Casadevall Nicole, Durieux Pierre, Dubois Stéphanie, Hemery François, Lepage Eric, Quarré Marie-Catherine, Damaj Gandhi, Giraudier Stéphane, Guerci Agnès, Laurent Guy, Dombret Hervé, Chomienne Christine, Ribrag Vincent, Stamatoullas Aspasia, Marie Jean-Pierre, Vekhoff Anne, Maloisel Frédéric, Navarro Robert, Dreyfus François, Fenaux Pierre
Hôtel Dieu, Service d'Hématologie Biologique, 1 place du parvis Notre Dame, 75004 Paris, France.
Blood. 2004 Jul 15;104(2):321-7. doi: 10.1182/blood-2003-07-2252. Epub 2004 Mar 30.
In myelodysplastic syndromes (MDS), anemia responds to recombinant human erythropoietin (rHuEPO) alone and in combination with recombinant human granulocyte-colony-stimulating factor (rHuGCSF) in 10% to 20% and in 35% to 40% of patients, respectively. We randomly divided 60 patients with low-grade anemic MDS and serum EPO levels lower than 500 IU/L (500 mU/mL) into 2 groups: rHuEPO + rHuG-CSF (arm A) and supportive care (arm B). After 12 weeks, those who had erythroid responses were given rHuEPO alone for 40 additional weeks. They were also given rHuG-CSF if they had relapses. A response was considered major if the hemoglobin (Hb) level was 115 g/L (11.5 g/dL) or higher and minor Hb increase was 15 g/L (1.5 g/dL) or more or if it remained stable without transfusion. Ten of 24 patients responded in arm A, and 0 of 26 responded in arm B (P =.01). Eight patients in arm A continued rHuEPO therapy alone, and 6 had relapses. Responses were always restored when rHuG-CSF was reintroduced. Mean direct costs per patient were 26,723 euros (arm A) and 8,746 euros (arm B). Quality of life was assessed with a Functional Assessment of Cancer Therapy-Anemia (FACT-An) scale. Similar percentages of patients from both arms showed significant clinical improvement. rHuEPO plus rHuG-CSF led to responses in 41.7% of MDS patients. This treatment was expensive. No effect on quality of life was demonstrated.
在骨髓增生异常综合征（MDS）中，单独使用重组人促红细胞生成素（rHuEPO）以及联合重组人粒细胞集落刺激因子（rHuGCSF）治疗时，分别有10%至20%和35%至40%的患者贫血症状得到缓解。我们将60例轻度贫血的MDS患者且血清促红细胞生成素水平低于500 IU/L（500 mU/mL）随机分为两组：rHuEPO + rHuG-CSF组（A组）和支持治疗组（B组）。12周后，对有红系反应的患者单独给予rHuEPO额外治疗40周。若复发则给予rHuG-CSF治疗。若血红蛋白（Hb）水平达到115 g/L（11.5 g/dL）或更高，或Hb轻度升高15 g/L（1.5 g/dL）或更多，或未输血且保持稳定，则视为主要反应。A组24例患者中有10例有反应，B组26例患者中0例有反应（P = 0.01）。A组8例患者继续单独接受rHuEPO治疗，6例复发。重新使用rHuG-CSF后反应总能恢复。每位患者的平均直接费用为26,723欧元（A组）和8,746欧元（B组）。使用癌症治疗功能评估-贫血（FACT-An）量表评估生活质量。两组中显示出显著临床改善的患者比例相似。rHuEPO加rHuG-CSF使41.7%的MDS患者有反应。这种治疗费用昂贵。未显示对生活质量有影响。
