Salvage lenalidomide in four rare oncological diseases.
Salvage lenalidomide in four rare oncological diseases.
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DOI：
10.1700/1377.15326
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发表时间：
2013-09
期刊：
影响因子：
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通讯作者：
P. Szturz;Z. Adam;Z. Řehák;R. Koukalová;L. Křen;M. Moulis;M. Krejci;J. Mayer
中科院分区：
文献类型：
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作者：
P. Szturz;Z. Adam;Z. Řehák;R. Koukalová;L. Křen;M. Moulis;M. Krejci;J. Mayer
在罕见的疾病中，通常没有标准的治疗建议。患有难治性疾病的患者可能需要新的实验方法。作为二线和四线治疗，来那度胺(28天周期中第1-21天口服10-25 mg)用于我们队列中4名患有侵袭性、多系统和复发性疾病的成人患者。朗格汉斯细胞组织细胞增多症(11个周期，联合地塞米松和依托泊苷，通过异基因造血干细胞移植巩固)和浆细胞Castleman病(15个周期，单一治疗)患者获得了完全和持久的缓解(1年以上，无需维持治疗)。Erdheim-Chester病(6个周期，单一治疗)出现混合反应，脑浸润物完全消失；多发性血管瘤病(9个周期，联合沙利度胺)胃肠道出血得到较好控制。为了评估疾病活动性，每个患者都接受了氟-18-氟脱氧葡萄糖正电子发射断层扫描/计算机断层扫描成像，并辅以临床和实验室检查。
In rare disorders, there are often no standard therapy recommendations. Patients with refractory disease may require novel experimental approaches. Applied as second- up to fourth-line treatment, lenalidomide (10-25 mg perorally on days 1-21 in a 28-day cycle) was used in our cohort of four adult patients with aggressive, multisystem and relapsing diseases. Complete and long-lasting remissions (more than 1 year, no maintenance therapy) were achieved in patients with Langerhans cell histiocytosis (11 cycles, combination with dexamethasone and etoposide, consolidated by allogeneic blood stem cell transplant) and plasma-cell Castleman disease (15 cycles, monotherapy). Mixed response with complete disappearance of brain infiltrates was reached in Erdheim-Chester disease (6 cycles, monotherapy) and gastrointestinal bleeding was well controlled in multiple angiomatosis (9 cycles, combination with thalidomide). For disease activity evaluation each patient underwent fluorine-18-fluorodeoxyglucose positron emission tomography/computed tomography scan imaging, which was complemented by clinical and laboratory investigations.