Improved outcomes for newly diagnosed AL amyloidosis between 2000 and 2014: cracking the glass ceiling of early death
Improved outcomes for newly diagnosed AL amyloidosis between 2000 and 2014: cracking the glass ceiling of early death
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DOI：
10.1182/blood-2016-11-751628
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发表时间：
2017-04-13
期刊：
影响因子：
20.3
通讯作者：
Dispenzieri, Angela
中科院分区：
文献类型：
--
作者：
Muchtar, Eli;Gertz, Morie A.;Dispenzieri, Angela
鉴于免疫球蛋白轻链（AL）淀粉样变的重大进展，我们评估了2000年至2014年在我院新诊断的1551例AL淀粉样变患者的表现、管理和结局趋势。与2000-2004年和2005-2009年的2个时间段相比，2010-2014年诊断的患者有>2受累器官的可能性较小。自体干细胞移植（ASCT）的使用在所有时期相似，约占患者的三分之一，但在2010-2014年，与早期相比，ASCT前硼替佐米诱导和未减毒的美伐兰调节的使用有所增加。非asct一线治疗方案发生变化，2010-2014年65%的患者接受硼替佐米为主的治疗，2005-2009年79%的患者接受美伐兰-地塞米松治疗，2000-2004年64%的患者接受美伐兰-泼尼松治疗。较好的部分缓解率（VGPR）在最近的时期更高（66% vs 58% vs 51%; P5.001），这一变化主要是由非asct人群中VGPR率的提高所驱动的。总生存期（OS）有所改善，ASCT组和非ASCT组改善的拐点有所不同。在ASCT人群中，最大的增长发生在2010年之后（4年生存率为91%，73%和65%）。在非asct组中，最大的增长发生在2005年之后（4年生存率分别为38%、32%和16%）。在2个后期，诊断后6个月内死亡的患者较少(24% vs25% vs 37%
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 stemcell transplant (ASCT) wassimilar 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 inmore recent periods (66% vs 58% vs 51%; P5.001), 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 gainswere after 2010 (4-year OS, 91% comparedwith 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 monthsof diagnosis in the 2 later periods(24% vs25% vs 37%; P