ISSN 1006-298X      CN 32-1425/R

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肾脏病与透析肾移植杂志 ›› 2022, Vol. 31 ›› Issue (4): 304-309.DOI: 10.3969/j.issn.1006298X.2022.04.002

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泊马度胺联合地塞米松治疗系统性轻链型淀粉样变性的临床观察

  

  • 出版日期:2022-08-29 发布日期:2022-08-26

Pomalidomide and dexamethasone in treatment of systemic light chain amyloidosis

  • Online:2022-08-29 Published:2022-08-26

摘要: 目的:观察泊马度胺联合地塞米松(PD)方案治疗系统性轻链型(AL型)淀粉样变性的疗效及安全性。
方法:回顾性研究国家肾脏疾病临床医学研究中心应用PD方案治疗的AL型淀粉样变性患者,观察疗效和不良反应。
结果:共纳入19例患者,其中男性9例、女性10例,起病年龄59±11岁,心脏受累比例3158%,肾脏受累100%。有2例初治患者,难治和复发患者各7例,3例一线治疗有效但不耐受。患者中位接受2(1,4)周期泊马度胺治疗,总血液学反应3158%,初治者血液学反应为50%,难治者2857%,复发者4286%,且血液学反应均在治疗4周期内获得;有2例(1053%)心脏受累程度进展,其余维持稳定;6例(3158%)肾脏进展,3例(1579%)部分缓解,2例(1053%)较好的部分缓解,8例(4211%)维持稳定。3级及以上不良发生发生率为2104%,主要为乏力、白细胞减低、血清肌酐升高和腹胀及水肿,停药及对症治疗可缓解。
结论:PD方案是治疗AL型淀粉样变性安全有效的候选方案之一,部分患者可获得早期快速的血液学缓解,长期疗效有待进一步观察。


Abstract: Objective:To observe the treatment response and adverse events of pomalidomide and dexamethasone(PD) in systemic light chain(AL) amyloidosis.
Methodology:A retrospective study was conducted on AL amyloidosis patients who were treated with PD in National Clinical Research Center of kidney disease, record clinical features of these patients, analyze effect and adverse effects.
Results:Two initial treated patients, 7 relapse patients, 7 refractory patients and 3 patients who were not tolerated to first line treatment were recruited. Nine (4737%) were males. The average age was 5874±1100 years. Heart and kidney involvement ratio were 3158% and 100%. The patients received 2(1, 4) cycles of PD treatment. The total hematological response rate was 3158% and 50% with initial treated ones, 2857% with refractory, 4286% with recovery patients. The hematological response was achieved in 4 treatment cycles. As for heart response, 2(1053%) patients had progress. And 6(3158%)patients’kidney condition progressed,3(1579%)patients had partial remission, 2(1053%)had very good partial remission, 8(4211%)had no remission. The adverse events (AEs) of PD were rare and controllable, including fatigue, myelosuppression, creatinine increasing, edema and gastrointestinal reactions. The prevalence of grade 3/4 AEs was 2104%. 
Conclusion:Pomalidomide and dexamethasone was an effective treatment of AL amyloidosis which could help patients getting earlier hematologic response. Further observation was needed to figure out the longterm effect.