Division of Haematology, Department of Oncology, Transplants and Advanced Technologies in Medicine, University of Pisa-AOUP, Pisa, Italy.
Am J Hematol. 2011 Sep;86(9):762-7. doi: 10.1002/ajh.22111.
We investigated the efficacy of alpha recombinant human erythropoietin (α-rHuEPO) administered as single agent to 133 patients affected by myelodysplastic syndromes referring to our Institution in the last 10 years. WPSS score was "very low" in 67%, "low" in 19%, "intermediate" in 14%. The starting schedule was: 40,000 IU bi-weekly, with reduction or suspension, when necessary, in responsive patients. According to new IWG criteria, response rate (RR) was 75%, 66%, 59% after 8, 16, 24 weeks, respectively. Comparing "very low" and "low/intermediate" risk, RR was 81% vs. 43% (P < 0.001); 70% vs. 45% (P = 0.040); 63% vs. 42% (P = NS) after 8, 16, 24 weeks. RR was significantly influenced by transfusion dependence (P = 0.039) and basal serum EPO level (P < 0.001). Mean Hb value was 94 ± 11 g/l before therapy; 114 ± 19 after 8 weeks (P < 0.001); 116 ± 18 after 16 weeks (P < 0.001); 114 ± 17 after 24 weeks (P < 0.001). Reduction or suspension of therapy significantly affected Hb level after 4 (P < 0.001) and 8 weeks (P < 0.01). Conversely, restart of full dosage significantly enhanced again Hb level after 4 (P < 0.01) and 8 weeks (P < 0.001). 65% patients are alive (mean survival: 74 weeks). Seventy percent are alive in the "very low risk" group and 38% in "low/intermediate risk" group (P < 0.001). Overall mean follow-up was 69 weeks (range, 8-376): it was 80 weeks in responsive patients (max 376) and 38 weeks in patients who progressively became unresponsive (max 168) (P < 0.01). Median response was 36 weeks, with 33% of patients still responding after one year. Treatment was well tolerated.
我们调查了重组人促红细胞生成素（α-rHuEPO）单一药物治疗 133 例骨髓增生异常综合征患者的疗效，这些患者在过去 10 年中曾到我院就诊。WPSS 评分为“极低”的占 67%，“低”的占 19%，“中”的占 14%。起始方案为：每周 2 次给予 40000IU，在有反应的患者中，必要时可减少或停止治疗。根据新的 IWG 标准，8、16、24 周后的缓解率（RR）分别为 75%、66%、59%。将“极低”和“低/中”风险进行比较，RR 分别为 81%比 43%（P<0.001）；70%比 45%（P=0.040）；63%比 42%（P=NS）。RR 受输血依赖性（P=0.039）和基础血清 EPO 水平（P<0.001）显著影响。治疗前平均血红蛋白（Hb）值为 94±11g/l；治疗 8 周后为 114±19g/l（P<0.001）；16 周后为 116±18g/l（P<0.001）；24 周后为 114±17g/l（P<0.001）。治疗的减少或暂停显著影响第 4 周（P<0.001）和第 8 周（P<0.01）的 Hb 水平。相反，完全剂量的重新开始显著提高了第 4 周（P<0.01）和第 8 周（P<0.001）的 Hb 水平。65%的患者存活（平均生存时间：74 周）。70%的患者在“极低风险”组中存活，38%的患者在“低/中风险”组中存活（P<0.001）。总的中位随访时间为 69 周（范围：8-376）：在有反应的患者中为 80 周（最长 376 周），在逐渐无反应的患者中为 38 周（最长 168 周）（P<0.01）。中位缓解时间为 36 周，1 年后仍有 33%的患者有反应。治疗耐受性良好。
