骨髓纖維化病患接受異體造血幹細胞移植之預後：單一中心回溯性研究

The Outcome of Allogeneic Hematopoietic Stem Cell Transplantation for Myelofibrosis: a Retrospective Study in a Single Center

侯美芳(Mei-Fang Hou) ； 侯信安(Hsin-An Hou) ； 林建廷(Chien-Ting Lin) ； 李啟誠(Chi-Cheng Li) ； 姚明(Ming Yao) ； 莊傑仰(Jie-Yang Jhuang) ； 林中梧(Chung-Wu Lin) ； 柯博升(Bor-Sheng Ko) ； 黃聖懿(Shang-Yi Huang) ； 周文堅(Wen-Chien Chou) ； 陳耀昌(Yao-Chang Chen) ； 田蕙芬(Hwei-Fang Tien) ； 唐季祿(Jih-Luh Tang)

《台灣癌症醫學雜誌》 2卷1期 (2015/03) Pp. 31-41

https://doi.org/10.6323/JCRP.2015.2.1.04

摘要

目的：異體造血幹細胞移植是唯一能根治骨髓纖維化之治療，本研究回溯分析台大醫院骨髓纖維化病患接受移植之預後。結果：11位病患接受移植，中位年齡為51歲，從診斷到移植的時間為10.1個月，病患都屬中高／高度風險族群，5位接受骨髓殲滅性化療，6位接受減低強度療法。移植後，全部病患均轉成全捐者基因嵌合體。3位病患移植後骨髓病理切片顯示纖維化有明顯改善。移植後100天之移植相關致死率為9%，4位病患發生第三至四級急性移植物抗宿主疾病。6位病患死亡，死因包括移植物抗宿主疾病、感染、次發性移植失敗，2年整體存活率為45.5%。結論：異體造血幹細胞移植對骨髓纖維化具有療效，但移植相關的毒性及併發症仍相對偏高；我們須積極克服移植物抗宿主疾病及感染所造成的併發症以改善病人存活。

關鍵字

骨髓纖維化 ； 異體造血幹細胞移植 ； 感染 ； 預後 ； 移植物抗宿主疾病

並列摘要

Purpose: Nowadays, myelofibrosis can be cured only by allogeneic hematopoietic stem cell transplantation (allo-HSCT), but the transplantation experience is very limited worldwide. We retrospectively investigated the treatment outcome of myelofibrosis patients post allo-HSCT at National Taiwan University Hospital. Results: Eleven myelofibrosis patients received allo-HSCT. The median age at transplantation was 51 years old and the median time from diagnosis to transplantation was 10.1 months. All patients were classified as intermediate-2 or high risk based on the International Prognostic Scoring System (IPSS). Conditioning regimens were myeloablative in 5 and reduced-intensity in 6 patients. All patients achieved full donor chimerism, and post-transplantation bone marrow biopsies showed significant improvement of myelofibrosis in 3 patients. The transplant- related mortality at 100 days post transplantation was 9%. Four patients had grade III-IV acute graft-versus-host disease. Six patients died, and the causes of death were GVHD (n=3), infection (n=2) and secondary graft failure (n=1). The estimated overall survival at two years was 45.5%. Conclusions: Allogeneic hematopoietic stem cell transplantation is effective in treating myelofibrosis and is associated with modest toxicity and post-transplantation complications. In order to improve the treatment outcome, we have to manage GVHD and infection more carefully.

並列關鍵字

myelofibrosis ； allogeneic hematopoietic stem cell transplantation ； infection ； prognosis ； graft-versus-host disease