Muchtar Eli, Gertz Morie A, Kumar Shaji K, Lacy Martha Q, Dingli David, Buadi Francis K, Grogan Martha, Hayman Suzanne R, Kapoor Prashant, Leung Nelson, Fonder Amie, Hobbs Miriam, Hwa Yi Lisa, Gonsalves Wilson, Warsame Rahma, Kourelis Taxiarchis V, Russell Stephen, Lust John A, Lin Yi, Go Ronald S, Zeldenrust Steven, Kyle Robert A, Rajkumar S Vincent, Dispenzieri Angela
Division of Hematology.
Department of Cardiovascular Diseases, and.
Blood. 2017 Apr 13;129(15):2111-2119. doi: 10.1182/blood-2016-11-751628. Epub 2017 Jan 26.
In light of major advances in immunoglobulin light chain (AL) amyloidosis, we evaluated the trends in presentation, management, and outcome among 1551 newly diagnosed AL amyloidosis patients seen in our institution from 2000 to 2014. As compared with the 2 intervals 2000-2004 and 2005-2009, patients diagnosed in 2010-2014 were less likely to have >2 involved organs. Utilization of autologous stem cell transplant (ASCT) was similar across all periods, about one-third of patients, but there was an increase in the use of pre-ASCT bortezomib induction and of unattenuated melphalan conditioning in 2010-2014 compared with earlier periods. Non-ASCT first-line regimen changed with 65% of patients in 2010-2014 received bortezomib-based therapy, 79% of patients in 2005-2009 received melphalan-dexamethasone, and 64% of patients in 2000-2004 received melphalan-prednisone. The rate of better than very good partial response (VGPR) was higher in more recent periods (66% vs 58% vs 51%; = .001), a change largely driven by improved VGPR rates in the non-ASCT population. Overall survival (OS) has improved, with inflection points for improvement differing for the ASCT and non-ASCT groups. In the ASCT population, the greatest gains were after 2010 (4-year OS, 91% compared with 73% and 65%). In the non-ASCT group, greatest gains were after 2005 (4-year OS, 38%, 32%, and 16%). Fewer patients died within 6 months of diagnosis in the 2 later periods (24% vs 25% vs 37%; < .001). Overall, outcomes among patients with AL amyloidosis have improved with earlier diagnosis, higher rates of VGPR, lower early mortality, and improved OS.
鉴于免疫球蛋白轻链（AL）淀粉样变性的重大进展，我们评估了2000年至2014年在我们机构就诊的1551例新诊断的AL淀粉样变性患者在临床表现、治疗和预后方面的趋势。与2000 - 2004年和2005 - 2009年这两个时间段相比，2010 - 2014年诊断的患者累及器官>2个的可能性较小。自体干细胞移植（ASCT）在所有时期的使用率相似，约为三分之一的患者，但与早期相比，2010 - 2014年ASCT前硼替佐米诱导治疗和未减量美法仑预处理的使用有所增加。非ASCT一线治疗方案有所变化，2010 - 2014年65%的患者接受基于硼替佐米的治疗，2005 - 2009年79%的患者接受美法仑 - 地塞米松治疗，2000 - 2004年64%的患者接受美法仑 - 泼尼松治疗。近期达到优于非常好的部分缓解（VGPR）的比例更高（66%对58%对51%；P = .001），这一变化主要由非ASCT人群中VGPR率的提高所驱动。总体生存率（OS）有所改善，ASCT组和非ASCT组改善的转折点不同。在ASCT人群中，最大的改善出现在2010年之后（4年OS，91%，而之前为73%和65%）。在非ASCT组中，最大的改善出现在2005年之后（4年OS，38%、32%和16%）。在后面两个时期，诊断后6个月内死亡的患者较少（24%对25%对3
