Hansen P B, Johnsen H E, Hippe E, Hellström-Lindberg E, Ralfkiaer E
Department of Internal Medicine and Haematology L, University of Copenhagen, Herlev Hospital, Denmark.
Am J Hematol. 1993 Dec;44(4):229-36. doi: 10.1002/ajh.2830440403.
The purpose of this study was to improve erythropoiesis in patients with anemia due to myelodysplastic syndromes (MDS). We treated 13 patients first with recombinant human granulocyte-macrophage colony-stimulating factor (rhGM-CSF) for 6 weeks, then with recombinant human erythropoietin (rhEpo) and rhGM-CSF for the next 12 weeks. Five patients had refractory anemia (RA), 3 refractory anemia with ringed sideroblasts (RAS), and 5 refractory anemia with excess of blasts (RAEB). Ten patients were transfusion-dependent at the time of inclusion. Eleven patients completed this phase II study. Five responded with an increase in hemoglobin level (3 patients) or a reduction in transfusion requirement (2 patients). We registered no response in the remaining 6 patients during treatment. Patients responding to combined treatment had relatively low concentrations of plasma Epo and plasma ferritin before treatment with rhEpo and a normal karyotype throughout the study. Long-term bone marrow cultures did not predict the response. Still, responders seemed to have a higher number of colony-forming progenitors than nonresponders. In conclusion, combined therapy with rhGM-CSF and rhEpo may stimulate hematopoiesis and correct or improve anemia in some patients with MDS.
本研究的目的是改善骨髓增生异常综合征（MDS）所致贫血患者的红细胞生成。我们首先对13例患者用重组人粒细胞巨噬细胞集落刺激因子（rhGM-CSF）治疗6周，然后在接下来的12周用重组人促红细胞生成素（rhEpo）和rhGM-CSF治疗。5例患者为难治性贫血（RA），3例为环形铁粒幼细胞难治性贫血（RAS），5例为原始细胞过多难治性贫血（RAEB）。纳入时10例患者依赖输血。11例患者完成了这项II期研究。5例患者有反应，表现为血红蛋白水平升高（3例患者）或输血需求减少（2例患者）。在治疗期间，其余6例患者无反应。对联合治疗有反应的患者在用rhEpo治疗前血浆Epo和血浆铁蛋白浓度相对较低，且在整个研究过程中核型正常。长期骨髓培养不能预测反应。不过，有反应者似乎比无反应者有更多的集落形成祖细胞。总之，rhGM-CSF和rhEpo联合治疗可能刺激某些MDS患者的造血功能并纠正或改善贫血。
