Evolution of the Treatment of Rare Hypersomnias in a Reference Center Certified Since 2005 (EPHyRa)
Central hypersomnias are rare diseases that include narcolepsy and idiopathic hypersomnia.
The Sleep and Vigilance Center at Hôtel-Dieu has been certified as a Rare Disease Reference Center (CRMR) for these conditions since 2005.
Scientific advances and changes to the ICSD (International Classification of Sleep Disorders) since 2005 (diagnostic criteria and drugs brought to market) have greatly improved the care of patients with central hypersomnia.
This retrospective and prospective study aims to evaluate how each advance translates into concrete terms in the care of patients followed at our center.
Main objective :
Determine whether new treatments are prescribed to replace treatments already on the market (reduction in prescriptions for treatments previously marketed) or whether they enable patients who have reached a therapeutic impasse to be treated (no reduction in prescriptions for treatments previously marketed).
Descripción general del estudio
Estado
Intervención / Tratamiento
Descripción detallada
Central hypersomnias are rare diseases that include narcolepsy and idiopathic hypersomnia.
The Sleep and Vigilance Center at Hôtel-Dieu has been certified as a Rare Disease Reference Center (CRMR) for these conditions since 2005.
Scientific advances and changes to the ICSD (International Classification of Sleep Disorders) since 2005 (diagnostic criteria and drugs brought to market) have greatly improved the care of patients with central hypersomnia.
This retrospective and prospective study aims to evaluate how each advance translates into concrete terms in the care of patients followed at our center.
Main objective :
Determine whether new treatments are prescribed to replace treatments already on the market (reduction in prescriptions for treatments previously marketed) or whether they enable patients who have reached a therapeutic impasse to be treated (no reduction in prescriptions for treatments previously marketed).
Tipo de estudio
Inscripción (Estimado)
Contactos y Ubicaciones
Estudio Contacto
- Nombre: Marie Benhammani-Godard
- Número de teléfono: 0033158411190
- Correo electrónico: marie.godard@aphp.fr
Copia de seguridad de contactos de estudio
- Nombre: Alexandre ROUEN
- Número de teléfono: +33 (0)1 42 64 86 26
- Correo electrónico: alexandre.rouen@aphp.fr
Ubicaciones de estudio
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IDF
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Paris, IDF, Francia, 75014
- Sleep and Vigilance Center, Hôtel-Dieu
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Criterios de participación
Criterio de elegibilidad
Edades elegibles para estudiar
Acepta Voluntarios Saludables
Géneros elegibles para el estudio
Método de muestreo
Población de estudio
Patients diagnosed with central hypersomnia and diagnosed and/or followed-up at our site.
Descripción
Inclusion Criteria:
- Patients who have had at least one consultation or examination at the Sleep and Vigilance Center at Hôtel-Dieu since January 1, 2005;
- Patients diagnosed with narcolepsy (type 1 or type 2) or idiopathic hypersomnia;
- Adult patients (aged 18 or over);
- Patients who have not objected to the processing of their data.
Exclusion Criteria:
- Minors;
- Patients who object to the processing of their data;
- Patients under guardianship or conservatorship.
Plan de estudios
¿Cómo está diseñado el estudio?
Detalles de diseño
- Modelos observacionales: COHORT
- Perspectivas temporales: OTHER
Cohortes e Intervenciones
Grupo / Cohorte |
Intervención / Tratamiento |
|---|---|
|
Central hypersomnia
Patients diagnosed with central hypersomnia |
Non applicable |
¿Qué mide el estudio?
Medidas de resultado primarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
|---|---|---|
|
Ratio of the number of patients who were prescribed this treatment during the year to the number of patients who had at least one consultation at the center during the same year.
Periodo de tiempo: For the 1st time for prescriptions from Jan 1, 2005, to Dec 31, 2025. Then, every three years: prescriptions until Dec 31, 2028, Dec 31, 2031, and Dec 31, 2034, in order to take into account marketing of new treatments currently in phase 3
|
Ratio of the number of patients who were prescribed this treatment during the year to the number of patients who had at least one consultation at the center during the same year. Annual change in prescriptions for each treatment |
For the 1st time for prescriptions from Jan 1, 2005, to Dec 31, 2025. Then, every three years: prescriptions until Dec 31, 2028, Dec 31, 2031, and Dec 31, 2034, in order to take into account marketing of new treatments currently in phase 3
|
|
Number of days of treatment
Periodo de tiempo: For the 1st time for prescriptions from Jan 1, 2005, to Dec 31, 2025. Then, every three years: prescriptions until Dec 31, 2028, Dec 31, 2031, and Dec 31, 2034, in order to take into account marketing of new treatments currently in phase 3
|
Number of days of treatment: mean + standard deviation for patients who received this treatment during the year, median and quartiles. Annual change in prescriptions for each treatment |
For the 1st time for prescriptions from Jan 1, 2005, to Dec 31, 2025. Then, every three years: prescriptions until Dec 31, 2028, Dec 31, 2031, and Dec 31, 2034, in order to take into account marketing of new treatments currently in phase 3
|
Medidas de resultado secundarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
|---|---|---|
|
Annual percentage of patients taking at least two treatments for their central hypersomnia.
Periodo de tiempo: This criterion will be assessed for the first time for prescriptions from January 1, 2005, to December 31, 2025. Then, every 3 years: prescriptions until December 31, 2028, December 31, 2031, and December 31, 2034.
|
Annual percentage of patients taking at least two treatments for their central hypersomnia. |
This criterion will be assessed for the first time for prescriptions from January 1, 2005, to December 31, 2025. Then, every 3 years: prescriptions until December 31, 2028, December 31, 2031, and December 31, 2034.
|
|
Number of diagnoses made between 2005 and 2023 that were modified by the new ICSD criteria in 2023.
Periodo de tiempo: During first analyses
|
Number of diagnoses made between 2005 and 2023 that were modified by the new ICSD criteria in 2023. |
During first analyses
|
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Percentage of diagnoses made between 2005 and 2023 that were modified by the new ICSD criteria in 2023.
Periodo de tiempo: During first analyses
|
Percentage of diagnoses made between 2005 and 2023 that were modified by the new ICSD criteria in 2023. |
During first analyses
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Number of patients with an initial diagnosis of NT2 who were reclassified as NT1 due to the onset of cataplexy.
Periodo de tiempo: This criterion will be assessed for the first time for prescriptions from January 1, 2005, to December 31, 2025. Then, every 3 years: prescriptions until December 31, 2028, December 31, 2031, and December 31, 2034.
|
Number of patients with an initial diagnosis of NT2 who were reclassified as NT1 due to the onset of cataplexy. |
This criterion will be assessed for the first time for prescriptions from January 1, 2005, to December 31, 2025. Then, every 3 years: prescriptions until December 31, 2028, December 31, 2031, and December 31, 2034.
|
|
Percentage of patients with an initial diagnosis of NT2 who were reclassified as NT1 due to the onset of cataplexy.
Periodo de tiempo: This criterion will be assessed for the first time for prescriptions from January 1, 2005, to December 31, 2025. Then, every 3 years: prescriptions until December 31, 2028, December 31, 2031, and December 31, 2034.
|
Percentage of patients with an initial diagnosis of NT2 who were reclassified as NT1 due to the onset of cataplexy. |
This criterion will be assessed for the first time for prescriptions from January 1, 2005, to December 31, 2025. Then, every 3 years: prescriptions until December 31, 2028, December 31, 2031, and December 31, 2034.
|
|
Epworth scale
Periodo de tiempo: This criterion will be assessed for the first time for prescriptions from January 1, 2005, to December 31, 2025. Then, every 3 years: prescriptions until December 31, 2028, December 31, 2031, and December 31, 2034.
|
Reduction in sleepiness (Epworth scale and wakefulness maintenance tests) average for each treatment and combination of treatments. |
This criterion will be assessed for the first time for prescriptions from January 1, 2005, to December 31, 2025. Then, every 3 years: prescriptions until December 31, 2028, December 31, 2031, and December 31, 2034.
|
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Wakefulness maintenance tests
Periodo de tiempo: This criterion will be assessed for the first time for prescriptions from January 1, 2005, to December 31, 2025. Then, every 3 years: prescriptions until December 31, 2028, December 31, 2031, and December 31, 2034.
|
Reduction in sleepiness (Epworth scale and wakefulness maintenance tests) average for each treatment and combination of treatments. |
This criterion will be assessed for the first time for prescriptions from January 1, 2005, to December 31, 2025. Then, every 3 years: prescriptions until December 31, 2028, December 31, 2031, and December 31, 2034.
|
|
Average age of these patients at the onset of cataplexy
Periodo de tiempo: This criterion will be assessed for the first time for prescriptions from January 1, 2005, to December 31, 2025. Then, every 3 years: prescriptions until December 31, 2028, December 31, 2031, and December 31, 2034.
|
Average age of these patients at the onset of cataplexy |
This criterion will be assessed for the first time for prescriptions from January 1, 2005, to December 31, 2025. Then, every 3 years: prescriptions until December 31, 2028, December 31, 2031, and December 31, 2034.
|
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Average number of years between initial diagnosis and onset of cataplexy
Periodo de tiempo: This criterion will be assessed for the first time for prescriptions from January 1, 2005, to December 31, 2025. Then, every 3 years: prescriptions until December 31, 2028, December 31, 2031, and December 31, 2034.
|
Average number of years between initial diagnosis and onset of cataplexy |
This criterion will be assessed for the first time for prescriptions from January 1, 2005, to December 31, 2025. Then, every 3 years: prescriptions until December 31, 2028, December 31, 2031, and December 31, 2034.
|
|
Average number of years between first symptoms and onset of cataplexy.
Periodo de tiempo: This criterion will be assessed for the first time for prescriptions from January 1, 2005, to December 31, 2025. Then, every 3 years: prescriptions until December 31, 2028, December 31, 2031, and December 31, 2034.
|
Average number of years between first symptoms and onset of cataplexy. |
This criterion will be assessed for the first time for prescriptions from January 1, 2005, to December 31, 2025. Then, every 3 years: prescriptions until December 31, 2028, December 31, 2031, and December 31, 2034.
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|
Adverse events collected during medical consultations
Periodo de tiempo: This criterion will be assessed for the first time for prescriptions from January 1, 2005, to December 31, 2025. Then, every 3 years: prescriptions until December 31, 2028, December 31, 2031, and December 31, 2034.
|
Adverse events collected during medical consultations |
This criterion will be assessed for the first time for prescriptions from January 1, 2005, to December 31, 2025. Then, every 3 years: prescriptions until December 31, 2028, December 31, 2031, and December 31, 2034.
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Effects on other components of the pathologies
Periodo de tiempo: This criterion will be assessed for the first time for prescriptions from January 1, 2005, to December 31, 2025. Then, every 3 years: prescriptions until December 31, 2028, December 31, 2031, and December 31, 2034.
|
Effects on other components of the pathologies (via IHSS scores, NSS, fatigue and brain fog scale, etc.). |
This criterion will be assessed for the first time for prescriptions from January 1, 2005, to December 31, 2025. Then, every 3 years: prescriptions until December 31, 2028, December 31, 2031, and December 31, 2034.
|
Colaboradores e Investigadores
Patrocinador
Colaboradores
Investigadores
- Investigador principal: Alexandre ROUEN, APHP
Fechas de registro del estudio
Fechas importantes del estudio
Inicio del estudio (Estimado)
Finalización primaria (Estimado)
Finalización del estudio (Estimado)
Fechas de registro del estudio
Enviado por primera vez
Primero enviado que cumplió con los criterios de control de calidad
Publicado por primera vez (Actual)
Actualizaciones de registros de estudio
Última actualización publicada (Actual)
Última actualización enviada que cumplió con los criterios de control de calidad
Última verificación
Más información
Términos relacionados con este estudio
Palabras clave
Términos MeSH relevantes adicionales
Otros números de identificación del estudio
- APHP251690
Plan de datos de participantes individuales (IPD)
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Información sobre medicamentos y dispositivos, documentos del estudio
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