Abstract

Background

Induction therapy is recommended before autologous stem cell transplantation (ASCT) for AL amyloidosis patients with high disease burden [bone marrow plasma cells (BMPCs) > 10%], but the role of induction therapy before ASCT in patients with low disease burden (BMPCs ≤ 10%) is still unknown.

Methods

A total of 227 patients with AL amyloidosis were included in this study. Among 227 patients, 124 patients received bortezomib-based induction prior to ASCT and were defined as group A, 35 patients received other chemotherapeutic induction and were defined as group B, and the other 68 patients without induction were defined as group C. We compared the differences of efficacy and prognosis between the three groups.

Results

The haematological overall response rates (ORR) of groups A, B and C were 91%, 67% and 75%, respectively. The complete response rates (CR) of groups A, B and C were 50%, 25% and 20%, respectively. Both the ORR and CR rates of group A were significantly higher than those of groups B and C. The renal response rates of groups A, B and C were 64%, 46% and 47%, respectively. The cardiac response rates of groups A, B and C were 74%, 45% and 40%, respectively. The renal and cardiac responses rates of group A were also significantly higher than those of the other two groups. After a median follow-up of 44 months, the median OS was not reached. The 5-year estimated overall survival (OS) rates of groups A, B and C were 81%, 57% and 67%, respectively. The median progression-free survival (PFS) was 83 months for all patients. The 5-year estimated PFS rates of groups A, B and C were 61%, 38% and 49%, respectively. Both the OS and PFS of group A were higher than those of both group B and group C. On multivariate analysis, baseline dFLC > 50 mg/L was associated with worse survival, but induction with bortezomib was associated with better survival.

Conclusion

Our study demonstrated that low disease burden AL patients who are eligible for ASCT may benefit from bortezomib-based induction therapy.

中文翻译：

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自体干细胞移植前诱导治疗在低疾病负担 AL 淀粉样变性患者中的作用

摘要

背景

对于疾病负担高[骨髓浆细胞（BMPCs）> 10%]的AL淀粉样变性患者，建议在自体干细胞移植（ASCT）前进行诱导治疗，但在疾病负担低（BMPCs≤BMPCs）的患者中，ASCT前诱导治疗的作用10%）仍然未知。

方法

本研究共纳入 227 名 AL 淀粉样变性患者。227 例患者中，124 例患者在 ASCT 前接受基于硼替佐米的诱导，定义为 A 组，35 例接受其他化疗诱导的患者定义为 B 组，其余 68 例未进行诱导的患者定义为 C 组。三组疗效及预后差异。

结果

A、B和C组的血液学总体反应率（ORR）分别为91%、67%和75%。A、B、C组的完全缓解率（CR）分别为50%、25%和20%。A组ORR和CR率均显着高于B、C组。A、B、C组肾脏反应率分别为64%、46%和47%。A、B、C组心脏反应率分别为74%、45%、40%。A组的肾脏和心脏反应率也明显高于其他两组。中位随访 44 个月后，未达到中位 OS。A、B和C组的5年估计总生存率（OS）分别为81%、57%和67%。所有患者的中位无进展生存期 (PFS) 为 83 个月。A、B 和 C 组的 5 年估计 PFS 率分别为 61%、38% 和 49%。A 组的 OS 和 PFS 均高于 B 组和 C 组。在多变量分析中，基线 dFLC > 50 mg/L 与较差的生存率相关，但硼替佐米诱导与更好的生存率相关。

结论

我们的研究表明，符合 ASCT 条件的低疾病负担 AL 患者可能会受益于基于硼替佐米的诱导治疗。