — |
| Unknown | 2 (14.3) | — |
| Previous aGVHD, n (%) | 10 (71.4) | 5 (83.3) |
| NIH cGVHD severity, n (%) | ||
| Moderate | 3 (21.4) | — |
| Severe | 11 (78.6) | 6 (100) |
| Mouth involvement per NIH-modified OMRS, mean (range) | ||
| Erythema score | 1.0 (0-2) | 1.0 (1-2) |
| Lichenoid score | 1.0 (0-3) | 0.5 (0-3) |
| Ulcer score | 0 (0-3) | 0 (0) |
| Total mouth score | 3.5 (1-7) | 1.5 (1-5) |
| Organ involvement | ||
| No. of organs involved, median (range) | 4.5 (3-6) | 3 (1-5) |
| ≥4 organs involved, n (%) | 11 (78.6) | 2 (33.3) |
| Skin, n (%) | 12 (85.7) | 4 (66.7) |
| Joints/fascia, n (%) | 12 (85.7) | 4 (66.7) |
| Eyes, n (%) | 9 (64.3) | 2 (33.3) |
| Lungs, n (%) | 6 (42.9) | 0 |
| Esophagus, n (%) | 4 (28.6) | 1 (16.7) |
| Upper GI tract, n (%) | 1 (7.1) | 0 |
| Lower GI tract, n (%) | 2 (14.3) | 0 |
| Liver, n (%) | 1 (7.1) | 1 (16.7) |
| Baseline global severity rating (0-10), n (%) | ||
| 3 | — | 1 (16.7) |
| 4 | — | — |
| 5 | 2 (14.3) | 1 (16.7) |
| 6 | 3 (21.4) | 1 (16.7) |
| 7 | 6 (42.9) | 3 (50.0) |
| 8 | 3 (21.4) | — |
| Median Karnofsky performance status, n (%) | ||
| 60-70 | 2 (14.3) | — |
| 80-90 | 12 (85.7) | 6 (100) |
| 100 | — | — |
| Time from HSCT to study enrollment, median (range), mo | 43.0 (13.6-89.7) | 33.2 (12.9-102.4) |
| Time from cGVHD diagnosis to study enrollment, median (range), mo | 13.9 (3.6-38.9) | 12.2 (5.1-38.7) |
| Previous therapies, median (range), n | 3.5 (2-7) | 3 (2-5) |
| Patients per treatment arm, n (%) | ||
| Once daily | 8 (57.1) | 3 (50.0) |
| Twice daily | 6 (42.9) | 3 (50.0) |
| Characteristic . | Mouth responders (n = 14) . | Mouth nonresponders (n = 6) . |
|---|---|---|
| Age, median (range), y | 49.5 (18-70) | 51.5 (27-71) |
| Sex, female, n (%) | 7 (50.0) | 2 (33.3) |
| Race/ethnicity, n (%) | ||
| White | 13 (92.9) | 5 (83.3) |
| Black | — | 1 (16.7) |
| Other | 1 (7.1) | — |
| HSCT indication, n (%) | ||
| AML | 6 (42.9) | 3 (50.0) |
| ALL | 1 (7.1) | 2 (33.3) |
| MDS | 2 (14.3) | — |
| CML | 1 (7.1) | — |
| Myelofibrosis | 1 (7.1) | — |
| CLL | 1 (7.1) | — |
| NHL | 1 (7.1) | — |
| Other | 1 (7.1) | 1 (16.7) |
| Conditioning, n (%) | ||
| Myeloablative | 11 (78.6) | 6 (100) |
| Nonmyeloablative | 2 (14.3) | — |
| TBI | — | — |
| Unknown | 1 (7.1) | — |
| HSCT graft source, peripheral blood, n (%) | 14 (100) | 6 (100) |
| HLA matching of donor/recipient, n (%) | ||
| Matched (related) | 6 (42.9) | 2 (33.3) |
| Matched (unrelated) | 7 (50.0) | 4 (66.7) |
| Partially matched (related) | 1 (7.1) | — |
| CMV serostatus (donor/recipient), n (%) | ||
| Positive/positive | — | 3 (50.0) |
| Positive/negative | 4 (28.6) | 1 (16.7) |
| Negative/positive | 3 (21.4) | 2 (33.3) |
| Negative/negative | 5 (35.7) | — |
| Unknown | 2 (14.3) | — |
| Previous aGVHD, n (%) | 10 (71.4) | 5 (83.3) |
| NIH cGVHD severity, n (%) | ||
| Moderate | 3 (21.4) | — |
| Severe | 11 (78.6) | 6 (100) |
| Mouth involvement per NIH-modified OMRS, mean (range) | ||
| Erythema score | 1.0 (0-2) | 1.0 (1-2) |
| Lichenoid score | 1.0 (0-3) | 0.5 (0-3) |
| Ulcer score | 0 (0-3) | 0 (0) |
| Total mouth score | 3.5 (1-7) | 1.5 (1-5) |
| Organ involvement | ||
| No. of organs involved, median (range) | 4.5 (3-6) | 3 (1-5) |
| ≥4 organs involved, n (%) | 11 (78.6) | 2 (33.3) |
| Skin, n (%) | 12 (85.7) | 4 (66.7) |
| Joints/fascia, n (%) | 12 (85.7) | 4 (66.7) |
| Eyes, n (%) | 9 (64.3) | 2 (33.3) |
| Lungs, n (%) | 6 (42.9) | 0 |
| Esophagus, n (%) | 4 (28.6) | 1 (16.7) |
| Upper GI tract, n (%) | 1 (7.1) | 0 |
| Lower GI tract, n (%) | 2 (14.3) | 0 |
| Liver, n (%) | 1 (7.1) | 1 (16.7) |
| Baseline global severity rating (0-10), n (%) | ||
| 3 | — | 1 (16.7) |
| 4 | — | — |
| 5 | 2 (14.3) | 1 (16.7) |
| 6 | 3 (21.4) | 1 (16.7) |
| 7 | 6 (42.9) | 3 (50.0) |
| 8 | 3 (21.4) | — |
| Median Karnofsky performance status, n (%) | ||
| 60-70 | 2 (14.3) | — |
| 80-90 | 12 (85.7) | 6 (100) |
| 100 | — | — |
| Time from HSCT to study enrollment, median (range), mo | 43.0 (13.6-89.7) | 33.2 (12.9-102.4) |
| Time from cGVHD diagnosis to study enrollment, median (range), mo | 13.9 (3.6-38.9) | 12.2 (5.1-38.7) |
| Previous therapies, median (range), n | 3.5 (2-7) | 3 (2-5) |
| Patients per treatment arm, n (%) | ||
| Once daily | 8 (57.1) | 3 (50.0) |
| Twice daily | 6 (42.9) | 3 (50.0) |
aGVHD, acute GVHD; ALL, acute lymphoblastic leukemia; AML, acute myeloid leukemia; CLL, chronic lymphocytic leukemia; CML, chronic myeloid leukemia; CMV, cytomegalovirus; GI, gastrointestinal; HSCT, hematopoietic stem cell transplant; MDS, myelodysplastic syndrome; NHL, non-Hodgkin lymphoma; TBI, total body irradiation.