Thalidomide produces transfusion independence in long-standing refractory anemias of patients with myelodysplastic syndromes
Thalidomide produces transfusion independence in long-standing refractory anemias of patients with myelodysplastic syndromes
复制标题
DOI：
10.1182/blood.v98.4.958
复制
发表时间：
2001-08-15
期刊：
影响因子：
20.3
通讯作者：
Zeldis, J
中科院分区：
文献类型：
--
作者：
Raza, A;Meyer, P;Zeldis, J
沙利度胺用于83例骨髓增生异常综合征（FADS）患者，起始剂量为每日口服100 mg，耐受后增加至400 mg。32名患者在12周（反应评估的最短时间）前停止治疗，51名患者完成了12周的治疗。使用MDS国际工作组缓解标准评价缓解。意向治疗（ITT）分析将所有退出研究的患者归类为无应答者。未参加研究的患者属于高风险类别（P = .002），其治疗前骨髓中的原始细胞百分比高于完成12周治疗的患者（P = .003）。未观察到细胞遗传学完全缓解，但16例患者显示血液学改善，其中10例先前依赖输血的患者变为不依赖输血。应答者治疗前原始细胞较低（P = 0.016），治疗前血小板输注持续时间较短（P = 0.013），治疗前血小板较高（P = 0.003）。在应答者中，9例患有难治性贫血（RA）; 5例患有RA伴环形铁粒幼细胞; 2例患有RA伴原始细胞过多。通过ITT分析，19%的患者（83例中的16例）应答，当仅分析可评价患者时，31%（51例中的16例）应答。结论：沙利度胺单药治疗可有效改善某些MDS患者的血细胞减少，尤其是那些无过多原始细胞的患者。(C)2001年，美国血液学会。
Thalidomide was administered to 83 patients with myelodysplastic syndrome (FADS), starting at 100 mg by mouth daily and increasing to 400 mg as tolerated. Thirty-two patients stopped therapy before 12 weeks (minimum period for response evaluation), and 51 completed 12 weeks of therapy. International Working Group response criteria for MDS were used to evaluate responses. Intent-to-treat (ITT) analysis classified all off-study patients as nonresponders. Off-study patients belonged to a higher risk category (P = .002) and had a higher percentage of blasts in their pretherapy bone marrow than patients who completed 12 weeks of therapy (P = .003). No cytogenetic br complete responses were seen, but 16 patients showed hematologic improvement, with 10 previously transfusion-dependent patients becoming transfusion independent. Responders had lower pretherapy blasts (P = .016), a lower duration of pretherapy platelet transfusions (P = .013), and higher pretherapy platelets (P = .003). Among responders, 9 had refractory anemia (RA); 5 had RA with ringed sideroblasts; and 2 had RA with excess blasts. By ITT analysis, 19% of patients (16 of 83) responded, and when only evaluable patients were analyzed, 31% (16 of 51) responded. It was concluded that thalidomide, as a single agent, is effective in improving cytopenias of some MDS patients, especially those who present without excess blasts. (C) 2001 by The American Society of Hematology.