HEME ARGINATE TREATMENT FOR MYELODYSPLASTIC SYNDROMES
HEME ARGINATE TREATMENT FOR MYELODYSPLASTIC SYNDROMES
复制标题
DOI：
10.1016/0145-2126(88)90062-8
复制
发表时间：
1988-01-01
影响因子：
2.7
通讯作者：
TENHUNEN, R
中科院分区：
文献类型：
--
作者：
VOLIN, L;RUUTU, T;TENHUNEN, R
26例骨髓增生异常综合征（MDS）患者每周输注精氨酸血红素2-3 mg/kg，共8-12周。大多数患者首先连续四天接受负荷剂量。6例患者在治疗期间血细胞减少症有所改善。在三个反应严重抑郁的血细胞计数恢复正常或接近正常。到目前为止，停止治疗后的最长缓解持续时间为25个月，两个持续缓解分别持续了11个月和12个月。在8例环形铁粒幼细胞超过15%的患者中，有2例反应者的环形铁粒幼细胞数量在治疗期间减少，但在6例无反应者中保持不变。应答者的特征在于在治疗期间增加的低或低正常血红素合酶活性，而无应答者显示出在治疗期间降低的较高平均血红素合酶活性。在一般情况下，反应者有显着较少的缺陷，血红素合成酶的活动比无反应者。造血祖细胞体外培养的FAB型、核型或生长模式不能预测反应。除了一例轻度静脉刺激外，未观察到其他不良反应。目前的研究表明，血红素精氨酸诱导有益的影响，在一些MDS患者的血细胞减少，并有很少的副作用。
Heme arginate was given to 26 patients with a myelodysplastic syndrome (MDS) as infusions of 2-3 mg/kg body weight weekly for 8-12 weeks. Most of the patients first received a loading dose on four consecutive days. Six of the patients showed improvement in cytopenias during the therapy. In three of the responders severely depressed blood cell counts recovered to normal or close to normal. So far the maximum duration of a response after the cessation of the treatment is 25 months, and the two ongoing responses have lasted for 11 and 12 months, respectively. In two responders of the eight patients with more than 15% ring sideroblasts the number of ring sideroblasts decreased during the treatment but remained unchanged in six non-responders. The responders were characterized by a low or low normal heme synthase activity which increased during the treatment, whereas the non-responders showed a higher mean heme synthase activity which decreased during the treatment. In general, the responders had significantly fewer defects in heme synthetic enzyme activities than the non-responders. FAB type, karyotype or growth pattern in in vitro cultures of hematopoietic progenitors did not predict the response. Apart from one case of mild venous irritation, no other adverse effects were seen. The present study shows that heme arginate induces beneficial affects on cytopenia in some MDS patients and has very few side-effects.