Hill LaQuisa, Alousi Amin, Kebriaei Partow, Mehta Rohtesh, Rezvani Katayoun, Shpall Elizabeth
Center for Cell and Gene Therapy, Baylor College of Medicine, Houston, TX, USA.
Department of Stem Cell Transplantation and Cellular Therapy, The University of Texas MD Anderson Cancer, Houston, TX, USA.
Ther Adv Hematol. 2018 Jan;9(1):21-46. doi: 10.1177/2040620717741860. Epub 2017 Nov 28.
Graft host disease (GVHD) remains a major cause of morbidity and mortality following allogeneic hematopoietic stem-cell transplantation (HSCT). Despite the use of prophylactic GVHD regimens, a significant proportion of transplant recipients will develop acute or chronic GVHD following HSCT. Corticosteroids are standard first-line therapy, but are only effective in roughly half of all cases with ~50% of patients going on to develop steroid-refractory disease, which increases the risk of nonrelapse mortality. While progress has been made with improvements in survival outcomes over time, corticosteroids are associated with significant toxicities, and many currently available salvage therapies are associated with increased immunosuppression, infectious complications, and potential loss of the graft leukemia (GVL) effect. Thus, there is an unmet need for development of newer treatment strategies for both acute and chronic GVHD to improve long-term post-transplant outcomes and quality of life for HSCT recipients. Here, we provide a concise review of major emerging therapies currently being studied in the treatment of acute and chronic GVHD.
移植物抗宿主病（GVHD）仍然是异基因造血干细胞移植（HSCT）后发病和死亡的主要原因。尽管使用了预防性GVHD方案，但相当一部分移植受者在HSCT后仍会发生急性或慢性GVHD。皮质类固醇是标准的一线治疗方法，但仅对大约一半的病例有效，约50%的患者会发展为类固醇难治性疾病，这增加了非复发死亡率的风险。虽然随着时间的推移，生存结果有所改善，但皮质类固醇具有显著的毒性，并且许多目前可用的挽救疗法与免疫抑制增加、感染并发症以及移植物抗白血病（GVL）效应的潜在丧失有关。因此，迫切需要开发针对急性和慢性GVHD的更新治疗策略，以改善HSCT受者的长期移植后结局和生活质量。在此，我们简要综述了目前正在研究的用于治疗急性和慢性GVHD的主要新兴疗法。
