轻链型淀粉样变性：维持治疗对自体干细胞移植结果的影响。
AL Amyloidosis: The Effect of Maintenance Therapy on Autologous Stem Cell Transplantation Outcomes.
作者信息
Ozga Michael, Zhao Qiuhong, Benson Don, Elder Patrick, Williams Nita, Bumma Naresh, Rosko Ashley, Chaudhry Maria, Khan Abdullah, Devarakonda Srinivas, Kahwash Rami, Vallakati Ajay, Campbell Courtney, Parikh Samir V, Almaani Salem, Prosek Jason, Bittengle Jordan, Pfund Katherine, LoRusso Samantha, Freimer Miriam, Redder Elyse, Efebera Yvonne, Sharma Nidhi
机构信息
Division of Hematology, Department of Internal Medicine, The Ohio State University Comprehensive Cancer Center, Columbus, OH 43210, USA.
Division of Cardiology, Department of Internal Medicine, The Ohio State University, Columbus, OH 43210, USA.
出版信息
J Clin Med. 2020 Nov 23;9(11):3778. doi: 10.3390/jcm9113778.
BACKGROUND
Autologous stem cell transplantation (ASCT) remains an effective treatment option for many patients with systemic light chain (AL) amyloidosis. While maintenance post ASCT in multiple myeloma is now standard, the decision to utilize maintenance in AL amyloidosis remains largely unexplored. The present study aims to determine the prognostic significance of utilizing maintenance therapy following ASCT and assess the impact of fluorescent in situ hybridization (FISH) abnormalities, bone marrow plasma cell burden (BMPC), and degree of organ involvement on this decision.
METHODS AND RESULTS
This is a retrospective analysis of fifty AL amyloidosis patients who underwent ASCT at The Ohio State University. Twenty-eight patients received maintenance and twenty-two did not. Kaplan-Meier survival analysis was used to compare the effect of maintenance therapy with no significant difference in PFS ( = 0.66) and OS ( = 0.32) between the two groups. There was no difference in survival based on maintenance when further categorized by FISH, PFS ( = 0.15), and OS ( = 0.65); BMPC ≥ 10%, PFS ( = 0.49), and OS ( = 0.32); or with 2 or more organs involved, PFS ( = 0.34) and OS ( = 0.80).
CONCLUSION
Maintenance therapy post ASCT did not impact PFS or OS when categorized by FISH abnormalities, increasing BMPC, or ≥2 organs involved in AL amyloidosis patients.
背景
自体干细胞移植（ASCT）仍是许多系统性轻链（AL）淀粉样变性患者的有效治疗选择。虽然多发性骨髓瘤ASCT后的维持治疗现已成为标准治疗，但AL淀粉样变性中使用维持治疗的决策在很大程度上仍未得到充分探索。本研究旨在确定ASCT后使用维持治疗的预后意义，并评估荧光原位杂交（FISH）异常、骨髓浆细胞负荷（BMPC）和器官受累程度对这一决策的影响。
方法与结果
这是一项对50例在俄亥俄州立大学接受ASCT的AL淀粉样变性患者的回顾性分析。28例患者接受了维持治疗，22例未接受。采用Kaplan-Meier生存分析比较维持治疗的效果，两组之间的无进展生存期（PFS）（P = 0.66）和总生存期（OS）（P = 0.32）无显著差异。根据FISH进一步分类时，维持治疗与否在生存方面无差异，PFS（P = 0.15）和OS（P = 0.65）；BMPC≥10%时，PFS（P = 0.49）和OS（P = 0.32）；或累及2个或更多器官时，PFS（P = 0.34）和OS（P = 0.80）。
结论
对于AL淀粉样变性患者，根据FISH异常、BMPC增加或累及≥2个器官进行分类时，ASCT后的维持治疗对PFS或OS没有影响。
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