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骨髓增生异常综合征中生长因子的治疗

Treatment with growth factors in myelodysplastic syndromes.

作者信息

Geissler R G, Schulte P, Ganser A

机构信息

Department of Hematology and Oncology, Hannover Medical School, Germany.

出版信息

Pathol Biol (Paris). 1997 Oct;45(8):656-67.

PMID:9569932
Abstract

In myelodysplastic syndromes (MDS), pancytopenia leads to a high risk of infectious and hemorrhagic complications. The progression to acute myeloid leukemia adds to morbidity and mortality. While transfusions of red blood cells and platelets are still a cornerstone of the therapy, the clinical use of recombinant hematopoietic growth factors has enlarged the range of therapeutic applications in patients with MDS. It is possible to reverse neutropenia by administration of G-CSF (granulocyte colony stimulating factor) or GM-CSF (granulocyte-monocyte colony stimulating factor). In the case of a severe infection, therapeutic administration of G-CSF together with antibiotics might be justified in otherwise neutropenic MDS patients. Since especially patients with only slight impairment of erythropoiesis and no transfusion dependency have the highest response rates but need erythropoietin (EPO) the least, pharmacoeconomic analyses are urgently needed. Controlled randomized trials will have to ascertain wether combinations of EPO with G-CSF or GM-CSF are of benefit. Clinical studies with thrombopoietin (megakaryocyte growth and differentiation factor) have to be initiated to find out whether thrombocytopenia in MDS can be reversed.

摘要

在骨髓增生异常综合征(MDS)中,全血细胞减少会导致感染和出血并发症的高风险。进展为急性髓系白血病会增加发病率和死亡率。虽然红细胞和血小板输血仍然是治疗的基石,但重组造血生长因子的临床应用扩大了MDS患者的治疗应用范围。通过给予G-CSF(粒细胞集落刺激因子)或GM-CSF(粒细胞-单核细胞集落刺激因子)可以逆转中性粒细胞减少。在严重感染的情况下,对于其他方面为中性粒细胞减少的MDS患者,联合使用G-CSF和抗生素进行治疗可能是合理的。由于尤其是那些红细胞生成仅有轻微损害且不依赖输血的患者反应率最高,但对促红细胞生成素(EPO)的需求最少,因此迫切需要进行药物经济学分析。对照随机试验必须确定EPO与G-CSF或GM-CSF联合使用是否有益。必须启动有关血小板生成素(巨核细胞生长和分化因子)的临床研究,以查明MDS中的血小板减少症是否可以逆转。

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