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).
研究概览
详细说明
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).
研究类型
注册 (估计的)
联系人和位置
学习联系方式
- 姓名：Marie Benhammani-Godard
- 电话号码：0033158411190
- 邮箱：marie.godard@aphp.fr
研究联系人备份
- 姓名：Alexandre ROUEN
- 电话号码：+33 (0)1 42 64 86 26
- 邮箱：alexandre.rouen@aphp.fr
学习地点
-
-
IDF
-
Paris、IDF、法国、75014
- Sleep and Vigilance Center, Hôtel-Dieu
-
-
参与标准
资格标准
适合学习的年龄
接受健康志愿者
有资格学习的性别
取样方法
研究人群
Patients diagnosed with central hypersomnia and diagnosed and/or followed-up at our site.
描述
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.
学习计划
研究是如何设计的？
设计细节
- 观测模型：COHORT
- 时间观点：OTHER
队列和干预
团体/队列 |
干预/治疗 |
|---|---|
|
Central hypersomnia
Patients diagnosed with central hypersomnia |
Non applicable |
研究衡量的是什么？
主要结果指标
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
|
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.
大体时间：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
大体时间：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
|
次要结果测量
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
|
Annual percentage of patients taking at least two treatments for their central hypersomnia.
大体时间：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.
大体时间：During first analyses
|
Number of diagnoses made between 2005 and 2023 that were modified by the new ICSD criteria in 2023. |
During first analyses
|
|
Percentage of diagnoses made between 2005 and 2023 that were modified by the new ICSD criteria in 2023.
大体时间：During first analyses
|
Percentage of diagnoses made between 2005 and 2023 that were modified by the new ICSD criteria in 2023. |
During first analyses
|
|
Number of patients with an initial diagnosis of NT2 who were reclassified as NT1 due to the onset of cataplexy.
大体时间：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.
大体时间：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
大体时间：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.
|
|
Wakefulness maintenance tests
大体时间：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
大体时间：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.
|
|
Average number of years between initial diagnosis and onset of cataplexy
大体时间：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.
大体时间：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.
|
|
Adverse events collected during medical consultations
大体时间：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.
|
|
Effects on other components of the pathologies
大体时间：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.
|
合作者和调查者
调查人员
- 首席研究员：Alexandre ROUEN、APHP
研究记录日期
研究主要日期
学习开始 (估计的)
初级完成 (估计的)
研究完成 (估计的)
研究注册日期
首次提交
首先提交符合 QC 标准的
首次发布 (实际的)
研究记录更新
最后更新发布 (实际的)
上次提交的符合 QC 标准的更新
最后验证
更多信息
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