Omoto E, Deguchi S, Takaba S, Kojima K, Yano T, Katayama Y, Sunami K, Takeuchi M, Kimura F, Harada M, Kimura I
Department of Medicine II, Okayama University Medical School, Okayama City, Japan.
Leukemia. 1996 Apr;10(4):609-14.
Twenty-one consecutive patients with high-risk myelodysplastic syndromes (MDS) including six with refractory anemia with excess blasts (RAEB) and 15 with RAEB in transformation (RAEBt) were treated with daily oral low-dose melphalan (2 mg/day). Seven patients achieved complete remission (CR), one patient partial response, and four minor response while the remaining eight did not respond. The median age of the patients was 65 (range 56-83 years). The mean total amount of melphalan given was 140+/-19 mg in patients who achieved CR. The median duration of CR was 14.5 months. Serious toxicity was not encountered in any of the cases. Neither marrow suppression nor pancytopenia was observed during the administration of melphalan in patients who achieved CR. The clinical features of CR patients included normal karyotype and hypocellular marrow in biopsied specimen from the lilac bone. These observations suggest that melphalan may exert some differentiation effects on leukemic cells in addition to cytotoxic effects. Our study indicates that daily administration of low-dose melphalan is worth trying in the treatment of elderly patients with high-risk MDS.
连续21例高危骨髓增生异常综合征（MDS）患者，其中包括6例伴有过多原始细胞的难治性贫血（RAEB）患者和15例转化中的RAEB（RAEBt）患者，接受每日口服低剂量美法仑（2毫克/天）治疗。7例患者达到完全缓解（CR），1例患者部分缓解，4例患者轻度缓解，其余8例无反应。患者的中位年龄为65岁（范围56 - 83岁）。达到CR的患者美法仑的平均总给药量为140±19毫克。CR的中位持续时间为14.5个月。所有病例均未出现严重毒性反应。达到CR的患者在美法仑给药期间未观察到骨髓抑制或全血细胞减少。CR患者的临床特征包括核型正常以及来自髂骨活检标本中的骨髓细胞减少。这些观察结果表明，美法仑除了具有细胞毒性作用外，可能还对白血病细胞发挥一些分化作用。我们的研究表明，每日给予低剂量美法仑在老年高危MDS患者的治疗中值得一试。
