Huang B, Li J, Xu X, Zheng D, Zhou Z, Liu J
Department of Hematology, The First Affiliated Hospital, Sun Yat-sen University, Guangzhou 510080, China.
Department of Hematology, Zhongshan City People Hospital, Zhongshan 528403, China.
Pathol Biol (Paris). 2015 Feb;63(1):17-20. doi: 10.1016/j.patbio.2014.10.001. Epub 2014 Nov 6.
To assess the efficacy and tolerability of bortezomib with dexamethasone for patients with renal light chain (AL) amyloidosis.
Twelve newly diagnosed patients with renal AL amyloidosis were treated with a combination of bortezomib (1.3mg/m(2)/d iv, d1, 4, 8, 11) and dexamethasone (20mg/d iv drip, d1-4).
Median follow-up time was 22.5 months (range, 2.1-53.6). Ten patients were evaluable. Five out of 10 (50%) patients achieved complete hematologic responses (CHR), and totally 8/10 (80%) achieved hematologic responses (HR). Median time to hematologic response was 1 month. All patients who received HR had no hematologic progression during follow-up period. Five patients (50%) had kidney responses and the other 5 patients (50%) were stable. Median time to kidney response was 3 months. No patients presented renal progression during follow-up. One patient achieved PR after 4 cycles of VD and then received autologous peripheral blood stem cell transplantation. Two out of 10 evaluable patients without hematologic response had died with median overall survival of 8.2 months. Eight of them who had HR were alive with median follow-up time of 28.5 months. Infection (6/12) and fatigue (5/12) were the most frequent side effects. Three patients developed herpes zoster and had to discontinue therapy.
VD produces rapid, deep and durable hematological responses and renal responses in the majority of patients with newly diagnosed renal AL. It is well tolerated. This treatment may be a good option as first-line treatment for renal AL amyloidosis patients.
评估硼替佐米联合地塞米松治疗肾轻链（AL）淀粉样变性患者的疗效和耐受性。
12例新诊断的肾AL淀粉样变性患者接受硼替佐米（1.3mg/m²/d静脉注射，第1、4、8、11天）联合地塞米松（20mg/d静脉滴注，第1 - 4天）治疗。
中位随访时间为22.5个月（范围2.1 - 53.6个月）。10例患者可评估。10例患者中有5例（50%）达到完全血液学缓解（CHR），10例中共有8例（80%）达到血液学缓解（HR）。血液学缓解的中位时间为1个月。所有达到HR的患者在随访期间均无血液学进展。5例患者（50%）肾脏情况有改善，另外5例患者（50%）病情稳定。肾脏情况改善的中位时间为3个月。随访期间无患者出现肾脏进展。1例患者在4个周期的VD治疗后达到部分缓解（PR），随后接受了自体外周血干细胞移植。10例可评估的无血液学缓解患者中有2例死亡，中位总生存期为8.2个月。8例达到HR的患者存活，中位随访时间为28.5个月。感染（6/12）和疲劳（5/12）是最常见的副作用。3例患者发生带状疱疹，不得不停止治疗。
VD方案能使大多数新诊断的肾AL患者迅速、深度且持久地获得血液学缓解和肾脏反应。耐受性良好。该治疗方案可能是肾AL淀粉样变性患者一线治疗的良好选择。
