STill 질환에서 대식세포 활성화 증후군의 평가 (AMETHYST)
STill 질환의 대식세포 활성화 증후군 평가: 환자 병력, 증상 해결 및 치료 특성에 대한 후향적 차트 분석
Assessment of Macrophage activation syndrome in STill's disease: retrospective chart analysis of patient History, Symptom resolution and Treatment characteristics
연구 개요
상세 설명
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.
연구 유형
등록 (실제)
연락처 및 위치
연구 장소
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Utrecht, 네덜란드
- Swedish Orphan Biovitrum Research Site
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Heidelberg, 독일
- Swedish Orphan Biovitrum Research Site
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California
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Los Angeles, California, 미국, 90095
- Swedish Orphan Biovitrum Research Site
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Georgia
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Atlanta, Georgia, 미국, 30329
- Swedish Orphan Biovitrum Research Site
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Ohio
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Cincinnati, Ohio, 미국, 45229
- Swedish Orphan Biovitrum Research Site
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Pennsylvania
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Philadelphia, Pennsylvania, 미국, 19104
- Swedish Orphan Biovitrum Research Site
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Barcelona, 스페인
- Swedish Orphan Biovitrum Research Site
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Milan, 이탈리아
- Swedish Orphan Biovitrum Research Site
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Rome, 이탈리아
- Swedish Orphan Biovitrum Research Site
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Calgary, 캐나다
- Swedish Orphan Biovitrum Research Site
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Paris, 프랑스
- Swedish Orphan Biovitrum Research Site
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참여기준
자격 기준
공부할 수 있는 나이
건강한 자원 봉사자를 받아들입니다
연구 대상 성별
샘플링 방법
연구 인구
The study population will include patients with Still's disease who were diagnosed with MAS that was refractory to GC treatment.
설명
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.
공부 계획
연구는 어떻게 설계됩니까?
디자인 세부사항
- 관찰 모델: OTHER
- 시간 관점: RETROSPECTIVE
연구는 무엇을 측정합니까?
주요 결과 측정
결과 측정 |
측정값 설명 |
기간 |
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Overall survival
기간: 1 year
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The proportion of patients alive after 1-year of follow-up |
1 year
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Time to laboratory value normalization
기간: From Index date up to 26 weeks
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Time to normalization for the key laboratory values |
From Index date up to 26 weeks
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Time to Macrophage Activation Syndrome (MAS) laboratory remission
기간: From Index date up to 26 weeks
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Time to normalization for all key laboratory values |
From Index date up to 26 weeks
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Time to partial MAS laboratory remission
기간: From Index date up to 26 weeks
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Time to normalization for at least 3 key laboratory values |
From Index date up to 26 weeks
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Time to tapering of Glucocorticoids (GCs)
기간: from index date to the last of 7 consecutive days
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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
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Number of recurrent MAS episodes
기간: through study completion
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Occurring any time after the end of the data collection period for the index MAS episode |
through study completion
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Administration of organ support care
기간: from the index date until either the end of hospitalization or 26 weeks, whichever occurs later
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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
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Characteristics of MAS treatment
기간: for MAS episodes occurring during the data collection period
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description of treatment patterns of MAS-related treatments for both index and recurrent MAS episodes. |
for MAS episodes occurring during the data collection period
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Clinical signs
기간: At index date and 8 weeks after index date or at the earliest time this data becomes available between 8 and 12 weeks
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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
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2차 결과 측정
결과 측정 |
측정값 설명 |
기간 |
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Time to hospital discharge
기간: Up to 26 weeks
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Defined as time from the index date to discharge from the hospital |
Up to 26 weeks
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Time to intensive care unit (ICU) discharge
기간: Up to 26 weeks
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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
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Duration of Clinical Response
기간: Until week 26
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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
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공동 작업자 및 조사자
수사관
- 연구 책임자: Uwe Ullman, MD, Swedish Orphan Biovitrum AG Riehenring 182, 4058 Basel, Switzerland
연구 기록 날짜
연구 주요 날짜
연구 시작 (실제)
기본 완료 (실제)
연구 완료 (실제)
연구 등록 날짜
최초 제출
QC 기준을 충족하는 최초 제출
처음 게시됨 (실제)
연구 기록 업데이트
마지막 업데이트 게시됨 (실제)
QC 기준을 충족하는 마지막 업데이트 제출
마지막으로 확인됨
추가 정보
이 연구와 관련된 용어
추가 관련 MeSH 약관
기타 연구 ID 번호
- Sobi.Emapalumab-105
약물 및 장치 정보, 연구 문서
미국 FDA 규제 의약품 연구
미국 FDA 규제 기기 제품 연구
미국에서 제조되어 미국에서 수출되는 제품
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