Thalidomide as initial therapy for early-stage myeloma
Thalidomide as initial therapy for early-stage myeloma
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DOI：
10.1038/sj.leu.2402866
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发表时间：
2003-04-01
期刊：
影响因子：
11.4
通讯作者：
Witzig, TE
中科院分区：
文献类型：
--
作者：
Rajkumar, SV;Gertz, MA;Witzig, TE
早期骨髓瘤患者通常不进行治疗，直到出现症状性疾病。然而，他们进展为症状性骨髓瘤的风险很高，进展的中位时间约为1-2年。我们报告了沙利度胺作为早期多发性骨髓瘤的初始治疗的11期试验的最终结果，该试验试图延缓进展为有症状的疾病。在梅奥诊所共研究了31例阴燃性或惰性多发性骨髓瘤患者。两名患者被认为不合格，因为他们之前接受过骨髓瘤治疗，除了毒性外，他们被排除在分析之外。沙利度胺的起始剂量为200mg/天。患者前6个月每月随访一次，此后每3个月随访一次。在29例符合条件的患者中，10例（34%）对治疗有部分反应，血清和尿液单克隆(M)蛋白减少至少50%或更多。当包括轻微反应（M蛋白减少25-49%）时，反应率为66%。3例患者在治疗期间病情进展。Kaplan-Meier估计1年无进展生存率为80%，2年为63%。3-4级主要毒性包括2例嗜睡和1例神经病变、深静脉血栓形成、听力丧失、虚弱、窦性心动过缓和水肿。沙利度胺在早期骨髓瘤中具有显著的活性，并有可能延缓疾病的进展。这种方法必须在随机试验中进一步检验。
Patients with early-stage myeloma are typically observed without therapy until symptomatic disease occurs. However, they are at high risk of progression to symptomatic myeloma, with a median time to progression of approximately 1-2 years. We report the final results of a phase 11 trial of thalidomide as initial therapy for early-stage multiple myeloma in an attempt to delay progression to symptomatic disease. In total, 31 patients with smoldering or indolent multiple myeloma were studied at the Mayo Clinic. Two patients were deemed ineligible because they were found to have received prior therapy for myeloma, and were excluded from analyses except for toxicity. Thalidomide was initiated at a starting dose of 200mg/day. Patients were followed-up monthly for the first 6 months and every 3 months thereafter. Of the 29 eligible patients, 10 (34%) had a partial response to therapy with at least 50% or greater reduction in serum and urine monoclonal (M) protein. When minor responses (25-49% decrease in M protein) were included, the response rate was 66%. Three patients had progressive disease while on therapy. Kaplan-Meier estimates of progression-free survival are 80% at 1 year and 63% at 2 years. major grade 3-4 toxicities included two patients with somnolence and one patient each with neuropathy, deep-vein thrombosis, hearing loss, weakness, sinus bradycardia, and edema. Thalidomide has significant activity in early-stage myeloma and has the potential to delay progression to symptomatic disease. This approach must be further tested in randomized trials.