Mikala G, Jákó J, Vályi-Nagy I
Egészségtudományi Kar, Belgyógyászati és Geriátriai Klinika, Semmelweis Egyetem, Budapest.
Orv Hetil. 2001 Aug 19;142(33):1789-98.
Multiple myeloma is a relatively common hematologic malignancy with no definitive treatment available. Although, therapy may include allogenic bone marrow transplantation, high-dose ablative chemotherapy followed by bone marrow or peripheral stem cell transplantation, melphalan/corticosteroid therapy, alpha-interferon treatment, and combined cytostatic chemotherapy, currently none of these alternatives offers cure for the disease. Thalidomide is an infamous molecule for its teratogenicity, yet it possesses potent immunomodulatory, anti-angiogeneic and, in higher concentrations, direct anti-myeloma-cell properties. At present, the drug is only approved for the treatment of erythema nodosum of leprosy, however, there are several preliminary results that show clinical efficacy in multiple myeloma. This drug has especially potent anti-myeloma effects in combinations with dexamethasone and certain cytostatic chemotherapeutic agents. The effects are evident both in polyresistant, and relapsing myeloma, a form with no accepted effective treatment options. In this paper, the fundamental molecular and cellular effects of thalidomide are summarized then the most important clinical studies with thalidomide are reviewed. It is the authors' hope that thalidomide will soon be a full member of the medical arsenal in the fight against multiple myeloma.
多发性骨髓瘤是一种相对常见的血液系统恶性肿瘤,目前尚无确切的治疗方法。虽然治疗方法可能包括异基因骨髓移植、大剂量清髓性化疗后进行骨髓或外周干细胞移植、美法仑/皮质类固醇疗法、α-干扰素治疗以及联合细胞毒性化疗,但目前这些方法都无法治愈该疾病。沙利度胺因其致畸性而臭名昭著,但它具有强大的免疫调节、抗血管生成特性,在较高浓度时还具有直接抗骨髓瘤细胞的特性。目前,该药物仅被批准用于治疗麻风结节性红斑,然而,有一些初步结果显示其在多发性骨髓瘤中具有临床疗效。该药物与地塞米松及某些细胞毒性化疗药物联合使用时,具有特别强大的抗骨髓瘤作用。在多药耐药和复发性骨髓瘤(一种尚无公认有效治疗方案的类型)中,这种作用都很明显。本文总结了沙利度胺的基本分子和细胞效应,然后综述了关于沙利度胺的最重要的临床研究。作者希望沙利度胺很快能成为对抗多发性骨髓瘤的药物武器库中的正式成员。