Baseline characteristics
| Characteristic . | All HSCT (N = 28) . | Mild-moderate-severe VOD/SOS (n = 11) . | Very severe VOD/SOS (n = 17) . |
|---|---|---|---|
| Sex, n (%) | |||
| Male | 22 (79) | 9 (82) | 13 (76) |
| Female | 6 (21) | 2 (18) | 4 (24) |
| Age at time of HSCT, mean (range), y | 58 (26-75) | 55 (29-72) | 55 (26-75) |
| Race, n (%) | |||
| White | 22 (79) | 11 (100) | 11 (65) |
| Asian | 2 (7) | 0 | 2 (12) |
| Unknown | 4 (14) | 0 | 4 (23) |
| Body mass index, mean (range), kg/m2 | 25.35 (17.5-35.5) | 25.78 (20.55-31.4) | 25.1 (17.6-35.5) |
| Transplant type, n (%) | |||
| Allogeneic | 28 (100) | 11 (100) | 17 (100) |
| Donor characteristics, n (%) | |||
| Matched, related | 5 (18) | 2 (18) | 3 (18) |
| Matched, unrelated | 17 (62) | 7 (64) | 10 (58) |
| Mismatched, related | 3 (10) | 0 | 3 (18) |
| Mismatched, unrelated | 3 (10) | 2 (18) | 1 (6) |
| Conditioning agents, n (%) | |||
| Cyclophosphamide/TBI 1200 cGy | 2 (7) | 1 (9) | 1 (6) |
| Cyclophosphamide, fludarabine/TBI 200 cGy | 1 (4) | 1 (9) | 0 |
| Myeloablative busulfan and fludarabine | 10 (36) | 1 (9) | 9 (53) |
| Reduced-intensity busulfan and fludarabine | 12 (43) | 7 (64) | 5 (29) |
| Melphalan and fludarabine | 3 (10) | 1 (9) | 2 (12) |
| GVHD prophylaxis, n (%) | |||
| Cyclophosphamide | 2 (7) | 1 (9) | 1 (6) |
| Cyclophosphamide, tacrolimus, and mycophenolate mofetil | 3 (10) | 1 (9) | 2 (12) |
| Tacrolimus and sirolimus | 9 (32) | 2 (18) | 7 (40) |
| Tacrolimus and methotrexate | 5 (18) | 2 (18) | 3 (18) |
| Tacrolimus, sirolimus, and methotrexate | 8 (29) | 5 (46) | 3 (18) |
| Tacrolimus and mycophenolate mofetil | 1 (4) | 0 | 1 (6) |
| Primary disease, n (%) | |||
| AML | 7 (25) | 2 (18) | 6 (34) |
| ALL | 8 (29) | 4 (36) | 3 (18) |
| MDS | 6 (22) | 3 (27) | 3 (18) |
| MPN | 3 (10) | 1 (9) | 2 (12) |
| Mixed MDS/MPN | 1 (4) | 0 | 1 (6) |
| NHL | 3 (10) | 1 (9) | 2 (12) |
| Type of conditioning regimen, n (%) | |||
| MAC | 12 (43) | 2 (18) | 10 (59) |
| RIC | 16 (57) | 9 (82) | 7 (41) |
| Prior HSCTs, n (%) | |||
| None | 24 (86) | 8 (73) | 16 (94) |
| ≥1 | 4 (14) | 3 (27) | 1 (6) |
| VOD/SOS onset after day 21, n (%) | 16 (57) | 7 (64) | 9 (53) |
| Time from diagnosis to start of defibrotide, n (%) | |||
| Day of diagnosis | 20 (71) | 8 (73) | 12 (70) |
| Day 1 | 4 (14) | 1 (9) | 3 (18) |
| Day 2 | 1 (4) | 1 (9) | 0 |
| Day 3 | 1 (4) | 0 | 1 (6) |
| Prophylactic defibrotide with VOD | 2 (7) | 1 (9) | 1 (6) |
| Immunoconjugate treatment history, n (%) | |||
| Inotuzumab ozogamicin | 4 (14) | 3 (27) | 1 (6) |
| Gemtuzumab ozogamicin | 0 | 0 | 0 |
| History of liver impairment, n (%) | |||
| Hepatitis C infection | 1 (4) | 0 | 1 (6) |
| Alcohol dependence | 2 (7) | 1 (9) | 1 (6) |
| Oncologic liver disease involvement | 1 (4) | 0 | 1 (6) |
| Gilbert’s syndrome | 1 (4) | 1 (9) | 0 |
| Fatty liver (nonalcoholic) | 1 (4) | 1 (9) | 0 |
| Cholangitis | 1 (4) | 0 | 1 (6) |
| Inotuzumab-induced transaminitis | 1 (4) | 1 (9) | 0 |
| Graft source | |||
| Bone marrow | 6 (21) | 1 (9) | 5 (29) |
| Peripheral blood | 22 (79) | 10 (91) | 12 (71) |
| Characteristic . | All HSCT (N = 28) . | Mild-moderate-severe VOD/SOS (n = 11) . | Very severe VOD/SOS (n = 17) . |
|---|---|---|---|
| Sex, n (%) | |||
| Male | 22 (79) | 9 (82) | 13 (76) |
| Female | 6 (21) | 2 (18) | 4 (24) |
| Age at time of HSCT, mean (range), y | 58 (26-75) | 55 (29-72) | 55 (26-75) |
| Race, n (%) | |||
| White | 22 (79) | 11 (100) | 11 (65) |
| Asian | 2 (7) | 0 | 2 (12) |
| Unknown | 4 (14) | 0 | 4 (23) |
| Body mass index, mean (range), kg/m2 | 25.35 (17.5-35.5) | 25.78 (20.55-31.4) | 25.1 (17.6-35.5) |
| Transplant type, n (%) | |||
| Allogeneic | 28 (100) | 11 (100) | 17 (100) |
| Donor characteristics, n (%) | |||
| Matched, related | 5 (18) | 2 (18) | 3 (18) |
| Matched, unrelated | 17 (62) | 7 (64) | 10 (58) |
| Mismatched, related | 3 (10) | 0 | 3 (18) |
| Mismatched, unrelated | 3 (10) | 2 (18) | 1 (6) |
| Conditioning agents, n (%) | |||
| Cyclophosphamide/TBI 1200 cGy | 2 (7) | 1 (9) | 1 (6) |
| Cyclophosphamide, fludarabine/TBI 200 cGy | 1 (4) | 1 (9) | 0 |
| Myeloablative busulfan and fludarabine | 10 (36) | 1 (9) | 9 (53) |
| Reduced-intensity busulfan and fludarabine | 12 (43) | 7 (64) | 5 (29) |
| Melphalan and fludarabine | 3 (10) | 1 (9) | 2 (12) |
| GVHD prophylaxis, n (%) | |||
| Cyclophosphamide | 2 (7) | 1 (9) | 1 (6) |
| Cyclophosphamide, tacrolimus, and mycophenolate mofetil | 3 (10) | 1 (9) | 2 (12) |
| Tacrolimus and sirolimus | 9 (32) | 2 (18) | 7 (40) |
| Tacrolimus and methotrexate | 5 (18) | 2 (18) | 3 (18) |
| Tacrolimus, sirolimus, and methotrexate | 8 (29) | 5 (46) | 3 (18) |
| Tacrolimus and mycophenolate mofetil | 1 (4) | 0 | 1 (6) |
| Primary disease, n (%) | |||
| AML | 7 (25) | 2 (18) | 6 (34) |
| ALL | 8 (29) | 4 (36) | 3 (18) |
| MDS | 6 (22) | 3 (27) | 3 (18) |
| MPN | 3 (10) | 1 (9) | 2 (12) |
| Mixed MDS/MPN | 1 (4) | 0 | 1 (6) |
| NHL | 3 (10) | 1 (9) | 2 (12) |
| Type of conditioning regimen, n (%) | |||
| MAC | 12 (43) | 2 (18) | 10 (59) |
| RIC | 16 (57) | 9 (82) | 7 (41) |
| Prior HSCTs, n (%) | |||
| None | 24 (86) | 8 (73) | 16 (94) |
| ≥1 | 4 (14) | 3 (27) | 1 (6) |
| VOD/SOS onset after day 21, n (%) | 16 (57) | 7 (64) | 9 (53) |
| Time from diagnosis to start of defibrotide, n (%) | |||
| Day of diagnosis | 20 (71) | 8 (73) | 12 (70) |
| Day 1 | 4 (14) | 1 (9) | 3 (18) |
| Day 2 | 1 (4) | 1 (9) | 0 |
| Day 3 | 1 (4) | 0 | 1 (6) |
| Prophylactic defibrotide with VOD | 2 (7) | 1 (9) | 1 (6) |
| Immunoconjugate treatment history, n (%) | |||
| Inotuzumab ozogamicin | 4 (14) | 3 (27) | 1 (6) |
| Gemtuzumab ozogamicin | 0 | 0 | 0 |
| History of liver impairment, n (%) | |||
| Hepatitis C infection | 1 (4) | 0 | 1 (6) |
| Alcohol dependence | 2 (7) | 1 (9) | 1 (6) |
| Oncologic liver disease involvement | 1 (4) | 0 | 1 (6) |
| Gilbert’s syndrome | 1 (4) | 1 (9) | 0 |
| Fatty liver (nonalcoholic) | 1 (4) | 1 (9) | 0 |
| Cholangitis | 1 (4) | 0 | 1 (6) |
| Inotuzumab-induced transaminitis | 1 (4) | 1 (9) | 0 |
| Graft source | |||
| Bone marrow | 6 (21) | 1 (9) | 5 (29) |
| Peripheral blood | 22 (79) | 10 (91) | 12 (71) |
ALL, acute lymphoblastic leukemia; AML, acute myeloid leukemia; MDS, myelodysplastic syndrome; MPN, myeloproliferative neoplasms; NHL, non-Hodgkin lymphoma; TBI, total body irradiation.