Hellström-Lindberg Eva, Gulbrandsen Nina, Lindberg Greger, Ahlgren Tomas, Dahl Inger Marie S, Dybedal Ingunn, Grimfors Gunnar, Hesse-Sundin Eva, Hjorth Martin, Kanter-Lewensohn Lena, Linder Olle, Luthman Michaela, Löfvenberg Eva, Oberg Gunnar, Porwit-MacDonald Anja, Rådlund Anders, Samuelsson Jan, Tangen Jon Magnus, Winquist Ingemar, Wisloff Finn
Department of Medicine, Karolinska Institutet at Huddinge University Hospital, Stockholm, Sweden.
Br J Haematol. 2003 Mar;120(6):1037-46. doi: 10.1046/j.1365-2141.2003.04153.x.
We have published previously a prototype of a decision model for anaemic patients with myelodysplastic syndromes (MDS), in which transfusion need and serum erythropoietin (S-Epo) were used to define three groups with different probabilities of erythroid response to treatment with granulocyte colony-stimulating factor (G-CSF) + Epo. S-Epo </= 500 U/l and a transfusion need of < 2 units/month predicted a high probability of response to treatment, S-Epo > 500 U/l and >/= 2 units/month for a poor response, whereas the presence of only one negative prognostic marker predicted an intermediate response. A total of 53 patients from a prospective study were included in our evaluation sample. Patients with good or intermediate probability of response were treated with G-CSF + Epo. The overall response rate was 42% with 28.3% achieving a complete and 13.2% a partial response to treatment. The response rates were 61% and 14% in the good and intermediate predictive groups respectively. The model retained a significant predictive value in the evaluation sample (P < 0.001). Median duration of response was 23 months. Scores for global health and quality of life (QOL) were significantly lower in MDS patients than in a reference population, and fatigue and dyspnoea was significantly more prominent. Global QOL improved in patients responding to treatment (P = 0.01). The validated decision model defined a subgroup of patients with a response rate of 61% (95% confidence interval 48-74%) to treatment with G-CSF + Epo. The majority of these patients have shown complete and durable responses.
我们之前发表过一种用于骨髓增生异常综合征（MDS）贫血患者的决策模型原型，该模型中使用输血需求和血清促红细胞生成素（S-Epo）来定义三组对粒细胞集落刺激因子（G-CSF）+促红细胞生成素治疗有不同红系反应概率的患者。S-Epo≤500 U/l且输血需求<2单位/月预示对治疗有高反应概率，S-Epo>500 U/l且≥2单位/月预示反应较差，而仅存在一个阴性预后标志物预示为中等反应。我们的评估样本纳入了一项前瞻性研究中的53例患者。反应概率为良好或中等的患者接受G-CSF+促红细胞生成素治疗。总体反应率为42%，其中28.3%达到完全缓解，13.2%达到部分缓解。良好和中等预测组的反应率分别为61%和14%。该模型在评估样本中保留了显著的预测价值（P<0.001）。反应的中位持续时间为23个月。MDS患者的总体健康和生活质量（QOL）评分显著低于参考人群，疲劳和呼吸困难更为突出。治疗有反应的患者总体QOL有所改善（P = 0.01）。经过验证的决策模型定义了一组对G-CSF+促红细胞生成素治疗反应率为61%（95%置信区间48-74%）的患者亚组。这些患者中的大多数已显示出完全且持久的反应。
