Jing Huang, Chunyan Sun, H. Su, Chun Zhang, J. Xiong
{"title":"硼替佐米化疗联合自体干细胞移植治疗肾脏单克隆γ病1例报告并文献复习","authors":"Jing Huang, Chunyan Sun, H. Su, Chun Zhang, J. Xiong","doi":"10.1159/000501314","DOIUrl":null,"url":null,"abstract":"Background/Aims: The term monoclonal gammopathy of renal significance (MGRS) was introduced in 2012 to emphasize kidney lesions in monoclonal gammopathy patients. Bortezomib-based chemotherapy has become the first-line treatment for MGRS. Objectives: The objective of this study was to investigate whether the strategy of combining chemotherapy with autologous stem cell transplantation (ASCT) could improve prognosis and decrease functional kidney impairment in patients with MGRS. Methods: We reported the case of a 44-year-old Asian patient who was diagnosed with MGRS and received 5 cycles of Velcade® (a trade name for bortezomib), thalidomide, and dexamethasone therapy (VTD therapy), and subsequently underwent ASCT. In addition, we performed a literature review and summarized the latest advances in the characterization, treatment, and prognosis of MGRS. Results: The patient was diagnosed with light chain deposition disease by renal biopsy. After 5 cycles of VTD therapy, the patient had a very good partial response characterized by the resolution of M-protein (20.2% before treatment vs. 2.5% after treatment), remission of the level of serum free lambda (FLAM; over 80% decline), and normalization of the serum free light chain (sFLC) ratio (κ to λ). He also had a renal response characterized by a decreased serum creatinine level (1.61 vs.1.34 mg/dL) and less severe proteinuria (6.77 g/24 h vs.1.264 g/24 h) after chemotherapy. Importantly, after ASCT, the patient achieved a complete response (CR) characterized by a negative serum immunofixation electrophoresis (IFE) result and a dramatic decrement in FLAM (over 90%). Furthermore, 6 months after ASCT, the patient still remained in stable condition with a negative IFE result, normal sFLC ratio, and low level of serum creatinine (1.31 mg/dL) and proteinuria (0.339 g/24 h). In our retrospective literature analysis, we found that MGRS patient survival time and renal outcome had been markedly improved by current therapies due to the popularization of bortezomib-based chemotherapy and ASCT. Conclusions: The patient successfully achieved CR after VTD therapy followed by ASCT. However, this treatment is controversial, and a standard therapy recommendation for MGRS has not been established. Bortezomib-based chemotherapy combined with ASCT may have prospects for the treatment of MGRS, but the exact effects of ASCT remain unclear and should be thoroughly investigated.","PeriodicalId":17810,"journal":{"name":"Kidney and Blood Pressure Research","volume":"12 1","pages":"858 - 869"},"PeriodicalIF":0.0000,"publicationDate":"2019-07-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"5","resultStr":"{\"title\":\"Bortezomib-Based Chemotherapy with Autologous Stem Cell Transplantation for Monoclonal Gammopathy of Renal Significance: A Case Report and Literature Review\",\"authors\":\"Jing Huang, Chunyan Sun, H. Su, Chun Zhang, J. Xiong\",\"doi\":\"10.1159/000501314\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"Background/Aims: The term monoclonal gammopathy of renal significance (MGRS) was introduced in 2012 to emphasize kidney lesions in monoclonal gammopathy patients. Bortezomib-based chemotherapy has become the first-line treatment for MGRS. Objectives: The objective of this study was to investigate whether the strategy of combining chemotherapy with autologous stem cell transplantation (ASCT) could improve prognosis and decrease functional kidney impairment in patients with MGRS. Methods: We reported the case of a 44-year-old Asian patient who was diagnosed with MGRS and received 5 cycles of Velcade® (a trade name for bortezomib), thalidomide, and dexamethasone therapy (VTD therapy), and subsequently underwent ASCT. In addition, we performed a literature review and summarized the latest advances in the characterization, treatment, and prognosis of MGRS. Results: The patient was diagnosed with light chain deposition disease by renal biopsy. After 5 cycles of VTD therapy, the patient had a very good partial response characterized by the resolution of M-protein (20.2% before treatment vs. 2.5% after treatment), remission of the level of serum free lambda (FLAM; over 80% decline), and normalization of the serum free light chain (sFLC) ratio (κ to λ). He also had a renal response characterized by a decreased serum creatinine level (1.61 vs.1.34 mg/dL) and less severe proteinuria (6.77 g/24 h vs.1.264 g/24 h) after chemotherapy. Importantly, after ASCT, the patient achieved a complete response (CR) characterized by a negative serum immunofixation electrophoresis (IFE) result and a dramatic decrement in FLAM (over 90%). Furthermore, 6 months after ASCT, the patient still remained in stable condition with a negative IFE result, normal sFLC ratio, and low level of serum creatinine (1.31 mg/dL) and proteinuria (0.339 g/24 h). In our retrospective literature analysis, we found that MGRS patient survival time and renal outcome had been markedly improved by current therapies due to the popularization of bortezomib-based chemotherapy and ASCT. Conclusions: The patient successfully achieved CR after VTD therapy followed by ASCT. However, this treatment is controversial, and a standard therapy recommendation for MGRS has not been established. Bortezomib-based chemotherapy combined with ASCT may have prospects for the treatment of MGRS, but the exact effects of ASCT remain unclear and should be thoroughly investigated.\",\"PeriodicalId\":17810,\"journal\":{\"name\":\"Kidney and Blood Pressure Research\",\"volume\":\"12 1\",\"pages\":\"858 - 869\"},\"PeriodicalIF\":0.0000,\"publicationDate\":\"2019-07-26\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"5\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Kidney and Blood Pressure Research\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://doi.org/10.1159/000501314\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"\",\"JCRName\":\"\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Kidney and Blood Pressure Research","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.1159/000501314","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"","JCRName":"","Score":null,"Total":0}
引用次数: 5
摘要
背景/目的:2012年引入肾脏意义单克隆伽玛病(monoclonal gammopathy of renal significance, MGRS)一词,以强调单克隆伽玛病患者的肾脏病变。以硼替佐米为基础的化疗已成为MGRS的一线治疗。目的:本研究的目的是探讨化疗联合自体干细胞移植(ASCT)策略是否可以改善MGRS患者的预后并减少功能性肾损害。方法:我们报告了一例44岁的亚洲患者,他被诊断为MGRS,接受了5个周期的Velcade®(硼替佐米的商品名称)、沙利度胺和地塞米松治疗(VTD治疗),随后进行了ASCT。此外,我们还进行了文献综述,总结了MGRS的表征、治疗和预后的最新进展。结果:患者经肾活检诊断为轻链沉积病。经过5个疗程的VTD治疗,患者获得了非常好的部分缓解,其特征是m蛋白的溶解(治疗前20.2%,治疗后2.5%),血清游离lambda (FLAM)水平缓解;下降80%以上),血清游离轻链(sFLC)比值(κ to λ)正常化。他也有肾脏反应,特点是化疗后血清肌酐水平下降(1.61 vs.1.34 mg/dL)和较轻的蛋白尿(6.77 g/24 h vs.1.264 g/24 h)。重要的是,ASCT后,患者实现了完全缓解(CR),其特征是血清免疫固定电泳(IFE)结果为阴性,FLAM显著下降(超过90%)。此外,ASCT后6个月,患者病情仍然稳定,IFE结果阴性,sFLC比率正常,血清肌酐(1.31 mg/dL)和蛋白尿(0.339 g/24 h)水平较低。在我们的回顾性文献分析中,我们发现,由于硼替唑米为主的化疗和ASCT的普及,目前的治疗方法明显改善了MGRS患者的生存时间和肾脏预后。结论:患者经VTD + ASCT治疗后,成功达到CR。然而,这种治疗是有争议的,对于MGRS的标准治疗建议尚未建立。以硼替佐米为基础的化疗联合ASCT治疗MGRS可能有前景,但ASCT的确切效果尚不清楚,应深入研究。
Bortezomib-Based Chemotherapy with Autologous Stem Cell Transplantation for Monoclonal Gammopathy of Renal Significance: A Case Report and Literature Review
Background/Aims: The term monoclonal gammopathy of renal significance (MGRS) was introduced in 2012 to emphasize kidney lesions in monoclonal gammopathy patients. Bortezomib-based chemotherapy has become the first-line treatment for MGRS. Objectives: The objective of this study was to investigate whether the strategy of combining chemotherapy with autologous stem cell transplantation (ASCT) could improve prognosis and decrease functional kidney impairment in patients with MGRS. Methods: We reported the case of a 44-year-old Asian patient who was diagnosed with MGRS and received 5 cycles of Velcade® (a trade name for bortezomib), thalidomide, and dexamethasone therapy (VTD therapy), and subsequently underwent ASCT. In addition, we performed a literature review and summarized the latest advances in the characterization, treatment, and prognosis of MGRS. Results: The patient was diagnosed with light chain deposition disease by renal biopsy. After 5 cycles of VTD therapy, the patient had a very good partial response characterized by the resolution of M-protein (20.2% before treatment vs. 2.5% after treatment), remission of the level of serum free lambda (FLAM; over 80% decline), and normalization of the serum free light chain (sFLC) ratio (κ to λ). He also had a renal response characterized by a decreased serum creatinine level (1.61 vs.1.34 mg/dL) and less severe proteinuria (6.77 g/24 h vs.1.264 g/24 h) after chemotherapy. Importantly, after ASCT, the patient achieved a complete response (CR) characterized by a negative serum immunofixation electrophoresis (IFE) result and a dramatic decrement in FLAM (over 90%). Furthermore, 6 months after ASCT, the patient still remained in stable condition with a negative IFE result, normal sFLC ratio, and low level of serum creatinine (1.31 mg/dL) and proteinuria (0.339 g/24 h). In our retrospective literature analysis, we found that MGRS patient survival time and renal outcome had been markedly improved by current therapies due to the popularization of bortezomib-based chemotherapy and ASCT. Conclusions: The patient successfully achieved CR after VTD therapy followed by ASCT. However, this treatment is controversial, and a standard therapy recommendation for MGRS has not been established. Bortezomib-based chemotherapy combined with ASCT may have prospects for the treatment of MGRS, but the exact effects of ASCT remain unclear and should be thoroughly investigated.