Table 2.
Overview of studies on therapy-refractory aGvHD
| Reference, year | Cohort | Diagnosis (n) | GvHD | Dose of MSC, cells/kg body weight | Response rate day +28 | Predicted survival | |
|---|---|---|---|---|---|---|---|
| Bader et al. [5], 2017 | n = 69 | Malignant (51) | aGvHD | 1–2 × 106 | CR = 31.9% | 6-month OS = 71 ± 6% | |
| ≤18 years:n = 51 | Nonmalignant (18) | II:n = 3 (4%) | PR = 50.7% | ||||
| >18 years:n = 18 | III:n = 25 (36%) IV:n = 41 (59%) |
OR = 82.6% | |||||
| Salmenniemi et al. [30], 2017 | n = 30 | Malignant and | aGvHD | 2 × 106 | CR = 23% | 6-month OS = 54% | |
| ≤18 years:n = 8 | nonmalignant | II:n = 2 (7%) | VGPR = 13% | 2-year OS = 29% | |||
| >18 years:n = 22 | III:n = 14 (47%) | PR = 17% | |||||
| IV:n = 10 (33%) cGvHD:n = 4 (13%) |
OS = 53% | ||||||
| Dotoli et al. [31], 2017 | n = 46 | Leukemia (22) | aGvHD | 6.81 × 106 | CR = 6.5% | 100-day OS = 34.4% | |
| ≤18 years:n = 16 | MDS (7) | III:n = 10 (21.7%) | PR = 43.5% | 2-year OS = 17.4% | |||
| >18 years:n = 30 | Nonmalignant (12) Others (5) |
IV:n = 36 (78.3%) | OR = 50% | ||||
| von Dalowski et al. [32], 2016 | 58 adults | Leukemia (39) | aGvHD | 0.99 × 106 | CR = 9% | 100-day OS = 34.5% | |
| MDS (5) | I:n = 1 (2%) | PR = 38% | 2-year OS = 16.6% | ||||
| Others (14) | II:n = 3 (5%) III:n = 8 (14%) IV:n = 46 (79%) |
OR = 47% | |||||
| Kurtzberg et al. [29], 2014 | 75 children | Leukemia (35) | aGvHD | 2 ×106 | OR = 61.3% | 100-day OS = 57.3% | |
| MDS (7) | B:n = 9 (12%) | ||||||
| Genetic disease (16) | C:n = 21 (28%) | ||||||
| Others (17) | D:n = 45 (60%) | ||||||
| Sánchez-Guijo et al. [26], 2014 | 25 adults | AML (6) | aGvHD | 1.1 × 106 | CR = 44% | 44% (11/25) alive after | |
| MDS (7) | II:n = 7 (28%) | PR = 27% | 12 months | ||||
| Others (12) | III:n = 13 (60%) IV:n = 3 (12%) |
OR = 71% | |||||
| Ball et al. [23], 2013 | 37 children | Leukemia (21) | aGvHD III-IV:n = 37 | 1–2×106 | CR = 65% | 6-year OS = 37% | |
| MDS (7) | PR = 21.5% | ||||||
| Nonmalignant (9) | OR = 86.5% | ||||||
| Introna et al. [28], 2014 | n = 40 | Malignant (36) | aGvHD | 1.5 × 106 | CR = 27.5% | 1-year OS = 50% | |
| <18 years:n = 15 | Nonmalignant (4) | II:n = 11 (27%) | PR = 40% | 2-year OS = 38.6% | |||
| >18 years:n = 25 | III-IV:n = 20 (50%) cGvHD:n = 3 (8%) overlap:n = 6 (15%) |
OR = 67.5% | |||||
| Resnick et al. [22], 2013 | n = 50 | Malignant (43) | aGvHD | 1.14 ± 0.47 × 106 | CR = 34% | 3.6-year DFS = 56% | |
| ≤18 years:n = 25 | Nonmalignant (7) | II—III:n = 8 (16%) | OR = 66% | ||||
| >18 years:n = 25 | IV:n = 42 (84%) | ||||||
| Hermann et al. [34], 2012 | 19 adults | Leukemia (15) | aGvHD:n = 12 (63%) | 1.7–2.3 × 106 | CR = 47.4% | 30-month OS 55% | |
| Other malignancies (4) | cGvHD:n = 7 (37%) | PR = 31.6% OR = 79% | |||||
| Prasad et al. [35], 2011 | 12 children | Malignant (7) | aGvHD | n = 10: 2 × 106 | CR = 17% | 2-year OS 40% | |
| Nonmalignant (5) | III:n = 5 (42%) | n = 2: 8 × 106 | PR = 50% | ||||
| IV:n = 7 (58%) | OR = 67% | ||||||
| Pérez-Simon et al. [36], 2011 | 18 adults | n/a | aGvHD | 1–2×106 | CR = 11% | 33% (6/18) alive at last | |
| II:n = 3 (17%) | PR = 50% | follow-up | |||||
| III–IV:n = 7 (39%) | cGvHD:n = 8 (44%) | OR = 61% | |||||
| Lucchini et al. [27], 2010 | 11 children | Leukemia (8) | aGvHD | 1.2 × 106 | CR = 23.8% | 73% (8/11) alive after | |
| Nonmalignant (3) | I–II:n = 4 (36.4%) | PR = 47.6% | 8 months | ||||
| III-IV:n = 4 (36.4%) cGvHD:n = 3 (27.2%) |
OS = 71.4% | ||||||
| von Bonin et al. [37], 2009 | 13 adults | Malignant (12) | aGvHD | 0.9 × 106 | OR = 45% | 45% (5/13) alive after | |
| SAA (1) | III:n = 11 (85%) | IV:n = 2 (15%) | 257 days | ||||
| Le Blanc et al. [21], 2008 | n = 55 | Leukemia (33) | aGvHD | 1.4 × 106 | CR = 54.5% | 2-year OS = 35% | |
| ≤18 years:n = 25 | MDS (6) | II:n = 5 (9%) | PR = 16% | ||||
| >18 years:n = 30 | Nonmalignant (10) | III:n = 25 (45.45%) | OR =70.5% | ||||
| Others (6) | IV:n = 25 (45.45%) |
aGvHD, acute graft-versus-host disease; cGvHD, chronic graft-versus-host disease; MSC, mesenchymal stromal cell; AML, acute myeloid leukemia; MDS, myelodysplastic syndrome; SAA, severe aplastic anemia; OS, overall survival; DFS, disease-free survival; CR, complete response; VGPR, very good partial response; PR, partial response; OR, overall response; n/a, not available.