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

Makoto Murata, Seitaro Terakura, Atsushi Wake, Kotaro Miyao, Kazuhiro Ikegame, Naoyuki Uchida, Keisuke Kataoka, Toshihiro Miyamoto, Makoto Onizuka, Tetsuya Eto, Noriko Doki, Shuichi Ota, Maho Sato, Yoshiko Hashii, Tatsuo Ichinohe, Takahiro Fukuda, Yoshiko Atsuta, Shinichiro Okamoto, Takanori Teshima

Research output: Contribution to journalArticlepeer-review

22 Citations (Scopus)


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.

Original languageEnglish
Pages (from-to)2355-2366
Number of pages12
JournalBone Marrow Transplantation
Issue number10
Publication statusPublished - 2021 Oct

ASJC Scopus subject areas

  • Hematology
  • Transplantation


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