Table 2.
Summary of studies with PTCY
| Bolanos-Meads et al12 |
Luznik et al13 |
Holtan et al14 |
Broers et al15 |
This study |
||||||
|---|---|---|---|---|---|---|---|---|---|---|
| (BMT CTN 1203) | (BMT CTN 1301) | (BMT CTN 1703) | (HOVON-96) | (Real world) | ||||||
| Type of study | Randomized phase 2 | Randomized phase 3 | Randomized phase 3 | Randomized phase 3 | Retrospective | |||||
| Transplant period | 2014-2016 | 2015-2018 | 2019-2021 | 2013-2018 | 2018-2021 | |||||
| Graft source | PBSC | BM | PBSC | PBSC | PBSC | |||||
| Conditioning intensity | RIC | MAC | RIC | NMA | MAC, RIC | |||||
| Conditioning regimen | Flu/Bu2, Flu/Cy, Flu/Mel, | Bu/Cy, Flu/Bu4, | Flu/Mel (57%), Flu/Bu2 (27%), | Flu/Cy/TBI for PTCA/CsA | Flu/Bu4 for MAC (80%) | |||||
| Flu/Cy/TBI | Cy/TBI, TBI/Etoposide | Flu/Cy/TBI (14%) | Flu/Bu2 for RIC (77%) | |||||||
| Donor type | 7/8-8/8 MUD, MRD | 8/8 MUD, MRD | 7/8-8/8 MUD, MRD | 8/8 MUD, MRD | 7/8-8/8 MUD | |||||
| Study arm | TAC/MTX | PTCY/TAC/MMF | TAC/MTX | PTCY | TAC/MTX | PTCY/TAC/MMF | CsA/MPA | PTCY/CsA | TAC based | PTCY/TAC/MMF |
| Sample size | 224 | 92 | 114 | 109 | 212 | 208 | 52 | 99 | 463 | 107 |
| Clinical outcome | ||||||||||
| Grade 2-4 aGVHD | 6 mo: 30% | 6 mo: 27% | D100: 29.8% | D100: 37.6% | D100: 51.9% | D100: 53.8% | 6 mo: 48% | 6 mo: 30%∗ | 6 mo: 20% | 6 mo: 20% |
| Grade 3-4 aGVHD | 6 mo: 13% | 6 mo: 2%∗ | D100: 3.5% | D100: 10.1% | D100: 14.7% | D100: 6.3%∗ | 6 mo: 12% | 6 mo: 6% | 6 mo: 8.6% | 6 mo: 7.5% |
| cGVHD | 1 y: 38% | 1 y: 28% | 1 y: 35.1% | 1 y: 21.9%∗ | 2 y: 65% | 2 y: 43%∗ | 2 y: 58% | 2 y: 32%∗ | ||
| Mod-severe cGVHD | 2 y: 33.7% | 2 y: 27% | 1 y: 15.6% | 1 y: 6.7%∗ | 2 y: 36% | 2 y: 12%∗ | ||||
| IS-requring cGVHD | 1 y: 37% | 1 y: 22%∗ | 1 y: 25% | 1 y: 12.5%∗ | ||||||
| Extensive cGVHD | 2 y: 48% | 2 y: 16%∗ | ||||||||
| NRM | 1 y: 16% | 1 y: 11% | 2 y: 7.9% | 2 y: 15.7% | 1 y: 17.2% | 1 y: 12.3% | 3 y: 14% | 3 y: 10% | 2 y: 12% | 2 y: 11% |
| Relapse | 1 y: 25% | 1 y: 28% | 2 y: 25.6% | 2 y: 13.9%∗ | 1 y: 20.2% | 1 y: 20.8% | 3 y: 24% | 3 y: 32% | 2 y: 34% | 2 y: 25%∗ |
| PFS | 1 y: 56% | 1 y: 60% | 2 y: 66.5% | 2 y: 70.3% | 1 y: 62.4% | 1 y: 67% | 3 y: 63% | 3 y: 59% | 2 y: 54% | 2 y: 64%∗ |
| OS | 1 y: 71% | 1 y: 71% | 2 y: 76.1% | 2 y: 76.2% | 1 y: 72.2% | 1 y: 77% | 3 y: 71% | 3 y: 65% | 2 y: 62% | 2 y: 66% |
aGVHD, acute GVHD; BM, bone marrow; cGVHD, chronic GVHD; CsA, cyclosporine A; mod-severe cGVHD, moderate-to-severe cGVHD; MPA, mycophenolic acid; IS-requiring cGVHD, chronic GVHD requiring systemic immunosuppressive treatment.
∗
P < .05, significantly different for the comparison between the PTCY arm and the control arm (TAC/MTX, CsA/MPA, or TAC based).