Fukutani H, Naoe T, Saito H, Ohshima T, Omine M, Miura Y, Mizoguchi H, Kimura I, Tomonaga M, Ohno R
Department of Medicine, Branch Hospital, Nagoya University School of Medicine.
Jpn J Clin Oncol. 1991 Aug;21(4):287-92.
N-(2S, 3R)-3-amino-2-hydroxy-4-phenylbutyryl-L-leucine (ubenimex) was administered orally, to patients with myelodysplastic syndromes (MDS) and acute leukemia derived from MDS, in a multi-institute study. Out of 77 patients evaluated, one achieved a complete remission, three a good response and two a partial response while 71 failed to respond to a daily oral administration of 30 mg ubenimex. The overall response rate was 7.8% (95% confidence limits; 3.6-16.0%); 7.0% (3.0-15.4%) in 71 MDS and 16.6% (3.0-56.3%) in acute six leukemias derived from MDS. Responses continued for six to 24 (median 10.5) weeks. No serious hematologic, biochemical or clinical toxicity was encountered, except for gastro-intestinal (GI) toxicity in one patient. The present study demonstrated ubenimex not to be generally beneficial for patients with MDS, and not to be recommended as a standard treatment for the disease.
在一项多机构研究中，对骨髓增生异常综合征（MDS）患者以及由MDS演变而来的急性白血病患者口服给予N-（2S，3R）-3-氨基-2-羟基-4-苯基丁酰-L-亮氨酸（ubenimex，乌苯美司）。在接受评估的77例患者中，1例达到完全缓解，3例有良好反应，2例有部分反应，而71例患者对每日口服30mg乌苯美司无反应。总缓解率为7.8%（95%置信区间：3.6-16.0%）；71例MDS患者的缓解率为7.0%（3.0-15.4%），由MDS演变而来的急性白血病患者的缓解率为16.6%（3.0-56.3%）。缓解持续6至24周（中位值10.5周）。除1例患者出现胃肠道毒性外，未遇到严重的血液学、生化或临床毒性。本研究表明，乌苯美司对MDS患者一般无益处，不推荐作为该病的标准治疗方法。
