文章：

多发性骨髓瘤中基于来那度胺治疗的异常应答者特征

Characteristics of exceptional responders to lenalidomide-based therapy in multiple myeloma

原文发布日期：2015-10-23

DOI: 10.1038/bcj.2015.91

类型: Original Article

开放获取: 是

英文摘要：

We studied all patients at our institution with a diagnosis of multiple myeloma (MM), from 1 January 2004 to 1 July 2009, who received lenalidomide–dexamethasone (Rd) as initial therapy and had a time to progression of 72 months or longer. Of 240 patients, we identified 33 exceptional responders. Twenty-five patients received primary therapy with Rd and eight patients received Rd induction followed by early stem cell transplantation (SCT). Seven of the eight patients who received SCT did not receive maintenance therapy; one patient received 9 months of lenalidomide post transplant. Fifteen (45%) patients had known clonal plasma cell disorder before the diagnosis of MM. The dominant mode of clinical presentation was with lytic lesions in 28 patients. Of those with informative cytogenetics (n=24), trisomies were present in 19 (79%), including one patient with concurrent trisomies and t(11;14). Overall, 21 of 24 patients (88%) had either trisomies or t(11;14). None of these exceptional responders had high-risk cytogenetic features at baseline. Twenty-five patients (76%) had a complete response (CR), whereas eight patients (24%) achieved the exceptional response state without ever achieving a CR. We identify a cohort of exceptional responders to Rd-based therapy, representing ~10–15% newly diagnosed MM patients with normal renal function.

摘要翻译： 

我们研究了所在机构2004年1月1日至2009年7月1日期间所有诊断为多发性骨髓瘤（MM）的患者，这些患者均以来那度胺-地塞米松（Rd）作为初始治疗且疾病进展时间达72个月或更长。在240例患者中，我们确定了33例特别应答者。其中25例患者接受Rd作为主要治疗，8例患者接受Rd诱导治疗后早期进行干细胞移植（SCT）。接受SCT的8例患者中有7例未接受维持治疗；1例患者在移植后接受了9个月的来那度胺治疗。15例（45%）患者在MM确诊前已知存在克隆性浆细胞疾病。28例患者的主要临床表现是溶骨性病变。在可获得细胞遗传学信息的患者（n=24）中，19例（79%）存在三体性，其中1例同时存在三体性和t(11;14)。总体而言，24例患者中有21例（88%）存在三体性或t(11;14)。这些特别应答者在基线时均未出现高危细胞遗传学特征。25例患者（76%）达到完全缓解（CR），而8例患者（24%）虽未达到CR但仍实现特别应答状态。我们确定了一组对Rd方案治疗具有特别应答的患者队列，约占肾功能正常的新诊断MM患者的10%-15%。

原文链接：

Characteristics of exceptional responders to lenalidomide-based therapy in multiple myeloma