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Off-the-shelf bone marrow-derived mesenchymal stem cell treatment for acute graft-versus-host disease: real-world evidence

Abstract

Temcell is a cryopreserved, human bone marrow-derived mesenchymal stem cell (MSC) product approved for the treatment of patients of all ages with acute graft-versus-host disease (GVHD). Initial experience with Temcell in a real-world setting from a cellular therapy registry in Japan is presented. A total of 381 consecutive patients were enrolled since its approval in 2016. The median cell number infused was 2.00 × 106/kg. The most common number of infusions was 8 in 100 patients. Of the 306 evaluable patients, the overall response rate (ORR) on day 28 after the start of MSC therapy was 56%. Of the 151 evaluable patients who received it as second-line therapy following first-line steroid therapy for classic acute GVHD, the ORR was 61%. Liver involvement of GVHD and ≥14 days from first-line steroid therapy to second-line MSC therapy was associated with a lower ORR. Day 28 ORR, patient age, GVHD grade, GVHD organ involvement, and a number of GVHD therapies before MSC therapy were associated with nonrelapse mortality. Overall survival at 6 months in 381 patients was 40%. This study suggests that Temcell is one of the treatment options for steroid-refractory acute GVHD until a new treatment with survival benefit is developed.

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Fig. 1: Characteristics of MSC therapy.
Fig. 2: ORR of acute GVHD with MSC therapy.
Fig. 3: NRM after MSC therapy.
Fig. 4: OS after MSC therapy.
Fig. 5: OS after MSC therapy in 381 patients.

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Acknowledgements

The authors would like to thank the physicians and data managers at each hospital who contributed valuable transplantation data to the Japanese Data Center for Hematopoietic Cell Transplantation.

Funding

This study was supported in part by a Grant-in-Aid for Scientific Research (18K08321 to M.M.) from the Japan Society for the Promotion of Science (JSPS) and a Practical Research Project for Allergic Diseases and Immunology (Research on Technology of Medical Transplantation) (JP20ek0510032 to M.M.) from the Japan Agency for Medical Research and Development (AMED).

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Correspondence to Makoto Murata.

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Murata, M., Terakura, S., Wake, A. et al. Off-the-shelf bone marrow-derived mesenchymal stem cell treatment for acute graft-versus-host disease: real-world evidence. Bone Marrow Transplant (2021). https://doi.org/10.1038/s41409-021-01304-y

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