Pidala Joseph, Kim Jongphil, Roman-Diaz Jaime, Shapiro Jamie, Nishihori Taiga, Bookout Ryan, Anasetti Claudio, Kharfan-Dabaja Mohamed A
Department of Blood and Marrow Transplantation, Moffitt Cancer Center and Research Institute, University of South Florida, Tampa, FL, USA.
Ann Transplant. 2010 Oct-Dec;15(4):21-9.
Despite scientific advances in hematopoietic cell allografting, glucocorticoid-refractory acute (aGVHD) and chronic graft-versus-host disease (cGVHD) represent major sources of transplant-related morbidity and mortality. We aimed to characterize the activity of pentostatin as rescue therapy for refractory GVHD.
MATERIAL/METHODS: In a retrospective analysis, we examined the activity of pentostatin as rescue therapy of glucocorticoid-refractory acute and chronic GVHD.
In 12 patients with advanced (overall aGVHD grade III/IV in 8/12) refractory aGVHD, overall response (ORR) was achieved in 6/12, and complete remission (CR) of aGVHD in 4/12 allowing additional rescue immunosuppressive agents. Median overall survival (OS) was 1.4 months (95% CI: 0.26-2.4). Causes of death included refractory aGVHD and infection. In 18 patients with refractory cGVHD (12/18 with severe cGVHD), pentostatin induced CR in 1/18, and partial response (PR) in 9/18. Activity was observed in all affected organs. The median decrease in glucocorticoid therapy over 24 months after pentostatin initiation for refractory cGVHD was 38% (range=0-100%). Median OS was 5 months (95% CI: 1.6 - NR).
Allowing for the utilization of additional immune suppressive agents, this series suggests the activity of pentostatin as rescue therapy of refractory GVHD.
尽管造血细胞移植在科学上取得了进展，但糖皮质激素难治性急性移植物抗宿主病（aGVHD）和慢性移植物抗宿主病（cGVHD）仍是移植相关发病率和死亡率的主要来源。我们旨在描述喷司他丁作为难治性移植物抗宿主病挽救治疗的活性。
材料/方法：在一项回顾性分析中，我们研究了喷司他丁作为糖皮质激素难治性急性和慢性移植物抗宿主病挽救治疗的活性。
在12例晚期（12例中有8例aGVHD总体为III/IV级）难治性aGVHD患者中，12例中有6例实现了总体缓解（ORR），12例中有4例aGVHD完全缓解（CR），从而可以使用额外的挽救性免疫抑制剂。中位总生存期（OS）为1.4个月（95%CI：0.26 - 2.4）。死亡原因包括难治性aGVHD和感染。在18例难治性cGVHD患者中（18例中有12例为重度cGVHD），喷司他丁使18例中的1例达到CR，18例中的9例达到部分缓解（PR）。在所有受累器官中均观察到活性。对于难治性cGVHD，喷司他丁开始使用后24个月内糖皮质激素治疗的中位减少量为38%（范围 = 0 - 100%）。中位OS为5个月（95%CI：1.6 - NR）。
考虑到可使用额外的免疫抑制剂，该系列研究提示喷司他丁作为难治性移植物抗宿主病挽救治疗具有活性。
