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首页> 外文期刊>Journal of experimental & clinical cancer research : >Successful treatment of refractory anemia with a combination regimen containing recombinant human erythropoietin, low-dose methylprednisolone and nandrolone.
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Successful treatment of refractory anemia with a combination regimen containing recombinant human erythropoietin, low-dose methylprednisolone and nandrolone.

机译：包含重组人促红细胞生成素，低剂量甲基强的松龙和Nandrolone的联合治疗方案成功治疗了难治性贫血。

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摘要

Myelodysplastic syndromes (MDS) are a heterogenous group of hematological clonal malignancies. Patients belonging to the refractory anemia (RA) subtype are usually treated with recombinant human erythropoietin (EPO). Not all patients respond to EPO administration and they are strictly dependent on supportive therapy with red cell blood (RBC) transfusions. The aim of this study was to investigate the efficacy of an alternative combination regimen containing EPO, low-dose methylprednisolone and nandrolone decanoate, in patients with RA unresponsive to EPO administration alone. Ten patients, 4 women and 6 men, median age: 70 years (range: 55-78 years) with refractory anemia unresponsive to EPO administration and RBC transfusion-dependent were included in the study. Median hematological data at baseline were Hb: 8.7 g/dl, (range 6.2-9.8), WBC: 3.35x10(9)/l (range 2.1-4), PLT: 82.5x10(9)/l (range 59-110). EPO 150 U/Kg three times/week subcutaneously, low-dose methylprednisolone 8 mg/day orally and nandrolone decanoate (Decadurabolin) 50 mg two-times/week intramuscularly were administered. As complete response (CR) to treatment was considered the normalization of the peripheral blood and bone marrow smears and biopsy. As partial response (PR) was considered increase in Hb level > or = 2 g/dl, or up to 10 g/dl and discontinuation of RBC transfusions. The response to therapy was evaluated on the 4th week after the initiation of the combination treatment. Bone marrow smear evaluation was carried out at baseline and every six months afterwards. After a 4-week treatment all patients achieved PR and discontinued RBC transfusions. Median and range hematological values on the 4th week after treatment initiation were Hb: 11.2 g/dl, (range: 9.8-12.8), WBC: 4.4x10(9)/l (3.5-6.6), PLT: 130x10(9)/l (95-160). The increase observed in hematological values was significant (p = 0.0001, 0.0004 and < 0.0001, respectively, for Hb, WBC and PLT counts). Treatment was well tolerated. Furthermore, two women, on treatment with the combination regimen, achieved CR one after six months and the second after 12 months. They are alive after 5 years from initiation of the combination treatment. After a median period of 18 months (range 12 to 20 months) in PR three men developed acute leukemia; they received intensive antileukemic chemotherapy without any response and died during the phase of pancytopenia. Three other men achieved CR, one after 6 and two after 12 months of therapy and they are on regular follow-up. Two women after 10 and 14 months in PR developed acute leukemia and died. In conclusion, combination therapy with EPO, nandrolone decanoate and low-dose methylprednisolone may be effective as an alternative treatment for RBC transfusion-dependent patients with RA unresponsive to EPO administration alone.
机译：骨髓增生异常综合症（MDS）是血液克隆性恶性肿瘤的异质性组。属于难治性贫血（RA）亚型的患者通常用重组人促红细胞生成素（EPO）治疗。并非所有患者都对EPO给药有反应，他们严格依赖红细胞输血（RBC）支持疗法。这项研究的目的是研究含EPO，低剂量甲基强的松龙和癸酸诺龙的替代组合方案在RA患者仅对EPO无效的情况下的疗效。该研究纳入了10名患者，其中4名女性和6名男性，中位年龄：70岁（范围：55-78岁），对EPO无效且依赖RBC输血而导致的难治性贫血。基线的血液学中位数数据为Hb：8.7 g / dl，（范围6.2-9.8），WBC：3.35x10（9）/ l（范围2.1-4），PLT：82.5x10（9）/ l（范围59-110） ）。皮下注射EPO 150 U / Kg /周3次，每周口服两次小剂量甲基强的松龙8 mg /天，癸酸诺龙（Decadurabolin）50 mg /周。作为对治疗的完全缓解（CR），可以认为是外周血和骨髓涂片及活检的正常化。由于部分反应（PR）被认为血红蛋白水平升高≥2 g / dl，或高达10 g / dl，并中断了RBC输血。在联合治疗开始后第4周评估对治疗的反应。在基线以及之后每六个月进行一次骨髓涂片评估。经过4周的治疗，所有患者均达到PR，并中断了RBC输血。治疗开始后第4周的血液中位数和范围血液学值为Hb：11.2 g / dl，（范围：9.8-12.8），WBC：4.4x10（9）/ l（3.5-6.6），PLT：130x10（9）/ l（95-160）。血液学值的增加是显着的（Hb，WBC和PLT计数分别为p = 0.0001、0.0004和<0.0001）。治疗耐受性良好。此外，两名妇女接受联合治疗后，一个月六个月后获得CR，第二个12个月后获得CR。从联合治疗开始5年后，他们还活着。在PR中位期18个月（12到20个月）后，三名男子患上了急性白血病。他们接受了无反应的强化抗白血病化疗，并在全血细胞减少症阶段死亡。另外三名男子则在治疗后六个月获得了CR，一个在6个月后获得了CR，在12个月后获得了CR，并且接受了定期随访。 PR中的10和14个月后有两名妇女发展为急性白血病并死亡。总之，EPO，癸酸诺龙和低剂量甲基泼尼松龙的联合治疗可能是对仅对EPO无效的RA输血依赖型RA患者的替代治疗。

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