Section of Hematology and Coagulation, Department of Medicine, Sahlgrenska University Hospital, Gothenburg, Sweden.
Eur J Haematol. 2011 Sep;87(3):244-52. doi: 10.1111/j.1600-0609.2011.01654.x. Epub 2011 Jul 26.
Anaemia in low-risk myelodysplastic syndromes (MDS) is associated with reduced quality of life (QoL). Response to treatment with erythropoietin ± granulocyte colony-stimulating factor (G-CSF) is associated with improved QoL, but whether transfusion therapy with higher haemoglobin (Hb) target levels has similar effects is unknown. The objective for this prospective phase II Nordic multicentre trial was to assess QoL, response rate and physical function in elderly anaemic MDS patients treated to a target Hb level of >120 g/L.
Thirty-six elderly patients with low- and intermediate-1 risk MDS received darbepoetin (DA) 300 μg/wk, with the addition of G-CSF if no response. If the Hb target was reached at 16 wk, treatment was maintained until week 26. Remaining patients were transfused to reach the target level for at least 8 wk.
Twenty-seven patients completed the study. Response rate to DA ± G-CSF was 67% in evaluable patients and 56% according to intention to treat. Eighteen patients reached the target Hb level according to protocol. QoL scores for fatigue, dyspnoea, constipation, and physical, role and social functioning improved significantly during study, with similar results for transfused and untransfused patients. Maintaining Hb >120 g/L did not confer a higher transfusion rate, once the target was reached. In two of fourteen patients, magnetic resonance imaging T2* indicated cardiac iron overload, however, without association with ferritin levels.
In elderly anaemic MDS patients, an increment in haemoglobin is associated with improved QoL, whether induced by growth factor treatment or transfusion therapy.
低危骨髓增生异常综合征(MDS)相关的贫血与生活质量(QoL)降低有关。用促红细胞生成素(EPO)±粒细胞集落刺激因子(G-CSF)治疗的反应与 QoL 的改善有关,但更高的血红蛋白(Hb)目标水平的输血治疗是否具有类似的效果尚不清楚。本前瞻性 II 期北欧多中心试验的目的是评估接受>120g/L 目标 Hb 水平治疗的老年贫血性 MDS 患者的 QoL、反应率和身体功能。
36 名低危和中危 1 风险 MDS 老年患者接受达贝泊汀(DA)300μg/周,如果没有反应,则加用 G-CSF。如果在 16 周达到 Hb 目标,则维持治疗至 26 周。其余患者接受输血以达到目标水平至少 8 周。
27 名患者完成了研究。可评估患者的 DA±G-CSF 反应率为 67%,根据意向治疗为 56%。根据方案,18 名患者达到了目标 Hb 水平。研究期间,疲劳、呼吸困难、便秘以及身体、角色和社会功能的 QoL 评分显著改善,输血和未输血患者的结果相似。一旦达到目标,维持 Hb>120g/L 并不会导致更高的输血率。在 14 名患者中的 2 名中,磁共振成像 T2*表明存在心脏铁过载,但与铁蛋白水平无关。
在老年贫血性 MDS 患者中,血红蛋白的增加与 QoL 的改善有关,无论是通过生长因子治疗还是输血治疗诱导的。