Table 1 Patient baseline demographic and disease characteristics
Characteristics | All patients |
|---|---|
Data are n (%) unless stated otherwise | (N = 30) |
Age (years), median (range) | 29.5 (18–50) |
Sex | |
Male | 21 (70.0%) |
Female | 9 (30.0%) |
Mean weight (SD), kg | 56.2 (13.5) |
Mean BMI (SD), kg/m2 | 19.8 (3.5) |
ECOG PS | |
0 | 9 (30.0%) |
1 | 21 (70.0%) |
Allo-HSCT | |
Yes | 30 (100.0%) |
Indication for transplant | |
Acute lymphoblastic leukemia | 10 (33.3%) |
Acute myeloid leukemia | 8 (26.7%) |
Myelodysplastic syndrome | 7 (23.3%) |
Chronic myelogenous leukemia | 4 (13.3%) |
Leukemia (other) | 1 (3.3%) |
Conditioning intensity | |
Myeloablative | 26 (86.7%) |
Unknown | 4 (13.3%) |
Type of donor | |
Related donor | 27 (90.0%) |
Unrelated donor | 3 (10.0%) |
Human leukocyte antigen matching of donor/recipient | |
Matched | 19 (63.3%) |
Partially matched | 10 (33.3%) |
Unknown | 1 (3.3%) |
Stem cell source | |
Peripheral blood stem cell | 21 (70.0%) |
Bone marrow | 2 (6.7%) |
Peripheral blood stem cell + bone marrow | 6 (20.0%) |
Cord blood | 1 (3.3%) |
Donor/recipient cytomegalovirus serological status | |
+ / + | 4 (13.3%) |
− / − | 2 (6.7%) |
Unknown/ + | 1 (3.3%) |
Unknown/ − | 16 (53.3%) |
Unknown/unknown | 7 (23.3%) |
History of aGVHD | |
Yes | 17 (56.7%) |
No | 13 (43.3%) |
Time from cGVHD diagnosis to enrollment, median (range), month | 24.2 (3.4–92.9) |
Number of organs involved, median (range) | 4 (1–6) |
Organ involvement | |
1 | 2 (6.7%) |
2 | 6 (20.0%) |
3 | 6 (20.0%) |
4 | 10 (33.3%) |
5 | 5 (16.7%) |
≥ 6 | 1 (3.3%) |
Eyes | 22 (73.3%) |
Mouth | 22 (73.3%) |
Skin | 20 (66.7%) |
Lungs | 13 (43.3%) |
Joints/fascia | 9 (30.0%) |
Liver | 9 (30.0%) |
Esophagus | 5 (16.7%) |
Upper GI tract | 3 (10.0%) |
cGVHD severity | |
Severe | 20 (66.7%) |
Moderate | 9 (30.0%) |
Mild | 1 (3.3%) |
Prior lines of therapies | |
1 | 3 (10.0%) |
2 | 8 (26.7%) |
3 | 6 (20.0%) |
4 | 10 (33.3%) |
5 | 3 (10.0%) |
Median, range | 3 (1–5) |
Refractory to the last systemic cGVHD treatment prior study enrollment | 23 (76.7%) |
Prior systemic cGVHD therapy type | |
Prednisone/methylprednisolone | 30 (100.0%) |
Cyclosporine | 19 (63.3%) |
Tacrolimus | 17 (56.7%) |
Ruxolitinib | 16 (53.3%) |
Mycophenolate mofetil | 13 (43.3%) |
Methotrexate | 9 (30.0%) |
Sirolimus | 6 (20.0%) |
Ibrutinib | 4 (13.3%) |
Imatinib | 3 (10.0%) |
Azathioprine | 3 (10.0%) |
Rituximab | 1 (3.3%) |
Stem cells (unspecified) | 1 (3.3%) |
Zanubrutinib | 1 (3.3%) |
Cyclophosphamide | 1 (3.3%) |
TNF receptor 2-Fc fusion protein | 1 (3.3%) |
Concomitant systemic cGVHD medications on cycle 1 day 1 (≥ 10% patients) | |
Prednisone/methylprednisolone | 30 (100.0%) |
Tacrolimus | 13 (43.3%) |
Cyclosporine | 7 (23.3%) |
Mycophenolate mofetil | 5 (16.7%) |