The clinical observation of sirolimus combined with calcineurin inhibitors for steroid-resistant/steroid-dependent extensive cGVHD
10.3760/cma.j.issn.0253-2727.2020.09.003
- VernacularTitle:西罗莫司联合钙调磷酸酶抑制剂治疗糖皮质激素耐药/依赖广泛型慢性移植物抗宿主病临床观察
- Author:
Wen ZHU
1
;
Yimei FENG
;
Ting CHEN
;
Han YAO
;
Yao QUAN
;
Jun RAO
;
Lei GAO
;
Cheng ZHANG
;
Yao LIU
;
Li GAO
;
Peiyan KONG
;
Xi ZHANG
Author Information
1. 陆军军医大学新桥医院血液病医学中心，创伤、烧伤与复合伤国家重点实验室，全军血液病中心，重庆市医学重点学科，重庆 400037
- Keywords:
Chronic graft versus host disease;
Glucocorticoid -resistant/dependent;
Sirolimus;
Calcineurin inhibitors
- From:
Chinese Journal of Hematology
2020;41(9):716-722
- CountryChina
- Language:Chinese
-
Abstract:
Objective:To observe the efficacy and safety of sirolimus combined with calcineurin inhibitor (CNI) in the treatment of glucocorticoid resistant/dependent extensive chronic graft-versus-host disease (cGVHD) .Methods:A total of 27 patients with steroid-resistant/steroid-dependent extensive cGVHD from November 2015 to January 2019 were enrolled and given sirolimus capsules combined with cyclosporine or tacrolimus to observe the clinical efficacy and adverse events.Results:The median duration of medication was 14.2 months and the mean duration was 16.7 months. The median follow-up time was 20.1 months (12.9-46.1 months) . Following the 6-month follow-up, 3 cases achieved complete response (CR) and 12 cases partial response (PR) . The overall response rate (ORR) was 55.6% ; for progression-free survival (PFS) , PFS-6 reached 88.9% (24/27) , and for overall survival (OS) , OS-6 was 100% . At the 1-year follow-up, there were 5 cases of CR and 11 cases of PR, ORR was 59.3% , PFS-12 reached 62.9% (17/27) , and OS-12 was 100% . The subgroup analysis found that the program was more effective for cGVHD in male donors and the target organ analysis had an advantage in the treatment of oral cavity, skin, and liver rejection. Adverse events were observed: hyperlipidemia 11.1% , oral ulcer 7.4% , fungal infection 11.1% , liver injury 3.7% , renal insufficiency 0, and no new CMV and EB viremia.Conclusion:Sirolimus combined with calcineurin inhibitors is effective in treating steroid-resistant/steroid-dependent extensive cGVHD, especially because adverse reactions (renal toxicity, CMV, EBV infection) are low in number, which is suitable for long-term treatment of cGVHD.
