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重组促红细胞生成素疗法在未获许可适应症中的新用途。

Novel uses for recombinant erythropoietin therapy in unlicensed indications.

作者信息

Macartney Christine A, Adgey A A Jennifer, Jones Frank G C, Morris Treen C M, McMullin Mary-Frances

机构信息

Department of Haematology, Belfast City Hospital, Lisburn Road, Belfast, Northern Ireland.

出版信息

Hematol J. 2004;5(2):181-5. doi: 10.1038/sj.thj.6200350.

DOI:10.1038/sj.thj.6200350
PMID:15048070
Abstract

Clinical uses for recombinant human erythropoietin (rHuEPO) therapy continue to expand. Initial use was in anaemia associated with end-stage renal disease, but more recently there have been many reports of the benefits of erythropoietin in other clinical situations such as cancer-related anaemia. Recombinant erythropoietin reduces the need for blood transfusion and hence exposure to donor blood products as well as improving quality of life. We report four patients who were transfusion dependent, none of whom had licensed indications for the use of recombinant erythropoietin. Two patients had microangiopathic haemolytic anaemia secondary to mechanical valve haemolysis and were unsuitable for any further cardiac intervention. One patient had anaemia of chronic disease and anti-Vel red cell antibodies, making compatible blood transfusions difficult to obtain. The fourth patient had primary thrombocythaemia and developed transfusion-dependent anaemia secondary to myelosuppressive agents. All four patients had a relative deficiency in endogenous erythropoietin levels ranging between 7 and 41 IU/l. After commencing recombinant erythropoietin therapy, all had a response in haemoglobin of at least 1 g/dl with an overall improvement in their quality of life. We conclude that rHuEPO is a very convenient and useful form of treatment in transfusion-dependent anaemia and in some cases beyond the licensed indications.

摘要

重组人促红细胞生成素(rHuEPO)疗法的临床应用范围不断扩大。最初用于治疗与终末期肾病相关的贫血,但最近有许多报告称促红细胞生成素在其他临床情况(如癌症相关性贫血)中也有好处。重组促红细胞生成素减少了输血需求,从而减少了接触供血者血液制品的机会,同时还改善了生活质量。我们报告了4例依赖输血的患者,他们均无使用重组促红细胞生成素的获批适应症。2例患者继发于机械瓣膜溶血的微血管病性溶血性贫血,不适合进行任何进一步的心脏干预。1例患者患有慢性病性贫血且有抗Vel红细胞抗体,难以获得相容的血液输血。第4例患者患有原发性血小板增多症,继发于骨髓抑制药物后出现依赖输血的贫血。所有4例患者内源性促红细胞生成素水平相对不足,范围在7至41 IU/l之间。开始重组促红细胞生成素治疗后,所有患者血红蛋白至少升高1 g/dl,生活质量总体改善。我们得出结论,rHuEPO在依赖输血的贫血治疗中是一种非常方便且有用的治疗方式,在某些情况下甚至超出了获批适应症范围。

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