The activity of lenalidomide with or without dexamethasone in patients with primary systemic amyloidosis
The activity of lenalidomide with or without dexamethasone in patients with primary systemic amyloidosis
复制标题
DOI：
10.1182/blood-2006-07-032987
复制
发表时间：
2007-01-15
期刊：
影响因子：
20.3
通讯作者：
Gertz, Morie A.
中科院分区：
文献类型：
--
作者：
Dispenzieri, Angela;Lacy, Martha Q.;Gertz, Morie A.
原发性系统性淀粉样变（AL）是一种无法治愈的浆细胞疾病。来那度胺，特别是与地塞米松联合使用，在多发性骨髓瘤患者中具有高度活性。我们研究了来那度胺在AL患者中的毒性和疗效。有症状的AL患者，可测量的浆细胞疾病，以及足够的血液和肾脏储备都符合条件。患者接受来那度胺单药治疗。如果3个月后无进展证据或3个周期后无血液学反应，则加用地塞米松。23例患者入组。13人曾接受过治疗。器官受累为心脏（64%）、肾脏（73%）、肝脏（23%）和神经（14%）。在治疗的前3个周期内，10例患者停止治疗：4例早期死亡，3例不良事件和3例其他原因。中位随访时间为17个月，10名患者对治疗有反应。在这些患者中，缓解包括9例血液学缓解、4例肾脏缓解、2例心脏缓解和2例肝脏缓解。除了一个应答者外，所有应答者都在他们的治疗计划中加入了地塞米松。至少可能归因于来那度胺的最常见的3级或4级不良事件是中性粒细胞减少症（45%）、血小板减少症（27%）、皮疹（18%）和疲劳（18%）。在AL患者中，我们观察到来那度胺单药治疗的活性有限，但与地塞米松联合治疗的活性显著，这需要进一步研究。
Primary systemic amylolclosis (AL) is an incurable plasma cell disorder. Lenalidomide, especially in conjunction with dexamethasone, is highly active in patients with multiple myeloma. We studied the toxicity and efficacy of lenalidomide in patients with AL. Patients with symptomatic AL, a measurable plasma cell disorder, and adequate hematologic and renal reserve were eligible. Patients received single-agent lenalidomide. If there was no evidence of progression after 3 months or of hematologic response after 3 cycles, dexamethasone was added. Twenty-three patients were enrolled. Thirteen were previously treated. Organ involvement was cardiac (64%), renal (73%), hepatic (23%), and nerve (14%). Within the first 3 cycles of therapy, 10 patients discontinued treatment: 4 early deaths, 3 adverse events, and 3 other causes. With a median follow-up of 17 months, 10 patients responded to treatment. In these patients, responses included 9 hematologic, 4 renal, 2 cardiac, and 2 hepatic. All but one of the responders had dexamethasone added to their treatment program. The most common grade 3 or 4 adverse events at least possibly attributable to lenalidomide were neutropenla (45%), thrombocytopenia (27%), rash (18%), and fatigue (18%). In AL patients, we saw limited activity of single-agent lenalidomide, but significant activity of the combination with dexamethasone, which warrants further investigation.