Ohno R
Department of Medicine III, Hamamatsu University School of Medicine, Japan.
Leuk Lymphoma. 1994 Aug;14(5-6):401-9. doi: 10.3109/10428199409049696.
Clinical trials of differentiation therapy on MDS with retinoic acid are reviewed, and it is discussed whether the differentiation therapy with this drug has scientific relevance for MDS. 13-Cis retinoic acid (13-CRA) may have moderate effect on 20-30% of patients with MDS. In general, the responses are short and toxicity is significant. Since the responses may take at least three weeks and sometimes several months to occur, only patients with a relatively indolent clinical course of MDS would be suitable candidates for this therapy. However, a placebo-controlled prospective randomized study could not confirm the effectiveness of this retinoic acid on MDS. There are only a few clinical trials of all-trans retinoic acid (ATRA) for treatment of MDS. ATRA may have a moderate effect on MDS, but no such a remarkable effect shown on acute promyelocytic leukemia. A placebo-controlled randomized study as having been conducted in 13-CRA is definitely needed to confirm the effectiveness of ATRA on MDS.
回顾了维甲酸对骨髓增生异常综合征（MDS）进行分化治疗的临床试验，并讨论了该药物的分化治疗对MDS是否具有科学相关性。13-顺式维甲酸（13-CRA）可能对20%-30%的MDS患者有中度疗效。一般来说，反应持续时间短且毒性显著。由于反应可能至少需要三周时间，有时甚至需要几个月才会出现，因此只有临床病程相对惰性的MDS患者才是该治疗的合适人选。然而，一项安慰剂对照的前瞻性随机研究未能证实这种维甲酸对MDS的有效性。全反式维甲酸（ATRA）治疗MDS的临床试验仅有少数几项。ATRA可能对MDS有中度疗效，但对急性早幼粒细胞白血病没有如此显著的效果。绝对需要像对13-CRA那样进行一项安慰剂对照的随机研究来证实ATRA对MDS的有效性。
