Rapamycin for the treatment of refractory extensive chronic graft-versus-host disease

doi:10.3969/j.issn.2095-4344.2013.18.009

Abstract

Abstract:

BACKGROUND: Refractory extensive chronic graft-versus-host disease is the mainly late complication of allogeneic hematopoietic stem cell transplantation and glucocorticoid is the first choice for the therapy. But now, there are few effective and less side effect methods for glucocorticoid-resistant or hormone-dependent patients.
OBJECTIVE: To observe clinical effects and side effects of rapamycin on patients suffering from refractory extensive chronic graft-versus-host disease that resistant or dependent on glucocorticoid.
METHODS: Seventy-two cases received allogenic hematopoietic stem cells transplantation in the Department of Hematology, the Third Affiliated Hospital of Sun Yat-sen University between January 1998 and June 2012. Among them, five patients with refractory extensive chronic graft-versus-host disease were resistant to glucocorticoid or dependent on hormone, and these five patients were treated with rapamycin. The treatment effects and side effects were observed.
RESULTS AND CONCLUSION: Among the five cases treated with rapamycin, one case received complete remission, three cases improved obviously, and the last one case received stable condition. The longest and median time for taking rapamycin was 14 and 9.4 (4-14) months respectively. The median follow-up time was 18 months, and five patients were all alive during the follow-up. The rapamycin had fewer side effects, hyperlipidemia and mild anemia were observed in one case separately, and the hepatic dysfunction and hyperglycemia were not observed. The results show that rapamycin has better effect on refractory extensive chronic graft-versus-host disease that resistant or dependent on glucocorticoid, and results in fewer side effects. Hematologic toxicity, hepatic dysfunction and high cholesterolcan can be tolerant well for patients.

Key words: organ transplantation, rapamycin, chronic-graft-versus host disease, hematopoietic stem cell transplantation, efficacy, side effects, glucocorticoids, provincial grants-supported paper

CLC Number:

R318

Li Xu-dong, He Yi, Wang Dong-ning, Hu Yuan, Wang Wen-wen, Zhang Xiang-zhong, . Rapamycin for the treatment of refractory extensive chronic graft-versus-host disease[J]. Chinese Journal of Tissue Engineering Research, 2013, 17(18): 3287-3294.

Figures/Tables 3

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