Wien Tale Norbye
Medisinsk avdeling Seksjon for nyresykdommer Rikshospitalet 0027 Oslo.
Tidsskr Nor Laegeforen. 2008 Jun 12;128(12):1397-400.
Treatment of amyloidosis is a substantial challenge. Various types of amyloidosis are associated with a broad spectrum of organ manifestations and considerable morbidity and mortality.
The present paper is based on a review of international literature, retrieved through a PubMed search on amyloidosis, and the author's clinical experience.
Current treatment of amyloidosis is first and foremost aimed at reducing the level of amyloid precursor proteins; e.g. immunosuppression in AA amyloidosis, chemotherapy in AL amyloidosis and supportive treatment of diminished or lost organ function. Aggressive immunomodulating treatment increases survival in AA amyloidosis associated with rheumatic disease, and high-dose melphalan with autologous stem-cell transplantation (HMAS) has in selected studies shown increased survival in AL amyloidosis. More recent knowledge on mechanisms of amyloid formation has given rise to novel therapeutic targets that may be useful for all amyloidoses. Examples of this are immunotherapy and inhibition of the interaction of extrafibrillary components, which have shown benefit in animal models for AA, AL and Abeta amyloidosis as well as in the first human studies. More studies are needed to define their place in treatment of amyloidosis.
淀粉样变性的治疗是一项重大挑战。各种类型的淀粉样变性与广泛的器官表现以及相当高的发病率和死亡率相关。
本文基于对国际文献的综述,通过在PubMed上搜索淀粉样变性获得,以及作者的临床经验。
目前淀粉样变性的治疗首先旨在降低淀粉样前体蛋白的水平;例如,AA型淀粉样变性采用免疫抑制治疗,AL型淀粉样变性采用化疗,以及对功能减退或丧失的器官进行支持性治疗。积极的免疫调节治疗可提高与风湿性疾病相关的AA型淀粉样变性患者的生存率,在一些研究中,高剂量美法仑联合自体干细胞移植(HMAS)已显示可提高AL型淀粉样变性患者的生存率。关于淀粉样蛋白形成机制的最新知识催生了可能对所有淀粉样变性都有用的新治疗靶点。例如免疫疗法以及抑制纤维外成分的相互作用,这些在AA型、AL型和β淀粉样变性的动物模型以及首批人体研究中已显示出益处。需要更多研究来确定它们在淀粉样变性治疗中的地位。