Regimen | No. of ALL | Mean age (range) | Donor | Stem cell source | Disease status at HSCT | Survival rate | Relapse rate | TRM/NRM | aGVHD | cGVHD | Remarks | Reference |
---|---|---|---|---|---|---|---|---|---|---|---|---|
ETP 60 mg/kg × 1 + TBI 1.2 Gy × 11 | 13 | 16 (6–36) | MRD | BM | CR2, IF, or relapse | 54 % (≧100 d) | 15 % | 31 % | 23 % | ND | It was not clear whether ETP 60 mg/kg was better than ETP 30 mg/kg according to a phase I/II (dose-finding) study using 25 to 70 mg/kg of ETP. | Blume KG, et al. Blood 1987;69:1015. |
ETP 50 to 70 mg/kg × 1 + TBI 2 Gy × 6 | 17 | 19 (4–38) | MRD | BM | Non-CR1, IF, or relapse except 1 | 65 % (≧182 d) | 6 % | 35 % | 85 % | 50 % | Anti-leukemic effect was observed in a phase I/II study with ETP + BI, but the rejection rate was high. Thus, the immunosuppressive effect was worse than CY + TBI. A dose of more than 60 mg/kg of ETP was too toxic. | Schmitz N, et al. Blood 1988;72: 1567. |
ETP 36 mg/kg or 52 mg/kg × 1 + CY 67 mg/kg or 103 mg/kg × 1 + TBI 2 Gy × 6 | 7 | 15 (6–35) | MRD | BM | Relapse | 0 % (≧899 d) | ND | ND | ND | ETP (36 mg/kg) + CY (67 mg/kg) + TBI was well tolerated for allogeneic HSCT (phase I study). | Petersen FB, et al. Bone Marrow Transplant. 1992; 10:83. | |
ETP 60 mg/kg × 1 + TBI 1.2 Gy × 11 | 25/122 | 2–48 | MRD | BM | CR2: 8 | 3-Y DFS: 63 % | 8 % | 4 % | ND | ND | ETP + TBI appeared to be better for a good-risk group than for a poor-risk one according to a randomized controlled study. Hepatic toxicity and severe mucositis were marked with ETP of 60 mg/kg. The age range for the patients with ALL was shown, but the mean was unclear. | Blume KG, et al. Blood 1993;81: 2187. |
CR3, Non-CR: 17 | 12 % | 40 % | 12 % | ND | ND | |||||||
BU 1 mg/kg × 16 + CY 60 mg/kg × 2 | 23/122 | 5–48 | CR2: 6 | 4 % | 17 % | 4 % | ND | ND | ||||
CR3, Non-CR: 17 | 17 % | 34 % | 22 % | ND | ND | |||||||
ETP 60 mg/kg × 1 + TBI 1.2 Gy × 11 | 34 | 27 (1–45) | MRD | BM | CR1 | 3-Y DFS: 64 % | 12 % | ND | 18 % (gr.≧II) | 38 % | A relatively good outcome was observed in a phase II study, but relapse and TRM rates were high. Thirty of the 34 patients were high-risk. | Snyder DS, et al. Blood 1993;82: 2920. |
High-risk | ||||||||||||
<20 y 3-Y DFS : 100 % | 0 % | |||||||||||
≧20 y 3-Y DFS: 54 % | 17 % | |||||||||||
ETP 30–60 mg/kg × 1 + CY 60–200 mg/kg × 2 + TBI 1.2Gy × 11 (1.5 Gy × 8) | 20/44 | 18 (1–54) | MRD | BM | ND | Early death: standard-risk 2/18 high-risk 2/26 | ND | ND | ND | ND | Fourty-four patients with hematological malignancies were analyzed in a retrospective study. Among them, 20 patients were ALL. Sixty to 65 mg/kg of ETP resulted in a fatal toxicity, but 30 to 50 mg/kg did not. Fifty mg/kg of ETP was considered to be the maximally tolerated dose. | Spitzer TR, et al. Int. J. Radiat. Oncol. Biol. Phys. 1994;29:39 |
ETP 25–60 mg/kg × 2 | 10/32 | 18 (3–49) | MRD | BM | ND | Early death: standard-risk 1/12 high-risk 5/20 | ND | |||||
BU 1 mg/kg × 12–16 | ||||||||||||
Standard | 20 (1–39) | MRD | BM | CR2: 20 | 7-Y OS: 52 % | 34 % | 25 % | 48 % (gr.≧II) | 41 % | A retrospective study suggested that high-dose conditioning regimens did not improve the outcome of patients transplanted for high-risk leukemia. | Mengarelli A, et al. Haematologica 2002;87:52. | |
TBI 2 Gy × 6 + CY 120 mg/kg × 2 | 7/38 | CR3: 9 | ||||||||||
1st Rel.: 5 | ||||||||||||
Adv.: 4 | ||||||||||||
BU 16 mg/kg × 4 + CY 120 mg/kg × 2 | 24/38 | |||||||||||
BU 16 mg/kg × 4 + CY 120 mg/kg × 4 | 7/38 | |||||||||||
Alternative | 23 (3–44) | MRD | BM: 60 | CR2: 47 | 7-Y OS: 25 % | 58 % | 32 % | 47 % (gr.≧II) | 44 % | |||
ETP 60 mg/kg × 1 + TBI 2 Gy × 6 TBI 2 Gy × 6 + CY 120 mg/kg × 2 | 43/66 | PB: 6 | CR3: 9 | |||||||||
1st Rel.: 6 | ||||||||||||
Adv.: 13 | ||||||||||||
BU 16 mg/kg × 4 + CY 120 mg/kg × 2 +IDA 42 mg/m2 × 2 | ||||||||||||
BU-CY + VP 20 mg/kg × 1 | ||||||||||||
BU-CY + Ara-C 2 g/m2 × 4 | ||||||||||||
ETP 40 mg/kg × 1 + CY 60 mg/kg × 2 + TBI 2 Gy × 6 | 39 | 34 (15–52) | MRD: 35 | BM | CR1 | 6-Y OS: 41 % | 10 % | 15 % | ND | ND | Autologous HSCT by ETP + CY + TBI regimen showed 41 % of 6-Y OS in a prospective study, whereas allogeneic BMT with the same regimen showed a 6-Y OS of 75 %. This result suggested a possibility of GVL effect. ETP was administered in 4 consecutive infusions of 10 mg/kg lasting 2 hours each. | Hunault M, et al. Blood 2004;104:3028. |
MUD: 4 | (<50 years old: 75 %) | |||||||||||
ETP 15 mg/kg × 2 + CY 60 mg/kg × 2 + TBI 2 Gy × 6 | 37 | 26 (15–58) | MRD: 13 | BM: 3 | CR1: 28 | 3-Y OS: 89 % | 8 % | 5 % | 78 % (gr.≧II: 41 %) | 55 % (ext.: 36 %) | Excellent outcome was observed in addition to low relapse and TRM rates in a retrospective study. | Shigematsu A, et al. Biol. Blood Marrow Transplant. 2008;14:568. |
MUD: 18 | PB: 4 | CR2: 7 | ||||||||||
MMRD: 2 | CB: 1 | Non-CR: 2 | ||||||||||
MMUD: 4 | ||||||||||||
ETP 15 mg/kg × 2 + CY 60 mg/kg × 2 + TBI 2 Gy × 6 or 3 Gy × 4 | 35 | 28 (15–58) | MRD:16 | BM: 29 | CR1: 28 | 5-Y OS:82 % | 14 % | 3 % | 71 % (gr.≧II: 37 %) | 46 % (ext.: 30 %) | A retrospective analysis in Japan showed ETP + CY + TBI was associated with lower relapse and NRM rates, resulting in better survival than that with CY + TBI. | Shigematsu A, et al. Int. J. Hematol. 2011;94:463. |
MUD: 11 | PB: 6 | CR2:7 | ||||||||||
MMUD: 6 | ||||||||||||
Unknown: 2 | ||||||||||||
CY 60 mg/kg × 2 + TBI 2 Gy × 6 or 3 Gy × 4 | 494 | 34 (15–59) | MRD: 235 | BM: 405 | CR1: 414 | 5-Y OS: 55 % | 29 % | 16 % | 62 % (gr.≧II: 37 %) | 45 % (ext.: 27 %) | ||
MUD: 180 | PB: 89 | CR2: 80 | ||||||||||
MMRD: 1 | ||||||||||||
MMUD: 70 | ||||||||||||
Unknown: 2 | ||||||||||||
ETP 15 mg/kg × 2 + CY 60 mg/kg × 2 + TBI 2 Gy × 6 | 50 | 34 (17–49) | MRD: 26 | BM: 40 | CR1: 47 | 1-Y OS: 80 % | 10 % | 14 % | 66 % (gr.≧II: 58 %; gr.≧III: 12 %) | 56 % (ext.: 38 %) | A prospective multi-center phase II study in Japan confirmed the excellent outcome of ETP + CY + TBI for adult ALL patients. | Shimemastu A, et al. Transplant. Direct. 2015;1:1 |
MUD: 24 | PB: 10 | CR2: 3 | 2-Y OS: 67 % | |||||||||
1-Y EFS: 76 % | ||||||||||||
2-Y EFS: 65 % |