Department of Hematology and Oncology, Nagoya University Graduate School of Medicine, Nagoya, Japan.
Department of Hematology, Toranomon Hospital Kajigaya, Kanagawa, Japan.
Bone Marrow Transplant. 2021 Oct;56(10):2355-2366. doi: 10.1038/s41409-021-01304-y. Epub 2021 May 11.
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 × 10/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.
泰美科是一种冷冻保存的、来源于人类骨髓的间充质干细胞（MSC）产品，已被批准用于治疗所有年龄段的急性移植物抗宿主病（GVHD）患者。本文介绍了日本细胞治疗注册研究中泰美科在真实世界环境下的初步应用经验。自 2016 年批准以来，共有 381 例连续患者入组。输注的中位数细胞数为 2.00×106/kg。100 例患者中最常见的输注次数为 8 次。在 306 例可评估患者中，MSC 治疗开始后第 28 天的总缓解率（ORR）为 56%。在 151 例可评估患者中，这些患者在一线类固醇治疗治疗经典急性 GVHD 后作为二线治疗接受了泰美科治疗，ORR 为 61%。GVHD 肝脏受累和从一线类固醇治疗到二线 MSC 治疗的时间≥14 天与较低的 ORR 相关。第 28 天 ORR、患者年龄、GVHD 分级、GVHD 器官受累以及在 MSC 治疗前接受的 GVHD 治疗次数与非复发死亡率相关。381 例患者的 6 个月总生存率为 40%。本研究表明，在开发具有生存获益的新疗法之前，泰美科是治疗类固醇难治性急性 GVHD 的一种选择。
