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硼替佐米/地塞米松联合自体干细胞移植作为轻链沉积病的一线治疗。

Bortezomib/dexamethasone followed by autologous stem cell transplantation as front line treatment for light-chain deposition disease.

机构信息

Department of Hematology, Amyloidosis and Myeloma Unit, Hospital Clínic, Barcelona, Spain.

出版信息

Eur J Haematol. 2012 Oct;89(4):340-4. doi: 10.1111/j.1600-0609.2012.01821.x. Epub 2012 Jul 10.

Abstract

Limited data has been published on the treatment results in patients with light-chain deposition disease (LCDD). Whenever possible, high-dose melphalan followed by autologous stem cell transplantation (ASCT) has been the first treatment option, achieving somehow better results than conventional therapy. However, and based on the promising results obtained by treating patients with light-chain amyloidosis with bortezomib/dexamethasone, new treatment options appear in LCDD. Herein, we describe three patients with LCDD treated with bortezomib/dexamethasone followed by high-dose melphalan and autologous transplantation. We believe that this new approach should be the treatment of choice in this disease. In addition, those patients achieving hematologic complete response after ASCT could benefit from a kidney transplant if the renal impairment requiring dialysis persists.

摘要

关于轻链沉积病(LCDD)患者的治疗结果,已有有限的数据发表。只要有可能,大剂量马法兰联合自体造血干细胞移植(ASCT)一直是首选的治疗方案,其疗效优于常规治疗。然而,基于硼替佐米/地塞米松治疗轻链淀粉样变性患者取得的有希望的结果,LCDD 出现了新的治疗选择。在此,我们描述了 3 例接受硼替佐米/地塞米松治疗后再接受大剂量马法兰和自体移植的 LCDD 患者。我们认为,这种新方法应该是这种疾病的治疗选择。此外,如果需要透析的肾功能损害持续存在,ASCT 后达到血液学完全缓解的患者可以从肾移植中获益。

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