自体干细胞移植对原发性难治性骨髓瘤有益？疾病进展与稳定的不同结果。
Benefit from autologous stem cell transplantation in primary refractory myeloma? Different outcomes in progressive versus stable disease.
机构信息
Hospital Clínic, Villarroel, 170, 08036 Barcelona, Spain.
出版信息
Haematologica. 2012 Apr;97(4):616-21. doi: 10.3324/haematol.2011.051441. Epub 2011 Nov 4.
BACKGROUND
Several studies of autologous stem cell transplantation in primary refractory myeloma have produced encouraging results. However, the outcome of primary refractory patients with stable disease has not been analyzed separately from the outcome of patients with progressive disease.
DESIGN AND METHODS
In the Spanish Myeloma Group 2000 trial, 80 patients with primary refractory myeloma (49 with stable disease and 31 with progressive disease), i.e. who were refractory to initial chemotherapy, were scheduled for tandem transplants (double autologous transplant or a single autologous transplant followed by an allogeneic transplant). Patients with primary refractory disease included those who never achieved a minimal response (≥ 25% M-protein decrease) or better. Responses were assessed using the European Bone Marrow Transplant criteria.
RESULTS
There were no significant differences in the rates of partial response or better between patients with stable or progressive disease. However, 38% of the patients with stable disease at the time of transplantation remained in a stable condition or achieved a minimal response after transplantation versus 7% in the group with progressive disease (P=0.0017) and the rate of early progression after transplantation was significantly higher among the group with progressive disease at the time of transplantation (22% versus 2%; P=0.0043). After a median follow-up of 6.6 years, the median survival after first transplant of the whole series was 2.3 years. Progression-free and overall survival from the first transplant were shorter in patients with progressive disease (0.6 versus 2.3 years, P=0.00004 and 1.1 versus 6 years, P=0.00002, respectively).
CONCLUSIONS
Our results show that patients with progressive refractory myeloma do not benefit from autologous transplantation, while patients with stable disease have an outcome comparable to those with chemosensitive disease.
背景
几项原发性难治性骨髓瘤自体干细胞移植的研究取得了令人鼓舞的结果。然而，稳定疾病原发性难治性患者的结果尚未与进展性疾病患者的结果分开分析。
设计和方法
在西班牙骨髓瘤组 2000 年的试验中，80 例原发性难治性骨髓瘤患者（49 例稳定疾病，31 例进展性疾病），即对初始化疗有耐药性，计划进行串联移植（双自体移植或单次自体移植后进行异基因移植）。原发性难治性疾病患者包括那些从未达到最小反应（≥25% M 蛋白减少）或更好的患者。反应使用欧洲骨髓移植标准进行评估。
结果
稳定疾病或进展性疾病患者的部分缓解或更好的比例没有显著差异。然而，38%的移植时稳定疾病的患者在移植后仍处于稳定状态或达到最小反应，而进展性疾病组为 7%（P=0.0017），且进展性疾病组移植后早期进展的发生率明显更高（22%比 2%；P=0.0043）。在中位数为 6.6 年的随访后，整个系列的首次移植后中位生存期为 2.3 年。进展性疾病患者的无进展生存期和总生存期从首次移植起更短（0.6 年与 2.3 年，P=0.00004 和 1.1 年与 6 年，P=0.00002）。
结论
我们的结果表明，进展性难治性骨髓瘤患者不能从自体移植中获益，而稳定疾病患者的结果与化疗敏感疾病患者相当。
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