STill病におけるマクロファージ活性化症候群Eの評価 (AMETHYST)
スティル病におけるマクロファージ活性化症候群の評価: 患者の病歴、症状の解決および治療の特徴の遡及的チャート分析
Assessment of Macrophage activation syndrome in STill's disease: retrospective chart analysis of patient History, Symptom resolution and Treatment characteristics
調査の概要
詳細な説明
This is an observational, retrospective cohort study on the treatment utilization and outcomes of Macrophage Activation Syndrome (MAS) refractory to glucocorticoids (GC) in patients with Still's disease. The study will be conducted entirely through medical chart abstraction; all data will be taken from the patient's medical record, with no additional assessments.
研究の種類
入学 (実際)
連絡先と場所
研究場所
-
-
California
-
Los Angeles、California、アメリカ、90095
- Swedish Orphan Biovitrum Research Site
-
-
Georgia
-
Atlanta、Georgia、アメリカ、30329
- Swedish Orphan Biovitrum Research Site
-
-
Ohio
-
Cincinnati、Ohio、アメリカ、45229
- Swedish Orphan Biovitrum Research Site
-
-
Pennsylvania
-
Philadelphia、Pennsylvania、アメリカ、19104
- Swedish Orphan Biovitrum Research Site
-
-
-
-
-
Milan、イタリア
- Swedish Orphan Biovitrum Research Site
-
Rome、イタリア
- Swedish Orphan Biovitrum Research Site
-
-
-
-
-
Utrecht、オランダ
- Swedish Orphan Biovitrum Research Site
-
-
-
-
-
Calgary、カナダ
- Swedish Orphan Biovitrum Research Site
-
-
-
-
-
Barcelona、スペイン
- Swedish Orphan Biovitrum Research Site
-
-
-
-
-
Heidelberg、ドイツ
- Swedish Orphan Biovitrum Research Site
-
-
-
-
-
Paris、フランス
- Swedish Orphan Biovitrum Research Site
-
-
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
The study population will include patients with Still's disease who were diagnosed with MAS that was refractory to GC treatment.
説明
Inclusion Criteria:
- Age >6 months and ≤80 years at the beginning of the index MAS episode.
- Diagnosis of Still's disease (sJIA (systemic Juvenile Idiopathic Arthritis) or AOSD (Adult Onset Stills Disease) diagnosis).
- Diagnosis of MAS according to treating physician in the medical record.
- Patients who have received at least 3 consecutive days of GC after diagnosis of MAS and/or are judged by the Investigator to be refractory to GC due to clinical worsening of patient's condition .
- The onset of the index MAS episode occurred between 01 January 2012 and 30 September 2022.
Exclusion Criteria:
- A diagnosis of primary Hemophagocytic Lymphohistiocytosis (HLH) prior to the beginning of the index MAS episode.
- Confirmed malignancy prior to the beginning of the index MAS episode.
- Patient treated with any investigational product as a part of clinical trial during the index MAS episode.
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：OTHER
- 時間の展望：RETROSPECTIVE
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 |
|---|---|---|
|
Overall survival
時間枠：1 year
|
The proportion of patients alive after 1-year of follow-up |
1 year
|
|
Time to laboratory value normalization
時間枠：From Index date up to 26 weeks
|
Time to normalization for the key laboratory values |
From Index date up to 26 weeks
|
|
Time to Macrophage Activation Syndrome (MAS) laboratory remission
時間枠：From Index date up to 26 weeks
|
Time to normalization for all key laboratory values |
From Index date up to 26 weeks
|
|
Time to partial MAS laboratory remission
時間枠：From Index date up to 26 weeks
|
Time to normalization for at least 3 key laboratory values |
From Index date up to 26 weeks
|
|
Time to tapering of Glucocorticoids (GCs)
時間枠：from index date to the last of 7 consecutive days
|
From index date to the last of 7 consecutive days receiving ≤1 mg/kg/day of prednisone (PDN). |
from index date to the last of 7 consecutive days
|
|
Number of recurrent MAS episodes
時間枠：through study completion
|
Occurring any time after the end of the data collection period for the index MAS episode |
through study completion
|
|
Administration of organ support care
時間枠：from the index date until either the end of hospitalization or 26 weeks, whichever occurs later
|
Proportion of patients receiving hemodialysis, assisted ventilation, cardiac support, or inotropic drugs |
from the index date until either the end of hospitalization or 26 weeks, whichever occurs later
|
|
Characteristics of MAS treatment
時間枠：for MAS episodes occurring during the data collection period
|
description of treatment patterns of MAS-related treatments for both index and recurrent MAS episodes. |
for MAS episodes occurring during the data collection period
|
|
Clinical signs
時間枠：At index date and 8 weeks after index date or at the earliest time this data becomes available between 8 and 12 weeks
|
No of MAS symptoms (Fever, Skin rash, Hemorrhagic manifestations, Evidence of CNS involvement) present |
At index date and 8 weeks after index date or at the earliest time this data becomes available between 8 and 12 weeks
|
二次結果の測定
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
|
Time to hospital discharge
時間枠：Up to 26 weeks
|
Defined as time from the index date to discharge from the hospital |
Up to 26 weeks
|
|
Time to intensive care unit (ICU) discharge
時間枠：Up to 26 weeks
|
For patients who are admitted to the ICU during the index MAS episode, this is defined as time from index date or ICU admission (whichever comes later) to ICU discharge |
Up to 26 weeks
|
|
Duration of Clinical Response
時間枠：Until week 26
|
defined as the time (in days) from the date of first documented MAS laboratory remission until the date of first documented occurrence of a new MAS episode as per investigator's assessment. |
Until week 26
|
協力者と研究者
スポンサー
捜査官
- スタディディレクター：Uwe Ullman, MD、Swedish Orphan Biovitrum AG Riehenring 182, 4058 Basel, Switzerland
研究記録日
主要日程の研究
研究開始 (実際)
一次修了 (実際)
研究の完了 (実際)
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
本研究に関する用語
追加の関連 MeSH 用語
その他の研究ID番号
- Sobi.Emapalumab-105
医薬品およびデバイス情報、研究文書
米国FDA規制医薬品の研究
米国FDA規制機器製品の研究
米国で製造され、米国から輸出された製品。
この情報は、Web サイト clinicaltrials.gov から変更なしで直接取得したものです。研究の詳細を変更、削除、または更新するリクエストがある場合は、register@clinicaltrials.gov。 までご連絡ください。 clinicaltrials.gov に変更が加えられるとすぐに、ウェブサイトでも自動的に更新されます。