Although the clinical outcome of newly diagnosed multiple myeloma has improved with maintenance therapy, maintenance with novel agents is not always available depending on medical expenses or drug accessibility. We intended to investigate the efficacy and toxicity of thalidomide/dexamethasone maintenance in Korean patients. In this multicenter phase 2 study, patients with newly diagnosed myeloma who underwent induction chemotherapy followed by autologous stem cell transplantation (ASCT) were enrolled to receive maintenance treatment of 100mg thalidomide daily for 28 days and 40mg dexamethasone daily for 4 days each cycle. Maintenance was given up to 12 cycles. The primary endpoint was a 1-year event free survival (EFS) rate. It was assumed that EFS at 1-year would be 91% with thalidomide and 1-year EFS below 82% would be of no effect. A total of 43 patients were consecutively enrolled (median age, 58 years [range, 34 – 65]; male, n = 31). With a median follow-up duration of 17.3 months (range, 1.1 – 32.2), EFS at 1 year was 65.1% (95% confidence interval [CI], 48.9 – 77.3). PFS and OS at 1 year was 85.6% (95% CI, 70.7 – 93.3) and 90.4 (95% CI, 76.3 – 96.3), respectively. In terms of side effects, 39 patients (90.7%) experienced adverse events (AEs) of any grade, and 14 patients (32.6%) experienced grade 3 or 4 adverse events. 15 patients (34.9%) failed to complete 12 cycles of maintenance, and the most common reason for premature termination was AEs (n = 6). In Korean patients the benefits of thalidomide maintenance does not seem to outweigh the toxicity of thalidomide, especially in high-risk MM. Considering the long clinical course of MM, preservation of quality of life and finances might be more beneficial for subsequent MM treatment.

尽管新诊断的多发性骨髓瘤的临床结果通过维持治疗有所改善，但根据医疗费用或药物可及性，并不总是可以使用新型药物进行维持。我们打算调查沙利度胺/地塞米松维持治疗对韩国患者的疗效和毒性。在这项多中心 2 期研究中，新诊断的骨髓瘤患者在接受诱导化疗后自体干细胞移植 (ASCT) 后被纳入接受每天 100mg 沙利度胺持续 28 天和每天 40mg 地塞米松持续治疗 4 天的维持治疗，每个周期。最多给予 12 个周期的维护。主要终点是 1 年无事件生存 (EFS) 率。假设沙利度胺的 1 年 EFS 为 91%，而 1 年 EFS 低于 82% 将无效。n  = 31)。中位随访时间为 17.3 个月（范围，1.1 – 32.2），1 年的 EFS 为 65.1%（95% 置信区间 [CI]，48.9 – 77.3）。1 年的 PFS 和 OS 分别为 85.6% (95% CI, 70.7 – 93.3) 和 90.4 (95% CI, 76.3 – 96.3)。在副作用方面，39 名患者 (90.7%) 经历了任何级别的不良事件 (AE)，14 名患者 (32.6%) 经历了 3 级或 4 级不良事件。15 名患者（34.9%）未能完成 12 个周期的维持，提前终止的最常见原因是 AE（n = 6)。在韩国患者中，维持沙利度胺的益处似乎并没有超过沙利度胺的毒性，尤其是在高危 MM 中。考虑到 MM 的长期临床病程，保持生活质量和财务状况可能对后续 MM 治疗更有利。