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硼替佐米联合化疗方案用于不适合自体干细胞移植的AL淀粉样变性患者的治疗结果

Outcomes of bortezomib combination chemotherapies in autologous stem cell transplantation-ineligible patients with AL amyloidosis.

作者信息

Hur Joon Young, Yoon Sang Eun, Kim Darae, Choi Jin-Oh, Min Ju-Hong, Kim Byung Jun, Kim Jung Sun, Lee Jung Eun, Choi Joon Young, Jeon Eun-Seok, Kim Seok Jin, Kim Kihyun

机构信息

Division of Hematology and Oncology, Department of Internal Medicine, Hanyang University Guri Hospital, Guri, Korea.

Division of Hematology-Oncology, Department of Medicine, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, Korea.

出版信息

Blood Res. 2021 Dec 31;56(4):266-278. doi: 10.5045/br.2021.2021121.

Abstract

BACKGROUND

Treatment protocols for light chain (AL) amyloidosis have been derived from myeloma treatment. Bortezomib is a key drug used for the treatment of myeloma and AL amyloidosis. We retrospectively investigated the efficacy and toxicity of bortezomib- based chemotherapy in patients with newly diagnosed AL amyloidosis.

METHODS

We reviewed the outcomes of newly diagnosed autologous stem cell transplantation (auto-SCT)-ineligible AL amyloidosis patients who received bortezomib-based chemotherapy at a referral center between 2011 and 2017.

RESULTS

Of 63 patients who received bortezomib-based chemotherapy, 32 were male, and the median age was 66 years (range, 42‒82 yr). The hematologic overall response rate (ORR) was 65.1%, and the chemotherapy regimen with the best hematologic response was VMP (75.7%, 28/37). Sixty patients had significant organ (heart or kidney) involvement; 28.3% of patients (N=17) had major organ responses after chemotherapy. With a median follow- up of 34 months, there was no significant difference in progression-free survival (=0.49) or overall survival ( =0.67) according to regimen. Most hematologic and non-hematologic problems were manageable.

CONCLUSION

Various chemotherapy combinations based on bortezomib are currently employed in the clinical setting, but no difference was found in terms of efficacy or toxicity.

摘要

背景

轻链(AL)淀粉样变性的治疗方案源自骨髓瘤治疗。硼替佐米是用于治疗骨髓瘤和AL淀粉样变性的关键药物。我们回顾性研究了以硼替佐米为基础的化疗在新诊断的AL淀粉样变性患者中的疗效和毒性。

方法

我们回顾了2011年至2017年间在一家转诊中心接受以硼替佐米为基础化疗的新诊断的自体干细胞移植(auto-SCT)不适合的AL淀粉样变性患者的治疗结果。

结果

在63例接受以硼替佐米为基础化疗的患者中,32例为男性,中位年龄为66岁(范围42 - 82岁)。血液学总缓解率(ORR)为65.1%,血液学反应最佳的化疗方案是VMP(75.7%;28/37)。60例患者有明显的器官(心脏或肾脏)受累;28.3%的患者(N = 17)化疗后有主要器官反应。中位随访34个月,根据治疗方案,无进展生存期(=0.49)或总生存期(=0.67)无显著差异。大多数血液学和非血液学问题是可控的。

结论

目前临床中采用了多种基于硼替佐米的化疗组合,但在疗效或毒性方面未发现差异。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/ff99/8721454/eb2b89c02a76/br-56-4-266-f1.jpg

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