接受家庭姑息治疗项目的肌萎缩侧索硬化患者的一年临床病程：一项扩展研究

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中文摘要

引言/目的： 关于接受家庭姑息治疗的ALS患者的长期数据很少。我们将一个6个月队列的随访延长至12个月，以描述功能、症状、生命支持（无创通气[NIV]、胃造瘘和/或气管切开）以及预先护理计划（ACP）的轨迹。
方法： 在这项前瞻性、单中心研究中，对连续34例参加家庭姑息治疗项目（SAMOT，巴勒莫）的ALS患者在基线、2、4、6和12个月时进行评估，使用Karnofsky、ALS功能评定量表修订版（ALSFRS-R）、爱丁堡认知和行为ALS筛查（ECAS）、埃德蒙顿症状评估量表（ESAS）、吞咽困难、生命支持和预先护理计划（ACP）。
结果： 10例患者（29.4%）在12个月内死亡（70%在家中死亡）；26例完成了12个月评估。Karnofsky（34.4-27.7）和ALSFRS-R（27.1-21.7）在整个期间显著下降（均p<0.0005）。ESAS总分从15.7升至23.7（p<0.0005）；焦虑在T4和T12之间显著增加。NIV使用率从20.6%升至50.0%（p=0.014），而气管切开保持稳定。记录的临终决定在6个月时升至70.4%，但趋于平稳（12个月时为69.2%）；没有完成生前预嘱。ECAS认知障碍约半数患者存在，随时间不变。没有患者被转诊或入住临终关怀机构。
讨论： 将随访延长至6个月以上显示持续的功能下降和症状负担增加，并突出迟发性焦虑和早期趋于平稳的ACP。尽管需求逐渐加重，在这个单中心、非对照队列中，这种家庭姑息治疗模式似乎在1年内支持大多数患者在家中直至死亡。

英文摘要

INTRODUCTION/AIMS: Longer term data on ALS patients receiving home palliative care are scarce. We extended follow-up of a 6-month cohort to 12 months to characterize the trajectory of function, symptoms, vital support (noninvasive ventilation [NIV], gastrostomy, and/or tracheostomy), and advance care planning (ACP).
METHODS: In this prospective, single-center study, 34 consecutive ALS patients in a home palliative care program (SAMOT, Palermo) were assessed at baseline, 2, 4, 6, and 12 months, using Karnofsky, ALS Functional Rating Scale-Revised (ALSFRS-R), Edinburgh Cognitive and Behavioural ALS Screen (ECAS), Edmonton Symptom Assessment Scale (ESAS), dysphagia, vital support, and advanced care planning (ACP).
RESULTS: Ten patients (29.4%) died over 12 months (70% at home); 26 completed the 12-month assessment. Karnofsky (34.4-27.7) and ALSFRS-R (27.1-21.7) declined significantly throughout (both p < 0.0005). Total ESAS rose from 15.7 to 23.7 (p < 0.0005); anxiety increased significantly between T4 and T12. NIV use rose from 20.6% to 50.0% (p = 0.014), while tracheostomy remained stable. Documented end-of-life decisions rose to 70.4% at 6 months but plateaued (69.2% at 12 months); no living wills were completed. Cognitive impairment on ECAS was present in about half of patients, unchanged over time. No patient was referred to or admitted to hospice.
DISCUSSION: Extending follow-up beyond 6 months shows continuous functional decline and rising symptom burden, and highlights late-onset anxiety and early-plateauing ACP. Despite progressive needs, this home palliative care model appeared to support most patients at home until death over 1 year, within this single-center, non-comparative cohort.