Clinical trial NCT06405152: Assessment of Macrophage Activation syndromE in STill's Disease
Official title: Assessment of Macrophage Activation syndromE in STill's Disease: Retrospective Chart Analysis of Patient HistorY, Symptom Resolution and Treatment Characteristics
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Macrophage Activation Syndrome
Still's Disease, Adult-Onset
Stills Disease, Juvenile-Onset
Lead sponsor: Swedish Orphan Biovitrum
Sponsor class: INDUSTRY
First posted: 2024-05-08
Enrollment: 75
Sex: ALL
Minimum age (years): 6
Maximum age (years): 80
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: OTHER
Time perspective: RETROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: The study population will include patients with Still's disease who were diagnosed with MAS that was refractory to GC treatment.

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.

Eligibility criteria: 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.

Primary outcomes:
measure: Overall survival
timeFrame: 1 year
measure: Time to laboratory value normalization
timeFrame: From Index date up to 26 weeks
measure: Time to Macrophage Activation Syndrome (MAS) laboratory remission
timeFrame: From Index date up to 26 weeks
measure: Time to partial MAS laboratory remission
timeFrame: From Index date up to 26 weeks
measure: Time to tapering of Glucocorticoids (GCs)
timeFrame: from index date to the last of 7 consecutive days
measure: Number of recurrent MAS episodes
timeFrame: through study completion
measure: Administration of organ support care
timeFrame: from the index date until either the end of hospitalization or 26 weeks, whichever occurs later
measure: Characteristics of MAS treatment
timeFrame: for MAS episodes occurring during the data collection period
measure: Clinical signs
timeFrame: At index date and 8 weeks after index date or at the earliest time this data becomes available between 8 and 12 weeks
Secondary outcomes:
measure: Time to hospital discharge
timeFrame: Up to 26 weeks
measure: Time to intensive care unit (ICU) discharge
timeFrame: Up to 26 weeks
measure: Duration of Clinical Response
timeFrame: Until week 26
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: Swedish Orphan Biovitrum Research Site
state: California
city: Atlanta
country: United States
facility: Swedish Orphan Biovitrum Research Site
state: Georgia
city: Cincinnati
country: United States
facility: Swedish Orphan Biovitrum Research Site
state: Ohio
city: Philadelphia
country: United States
facility: Swedish Orphan Biovitrum Research Site
state: Pennsylvania
city: Calgary
country: Canada
facility: Swedish Orphan Biovitrum Research Site
city: Paris
country: France
facility: Swedish Orphan Biovitrum Research Site
city: Heidelberg
country: Germany
facility: Swedish Orphan Biovitrum Research Site
city: Milan
country: Italy
facility: Swedish Orphan Biovitrum Research Site
city: Rome
country: Italy
facility: Swedish Orphan Biovitrum Research Site
city: Utrecht
country: Netherlands
facility: Swedish Orphan Biovitrum Research Site
city: Barcelona
country: Spain
facility: Swedish Orphan Biovitrum Research Site
Study officials:
affiliation: Swedish Orphan Biovitrum AG Riehenring 182, 4058 Basel, Switzerland
name: Uwe Ullman, MD
role: STUDY_DIRECTOR
References: