Assessment of Macrophage Activation syndromE in STill's Disease: Retrospective Chart Analysis of Patient HistorY, Symptom Resolution and Treatment Characteristics
Trial Snapshot
- Phase
- Not Applicable
- Status
- Completed
- Sponsor
- Enrollment
- 75
- Locations
- 1
- Primary Endpoint
- Time to Macrophage Activation Syndrome (MAS) laboratory remission
Study Overview
Brief Summary
Assessment of Macrophage activation syndrome in STill's disease: retrospective chart analysis of patient History, Symptom resolution and Treatment characteristics
Detailed Description
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.
Study Design
- Study Type
- Observational
- Observational Model
- Other
- Time Perspective
- Retrospective
Eligibility Criteria
- Ages
- 6 Months to 80 Years (Child, Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
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.
Outcomes
Primary Outcomes
Time to Macrophage Activation Syndrome (MAS) laboratory remission
Time Frame: From Index date up to 26 weeks
Time to normalization for all key laboratory values
Time to partial MAS laboratory remission
Time Frame: From Index date up to 26 weeks
Time to normalization for at least 3 key laboratory values
Clinical signs
Time Frame: 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
Number of recurrent MAS episodes
Time Frame: through study completion
Occurring any time after the end of the data collection period for the index MAS episode
Overall survival
Time Frame: 1 year
The proportion of patients alive after 1-year of follow-up
Administration of organ support care
Time Frame: 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
Time to laboratory value normalization
Time Frame: From Index date up to 26 weeks
Time to normalization for the key laboratory values
Time to tapering of Glucocorticoids (GCs)
Time Frame: 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).
Characteristics of MAS treatment
Time Frame: for MAS episodes occurring during the data collection period
description of treatment patterns of MAS-related treatments for both index and recurrent MAS episodes.
Secondary Outcomes
- Duration of Clinical Response(Until week 26)
- Time to intensive care unit (ICU) discharge(Up to 26 weeks)
- Time to hospital discharge(Up to 26 weeks)