This table describes key modern studies investigating the optimal use of HSCT in the management of R/R ALL.
| Reference . | Design . | N; Median age; Range . | Indication . | Staging . | Graft source . | Conditioning . | GVHD prophylaxis . | aGVHD . | cGVHD . | Relapse . | TRM . | Survival . |
|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Bethge et al (2022)19 | Prospective | 60; 18.5; 1-63 | ALL AML MDS MPS MM NM ST | CR1 20% >CR1 22% NR 13% | MMRD | ATG/Flu/ TT/Mel | TCRαβ/ CD19 depletion | Grade II-IV 10% Grade III-IV 0% | Overall 31% Severe 8% | 20% | 17% | 2 year OS63% DFS 50% GRFS 39% |
| Pulsipher et al (2022)17 | Prospective | 51; 35; 6-61 | ALL AML MDS | CR1 53% CR2 37.3% | MMRD PBSC | MAC-TBI 31%, MAC-Bu 22% RIC-Mel 43% RIC-TLI 4% | TCRαβ/ CD19 depletion | 2 year OS 75% DFS 75% EFS 69% | ||||
| Peters et al (2021)33 | Randomized, controlled, international, multicentered, phase 3 | 417; 4-21 | ALL | CR1 54% CR2 40% CR3 4% | MSD or MD BM, PBSC or Cord | MAC-TBI 50% MAC-Bu 24% Treo 23% Other 3% | MSD-CSA only MD-ATG/ MTX/ CSA | Grade II-IV TBI 37% Chemo29% | Overall-TBI 16% Overall-Chemo 11% | TBI-12% Chemo-33% | TBI-2% Chemo-9% | 2-year OS-TBI 91% OS-chemo 75% GRFS-TBI 72% GRFS-chemo 51% |
| Ruggeri et al (2021)16 | Retrospective | 180; 9.25 | ALL | CR1 24% CR2 45% CR3 12% NR 19% | MMRD BM or PBSC | MAC-TBI 25.6% MAC-Chemo 51.6% RIC 22.78% | PtCy | Grade II-IV 28.3% Grade III-IV 12.4% | Overall 21.9% Extensive 9.5% | CR1 25% CR2 37% CR3 50% NR 70% | 19.6% | 2 year OS 50.8% LFS 38.5% GRFS 29% |
| Symons et al (2020)44 | Prospective, single center, phase 2 | 96; 42; 1-65 | ALL AML MDS Lymphoma | CR1 41% >CR1 18% | MMRDBM | MAC-TBI MAC-Bu | PtCy | Grade II-IV 11% Grade III-IV 4% | Overall 15% Moderate to severe 6% | 43% | 11% | 3 year OS 54% EFS 48% |
| Bertaina et al (2018)43 | Retrospective | 98: 6.6; 0.1-17.3 | AL | CR1 43% CR2 47% Other CR 10% | MMRD PBSC | MAC-TBI 74% | TCRαβ/ CD19 depletion | Grade II-IV 16% Grade III-IV 0% | Overall 6%, Extensive 1% | 29% | 9% | 5 year OS 68% LFS 62% |
| Locatelli et al (2017)18 | Prospective | 80; 9.7; 0.9-20.9 | ALL AML | CR1 39% CR2 56% | MMRD PBSC | TBI/TT/Flu TBI/TT/Mel Bu/TT/Flu Bu/Cy/Mel with ATG/Ritux | TCRαβ/ CD19 depletion | Grade I-II 30% | Limited 5% | 24% | 5% | 5 year OS 72% DFS 71% |
| Reference . | Design . | N; Median age; Range . | Indication . | Staging . | Graft source . | Conditioning . | GVHD prophylaxis . | aGVHD . | cGVHD . | Relapse . | TRM . | Survival . |
|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Bethge et al (2022)19 | Prospective | 60; 18.5; 1-63 | ALL AML MDS MPS MM NM ST | CR1 20% >CR1 22% NR 13% | MMRD | ATG/Flu/ TT/Mel | TCRαβ/ CD19 depletion | Grade II-IV 10% Grade III-IV 0% | Overall 31% Severe 8% | 20% | 17% | 2 year OS63% DFS 50% GRFS 39% |
| Pulsipher et al (2022)17 | Prospective | 51; 35; 6-61 | ALL AML MDS | CR1 53% CR2 37.3% | MMRD PBSC | MAC-TBI 31%, MAC-Bu 22% RIC-Mel 43% RIC-TLI 4% | TCRαβ/ CD19 depletion | 2 year OS 75% DFS 75% EFS 69% | ||||
| Peters et al (2021)33 | Randomized, controlled, international, multicentered, phase 3 | 417; 4-21 | ALL | CR1 54% CR2 40% CR3 4% | MSD or MD BM, PBSC or Cord | MAC-TBI 50% MAC-Bu 24% Treo 23% Other 3% | MSD-CSA only MD-ATG/ MTX/ CSA | Grade II-IV TBI 37% Chemo29% | Overall-TBI 16% Overall-Chemo 11% | TBI-12% Chemo-33% | TBI-2% Chemo-9% | 2-year OS-TBI 91% OS-chemo 75% GRFS-TBI 72% GRFS-chemo 51% |
| Ruggeri et al (2021)16 | Retrospective | 180; 9.25 | ALL | CR1 24% CR2 45% CR3 12% NR 19% | MMRD BM or PBSC | MAC-TBI 25.6% MAC-Chemo 51.6% RIC 22.78% | PtCy | Grade II-IV 28.3% Grade III-IV 12.4% | Overall 21.9% Extensive 9.5% | CR1 25% CR2 37% CR3 50% NR 70% | 19.6% | 2 year OS 50.8% LFS 38.5% GRFS 29% |
| Symons et al (2020)44 | Prospective, single center, phase 2 | 96; 42; 1-65 | ALL AML MDS Lymphoma | CR1 41% >CR1 18% | MMRDBM | MAC-TBI MAC-Bu | PtCy | Grade II-IV 11% Grade III-IV 4% | Overall 15% Moderate to severe 6% | 43% | 11% | 3 year OS 54% EFS 48% |
| Bertaina et al (2018)43 | Retrospective | 98: 6.6; 0.1-17.3 | AL | CR1 43% CR2 47% Other CR 10% | MMRD PBSC | MAC-TBI 74% | TCRαβ/ CD19 depletion | Grade II-IV 16% Grade III-IV 0% | Overall 6%, Extensive 1% | 29% | 9% | 5 year OS 68% LFS 62% |
| Locatelli et al (2017)18 | Prospective | 80; 9.7; 0.9-20.9 | ALL AML | CR1 39% CR2 56% | MMRD PBSC | TBI/TT/Flu TBI/TT/Mel Bu/TT/Flu Bu/Cy/Mel with ATG/Ritux | TCRαβ/ CD19 depletion | Grade I-II 30% | Limited 5% | 24% | 5% | 5 year OS 72% DFS 71% |
AL, acute leukemia; AML, acute myeloid leukemia; ATG, antithymocyte globulin; Bu, busulfan; DFS, disease-free survival; Flu, fludarabine; GRFS, GVHD relapse-free survival; LFS, leukemia-free survival; MAC, myeloablative conditioning; MD, matched related or unrelated donor; MDS, myelodysplastic syndrome; Mel, melphalan; MM, multiple myeloma; MMRD, mismatched related donor; MPS, myeloproliferative syndrome; MSD, matched sibling donor; NM, nonmalignant disease; NR, not in remission; RIC, reduced intensity conditioning; Treo, treosulfan; TT, thiotepa; ST, solid tumor.