Assessment of Macrophage Activation syndromE in STill's Disease: Retrospective Chart Analysis of Patient hIstory, Symptom Resolution and TreAtment Characteristics in Italy (AMETISTA)
Trial Snapshot
- Phase
- Not Applicable
- Status
- Completed
- Sponsor
- Enrollment
- 32
- Locations
- 2
- Primary Endpoint
- Overall survival
Study Overview
Brief Summary
Assessment of macrophage activation syndrome in Still's disease:
retrospective chart analysis of patient history, symptom resolution and treatment characteristics in Italy
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. The study will be conducted in Italy.
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 or AOSD 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
- •According to local regulations, waivers of consent will be sought for study participants from the appropriate regulatory authorities and/or the independent ethics committee (IEC)/institutional review board (IRB). o For patients not covered by waivers of consent, signed, and dated informed consent provided by the patient, or the patient's legally authorized representative(s) for patients under the legal age (with patient assent, as applicable) or who have died, should be obtained before any study-related activities are undertaken.
Exclusion Criteria
- •A diagnosis of primary 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
Overall survival
Time Frame: from 2012 to 2022 (data abdstraction period)
defined as the survival time of patients from the index date
Time to laboratory value normalization
Time Frame: up to 24 weeks
defined as the time to normalization for key laboratory values
Time to MAS laboratory remission
Time Frame: up to 24 weeks
defined as the time from index date to the last date of normalization of key laboratory assessments
Time to partial MAS laboratory remission
Time Frame: up to 24 weeks
defined as the time from index date to the last date of normalization of at least 3 of the key laboratory assessments
Time to tapering of GCs
Time Frame: from index date to the last of 7 consecutive days receiving ≤1 mg/kg/day of prednisone (PDN) equivalent dose
defined as the time from index date to the last of 7 consecutive days receiving ≤1 mg/kg/day of prednisone (PDN) equivalent dose
Number of recurrent MAS episodes
Time Frame: from hospital discharge or 26-weeks from the index date, whichever occurs later, until the end of study data collection]
defined as the number of MAS episodes occurring any time after the end of the data collection period for the index MAS episode (hospital discharge or 26-weeks from the index date, whichever occurs later) until the end of study data collection
Administration of organ support care
Time Frame: from the index date until either the hospital discharge or 26 weeks, whichever occurs later
defined as the proportion of patients receiving hemodialysis, assisted ventilation, cardiac support, or inotropic drugs at any time from the index date until either the hospital discharge or 26 weeks, whichever occurs later
Characteristics of MAS treatment
Time Frame: up to 24 weeks
Concomitant medications
Clinical signs
Time Frame: at index date and 8 weeks after index date (allowed time window 6 to 12 weeks)
Patients experiencing MAS symptoms if present (documentation in free text)
Secondary Outcomes
- Time to hospital discharge(up to 24 weeks)
- Time to intensive care unit (ICU) discharge(up to 24 weeks)
- Normalization of key laboratory values(Week 8 after index date (allowed time window 6 to 12 weeks))
- MAS laboratory remission(at Week 8 after index date (allowed time window 6 to 12 weeks).)
- Partial MAS laboratory remission(at Week 8 after index date (allowed time window 6 to 12 weeks))
- Duration of Clinical Response(up to 24 weeks)