Long-term disease history, clinical symptoms, health status, and healthcare utilization in patients suffering from Lambert Eaton myasthenic syndrome: Results of a patient interview survey in Germany

Abstract

Introduction:

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.

Methods:

Semi-structured, face-to-face interviews with LEMS patients from two specialized centres in Germany between September and December 2010.

Results:

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.

Conclusion:

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.

Key words::

• Lambert Eaton Myasthenic Syndrome
• LEMS
• Quality-of-life
• Health status
• Burden of disease

Abbreviations::

• 3,4-DAP, 3,4-diaminopyridine; ADL, Activities of daily living; DGM, German Society for Muscle Patients; EURORDIS, European Organisation for Rare Diseases; HRQoL, Health-related quality-of-life; LEMS, Lambert-Eaton myasthenic syndrome; MG, Myasthenia gravis; SCLC, Small cell lung cancer; VAS, Visual analogue scale; WPAI, Work Productivity and Activity Impairment Questionnaire

Transparency

Declaration of funding

This survey was funded by BioMarin Europe Limited, UK. Editorial support was provided by Tricia Dixon, JB Medical Ltd, UK.

Declaration of financial/other relationships

JPS and LH have disclosed that they were paid consultants for BioMarin regarding the planning of this study. AEW and RG have disclosed that they are employees of BioMarin Europe Limited, UK. RS, VC, and CP have disclosed that they are employees of IMS Health, a company that received payment for their involvement in this project.