Ulu Bahar Uncu, Hindilerden Ipek Yonal, Yigenoglu Tugce Nur, Tiryaki Tarik Onur, Erkurt Mehmet Ali, Korkmaz Gulten, Namdaroglu Sinem, Aksoy Elif, Korkmaz Serdal, Seyhan Mert, Yilmaz Seda, Besisik Sevgi Kalayoglu, Dal Mehmet Sinan, Ulas Turgay, Altuntas Fevzi
University of Health Sciences, Ankara Oncology Training and Research Hospital, Department of Hematology & Apheresis Unit, Ankara, Turkey.
Istanbul University Istanbul Medical Faculty, Department of Internal Medicine, Division of Hematology, Istanbul, Turkey.
Transfus Apher Sci. 2025 Feb;64(1):104051. doi: 10.1016/j.transci.2024.104051. Epub 2024 Dec 16.
Graft-versus-host disease (GvHD) is a common and serious complication following allogeneic hematopoietic stem cell transplantation (allo-HSCT), significantly impacting transplant efficacy. In the treatment of GvHD, numerous therapeutic approaches have been explored, with mesenchymal stem cells (MSCs) emerging as a prominent immunomodulatory option. We aimed to evaluate efficacy and outcomes of using MSCs for steroid refractory acute GVHD (SR-aGvHD) management.
We retrospectively analyzed data from 36 patients' who received MSCs for treatment of SR-aGvHD following allo-HSCT between 2018 and 2024 from nine transplantation centers in Türkiye. The product consisted of umbilical cord-derived allogeneic MSCs, which were administered intravenously.
Our cohort was at the median age of 39 years (range: 19-61 years), with aGvHD diagnosed at a median of two months after allo-HSCT. More than half of the patients (58.3 %) classified as high-grade aGvHD according to the Minnesota risk scoring. Cord blood-derived MSCs were administered at a median dose of 3.45 (range: 0.8-5) million MSCs/kg, with a median of 3th (range: 2-5) line treatment. The rate of responses exceeding partial response (PR) was approximately 20 % at the first month, increasing to 24 % at the second month. The six-month survival rate was 33 %, with 46 % of mortality attributed to sepsis and 12.5 % related to GvHD. Multivariate analysis indicated that increasing age (≥35 years) and lower platelet counts (≤75 x10/L) were associated with higher mortality (p < 0.05).
MSC therapy has shown promising potential in improving response rates in aGvHD treatment, with efficacy enhanced by younger age and higher platelet counts.
移植物抗宿主病（GvHD）是异基因造血干细胞移植（allo-HSCT）后常见且严重的并发症，对移植疗效有显著影响。在GvHD的治疗中，人们探索了多种治疗方法，间充质干细胞（MSCs）成为一种突出的免疫调节选择。我们旨在评估使用MSCs治疗类固醇难治性急性移植物抗宿主病（SR-aGvHD）的疗效和结果。
我们回顾性分析了2018年至2024年间来自土耳其九个移植中心的36例接受MSCs治疗allo-HSCT后SR-aGvHD患者的数据。产品为由脐带血来源的异体MSCs，通过静脉注射给药。
我们的队列中位年龄为39岁（范围：19 - 61岁），allo-HSCT后中位两个月诊断为急性移植物抗宿主病。根据明尼苏达风险评分，超过一半的患者（58.3%）被归类为高级别急性移植物抗宿主病。脐带血来源的MSCs中位剂量为345万（范围：80万 - 500万）个MSCs/kg，中位接受第3（范围：2 - 5）线治疗。第一个月超过部分缓解（PR）的反应率约为20%，第二个月增至24%。六个月生存率为33%，46%的死亡归因于败血症，12.5%与移植物抗宿主病相关。多因素分析表明，年龄增加（≥35岁）和血小板计数较低（≤75×10⁹/L）与较高死亡率相关（p < 0.05）。
间充质干细胞疗法在提高急性移植物抗宿主病治疗反应率方面显示出有前景的潜力，年轻和较高的血小板计数可增强疗效。
