TY - JOUR
T1 - Off-the-shelf bone marrow-derived mesenchymal stem cell treatment for acute graft-versus-host disease
T2 - real-world evidence
AU - Murata, Makoto
AU - Terakura, Seitaro
AU - Wake, Atsushi
AU - Miyao, Kotaro
AU - Ikegame, Kazuhiro
AU - Uchida, Naoyuki
AU - Kataoka, Keisuke
AU - Miyamoto, Toshihiro
AU - Onizuka, Makoto
AU - Eto, Tetsuya
AU - Doki, Noriko
AU - Ota, Shuichi
AU - Sato, Maho
AU - Hashii, Yoshiko
AU - Ichinohe, Tatsuo
AU - Fukuda, Takahiro
AU - Atsuta, Yoshiko
AU - Okamoto, Shinichiro
AU - Teshima, Takanori
N1 - Funding Information:
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).
Publisher Copyright:
© 2021, The Author(s), under exclusive licence to Springer Nature Limited.
PY - 2021/10
Y1 - 2021/10
N2 - 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.
AB - 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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U2 - 10.1038/s41409-021-01304-y
DO - 10.1038/s41409-021-01304-y
M3 - Article
AN - SCOPUS:85105477716
SN - 0268-3369
VL - 56
SP - 2355
EP - 2366
JO - Bone Marrow Transplantation
JF - Bone Marrow Transplantation
IS - 10
ER -
