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HOVON 104研究:硼替佐米联合地塞米松诱导治疗后进行自体干细胞移植的新诊断AL淀粉样变性患者的长期随访

HOVON 104, long-term follow-up of bortezomib-dexamethasone induction therapy followed by autologous stem cell transplantation in newly diagnosed AL amyloidosis patients.

作者信息

Minnema Monique C, Nasserinejad Kazem, Hegenbart Ute, Ypma Paula F, Wu Ka Lung, Kersten Marie Jose, Croockewit Sandra, Zweegman Sonja, Tick Lidwine, Broijl Annemiek, Koene Harry, Bos Gerard M J, Sonneveld Pieter, Schönland Stefan O

机构信息

Department of Hematology UMC Utrecht University Utrecht Utrecht The Netherlands.

Department of Hematology HOVON Data Center Erasmus MC Cancer Institute Rotterdam The Netherlands.

出版信息

EJHaem. 2024 Jun 17;5(4):815-819. doi: 10.1002/jha2.918. eCollection 2024 Aug.

DOI:10.1002/jha2.918
PMID:39157610
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC11327711/
Abstract

The HOVON 104 studied bortezomib-dexamethasone induction therapy and autologous stem cell transplantation in 50 patients, of whom 35 received an autologous stem cell transplantation (ASCT). We demonstrate a 5-year overall survival (OS) of 73% and progression-free survival (PFS) of 52% for all 50 patients with a median follow-up of 61.3 months. For the 35 transplanted patients, calculated from the date of ASCT, the 5-year OS and PFS were 91% and 68%, respectively. After ASCT, the rate of organ response improved over time but stabilized around 3 years. A complete cardiac response was seen in around 60% of patients and remained stable from 2 years onward. Reaching complete renal response was slower over time and achieved by 61% of the renal-affected patients at 5 years. We confirm the excellent outcomes after ASCT and demonstrate a 60% complete organ response with longer follow-up.

摘要

HOVON 104研究了50例患者接受硼替佐米-地塞米松诱导治疗及自体干细胞移植,其中35例接受了自体干细胞移植(ASCT)。我们证明,50例患者的5年总生存率(OS)为73%,无进展生存率(PFS)为52%,中位随访时间为61.3个月。对于35例移植患者,从ASCT日期算起,5年OS和PFS分别为91%和68%。ASCT后,器官反应率随时间改善,但在3年左右稳定下来。约60%的患者出现完全心脏反应,从2年起保持稳定。随着时间推移,达到完全肾脏反应较慢,5年时61%的肾脏受累患者实现了完全肾脏反应。我们证实了ASCT后的良好结果,并通过更长时间的随访证明了60%的完全器官反应。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/96e0/11327711/e4bc469631ed/JHA2-5-815-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/96e0/11327711/4f33f002b880/JHA2-5-815-g002.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/96e0/11327711/e4bc469631ed/JHA2-5-815-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/96e0/11327711/4f33f002b880/JHA2-5-815-g002.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/96e0/11327711/e4bc469631ed/JHA2-5-815-g001.jpg

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本文引用的文献

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Role of autologous haematopoietic cell transplantation in the treatment of systemic light chain amyloidosis in the era of anti-CD38 monoclonal antibodies.自体造血细胞移植在抗 CD38 单克隆抗体时代治疗系统性轻链淀粉样变性中的作用。
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Predictors of hematologic response and survival with stem cell transplantation in AL amyloidosis: A 25-year longitudinal study.
干细胞移植治疗淀粉样变性的血液学反应和生存预测因素:25 年的纵向研究。
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Daratumumab-Based Treatment for Immunoglobulin Light-Chain Amyloidosis.达雷妥尤单抗治疗免疫球蛋白轻链淀粉样变性。
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Bortezomib-Based Induction Is Associated with Superior Outcomes in Light Chain Amyloidosis Patients Treated with Autologous Hematopoietic Cell Transplantation Regardless of Plasma Cell Burden.无论浆细胞负荷如何,基于硼替佐米的诱导治疗与接受自体造血细胞移植的轻链淀粉样变性患者的更好预后相关。
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Haematologica. 2019 Nov;104(11):2274-2282. doi: 10.3324/haematol.2018.213900. Epub 2019 Mar 28.
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Biol Blood Marrow Transplant. 2019 May;25(5):e169-e173. doi: 10.1016/j.bbmt.2019.01.007. Epub 2019 Jan 11.