Schouten H C, Vellenga E, van Rhenen D J, de Wolf J T, Coppens P J, Blijham G H
Department of Internal Medicine, University Hospital Maastricht, The Netherlands.
Leukemia. 1991 May;5(5):432-6.
As anemia is frequently the main problem in myelodysplastic syndromes (MDS), we studied the efficacy of human erythropoietin (rhEpo) in stimulating the erythroid lineage in 14 patients, starting with 40 U/kg three times a week and doubling the dose every 6 weeks until a response was observed. The highest doses administered were 80 (n = 1), 160 (n = 4), 320 (n = 8) and 640 U/kg (n = 1). One patient (refractory anemia with an excess of blasts, RAEB) showed an increase of hemoglobin, white blood cells and platelets with 80 U/kg rhEpo. However, this patient developed acute leukemia while on therapy. Two other patients (RAEB and RAEB in transformation) also transformed to acute leukemia. In the other 11 patients no response was observed. There was no correlation between in vitro culture data and in vivo responsiveness. The treatment was well tolerated and no nonhematological side effects were observed. From this study we conclude that rhEpo, even when given at high doses, has a low response rate in patients with MDS. Further investigation is needed in order to clarify whether rhEpo increases the potential risk of transformation to acute leukemia.
由于贫血常常是骨髓增生异常综合征（MDS）的主要问题，我们研究了人促红细胞生成素（rhEpo）刺激14例患者红系造血的疗效，起始剂量为每周3次，每次40 U/kg，每6周剂量加倍，直至观察到反应。给予的最高剂量分别为80 U/kg（1例）、160 U/kg（4例）、320 U/kg（8例）和640 U/kg（1例）。1例患者（原始细胞过多的难治性贫血，RAEB）使用80 U/kg rhEpo后血红蛋白、白细胞和血小板增加。然而，该患者在治疗期间发生了急性白血病。另外2例患者（RAEB和转化中的RAEB）也转化为急性白血病。其他11例患者未观察到反应。体外培养数据与体内反应性之间无相关性。治疗耐受性良好，未观察到非血液学副作用。从本研究我们得出结论，rhEpo即使大剂量使用，在MDS患者中的反应率也很低。需要进一步研究以阐明rhEpo是否增加转化为急性白血病的潜在风险。
