Verhoef G E, Zachée P, Ferrant A, Demuynck H, Selleslag D, Van Hove L, Deckers F, Boogaerts M A
Department of Hematology, University Hospital Gasthuisberg, Leuven, Belgium.
Ann Hematol. 1992 Jan;64(1):16-21. doi: 10.1007/BF01811466.
The clinical and ferrokinetic effects of escalating doses of subcutaneously administered recombinant human erythropoietin (rh-EPO) were studied in ten patients with myelodysplastic syndromes and severe transfusion-dependent anemia. Red blood cell transfusion requirements diminished in four patients, and one of the patients eventually became transfusion independent with an EPO-induced rise of Hb from 7.7 g/dl to 12.3 g/dl. Endogenous serum levels of EPO were significantly increased in all patients (100-5700 mU/ml), but three of four responders had a relatively low baseline level. The effective red cell iron turnover (RCIT) improved in two responding patients and even normalized in one patient. This increase in RCIT was accompanied with a decline in the ineffective red cell iron turnover (IIT). The other responding patients had a relatively preserved RCIT before EPO treatment. EPO therapy further increased the fraction of IIT in the latter patients. Red cell survival time did not increase during EPO therapy, even in the responding patients. One transient and one maintained increase in platelet count were observed. Disease progression with a sustained increase in blast cells in one patient and a transient elevation of blasts in another patient was seen. No other side effects of EPO therapy were observed. These results suggest that anemic MDS patients with low serum EPO levels and relatively spared effective erythropoiesis as measured by ferrokinetic studies may be the best candidates for treatment with recombinant human EPO.
对10例骨髓增生异常综合征和严重依赖输血的贫血患者皮下注射递增剂量的重组人促红细胞生成素（rh-EPO），研究其临床和铁动力学效应。4例患者的红细胞输血需求减少，其中1例患者最终不再依赖输血，EPO使血红蛋白从7.7 g/dl升至12.3 g/dl。所有患者的内源性血清EPO水平均显著升高（100 - 5700 mU/ml），但4例有反应的患者中有3例基线水平相对较低。2例有反应的患者有效红细胞铁周转率（RCIT）改善，1例患者甚至恢复正常。RCIT的增加伴随着无效红细胞铁周转率（IIT）的下降。其他有反应的患者在EPO治疗前RCIT相对保持稳定。EPO治疗使后一组患者的IIT比例进一步增加。即使在有反应的患者中，EPO治疗期间红细胞存活时间也未增加。观察到1例血小板计数短暂增加，1例持续增加。1例患者出现原始细胞持续增加的疾病进展，另1例患者出现原始细胞短暂升高。未观察到EPO治疗的其他副作用。这些结果表明，血清EPO水平低且通过铁动力学研究测量有效红细胞生成相对保留的贫血MDS患者可能是重组人EPO治疗的最佳候选者。
