Baseline characteristics
| . | Prednisone (n = 64), N (%) . | Sirolimus (n = 58), N (%) . | Total (n = 122), N (%) . |
|---|---|---|---|
| AA score | |||
| AA1 | 45 (70.3%) | 38 (65.5%) | 83 (68.0%) |
| AA2 | 19 (29.7%) | 20 (34.5%) | 39 (32.0%) |
| Male sex | 33 (51.6%) | 37 (63.8%) | 70 (57.4%) |
| Age, y | |||
| Median (range) | 52.4 (0.9-72.7) | 58.0 (7.8-74.7) | 54.6 (0.9-74.7) |
| Pediatric (<18 y) | 3 (4.7%) | 1 (1.7%) | 4 (3.3%) |
| Median days of follow-up since enrollment (range) | 365.0 (13.0-365.0) | 365.0 (1.0-365.0) | 365.0 (1.0-365.0) |
| Skin GVHD stage at enrollment* | |||
| 0 | 19 (29.7%) | 20 (34.5%) | 39 (32.0%) |
| 1 | 11 (17.2%) | 8 (13.8%) | 19 (15.6%) |
| 2 | 13 (20.3%) | 14 (24.1%) | 27 (22.1%) |
| 3 | 21 (32.8%) | 16 (27.6%) | 37 (30.3%) |
| Upper GI GVHD stage at enrollment† | |||
| 0 | 36 (56.3%) | 32 (55.2%) | 68 (55.7%) |
| 1 | 28 (43.8%) | 26 (44.8%) | 54 (44.3%) |
| Lower GI GVHD stage at enrollment‡ | |||
| 0 | 56 (87.5%) | 56 (96.6%) | 112 (91.8%) |
| 1 | 7 (10.9%) | 2 (3.4%) | 9 (7.4%) |
| 2 | 1 (1.6%) | 0 (0.0%) | 1 (0.8%) |
| Liver GVHD stage at enrollment¶ | |||
| 0 | 63 (98.4%) | 58 (100.0%) | 121 (99.2%) |
| 1 | 1 (1.6%) | 0 (0.0%) | 1 (0.8%) |
| MN risk category | |||
| Stage 1-3 Skin | 32 (50.0%) | 31 (53.4%) | 63 (51.6%) |
| Stage 1-2 GI | 19 (29.7%) | 20 (34.5%) | 39 (32.0%) |
| Stage 1-3 Skin and stage 1 GI | 12 (18.8%) | 7 (12.1%) | 19 (15.6%) |
| Stage 1-3 Skin and stage 1-4 Liver | 1 (1.6%) | 0 (0.0%) | 1 (0.8%) |
| Acute GVHD grade at enrollment (consensus conference) | |||
| I | 16 (25.0%) | 16 (27.6%) | 32 (26.2%) |
| II | 47 (73.4%) | 42 (72.4%) | 89 (73.0%) |
| III | 1 (1.6%) | 0 (0.0%) | 1 (0.8%) |
| Organ involvement at enrollment | |||
| Skin stage 1 only | 6 (9.4%) | 5 (8.6%) | 11 (9.0%) |
| Skin stage 2 only | 8 (12.5%) | 11 (19.0%) | 19 (15.6%) |
| Skin stage 3 only | 18 (28.1%) | 15 (25.9%) | 33 (27.0%) |
| Upper GI only | 13 (20.3%) | 18 (31.0%) | 31 (25.4%) |
| Lower GI only | 1 (1.6%) | 1 (1.7%) | 2 (1.6%) |
| Multiple organs | 18 (28.1%) | 8 (13.8%) | 26 (21.3%) |
| Patients using topical steroids at enrollment | 36 (56.3%) | 32 (55.2%) | 68 (55.7%) |
| Donor type | |||
| Related BM or PB | 31 (48.4%) | 23 (39.7%) | 54 (44.3%) |
| Unrelated BM or PB | 29 (45.3%) | 32 (55.2%) | 61 (50.0%) |
| Unrelated Cord Blood | 4 (6.3%) | 3 (5.2%) | 7 (5.7%) |
| Karnofsky Performance Score | |||
| 90-100 | 42 (65.6%) | 26 (44.8%) | 68 (55.7%) |
| <90 | 21 (32.8%) | 32 (55.2%) | 53 (43.4%) |
| Missing | 1 (1.6%) | 0 (0.0%) | 1 (0.8%) |
| Primary disease | |||
| Acute leukemias | 34 (53.1%) | 28 (48.3%) | 62 (50.8%) |
| Hodgkin and non-Hodgkin lymphomas | 9 (14.1%) | 3 (5.2%) | 12 (9.8%) |
| Plasma cell disorder/multiple myeloma | 2 (3.1%) | 2 (3.4%) | 4 (3.3%) |
| Other leukemias | 7 (10.9%) | 6 (10.3%) | 13 (10.7%) |
| Nonmalignant disorders | 2 (3.1%) | 0 (0.0%) | 2 (1.6%) |
| Myelodysplastic/myeloproliferative disorders | 10 (15.6%) | 19 (32.8%) | 29 (23.8%) |
| Conditioning regimen intensity | |||
| Myeloablative | 35 (54.7%) | 32 (55.2%) | 67 (54.9%) |
| NMA/RIC | 29 (45.3%) | 26 (44.8%) | 55 (45.1%) |
| HLA match score | |||
| 8/8 BM or PB | 46 (71.9%) | 46 (79.3%) | 92 (75.4%) |
| 7/8 BM or PB | 2 (3.1%) | 3 (5.2%) | 5 (4.1%) |
| ≤6/8 BM or PB | 12 (18.8%) | 6 (10.3%) | 18 (14.8%) |
| Cord blood | 4 (6.3%) | 3 (5.2%) | 7 (5.7%) |
| Graft source | |||
| Bone marrow | 14 (21.9%) | 8 (13.8%) | 22 (18.0%) |
| Peripheral blood | 46 (71.9%) | 47 (81.0%) | 93 (76.2%) |
| Cord blood | 4 (6.3%) | 3 (5.2%) | 7 (5.7%) |
| GVHD prophylaxis | |||
| CNI-based | 49 (76.6%) | 44 (75.9%) | 93 (76.2%) |
| Post-CY + other(s) | 15 (23.4%) | 14 (24.1%) | 29 (23.8%) |
| Donor-recipient CMV status | |||
| ± | 18 (28.1%) | 25 (43.1%) | 43 (35.2%) |
| Others | 42 (65.6%) | 30 (51.7%) | 72 (59.0%) |
| Cord blood | 4 (6.3%) | 3 (5.2%) | 7 (5.7%) |
| Donor-recipient sex matching | |||
| Female donor, male recipient | 12 (18.8%) | 9 (15.5%) | 21 (17.2%) |
| Others | 52 (81.3%) | 49 (84.5%) | 101 (82.8%) |
| . | Prednisone (n = 64), N (%) . | Sirolimus (n = 58), N (%) . | Total (n = 122), N (%) . |
|---|---|---|---|
| AA score | |||
| AA1 | 45 (70.3%) | 38 (65.5%) | 83 (68.0%) |
| AA2 | 19 (29.7%) | 20 (34.5%) | 39 (32.0%) |
| Male sex | 33 (51.6%) | 37 (63.8%) | 70 (57.4%) |
| Age, y | |||
| Median (range) | 52.4 (0.9-72.7) | 58.0 (7.8-74.7) | 54.6 (0.9-74.7) |
| Pediatric (<18 y) | 3 (4.7%) | 1 (1.7%) | 4 (3.3%) |
| Median days of follow-up since enrollment (range) | 365.0 (13.0-365.0) | 365.0 (1.0-365.0) | 365.0 (1.0-365.0) |
| Skin GVHD stage at enrollment* | |||
| 0 | 19 (29.7%) | 20 (34.5%) | 39 (32.0%) |
| 1 | 11 (17.2%) | 8 (13.8%) | 19 (15.6%) |
| 2 | 13 (20.3%) | 14 (24.1%) | 27 (22.1%) |
| 3 | 21 (32.8%) | 16 (27.6%) | 37 (30.3%) |
| Upper GI GVHD stage at enrollment† | |||
| 0 | 36 (56.3%) | 32 (55.2%) | 68 (55.7%) |
| 1 | 28 (43.8%) | 26 (44.8%) | 54 (44.3%) |
| Lower GI GVHD stage at enrollment‡ | |||
| 0 | 56 (87.5%) | 56 (96.6%) | 112 (91.8%) |
| 1 | 7 (10.9%) | 2 (3.4%) | 9 (7.4%) |
| 2 | 1 (1.6%) | 0 (0.0%) | 1 (0.8%) |
| Liver GVHD stage at enrollment¶ | |||
| 0 | 63 (98.4%) | 58 (100.0%) | 121 (99.2%) |
| 1 | 1 (1.6%) | 0 (0.0%) | 1 (0.8%) |
| MN risk category | |||
| Stage 1-3 Skin | 32 (50.0%) | 31 (53.4%) | 63 (51.6%) |
| Stage 1-2 GI | 19 (29.7%) | 20 (34.5%) | 39 (32.0%) |
| Stage 1-3 Skin and stage 1 GI | 12 (18.8%) | 7 (12.1%) | 19 (15.6%) |
| Stage 1-3 Skin and stage 1-4 Liver | 1 (1.6%) | 0 (0.0%) | 1 (0.8%) |
| Acute GVHD grade at enrollment (consensus conference) | |||
| I | 16 (25.0%) | 16 (27.6%) | 32 (26.2%) |
| II | 47 (73.4%) | 42 (72.4%) | 89 (73.0%) |
| III | 1 (1.6%) | 0 (0.0%) | 1 (0.8%) |
| Organ involvement at enrollment | |||
| Skin stage 1 only | 6 (9.4%) | 5 (8.6%) | 11 (9.0%) |
| Skin stage 2 only | 8 (12.5%) | 11 (19.0%) | 19 (15.6%) |
| Skin stage 3 only | 18 (28.1%) | 15 (25.9%) | 33 (27.0%) |
| Upper GI only | 13 (20.3%) | 18 (31.0%) | 31 (25.4%) |
| Lower GI only | 1 (1.6%) | 1 (1.7%) | 2 (1.6%) |
| Multiple organs | 18 (28.1%) | 8 (13.8%) | 26 (21.3%) |
| Patients using topical steroids at enrollment | 36 (56.3%) | 32 (55.2%) | 68 (55.7%) |
| Donor type | |||
| Related BM or PB | 31 (48.4%) | 23 (39.7%) | 54 (44.3%) |
| Unrelated BM or PB | 29 (45.3%) | 32 (55.2%) | 61 (50.0%) |
| Unrelated Cord Blood | 4 (6.3%) | 3 (5.2%) | 7 (5.7%) |
| Karnofsky Performance Score | |||
| 90-100 | 42 (65.6%) | 26 (44.8%) | 68 (55.7%) |
| <90 | 21 (32.8%) | 32 (55.2%) | 53 (43.4%) |
| Missing | 1 (1.6%) | 0 (0.0%) | 1 (0.8%) |
| Primary disease | |||
| Acute leukemias | 34 (53.1%) | 28 (48.3%) | 62 (50.8%) |
| Hodgkin and non-Hodgkin lymphomas | 9 (14.1%) | 3 (5.2%) | 12 (9.8%) |
| Plasma cell disorder/multiple myeloma | 2 (3.1%) | 2 (3.4%) | 4 (3.3%) |
| Other leukemias | 7 (10.9%) | 6 (10.3%) | 13 (10.7%) |
| Nonmalignant disorders | 2 (3.1%) | 0 (0.0%) | 2 (1.6%) |
| Myelodysplastic/myeloproliferative disorders | 10 (15.6%) | 19 (32.8%) | 29 (23.8%) |
| Conditioning regimen intensity | |||
| Myeloablative | 35 (54.7%) | 32 (55.2%) | 67 (54.9%) |
| NMA/RIC | 29 (45.3%) | 26 (44.8%) | 55 (45.1%) |
| HLA match score | |||
| 8/8 BM or PB | 46 (71.9%) | 46 (79.3%) | 92 (75.4%) |
| 7/8 BM or PB | 2 (3.1%) | 3 (5.2%) | 5 (4.1%) |
| ≤6/8 BM or PB | 12 (18.8%) | 6 (10.3%) | 18 (14.8%) |
| Cord blood | 4 (6.3%) | 3 (5.2%) | 7 (5.7%) |
| Graft source | |||
| Bone marrow | 14 (21.9%) | 8 (13.8%) | 22 (18.0%) |
| Peripheral blood | 46 (71.9%) | 47 (81.0%) | 93 (76.2%) |
| Cord blood | 4 (6.3%) | 3 (5.2%) | 7 (5.7%) |
| GVHD prophylaxis | |||
| CNI-based | 49 (76.6%) | 44 (75.9%) | 93 (76.2%) |
| Post-CY + other(s) | 15 (23.4%) | 14 (24.1%) | 29 (23.8%) |
| Donor-recipient CMV status | |||
| ± | 18 (28.1%) | 25 (43.1%) | 43 (35.2%) |
| Others | 42 (65.6%) | 30 (51.7%) | 72 (59.0%) |
| Cord blood | 4 (6.3%) | 3 (5.2%) | 7 (5.7%) |
| Donor-recipient sex matching | |||
| Female donor, male recipient | 12 (18.8%) | 9 (15.5%) | 21 (17.2%) |
| Others | 52 (81.3%) | 49 (84.5%) | 101 (82.8%) |
BM, bone marrow; BSA, body surface area; CB, cord blood; CNI, calcineurin inhibitor; CMV, cytomegalovirus; CY, cyclophosphamide; GI, gastrointestinal; GVHD, graft-versus-host disease; NMA/RIC, nonmyeloablative/reduced-intensity conditioning; PB, peripheral blood.
Stage 0 = no active (erythematous) GVHD rash, stage 1, maculopapular rash <25% BSA; stage 2, maculopapular rash 25% to 50% BSA; stage 3, maculopapular rash >50% BSA.
Stage 0, no or intermittent nausea, vomiting, or anorexia; stage 1, persistent nausea, vomiting, or anorexia.
Stage 0, diarrhea, adult: 0 to 499 mL/day, 0 to 2 episodes/day; child: 0 to 9 mL/kg/day, 0 to 3 episodes/day; stage 1, diarrhea, adult: 500 to 999 mL/day, 3 to 4 episodes/day; child: 10 to 19.9 mL/kg/day, 4 to 6 episodes/day; stage 2, diarrhea, adult: 1000 to 1500 mL/day, 5 to 7 episodes/day; child: 20 to 30 mL/kg/day, 7 to 10 episodes/day
Stage 0, bilirubin <2.0 mg/dL; stage 1, bilirubin 2.0-3.0 mg/dL.