The European LEMS Registry: Baseline Demographics and Treatment Approaches
Introduction Lambert–Eaton myasthenic syndrome (LEMS) is a rare autoimmune disorder affecting the neuromuscular junction, clinically characterized by proximal muscle weakness and autonomic changes. LEMS is often associated with an underlying tumor (paraneoplastic form) but also occurs in the absence...
Ausführliche Beschreibung
Gespeichert in:
| Autor*in: |
Mantegazza, Renato [verfasserIn] Meisel, Andreas [verfasserIn] Sieb, Joern P. [verfasserIn] Le Masson, Gwendal [verfasserIn] Desnuelle, Claude [verfasserIn] Essing, Mirko [verfasserIn] |
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| Format: |
E-Artikel |
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| Sprache: |
Englisch |
| Erschienen: |
2015 |
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| Übergeordnetes Werk: |
Enthalten in: Neurology and Therapy - Berlin : Springer, 2012, 4(2015), 2 vom: 02. Nov., Seite 105-124 |
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| Übergeordnetes Werk: |
volume:4 ; year:2015 ; number:2 ; day:02 ; month:11 ; pages:105-124 |
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| DOI / URN: |
10.1007/s40120-015-0034-0 |
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| Katalog-ID: |
SPR032876602 |
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| 520 | |a Introduction Lambert–Eaton myasthenic syndrome (LEMS) is a rare autoimmune disorder affecting the neuromuscular junction, clinically characterized by proximal muscle weakness and autonomic changes. LEMS is often associated with an underlying tumor (paraneoplastic form) but also occurs in the absence of cancer (idiopathic form). Treatment consists of immunomodulation (immunosuppression), anticancer treatment when carcinoma is present, and symptomatic treatment [acetylcholinesterase inhibitors and potassium channel blockers, e.g., amifampridine (3,4-diaminopyridine, i.e., 3,4-DAP), to improve neurotransmission]. Although there has long been information from case reports, several randomized controlled trials, and treatment guidelines, population data are still scarce. Methods The LEMS patient registry was launched in the European community in mid-2010 as a voluntary, multinational, observational, non-interventional program to collect structured empirical data on clinical course, treatment utilization, and safety and efficacy from the use of LEMS-specific treatments. Results Sixty-nine patients have been enrolled [36 males, 32 females, 1 gender not reported; mean age 61.5 (27–84) years]. Eighteen patients (26%) were diagnosed with an associated carcinoma. At the time of enrollment, the majority of patients (65%) were receiving amifampridine [either compounded 3,4-DAP (22%) or 3,4-DAP phosphate, $ Firdapse^{®} $ (43%)]. At enrollment, most patients demonstrate a profile of mild-to-moderate deficits in daily functioning but generally have good muscle strength, albeit with reduced deep tendon reflexes, frequent ataxia during walking, and signs of autonomic dysfunction including dry mouth, bladder dysfunction, and constipation. Conclusion The LEMS European Union registry will continue to enroll patients and periodically report the accrued longitudinal data obtained on clinical assessments and laboratory findings, treatment practices, the safety and efficacy of treatment approaches, and long-term clinical outcomes. Funding BioMarin Pharmaceutical Inc., Novato, CA, USA. | ||
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10.1007/s40120-015-0034-0 doi (DE-627)SPR032876602 (SPR)s40120-015-0034-0-e DE-627 ger DE-627 rakwb eng 610 ASE Mantegazza, Renato verfasserin aut The European LEMS Registry: Baseline Demographics and Treatment Approaches 2015 Text txt rdacontent Computermedien c rdamedia Online-Ressource cr rdacarrier Introduction Lambert–Eaton myasthenic syndrome (LEMS) is a rare autoimmune disorder affecting the neuromuscular junction, clinically characterized by proximal muscle weakness and autonomic changes. LEMS is often associated with an underlying tumor (paraneoplastic form) but also occurs in the absence of cancer (idiopathic form). Treatment consists of immunomodulation (immunosuppression), anticancer treatment when carcinoma is present, and symptomatic treatment [acetylcholinesterase inhibitors and potassium channel blockers, e.g., amifampridine (3,4-diaminopyridine, i.e., 3,4-DAP), to improve neurotransmission]. Although there has long been information from case reports, several randomized controlled trials, and treatment guidelines, population data are still scarce. Methods The LEMS patient registry was launched in the European community in mid-2010 as a voluntary, multinational, observational, non-interventional program to collect structured empirical data on clinical course, treatment utilization, and safety and efficacy from the use of LEMS-specific treatments. Results Sixty-nine patients have been enrolled [36 males, 32 females, 1 gender not reported; mean age 61.5 (27–84) years]. Eighteen patients (26%) were diagnosed with an associated carcinoma. At the time of enrollment, the majority of patients (65%) were receiving amifampridine [either compounded 3,4-DAP (22%) or 3,4-DAP phosphate, $ Firdapse^{®} $ (43%)]. At enrollment, most patients demonstrate a profile of mild-to-moderate deficits in daily functioning but generally have good muscle strength, albeit with reduced deep tendon reflexes, frequent ataxia during walking, and signs of autonomic dysfunction including dry mouth, bladder dysfunction, and constipation. Conclusion The LEMS European Union registry will continue to enroll patients and periodically report the accrued longitudinal data obtained on clinical assessments and laboratory findings, treatment practices, the safety and efficacy of treatment approaches, and long-term clinical outcomes. Funding BioMarin Pharmaceutical Inc., Novato, CA, USA. 3,4-Diaminopyridine (3,4-DAP) (dpeaa)DE-He213 Amifampridine (dpeaa)DE-He213 Firdapse (dpeaa)DE-He213 Lambert–Eaton (dpeaa)DE-He213 LEMS (dpeaa)DE-He213 Registry (dpeaa)DE-He213 Meisel, Andreas verfasserin aut Sieb, Joern P. verfasserin aut Le Masson, Gwendal verfasserin aut Desnuelle, Claude verfasserin aut Essing, Mirko verfasserin aut Enthalten in Neurology and Therapy Berlin : Springer, 2012 4(2015), 2 vom: 02. Nov., Seite 105-124 (DE-627)726126209 (DE-600)2682228-3 2193-6536 nnns volume:4 year:2015 number:2 day:02 month:11 pages:105-124 https://dx.doi.org/10.1007/s40120-015-0034-0 kostenfrei Volltext GBV_USEFLAG_A SYSFLAG_A GBV_SPRINGER SSG-OLC-PHA GBV_ILN_20 GBV_ILN_22 GBV_ILN_23 GBV_ILN_24 GBV_ILN_31 GBV_ILN_39 GBV_ILN_40 GBV_ILN_60 GBV_ILN_62 GBV_ILN_63 GBV_ILN_65 GBV_ILN_69 GBV_ILN_73 GBV_ILN_74 GBV_ILN_95 GBV_ILN_105 GBV_ILN_110 GBV_ILN_151 GBV_ILN_161 GBV_ILN_170 GBV_ILN_206 GBV_ILN_213 GBV_ILN_230 GBV_ILN_285 GBV_ILN_293 GBV_ILN_602 GBV_ILN_2014 GBV_ILN_4012 GBV_ILN_4037 GBV_ILN_4112 GBV_ILN_4125 GBV_ILN_4126 GBV_ILN_4249 GBV_ILN_4305 GBV_ILN_4306 GBV_ILN_4307 GBV_ILN_4313 GBV_ILN_4322 GBV_ILN_4323 GBV_ILN_4324 GBV_ILN_4325 GBV_ILN_4338 GBV_ILN_4367 GBV_ILN_4700 AR 4 2015 2 02 11 105-124 |
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10.1007/s40120-015-0034-0 doi (DE-627)SPR032876602 (SPR)s40120-015-0034-0-e DE-627 ger DE-627 rakwb eng 610 ASE Mantegazza, Renato verfasserin aut The European LEMS Registry: Baseline Demographics and Treatment Approaches 2015 Text txt rdacontent Computermedien c rdamedia Online-Ressource cr rdacarrier Introduction Lambert–Eaton myasthenic syndrome (LEMS) is a rare autoimmune disorder affecting the neuromuscular junction, clinically characterized by proximal muscle weakness and autonomic changes. LEMS is often associated with an underlying tumor (paraneoplastic form) but also occurs in the absence of cancer (idiopathic form). Treatment consists of immunomodulation (immunosuppression), anticancer treatment when carcinoma is present, and symptomatic treatment [acetylcholinesterase inhibitors and potassium channel blockers, e.g., amifampridine (3,4-diaminopyridine, i.e., 3,4-DAP), to improve neurotransmission]. Although there has long been information from case reports, several randomized controlled trials, and treatment guidelines, population data are still scarce. Methods The LEMS patient registry was launched in the European community in mid-2010 as a voluntary, multinational, observational, non-interventional program to collect structured empirical data on clinical course, treatment utilization, and safety and efficacy from the use of LEMS-specific treatments. Results Sixty-nine patients have been enrolled [36 males, 32 females, 1 gender not reported; mean age 61.5 (27–84) years]. Eighteen patients (26%) were diagnosed with an associated carcinoma. At the time of enrollment, the majority of patients (65%) were receiving amifampridine [either compounded 3,4-DAP (22%) or 3,4-DAP phosphate, $ Firdapse^{®} $ (43%)]. At enrollment, most patients demonstrate a profile of mild-to-moderate deficits in daily functioning but generally have good muscle strength, albeit with reduced deep tendon reflexes, frequent ataxia during walking, and signs of autonomic dysfunction including dry mouth, bladder dysfunction, and constipation. Conclusion The LEMS European Union registry will continue to enroll patients and periodically report the accrued longitudinal data obtained on clinical assessments and laboratory findings, treatment practices, the safety and efficacy of treatment approaches, and long-term clinical outcomes. Funding BioMarin Pharmaceutical Inc., Novato, CA, USA. 3,4-Diaminopyridine (3,4-DAP) (dpeaa)DE-He213 Amifampridine (dpeaa)DE-He213 Firdapse (dpeaa)DE-He213 Lambert–Eaton (dpeaa)DE-He213 LEMS (dpeaa)DE-He213 Registry (dpeaa)DE-He213 Meisel, Andreas verfasserin aut Sieb, Joern P. verfasserin aut Le Masson, Gwendal verfasserin aut Desnuelle, Claude verfasserin aut Essing, Mirko verfasserin aut Enthalten in Neurology and Therapy Berlin : Springer, 2012 4(2015), 2 vom: 02. Nov., Seite 105-124 (DE-627)726126209 (DE-600)2682228-3 2193-6536 nnns volume:4 year:2015 number:2 day:02 month:11 pages:105-124 https://dx.doi.org/10.1007/s40120-015-0034-0 kostenfrei Volltext GBV_USEFLAG_A SYSFLAG_A GBV_SPRINGER SSG-OLC-PHA GBV_ILN_20 GBV_ILN_22 GBV_ILN_23 GBV_ILN_24 GBV_ILN_31 GBV_ILN_39 GBV_ILN_40 GBV_ILN_60 GBV_ILN_62 GBV_ILN_63 GBV_ILN_65 GBV_ILN_69 GBV_ILN_73 GBV_ILN_74 GBV_ILN_95 GBV_ILN_105 GBV_ILN_110 GBV_ILN_151 GBV_ILN_161 GBV_ILN_170 GBV_ILN_206 GBV_ILN_213 GBV_ILN_230 GBV_ILN_285 GBV_ILN_293 GBV_ILN_602 GBV_ILN_2014 GBV_ILN_4012 GBV_ILN_4037 GBV_ILN_4112 GBV_ILN_4125 GBV_ILN_4126 GBV_ILN_4249 GBV_ILN_4305 GBV_ILN_4306 GBV_ILN_4307 GBV_ILN_4313 GBV_ILN_4322 GBV_ILN_4323 GBV_ILN_4324 GBV_ILN_4325 GBV_ILN_4338 GBV_ILN_4367 GBV_ILN_4700 AR 4 2015 2 02 11 105-124 |
| allfields_unstemmed |
10.1007/s40120-015-0034-0 doi (DE-627)SPR032876602 (SPR)s40120-015-0034-0-e DE-627 ger DE-627 rakwb eng 610 ASE Mantegazza, Renato verfasserin aut The European LEMS Registry: Baseline Demographics and Treatment Approaches 2015 Text txt rdacontent Computermedien c rdamedia Online-Ressource cr rdacarrier Introduction Lambert–Eaton myasthenic syndrome (LEMS) is a rare autoimmune disorder affecting the neuromuscular junction, clinically characterized by proximal muscle weakness and autonomic changes. LEMS is often associated with an underlying tumor (paraneoplastic form) but also occurs in the absence of cancer (idiopathic form). Treatment consists of immunomodulation (immunosuppression), anticancer treatment when carcinoma is present, and symptomatic treatment [acetylcholinesterase inhibitors and potassium channel blockers, e.g., amifampridine (3,4-diaminopyridine, i.e., 3,4-DAP), to improve neurotransmission]. Although there has long been information from case reports, several randomized controlled trials, and treatment guidelines, population data are still scarce. Methods The LEMS patient registry was launched in the European community in mid-2010 as a voluntary, multinational, observational, non-interventional program to collect structured empirical data on clinical course, treatment utilization, and safety and efficacy from the use of LEMS-specific treatments. Results Sixty-nine patients have been enrolled [36 males, 32 females, 1 gender not reported; mean age 61.5 (27–84) years]. Eighteen patients (26%) were diagnosed with an associated carcinoma. At the time of enrollment, the majority of patients (65%) were receiving amifampridine [either compounded 3,4-DAP (22%) or 3,4-DAP phosphate, $ Firdapse^{®} $ (43%)]. At enrollment, most patients demonstrate a profile of mild-to-moderate deficits in daily functioning but generally have good muscle strength, albeit with reduced deep tendon reflexes, frequent ataxia during walking, and signs of autonomic dysfunction including dry mouth, bladder dysfunction, and constipation. Conclusion The LEMS European Union registry will continue to enroll patients and periodically report the accrued longitudinal data obtained on clinical assessments and laboratory findings, treatment practices, the safety and efficacy of treatment approaches, and long-term clinical outcomes. Funding BioMarin Pharmaceutical Inc., Novato, CA, USA. 3,4-Diaminopyridine (3,4-DAP) (dpeaa)DE-He213 Amifampridine (dpeaa)DE-He213 Firdapse (dpeaa)DE-He213 Lambert–Eaton (dpeaa)DE-He213 LEMS (dpeaa)DE-He213 Registry (dpeaa)DE-He213 Meisel, Andreas verfasserin aut Sieb, Joern P. verfasserin aut Le Masson, Gwendal verfasserin aut Desnuelle, Claude verfasserin aut Essing, Mirko verfasserin aut Enthalten in Neurology and Therapy Berlin : Springer, 2012 4(2015), 2 vom: 02. Nov., Seite 105-124 (DE-627)726126209 (DE-600)2682228-3 2193-6536 nnns volume:4 year:2015 number:2 day:02 month:11 pages:105-124 https://dx.doi.org/10.1007/s40120-015-0034-0 kostenfrei Volltext GBV_USEFLAG_A SYSFLAG_A GBV_SPRINGER SSG-OLC-PHA GBV_ILN_20 GBV_ILN_22 GBV_ILN_23 GBV_ILN_24 GBV_ILN_31 GBV_ILN_39 GBV_ILN_40 GBV_ILN_60 GBV_ILN_62 GBV_ILN_63 GBV_ILN_65 GBV_ILN_69 GBV_ILN_73 GBV_ILN_74 GBV_ILN_95 GBV_ILN_105 GBV_ILN_110 GBV_ILN_151 GBV_ILN_161 GBV_ILN_170 GBV_ILN_206 GBV_ILN_213 GBV_ILN_230 GBV_ILN_285 GBV_ILN_293 GBV_ILN_602 GBV_ILN_2014 GBV_ILN_4012 GBV_ILN_4037 GBV_ILN_4112 GBV_ILN_4125 GBV_ILN_4126 GBV_ILN_4249 GBV_ILN_4305 GBV_ILN_4306 GBV_ILN_4307 GBV_ILN_4313 GBV_ILN_4322 GBV_ILN_4323 GBV_ILN_4324 GBV_ILN_4325 GBV_ILN_4338 GBV_ILN_4367 GBV_ILN_4700 AR 4 2015 2 02 11 105-124 |
| allfieldsGer |
10.1007/s40120-015-0034-0 doi (DE-627)SPR032876602 (SPR)s40120-015-0034-0-e DE-627 ger DE-627 rakwb eng 610 ASE Mantegazza, Renato verfasserin aut The European LEMS Registry: Baseline Demographics and Treatment Approaches 2015 Text txt rdacontent Computermedien c rdamedia Online-Ressource cr rdacarrier Introduction Lambert–Eaton myasthenic syndrome (LEMS) is a rare autoimmune disorder affecting the neuromuscular junction, clinically characterized by proximal muscle weakness and autonomic changes. LEMS is often associated with an underlying tumor (paraneoplastic form) but also occurs in the absence of cancer (idiopathic form). Treatment consists of immunomodulation (immunosuppression), anticancer treatment when carcinoma is present, and symptomatic treatment [acetylcholinesterase inhibitors and potassium channel blockers, e.g., amifampridine (3,4-diaminopyridine, i.e., 3,4-DAP), to improve neurotransmission]. Although there has long been information from case reports, several randomized controlled trials, and treatment guidelines, population data are still scarce. Methods The LEMS patient registry was launched in the European community in mid-2010 as a voluntary, multinational, observational, non-interventional program to collect structured empirical data on clinical course, treatment utilization, and safety and efficacy from the use of LEMS-specific treatments. Results Sixty-nine patients have been enrolled [36 males, 32 females, 1 gender not reported; mean age 61.5 (27–84) years]. Eighteen patients (26%) were diagnosed with an associated carcinoma. At the time of enrollment, the majority of patients (65%) were receiving amifampridine [either compounded 3,4-DAP (22%) or 3,4-DAP phosphate, $ Firdapse^{®} $ (43%)]. At enrollment, most patients demonstrate a profile of mild-to-moderate deficits in daily functioning but generally have good muscle strength, albeit with reduced deep tendon reflexes, frequent ataxia during walking, and signs of autonomic dysfunction including dry mouth, bladder dysfunction, and constipation. Conclusion The LEMS European Union registry will continue to enroll patients and periodically report the accrued longitudinal data obtained on clinical assessments and laboratory findings, treatment practices, the safety and efficacy of treatment approaches, and long-term clinical outcomes. Funding BioMarin Pharmaceutical Inc., Novato, CA, USA. 3,4-Diaminopyridine (3,4-DAP) (dpeaa)DE-He213 Amifampridine (dpeaa)DE-He213 Firdapse (dpeaa)DE-He213 Lambert–Eaton (dpeaa)DE-He213 LEMS (dpeaa)DE-He213 Registry (dpeaa)DE-He213 Meisel, Andreas verfasserin aut Sieb, Joern P. verfasserin aut Le Masson, Gwendal verfasserin aut Desnuelle, Claude verfasserin aut Essing, Mirko verfasserin aut Enthalten in Neurology and Therapy Berlin : Springer, 2012 4(2015), 2 vom: 02. Nov., Seite 105-124 (DE-627)726126209 (DE-600)2682228-3 2193-6536 nnns volume:4 year:2015 number:2 day:02 month:11 pages:105-124 https://dx.doi.org/10.1007/s40120-015-0034-0 kostenfrei Volltext GBV_USEFLAG_A SYSFLAG_A GBV_SPRINGER SSG-OLC-PHA GBV_ILN_20 GBV_ILN_22 GBV_ILN_23 GBV_ILN_24 GBV_ILN_31 GBV_ILN_39 GBV_ILN_40 GBV_ILN_60 GBV_ILN_62 GBV_ILN_63 GBV_ILN_65 GBV_ILN_69 GBV_ILN_73 GBV_ILN_74 GBV_ILN_95 GBV_ILN_105 GBV_ILN_110 GBV_ILN_151 GBV_ILN_161 GBV_ILN_170 GBV_ILN_206 GBV_ILN_213 GBV_ILN_230 GBV_ILN_285 GBV_ILN_293 GBV_ILN_602 GBV_ILN_2014 GBV_ILN_4012 GBV_ILN_4037 GBV_ILN_4112 GBV_ILN_4125 GBV_ILN_4126 GBV_ILN_4249 GBV_ILN_4305 GBV_ILN_4306 GBV_ILN_4307 GBV_ILN_4313 GBV_ILN_4322 GBV_ILN_4323 GBV_ILN_4324 GBV_ILN_4325 GBV_ILN_4338 GBV_ILN_4367 GBV_ILN_4700 AR 4 2015 2 02 11 105-124 |
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10.1007/s40120-015-0034-0 doi (DE-627)SPR032876602 (SPR)s40120-015-0034-0-e DE-627 ger DE-627 rakwb eng 610 ASE Mantegazza, Renato verfasserin aut The European LEMS Registry: Baseline Demographics and Treatment Approaches 2015 Text txt rdacontent Computermedien c rdamedia Online-Ressource cr rdacarrier Introduction Lambert–Eaton myasthenic syndrome (LEMS) is a rare autoimmune disorder affecting the neuromuscular junction, clinically characterized by proximal muscle weakness and autonomic changes. LEMS is often associated with an underlying tumor (paraneoplastic form) but also occurs in the absence of cancer (idiopathic form). Treatment consists of immunomodulation (immunosuppression), anticancer treatment when carcinoma is present, and symptomatic treatment [acetylcholinesterase inhibitors and potassium channel blockers, e.g., amifampridine (3,4-diaminopyridine, i.e., 3,4-DAP), to improve neurotransmission]. Although there has long been information from case reports, several randomized controlled trials, and treatment guidelines, population data are still scarce. Methods The LEMS patient registry was launched in the European community in mid-2010 as a voluntary, multinational, observational, non-interventional program to collect structured empirical data on clinical course, treatment utilization, and safety and efficacy from the use of LEMS-specific treatments. Results Sixty-nine patients have been enrolled [36 males, 32 females, 1 gender not reported; mean age 61.5 (27–84) years]. Eighteen patients (26%) were diagnosed with an associated carcinoma. At the time of enrollment, the majority of patients (65%) were receiving amifampridine [either compounded 3,4-DAP (22%) or 3,4-DAP phosphate, $ Firdapse^{®} $ (43%)]. At enrollment, most patients demonstrate a profile of mild-to-moderate deficits in daily functioning but generally have good muscle strength, albeit with reduced deep tendon reflexes, frequent ataxia during walking, and signs of autonomic dysfunction including dry mouth, bladder dysfunction, and constipation. Conclusion The LEMS European Union registry will continue to enroll patients and periodically report the accrued longitudinal data obtained on clinical assessments and laboratory findings, treatment practices, the safety and efficacy of treatment approaches, and long-term clinical outcomes. Funding BioMarin Pharmaceutical Inc., Novato, CA, USA. 3,4-Diaminopyridine (3,4-DAP) (dpeaa)DE-He213 Amifampridine (dpeaa)DE-He213 Firdapse (dpeaa)DE-He213 Lambert–Eaton (dpeaa)DE-He213 LEMS (dpeaa)DE-He213 Registry (dpeaa)DE-He213 Meisel, Andreas verfasserin aut Sieb, Joern P. verfasserin aut Le Masson, Gwendal verfasserin aut Desnuelle, Claude verfasserin aut Essing, Mirko verfasserin aut Enthalten in Neurology and Therapy Berlin : Springer, 2012 4(2015), 2 vom: 02. Nov., Seite 105-124 (DE-627)726126209 (DE-600)2682228-3 2193-6536 nnns volume:4 year:2015 number:2 day:02 month:11 pages:105-124 https://dx.doi.org/10.1007/s40120-015-0034-0 kostenfrei Volltext GBV_USEFLAG_A SYSFLAG_A GBV_SPRINGER SSG-OLC-PHA GBV_ILN_20 GBV_ILN_22 GBV_ILN_23 GBV_ILN_24 GBV_ILN_31 GBV_ILN_39 GBV_ILN_40 GBV_ILN_60 GBV_ILN_62 GBV_ILN_63 GBV_ILN_65 GBV_ILN_69 GBV_ILN_73 GBV_ILN_74 GBV_ILN_95 GBV_ILN_105 GBV_ILN_110 GBV_ILN_151 GBV_ILN_161 GBV_ILN_170 GBV_ILN_206 GBV_ILN_213 GBV_ILN_230 GBV_ILN_285 GBV_ILN_293 GBV_ILN_602 GBV_ILN_2014 GBV_ILN_4012 GBV_ILN_4037 GBV_ILN_4112 GBV_ILN_4125 GBV_ILN_4126 GBV_ILN_4249 GBV_ILN_4305 GBV_ILN_4306 GBV_ILN_4307 GBV_ILN_4313 GBV_ILN_4322 GBV_ILN_4323 GBV_ILN_4324 GBV_ILN_4325 GBV_ILN_4338 GBV_ILN_4367 GBV_ILN_4700 AR 4 2015 2 02 11 105-124 |
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Introduction Lambert–Eaton myasthenic syndrome (LEMS) is a rare autoimmune disorder affecting the neuromuscular junction, clinically characterized by proximal muscle weakness and autonomic changes. LEMS is often associated with an underlying tumor (paraneoplastic form) but also occurs in the absence of cancer (idiopathic form). Treatment consists of immunomodulation (immunosuppression), anticancer treatment when carcinoma is present, and symptomatic treatment [acetylcholinesterase inhibitors and potassium channel blockers, e.g., amifampridine (3,4-diaminopyridine, i.e., 3,4-DAP), to improve neurotransmission]. Although there has long been information from case reports, several randomized controlled trials, and treatment guidelines, population data are still scarce. Methods The LEMS patient registry was launched in the European community in mid-2010 as a voluntary, multinational, observational, non-interventional program to collect structured empirical data on clinical course, treatment utilization, and safety and efficacy from the use of LEMS-specific treatments. Results Sixty-nine patients have been enrolled [36 males, 32 females, 1 gender not reported; mean age 61.5 (27–84) years]. Eighteen patients (26%) were diagnosed with an associated carcinoma. At the time of enrollment, the majority of patients (65%) were receiving amifampridine [either compounded 3,4-DAP (22%) or 3,4-DAP phosphate, $ Firdapse^{®} $ (43%)]. At enrollment, most patients demonstrate a profile of mild-to-moderate deficits in daily functioning but generally have good muscle strength, albeit with reduced deep tendon reflexes, frequent ataxia during walking, and signs of autonomic dysfunction including dry mouth, bladder dysfunction, and constipation. Conclusion The LEMS European Union registry will continue to enroll patients and periodically report the accrued longitudinal data obtained on clinical assessments and laboratory findings, treatment practices, the safety and efficacy of treatment approaches, and long-term clinical outcomes. Funding BioMarin Pharmaceutical Inc., Novato, CA, USA. |
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Introduction Lambert–Eaton myasthenic syndrome (LEMS) is a rare autoimmune disorder affecting the neuromuscular junction, clinically characterized by proximal muscle weakness and autonomic changes. LEMS is often associated with an underlying tumor (paraneoplastic form) but also occurs in the absence of cancer (idiopathic form). Treatment consists of immunomodulation (immunosuppression), anticancer treatment when carcinoma is present, and symptomatic treatment [acetylcholinesterase inhibitors and potassium channel blockers, e.g., amifampridine (3,4-diaminopyridine, i.e., 3,4-DAP), to improve neurotransmission]. Although there has long been information from case reports, several randomized controlled trials, and treatment guidelines, population data are still scarce. Methods The LEMS patient registry was launched in the European community in mid-2010 as a voluntary, multinational, observational, non-interventional program to collect structured empirical data on clinical course, treatment utilization, and safety and efficacy from the use of LEMS-specific treatments. Results Sixty-nine patients have been enrolled [36 males, 32 females, 1 gender not reported; mean age 61.5 (27–84) years]. Eighteen patients (26%) were diagnosed with an associated carcinoma. At the time of enrollment, the majority of patients (65%) were receiving amifampridine [either compounded 3,4-DAP (22%) or 3,4-DAP phosphate, $ Firdapse^{®} $ (43%)]. At enrollment, most patients demonstrate a profile of mild-to-moderate deficits in daily functioning but generally have good muscle strength, albeit with reduced deep tendon reflexes, frequent ataxia during walking, and signs of autonomic dysfunction including dry mouth, bladder dysfunction, and constipation. Conclusion The LEMS European Union registry will continue to enroll patients and periodically report the accrued longitudinal data obtained on clinical assessments and laboratory findings, treatment practices, the safety and efficacy of treatment approaches, and long-term clinical outcomes. Funding BioMarin Pharmaceutical Inc., Novato, CA, USA. |
| abstract_unstemmed |
Introduction Lambert–Eaton myasthenic syndrome (LEMS) is a rare autoimmune disorder affecting the neuromuscular junction, clinically characterized by proximal muscle weakness and autonomic changes. LEMS is often associated with an underlying tumor (paraneoplastic form) but also occurs in the absence of cancer (idiopathic form). Treatment consists of immunomodulation (immunosuppression), anticancer treatment when carcinoma is present, and symptomatic treatment [acetylcholinesterase inhibitors and potassium channel blockers, e.g., amifampridine (3,4-diaminopyridine, i.e., 3,4-DAP), to improve neurotransmission]. Although there has long been information from case reports, several randomized controlled trials, and treatment guidelines, population data are still scarce. Methods The LEMS patient registry was launched in the European community in mid-2010 as a voluntary, multinational, observational, non-interventional program to collect structured empirical data on clinical course, treatment utilization, and safety and efficacy from the use of LEMS-specific treatments. Results Sixty-nine patients have been enrolled [36 males, 32 females, 1 gender not reported; mean age 61.5 (27–84) years]. Eighteen patients (26%) were diagnosed with an associated carcinoma. At the time of enrollment, the majority of patients (65%) were receiving amifampridine [either compounded 3,4-DAP (22%) or 3,4-DAP phosphate, $ Firdapse^{®} $ (43%)]. At enrollment, most patients demonstrate a profile of mild-to-moderate deficits in daily functioning but generally have good muscle strength, albeit with reduced deep tendon reflexes, frequent ataxia during walking, and signs of autonomic dysfunction including dry mouth, bladder dysfunction, and constipation. Conclusion The LEMS European Union registry will continue to enroll patients and periodically report the accrued longitudinal data obtained on clinical assessments and laboratory findings, treatment practices, the safety and efficacy of treatment approaches, and long-term clinical outcomes. Funding BioMarin Pharmaceutical Inc., Novato, CA, USA. |
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The European LEMS Registry: Baseline Demographics and Treatment Approaches |
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Meisel, Andreas Sieb, Joern P. Le Masson, Gwendal Desnuelle, Claude Essing, Mirko |
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LEMS is often associated with an underlying tumor (paraneoplastic form) but also occurs in the absence of cancer (idiopathic form). Treatment consists of immunomodulation (immunosuppression), anticancer treatment when carcinoma is present, and symptomatic treatment [acetylcholinesterase inhibitors and potassium channel blockers, e.g., amifampridine (3,4-diaminopyridine, i.e., 3,4-DAP), to improve neurotransmission]. Although there has long been information from case reports, several randomized controlled trials, and treatment guidelines, population data are still scarce. Methods The LEMS patient registry was launched in the European community in mid-2010 as a voluntary, multinational, observational, non-interventional program to collect structured empirical data on clinical course, treatment utilization, and safety and efficacy from the use of LEMS-specific treatments. 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