Changes in patient-reported outcomes in light chain amyloidosis in the first year after diagnosis and relationship to NT-proBNP change.
Changes in patient-reported outcomes in light chain amyloidosis in the first year after diagnosis and relationship to NT-proBNP change.
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DOI：
10.1038/s41408-021-00412-8
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发表时间：
2021-02-01
影响因子：
12.8
通讯作者：
Flynn KE
中科院分区：
文献类型：
--
作者：
D'Souza A;Brazauskas R;Dispenzieri A;Panepinto J;Flynn KE
我们对新诊断的系统性轻链（AL）淀粉样变性患者（N = 59）进行了一项前瞻性队列研究，以研究患者第一年报告的预后（PROs）。中位年龄为68岁，42%为女性，8%为黑人，78%为lambda亚型。器官受累66%为心脏，58%为肾脏，25%有3个或以上器官受累。在基线至3个月期间，除焦虑改善2.1分外，所有PROMIS®-29领域得分均恶化了0.4-4.1分。1年后，与3个月时的最大下降相比，得分有所提高，总体身体健康、身体功能和疲劳的统计意义最大。在分期调整生存分析中，除了基线整体身心健康外，测量身体功能、疲劳、焦虑、抑郁和社会角色的领域与1年生存率相关。1年时，PROMIS测量与NT-proBNP变化和血液学反应相关。在NT-proBNP反应的患者中，身体功能、社会角色、整体心理健康和焦虑都有所改善。在NT-proBNP进展的患者中，焦虑、抑郁、睡眠和整体心理健康均出现恶化。测量和跟踪AL淀粉样变性患者的pro是很重要的，这些重要的结果可以作为临床护理/研究的相关终点。
We conducted a prospective cohort study in newly diagnosed systemic light chain (AL) amyloidosis patients (N = 59) to study patient-reported outcomes (PROs) through the first year. The median age was 68 years with 42% female, 8% Black, and 78% lambda subtype. Organ involvement was cardiac in 66%, renal in 58%, with 25% having 3 or greater organs involved. Between baseline and 3 months, all PROMIS®-29 domain scores worsened by 0.4–4.1 points except anxiety which improved by 2.1 points. By 1 year, scores improved compared to the greatest decline at 3 months, most statistically significant for global physical health, physical function, and fatigue. On stage-adjusted survival analysis, in addition to baseline global physical and mental health, domains measuring physical function, fatigue, anxiety, depression, and social roles were associated with 1-year survival. At 1 year, PROMIS measures were associated with NT-proBNP changes and hematologic response. Among patients with an NT-proBNP response, the improvement was seen in physical function, social roles, global mental health, and anxiety. Among patients with an NT-proBNP progression, worsening was seen with anxiety, depression, sleep, and global mental health. Measuring and tracking PROs in patients with AL amyloidosis is important and these important outcomes can be used as correlative endpoints in clinical care/research.
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影响因子：
12.8
作者：
Warsame, Rahma;Kumar, Shaji K.;Dispenzieri, Angela
通讯作者：
Dispenzieri, Angela
影响因子：
20.3
作者：
Muchtar, Eli;Gertz, Morie A.;Dispenzieri, Angela
通讯作者：
Dispenzieri, Angela
影响因子：
6.5
作者：
Sanchorawala V;McCausland KL;White MK;Bayliss MS;Guthrie SD;Lo S;Skinner M
通讯作者：
Skinner M
影响因子：
51.1
作者：
Efficace, Fabio;Gaidano, Gianluca;Mandelli, Franco
通讯作者：
Mandelli, Franco
影响因子：
3.7
作者：
Morel, Thomas;Cano, Stefan J.
通讯作者：
Cano, Stefan J.