Abstract

Pomalidomide demonstrated activity in the treatment of AL amyloidosis in three phase II clinical trials. We evaluated the safety and efficacy of 28-day cycles of pomalidomide and dexamethasone in 153 previously treated patients with systemic AL amyloidosis. Ninety-nine (65%) were refractory to the last line of therapy and 54 (35%) had relapsed. The median number of previous lines of therapy was 3 (range: 2–7): 143 patients (93%) previously received bortezomib, 124 (81%) lenalidomide, 114 (75%) oral melphalan, and 37 (24%) underwent autologous stem cell transplant. At the completion of cycle 6, 68 (44%) patients obtained at least partial haematologic response, with 5 complete responses (CR, 3%), 35 very good partial responses (VGPR, 23%). Haematologic response resulted in improved overall survival (median survival 50 vs. 27 months, p = .033) in a 6 months landmark analysis. Obtaining at least partial response was also associated with a significant improvement of the progression-free survival (median PFS 37 vs. 18 months, p < .001). Pomalidomide is an effective treatment for heavily pre-treated patients with AL amyloidosis. Haematologic responses are associated with an overall survival advantage.

摘要

泊马度胺在三项 II 期临床试验中显示出治疗 AL 淀粉样变性的活性。我们在 153 名既往接受过治疗的全身性 AL 淀粉样变性患者中评估了泊马度胺和地塞米松 28 天周期的安全性和有效性。99 人 (65%) 对最后一线治疗无效，54 人 (35%) 复发。既往治疗线的中位数为 3（范围：2-7）：143 名患者 (93%) 之前接受过硼替佐米，124 名 (81%) 来那度胺，114 名 (75%) 口服美法仑，37 名 (24%) 接受过自体干细胞移植。在第 6 周期结束时，68 名 (44%) 患者获得至少部分血液学缓解，其中 5 名完全缓解（CR，3%），35 名非常好的部分缓解（VGPR，23%）。血液学反应导致总生存期提高（中位生存期为 50 个月与 27 个月，p  = .033) 在 6 个月的里程碑分析中。获得至少部分缓解还与无进展生存期的显着改善相关（中位 PFS 37 个月与 18 个月，p  < .001）。Pomalidomide 是一种有效的治疗方法，用于治疗大量预先治疗的 AL 淀粉样变性患者。血液学反应与总体生存优势相关。