Klinik für Neurologie, Charité - Universitätsmedizin Berlin, Clinic for Neurology, Berlin, Germany.
J Med Econ. 2012;15(3):521-30. doi: 10.3111/13696998.2012.660897. Epub 2012 Feb 21.
The burden of disease in Lambert-Eaton myasthenic syndrome (LEMS) patients is unclear. This study focused on the patient's perspective to obtain patient-reported information on clinical symptoms, burden of illness, impact of LEMS on activities of daily living (ADL), and management of LEMS.
Semi-structured, face-to-face interviews with LEMS patients from two specialized centres in Germany between September and December 2010.
Twelve patients participated; mean age 66.7 ± 9.8 years. First symptoms occurred at age 52.5 ± 14.0 years. Mean time between first symptoms and diagnosis was 4.4 ± 6.2 years. Patients reported neuromuscular, cranial, and autonomic symptoms plus general fatigue. Two-thirds of patients reported 10 or more symptoms. The most frequent symptoms were leg weakness (91.7%) and general fatigue (83.3%). Restrictions in ADL were reported always or often in 75% of patients. Over half of the patients (n = 7) reported poor or very poor health status. Mean EQ-5D utility scores were 0.34 ± 0.35, with little day-to-day variation. Patients visited a number of different clinicians; most had been hospitalized at some point in the course of their disease. The most frequent drug treatments were 3,4-diaminopyridine (3,4-DAP) (83.3%) and pyridostigmine (41.5%). The study has several limitations, including small sample size and the potential influence of recall bias.
LEMS patients report long individual disease histories. Most patients suffer multiple symptoms which are frequently severe and troublesome, and almost all are restricted in ADL with poor health status. There is high utilization of healthcare resources from diagnosis to ongoing treatment. Physicians should be aware of this rare disease to ensure that patients receive an early diagnosis and prompt and appropriate treatment.
Lambert-Eaton 肌无力综合征（LEMS）患者的疾病负担尚不清楚。本研究从患者的角度出发，获得了患者对临床症状、疾病负担、LEMS 对日常生活活动（ADL）的影响以及 LEMS 管理的报告信息。
2010 年 9 月至 12 月，在德国的两个专门中心对 LEMS 患者进行了半结构式的面对面访谈。
12 名患者参与了研究；平均年龄 66.7±9.8 岁。首发症状发生于 52.5±14.0 岁。首发症状与诊断之间的平均时间为 4.4±6.2 年。患者报告了神经肌肉、颅神经和自主神经症状以及全身疲劳。三分之二的患者报告了 10 种或更多症状。最常见的症状是腿部无力（91.7%）和全身疲劳（83.3%）。75%的患者报告在日常生活中经常或总是受限。超过一半的患者（n=7）报告健康状况较差或非常差。平均 EQ-5D 效用评分 0.34±0.35，每天的差异较小。患者就诊于许多不同的临床医生；大多数患者在疾病过程中曾住院治疗。最常见的药物治疗是 3,4-二氨基吡啶（3,4-DAP）（83.3%）和吡啶斯的明（41.5%）。本研究存在一些局限性，包括样本量小和潜在的回忆偏倚影响。
LEMS 患者报告了个体的长病史。大多数患者患有多种症状，这些症状常常严重且令人困扰，几乎所有患者的日常生活活动都受限，健康状况较差。从诊断到持续治疗，医疗资源的利用率很高。医生应了解这种罕见疾病，以确保患者得到及时的诊断和及时、适当的治疗。
