移植物抗宿主?。℅vHD）是異基因造血干細胞移植（Allo-HSCT）后主要的并發(fā)癥和死亡原因。根據(jù)臨床特征GvHD主要分為急性GvHD和慢性GvHD，可累及皮膚、肝臟、胃腸道等全身多個器官，是一種嚴重且異質(zhì)性高的疾病。糖皮質(zhì)激素是GvHD一線系統(tǒng)治療，激素難治GvHD的患者預后較差。近年來，GvHD新藥研發(fā)成為熱點，伊布替尼、蘆可替尼、貝舒地爾相繼在國際獲批用于GvHD二線和/或后線治療用藥，國內(nèi)也有多個創(chuàng)新藥正處于臨床研發(fā)階段。在總結(jié)近年上市藥物臨床研發(fā)經(jīng)驗以及國外監(jiān)管考量的基礎上，從技術(shù)審評角度，結(jié)合GvHD臨床需求、疾病和藥物特點、國內(nèi)外差異等，對GvHD治療藥物的臨床研發(fā)和評價中關(guān)注的重點內(nèi)容進行討論，以期為此類藥物的臨床研發(fā)和評價提供參考，加快創(chuàng)新藥物研發(fā)。

Graft-versus-host disease (GvHD) is a major complication and cause of death in allogeneic hematopoietic stem cell transplantation recipients. According to clinical characteristics, it is mainly divided into acute GvHD and chronic GvHD, which may impair multiple organs such as skin, liver, and gastrointestinal tract. GvHD is a serious and highly heterogeneous disease. Corticosteroids are the first-line systemic treatment for GvHD, and steroid refractory GvHD patients have a poor prognosis. In recent years, the development of GvHD new drugs has become a hot topic. Ibrutinib, ruxolitinib, and belumosudil have been approved for the treatment of GvHD over the world. There are also several innovative drugs in different phases of clinical trials in China. This paper summarizes the clinical development experiences of recently listed drugs and the regulatory considerations of foreign countries. Some considerations are put forward from the perspective of drug evaluation based on clinical needs, disease and drug characteristics, differences at home and abroad, in order to provide reference for the clinical research and evaluation.

R926