Table 2.
A summary of reviewed MSC-based therapies used for treatment aGVHD after allogeneic hematopoietic stem cell transplant
| MSC source | MSC dose per kg (no. doses/pt) | Age range, y (n) | Clinical context | Outcome | Phase |
|---|---|---|---|---|---|
| BM | 4-9 × 106 (1-5) | 0-64 (55) | Grade II-IV aGVHD; ppx: CNI/MTX/MMF/prednisolone | 69% OS; ↑OS in responders; no effect of HLA match | 272 |
| BM | 2 × 106 (6) | 2-66 (30) | Grade II-IV SR c/aGVHD; cond: 50% myeloablative; ppx: CNI/MTX/prednisolone | 62% OS; ↑OS in pediatric vs adult responders | 1/273 |
| BM | 2 × 106 (6) | 5-66 (25) | Grade III-IV SR aGVHD; cond: 56% myeloablative; ppx: CNI/MTX/prednisolone | 80% CR; ↑OS in responders, CNI/MTX/prednisolone | 2/374 |
| BM | 2 × 106 (6) | 0-70 (260) | Grade III-IV SR aGVHD* | Not different from second-line placebo. ↑OR in children and pts with liver GVHD. | 375,† |
| BM | 2 × 106 (6) | 0-17 (55) | Grade III-IV SR aGVHD* | Day +28 response predicted OS: 69.1% day +28 OR; 69.5% OS | 325 |
| Placenta | 1 × 106 (2) | 0-65 (28) | Grade II-IV aGVHD; cond: 43% myeloablative; ppx: CNI/MTX/prednisolone | 100% OR (48% PR; 52% CR); 76% OS | 1/226 |
All patients had GVHD organ involvement in ≥1 organ (skin, gut, liver). All MSCs were from a third-party donor and were expanded, cultured, and prepared according to good manufacturing process standards. Immune phenotyping proving MSC expression profile was performed per Horwitz et al.80
BM, bone marrow; CNI, calcineurin inhibitor; cond, conditioning regimen; CR, complete response; MMF, mycophenolate mofetil; MTX, methotrexate; OR, overall response; OS, overall survival; ppx, GVHD prophylaxis; PR, partial response; pt(s), patient(s); SR, steroid refractory.
Institution-specific GVHD prophylactic standard of care (majority of patients receiving CNI/MTX/prednisolone).
Randomized clinical trial.