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).
調査の概要
詳細な説明
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).
研究の種類
入学 (推定)
連絡先と場所
研究連絡先
- 名前：Marie Benhammani-Godard
- 電話番号：0033158411190
- メール：marie.godard@aphp.fr
研究連絡先のバックアップ
- 名前：Alexandre ROUEN
- 電話番号：+33 (0)1 42 64 86 26
- メール：alexandre.rouen@aphp.fr
研究場所
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-
IDF
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Paris、IDF、フランス、75014
- Sleep and Vigilance Center, Hôtel-Dieu
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参加基準
適格基準
就学可能な年齢
健康ボランティアの受け入れ
受講資格のある性別
サンプリング方法
調査対象母集団
Patients diagnosed with central hypersomnia and diagnosed and/or followed-up at our site.
説明
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.
研究計画
研究はどのように設計されていますか？
デザインの詳細
- 観測モデル：COHORT
- 時間の展望：OTHER
コホートと介入
グループ/コホート |
介入・治療 |
|---|---|
|
Central hypersomnia
Patients diagnosed with central hypersomnia |
Non applicable |
この研究は何を測定していますか？
主要な結果の測定
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
|
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.
時間枠：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
時間枠：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
|
二次結果の測定
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
|
Annual percentage of patients taking at least two treatments for their central hypersomnia.
時間枠：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.
時間枠：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.
時間枠：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.
時間枠：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.
時間枠：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
時間枠：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
時間枠：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
時間枠：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
時間枠：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.
時間枠：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
時間枠：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
時間枠：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.
|
協力者と研究者
捜査官
- 主任研究者：Alexandre ROUEN、APHP
研究記録日
主要日程の研究
研究開始 (推定)
一次修了 (推定)
研究の完了 (推定)
試験登録日
最初に提出
QC基準を満たした最初の提出物
最初の投稿 (実際)
学習記録の更新
投稿された最後の更新 (実際)
QC基準を満たした最後の更新が送信されました
最終確認日
詳しくは
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