Ghio R, Balleari E, Ballestrero A, Gatti A M, Mareni C, Massa G, Patrone F, Sessarego M, Timitilli S
Department of Internal Medicine, University of Genoa, Italy.
Acta Haematol. 1993;90(2):58-64. doi: 10.1159/000204377.
Recombinant human erythropoietin (rhEPO) was administered subcutaneously to 13 anemic (Hb < 10 g/dl) patients with myelodysplasia (MDS). rhEPO was given 3 times a week at doses of 75-250 U/kg body weight, over a maximum period of 24 weeks. Five patients (38%) showed a response to rhEPO treatment. rhEPO was well tolerated and without relevant side effects throughout the study. All responding patients had low but detectable pretreatment circulating erythroid progenitor cells (BFU-E) and the response to rhEPO was associated with a significant increase in BFU-E (p < 0.01); concentrations of serum transferrin receptor (TfR) also consistently rose in all responding patients. Baseline erythropoietin (EPO) concentrations did not significantly differ between responders and nonresponders, although 4 out of the 5 responders had relatively low levels of EPO. In conclusion, subcutaneous rhEPO administration appears to be an effective treatment of anemia in a substantial subset of patients with MDS. Relatively low baseline EPO concentrations, detectable pretreatment circulating BFU-E and an early increase in the serum concentrations of TfR seem to be criteria for predicting response to rhEPO in patients with MDS.
对13例骨髓增生异常综合征（MDS）贫血（血红蛋白<10g/dl）患者皮下注射重组人促红细胞生成素（rhEPO）。rhEPO每周给药3次，剂量为75 - 250U/kg体重，最长给药24周。5例患者（38%）对rhEPO治疗有反应。在整个研究过程中，rhEPO耐受性良好，无相关副作用。所有有反应的患者治疗前循环红细胞祖细胞（BFU-E）水平较低但可检测到，对rhEPO的反应与BFU-E显著增加相关（p<0.01）；所有有反应的患者血清转铁蛋白受体（TfR）浓度也持续升高。有反应者和无反应者的基线促红细胞生成素（EPO）浓度无显著差异，尽管5例有反应者中有4例EPO水平相对较低。总之，皮下注射rhEPO似乎是治疗相当一部分MDS患者贫血的有效方法。相对较低的基线EPO浓度、治疗前可检测到的循环BFU-E以及血清TfR浓度早期升高似乎是预测MDS患者对rhEPO反应的标准。
