Questionnaire survey to systematically assess and characterize quality of life limitations in the context of Lambert-Eaton myasthenia syndrome (LEMS).
Organizational Data
- DRKS-ID:
- DRKS00024527
- Recruitment Status:
- Recruiting complete, study complete
- Date of registration in DRKS:
- 2021-02-15
- Last update in DRKS:
- 2024-02-29
- Registration type:
- Prospective
Acronym/abbreviation of the study
LEMS BoD
URL of the study
https://neurocure.de/klinisches-zentrum/forschung-am-ncrc/laufende-studien/lems-bod-studie.html
Brief summary in lay language
The limitation of quality of life in the context of Lambert-Eaton myasthenia syndrome (LEMS) has been insufficiently studied so far. Therefore, in this project we aim to systematically assess and characterize quality of life in patients with LEMS, including education and employment, economic aspects, disease-specific characteristics, social situation and support received. In order to obtain data on this question that are as representative as possible for the LEMS patient population in Germany, we would like to survey LEMS patients who are members of the German Myasthenia Society (DMG) on a one-time basis using a questionnaire.
Brief summary in scientific language
Lambert Eaton Myasthenia Syndrome (LEMS) is a very rare, chronic neurological autoimmune disorder that can affect people of any age. The incidence is approximately 0.45/million, and the prevalence is approximately 2.32/million. The cause is antibody-mediated inactivation of the VGCC (voltage gated calcium channels) at the presynapse of the so-called neuromuscular endplate, followed by proximal muscle exhaustion. The impaired function of the receptors results in a blockade of the transmission between nerve and muscle. This manifests itself, for example, in the form of general weakness, fatigue or reduced performance as well as autonomic symptoms. These very unspecific symptoms often result in a diagnosis that is delayed by years. The most common differential diagnosis is myasthenia gravis. The occurrence of LEMS is often paraneoplastic, especially associated with small cell lung cancer (SCLC). Prognosis then depends on the primary tumor. Symptomatic treatment is typically carried out with drugs that improve neurotransmission, e.g. with the potassium channel blocker amifampridine (3,4-diaminopyridine). Chronic courses are treated with immunosuppressants (such as prednisolone and azathioprine). In the presence of a tumor, treatment is primarily oncological. Despite treatment of neurological symptoms, LEMS patients often suffer from additional physical impairments such as fatigue, or accompanying psychological disorders such as anxiety, depression and sleep disorders. These concomitant diseases may partly precede the myasthenic symptoms, but also accompany the course of the disease continuously, which can result in a severe impairment of the quality of life and, in younger patients, often leads to inability to work and to early retirement. The impact of LEMS on daily life and relevant issues such as family planning is large, but it has not yet been well understood what these impairments look like and with which clinical parameters they correlate.
Health condition or problem studied
- ICD10:
- G70 - Myasthenia gravis and other myoneural disorders
- ICD10:
- G73.1 - Lambert-Eaton syndrome
- Healthy volunteers:
- No Entry
Interventions, Observational Groups
- Arm 1:
- Patients with LEMS are surveyed once about their quality of life using a questionnaire developed for this purpose.
Endpoints
- Primary outcome:
- Self-reported quality of life (questionnaire SF36). Summated scales for physical and psychological quality of life.
- Secondary outcome:
- 1. Mya Quality of Life 15 (Mya Qol 15), self-reported information on disease severity (mild, moderate, severe). 2. Mya Qol 15, Myasthenia Gravis Activities of Daily Living Profile (MG-ADL). 3. MG-ADL, self-reported information on disease severity (mild, moderate, severe). 4. Mya Qol 15,self-reported history of onset of disease and time of diagnosis. 5. Mya Qol 15, ENRICHD Social Support Inventory – In German: ESSI-D. 6. Mya Qol 15, self-reported information on net income, loss of income due to MG (yes, no), pension payments (yes, no), and social security benefits (yes, no). 7. Mya Qol 15,self-reported information on side effects of drug therapies (yes, no). 8. HADS (Hospital Anxiety and Depression Scale) and comparison with data from the literature. 9. HADS, Mya Qol 15. 10. Chalder Fatigue Scale (CFQ) and comparison with data from the literature. 11. CFQ, Mya Qol 15.
Study Design
- Purpose:
- Supportive care
- Retrospective/prospective:
- No Entry
- Study type:
- Non-interventional
- Longitudinal/cross-sectional:
- No Entry
- Study type non-interventional:
- Epidemiological study
Recruitment
- Recruitment Status:
- Recruiting complete, study complete
- Reason if recruiting stopped or withdrawn:
- No Entry
Recruitment Locations
- Recruitment countries:
-
- Germany
- Number of study centers:
- Monocenter study
- Recruitment location(s):
-
- Other Deutsche Myasthenie Gesellschaft e.V. Bremen
Recruitment period and number of participants
- Planned study start date:
- 2021-02-28
- Actual study start date:
- 2021-03-09
- Planned study completion date:
- No Entry
- Actual Study Completion Date:
- 2021-05-31
- Target Sample Size:
- 40
- Final Sample Size:
- 47
Inclusion Criteria
- Sex:
- All
- Minimum Age:
- 18 Years
- Maximum Age:
- no maximum age
- Additional Inclusion Criteria:
- Diagnosis of a LEMS
Exclusion Criteria
Under 18 years of age
Addresses
Primary Sponsor
- Address:
- Klinik für NeurologieNeuroCure Clinical Research Center (NCRC)Charité Universitätsmedizin BerlinCharité Campus MitteProf. Andreas MeiselCharitéplatz 110117 BerlinGermany
- Telephone:
- +49 30 450 560026
- Fax:
- No Entry
- Contact per E-Mail:
- Contact per E-Mail
- URL:
- https://neurocure.de/ncrc/ueber-uns.html
- Investigator Sponsored/Initiated Trial (IST/IIT):
- Yes
Contact for Scientific Queries
- Address:
- Klinik für NeurologieNeuroCure Clinical Research Center (NCRC)Charité Universitätsmedizin BerlinCharité Campus MitteProf. Andreas MeiselCharitéplatz 110117 BerlinGermany
- Telephone:
- +49 30 450 560026
- Fax:
- No Entry
- Contact per E-Mail:
- Contact per E-Mail
- URL:
- https://neurocure.de/ncrc/ueber-uns.html
Contact for Public Queries
- Address:
- Klinik für Neurologie mit Experimenteller Neurologie und Integriertes Myasthenie Zentrum, (Charité – Universitätsmedizin)Dr. Sophie LehnererChariteplatz 110117 BerlinGermany
- Telephone:
- +49 30 450 539734
- Fax:
- No Entry
- Contact per E-Mail:
- Contact per E-Mail
- URL:
- https://neurologie.charite.de/leistungen/klinische_schwerpunkte/myasthenien/
Principal Investigator
- Address:
- Klinik für NeurologieNeuroCure Clinical Research Center (NCRC)Charité Universitätsmedizin BerlinCharité Campus MitteProf. Andreas MeiselCharitéplatz 110117 BerlinGermany
- Telephone:
- +49 30 450 560026
- Fax:
- No Entry
- Contact per E-Mail:
- Contact per E-Mail
- URL:
- https://neurocure.de/ncrc/ueber-uns.html
Sources of Monetary or Material Support
Institutional budget, no external funding (budget of sponsor/PI)
- Address:
- Klinik für NeurologieNeuroCure Clinical Research Center (NCRC)Charité Universitätsmedizin BerlinCharité Campus MitteCharitéplatz 110117 BerlinGermany
- Telephone:
- +49 30 450 560026
- Fax:
- No Entry
- Contact per E-Mail:
- Contact per E-Mail
- URL:
- https://neurocure.de/ncrc/ueber-uns.html
Ethics Committee
Address Ethics Committee
- Address:
- Ethikkommission der Charité – Universitätsmedizin BerlinCharitéplatz 110117 BerlinGermany
- Telephone:
- (+49)30-450517222
- Fax:
- (+49)30-450517952
- Contact per E-Mail:
- Contact per E-Mail
- URL:
- No Entry
Vote of leading Ethics Committee
- Vote of leading Ethics Committee
- Date of ethics committee application:
- 2020-12-02
- Ethics committee number:
- EA1/390/20
- Vote of the Ethics Committee:
- Approved
- Date of the vote:
- 2021-01-21
Further identification numbers
- Other primary registry ID:
- No Entry
- EudraCT Number:
- No Entry
IPD - Individual Participant Data
- Do you plan to make participant-related data (IPD) available to other researchers in an anonymized form?:
- Yes
- IPD Sharing Plan:
- 1 Will individual participant data be available (including data dictionaries)? Yes. 2. What data in particular will be shared? Individual participant data after de-identification, including results reported in published articles as text, tables, figures, and appendices. 3. What other documents will be available? Study protocol, statistical analysis plan, informed consent, clinical study report, analytical code. 4. When will data be available (start and end dates)? Beginning 3 months and ending 5 years after publication of the article. 5. With whom? With interested third parties and researchers making a methodologically sound proposal, including a precise description of the objective of the evaluation. 6. For what types of analyses? For the scientific evaluation of LEMS burden of disease questions. 7. By what mechanism will data be made available? The proposal should be directed to the principal investigator. To gain access to the data, proposers must sign a Data Access Agreement. The data will be available for 5 years on a third-party website to be determined at a later date.
Study protocol and other study documents
- Study protocols:
- No Entry
- Study abstract:
- No Entry
- Other study documents:
- No Entry
- Background literature:
- No Entry
- Related DRKS studies:
- No Entry
Publication of study results
- Planned publication:
- No Entry
- Publikationen/Studienergebnisse:
- Lehnerer, S., Herdick, M., Stegherr, R. et al. Burden of disease in Lambert-Eaton myasthenic syndrome: taking the patient’s perspective. J Neurol (2024). https://doi.org/10.1007/s00415-024-12206-6
- Date of first publication of study results:
- 2024-02-29
- DRKS entry published for the first time with results:
- 2024-02-29
Basic reporting
- Basic Reporting / Results tables:
- No Entry
- Brief summary of results:
- Abstract Background Lambert-Eaton myasthenic syndrome (LEMS) is an autoimmune-mediated neuromuscular disorder leading to muscle weakness, autonomic dysregulation and hyporeflexia. Psychosocial well-being is affected. Previously, we assessed burden of disease for Myasthenia gravis (MG). Here, we aim to elucidate burden of disease by comparing health-related quality of life (HRQoL) of patients with LEMS to the general population (genP) as well as MG patients. Methods A questionnaire-based survey included sociodemographic and clinical data along with standardized questionnaires, e.g. the Short Form Health (SF-36). HRQoL was evaluated through matched-pairs analyses. Participants from a general health survey served as control group. Results 46 LEMS patients matched by age and gender were compared to 92 controls from the genP and a matched cohort of 92 MG patients. LEMS participants showed lower levels of physical functioning (SF-36 mean 34.2 SD 28.6) compared to genP (mean 78.6 SD 21.1) and MG patients (mean 61.3 SD 31.8). LEMS patients showed lower mental health sub-scores compared to genP (SF-36 mean 62.7 SD 20.2, vs. 75.7 SD 15.1) and MG patients (SF-36 mean 62.7 SD 20.2, vs. 66.0 SD 18.). Depression, anxiety and fatigue were prevalent. Female gender, low income, lower activities of daily living, symptoms of depression, anxiety and fatigue were associated with a lower HRQoL in LEMS. Discussion HRQoL is lower in patients with LEMS compared to genP and MG in a matched pair-analysis. The burden of LEMS includes economic and social aspects as well as emotional well-being.