The Role of Autologous Hematopoietic Stem Cell Transplantation in the Therapy of Systemic AL Amyloidosis

Q4 Medicine
Ольга Владиславовна Пирогова, О. В. Кудяшева, А. Г. Смирнова, В. В. Порунова, С. В. Толстова, К. Р. Калимулина, М. В. Черноус, Ю. Ю. Власова, И. С. Моисеев, В. А. Добронравов, А. Д. Кулагин
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引用次数: 0

Abstract

Aim. To assess the outcomes of autologous hematopoietic stem cell transplantation (auto-HSCT) in systemic AL Amyloidosis patients treated at the R.M. Gorbacheva Scientific Research Institute of Pediatric Oncology, Hematology and Transplantation. Materials & Methods. In the period from 2005 to 2022, auto-HSCT was performed in 33 patients with systemic AL Amyloidosis. In 7 of them, auto-HSCT was not preceded by the induction therapy “upfront”. From 2012 all patients received induction therapy prior to transplantation. The median age of patients was 54 years (range 38–68 years); among them there were 17 women and 16 men. Results. The 3-year follow-up period showed hematological response rate of 76 % (95% confidence interval [95% CI] 50–90 %), heart response rate of 27 % (95% CI 6–55 %), renal response rate of 76 % (95% CI 41–93 %), and hepatic response rate of 26 % (95% CI 8–50 %). The 5-year overall (OS) and progression-free (PFS) survivals were 71 % (95% CI 49–85 %) and 53 % (95% CI 32–71 %), respectively. The OS parameters in the group with delayed auto-HSCT, i.e., after induction therapy, were better than in the “upfront” group: 82 % (95% CI 60–93 %) vs. 43 % (95% CI 10–73 %) (p = 0.03). The OS parameters were affected by health status (p = 0.03), reduced left ventricular ejection fraction < 60 % (p = 0.006), stage of heart disease (p = 0.016), and stage III kidney disease (p = 0.007). The PFS parameters depended on ECOG performance status (p = 0.004) and stage of heart disease (p = 0.041). Conclusion. The presented data confirm the results of the studies emphasizing the importance of induction therapy prior to auto-HSCT in the treatment of systemic AL Amyloidosis. More stringent parameters of renal function, left ventricular ejection fraction, and ECOG performance status can be used as criteria for auto-HSCT eligibility. Reduced melphalan doses, as conditioning regimen, can be administered to patients with pronounced comorbidity.
自体造血干细胞移植在全身性AL淀粉样变性治疗中的作用
的目标。评估在戈尔巴乔夫儿科肿瘤、血液和移植科学研究所治疗的全身性AL淀粉样变性患者的自体造血干细胞移植(auto-HSCT)的疗效。 材料,方法。在2005年至2022年期间,对33例系统性AL淀粉样变性患者进行了自体造血干细胞移植。其中7例自体造血干细胞移植前未“预先”进行诱导治疗。从2012年起,所有患者在移植前接受诱导治疗。患者年龄中位数为54岁(38-68岁);其中女性17人,男性16人。结果。3年随访期间,血液反应率为76%(95%可信区间[95% CI] 50 - 90%),心脏反应率为27% (95% CI 6 - 55%),肾脏反应率为76% (95% CI 41 - 93%),肝脏反应率为26% (95% CI 8 - 50%)。5年总生存率(OS)和无进展生存率(PFS)分别为71% (95% CI 49 - 85%)和53% (95% CI 32 - 71%)。延迟自体造血干细胞移植组(即诱导治疗后)的OS参数优于“前期”组:82% (95% CI 60 - 93%) vs 43% (95% CI 10 - 73%) (p = 0.03)。健康状况(p = 0.03)、左室射血分数降低及lt;60% (p = 0.006),心脏疾病(p = 0.016)和肾脏疾病III期(p = 0.007)。PFS参数取决于ECOG功能状态(p = 0.004)和心脏病分期(p = 0.041)。 结论。目前的数据证实了强调自体造血干细胞移植前诱导治疗在治疗全身性AL淀粉样变性中的重要性的研究结果。肾功能、左心室射血分数和ECOG表现状态等更严格的参数可作为自动造血干细胞移植资格的标准。减少美法仑剂量,作为调理方案,可给予患者明显的合并症。
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来源期刊
CiteScore
0.80
自引率
0.00%
发文量
20
审稿时长
12 weeks
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