Macrophage Activation Syndrome, Still's Disease, Adult-Onset, Stills Disease, Juvenile-Onset
Conditions
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.
Interventions
OTHERChart review
Retrospective Chart Review
Sponsors
Swedish Orphan Biovitrum
Study design
Observational model
OTHER
Time perspective
RETROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
6 Months to 80 Years
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Clinical signs | 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 | through study completion | Occurring any time after the end of the data collection period for the index MAS episode |
| Administration of organ support care | 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 |
| Characteristics of MAS treatment | for MAS episodes occurring during the data collection period | description of treatment patterns of MAS-related treatments for both index and recurrent MAS episodes. |
| Overall survival | 1 year | The proportion of patients alive after 1-year of follow-up |
| Time to laboratory value normalization | From Index date up to 26 weeks | Time to normalization for the key laboratory values |
| Time to Macrophage Activation Syndrome (MAS) laboratory remission | From Index date up to 26 weeks | Time to normalization for all key laboratory values |
| Time to partial MAS laboratory remission | From Index date up to 26 weeks | Time to normalization for at least 3 key laboratory values |
| Time to tapering of Glucocorticoids (GCs) | 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). |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Time to intensive care unit (ICU) discharge | 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 |
| Duration of Clinical Response | 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. |
| Time to hospital discharge | Up to 26 weeks | Defined as time from the index date to discharge from the hospital |
Countries
Canada, France, Germany, Italy, Netherlands, Spain, United States
Outcome results
None listed