Elgaz Sümeyye, Kuçi Zyrafete, Kuçi Selim, Bönig Halvard, Bader Peter
Division for Stem Cell Transplantation and Immunology, Department for Children and Adolescents, University Hospital Frankfurt, Frankfurt am Main, Germany.
German Red Cross Blood Center Frankfurt and Institute of Transfusion Medicine and Immunohematology, Goethe University Medical Center, Frankfurt am Main, Germany.
Transfus Med Hemother. 2019 Feb;46(1):27-34. doi: 10.1159/000496809. Epub 2019 Feb 4.
Acute graft-versus-host disease (aGvHD) continues to impact morbidity and mortality after allogeneic stem cell transplantation (allo-SCT). First-line therapy for aGvHD still remains the use of high-dose corticosteroids. Unfortunately, 40-60% of patients with aGvHD exhibit steroid resistance, which is associated with a very poor prognosis. As no effective second-line therapy existed, in recent decades various treatment options were considered for the treatment of therapy-refractory GvHD. Based on their in vitro immunomodulatory properties, the use of mesenchymal stromal cells (MSCs) in the treatment of aGvHD has been introduced. However, most of the clinical data are generated from uncontrolled trials and case series, showing clinical responses to MSCs. Clinical results are more consistent in children despite the use of MSC preparations of various provenance and manufacturing protocols. While these data support the therapeutic principle, the great variability of outcomes strongly suggests that not all MSC preparations are equal and that the specific manufacturing protocols influence therapeutic success in vivo.
急性移植物抗宿主病(aGvHD)仍然影响异基因干细胞移植(allo-SCT)后的发病率和死亡率。aGvHD的一线治疗仍然是使用大剂量皮质类固醇。不幸的是,40%-60%的aGvHD患者表现出类固醇耐药性,这与非常差的预后相关。由于不存在有效的二线治疗方法,近几十年来,人们考虑了各种治疗方案来治疗难治性移植物抗宿主病(GvHD)。基于间充质基质细胞(MSCs)的体外免疫调节特性,其已被引入用于治疗aGvHD。然而,大多数临床数据来自非对照试验和病例系列,显示了对MSCs的临床反应。尽管使用了不同来源和生产方案的MSC制剂,但儿童的临床结果更为一致。虽然这些数据支持了治疗原则,但结果的巨大差异强烈表明,并非所有的MSC制剂都是相同的,并且特定的生产方案会影响体内治疗的成功。