Assessment of Macrophage Activation syndromE in STill's Disease

Completed

• Conditions: Stills Disease, Juvenile-Onset; Still's Disease, Adult-Onset; Macrophage Activation Syndrome

• Registration Number: NCT06405152
• Lead Sponsor: Swedish Orphan Biovitrum

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 Timeline

• Study Start: 2023-09-27
• Study First Submitted: 2024-04-09
• Study First Submitted QC: 2024-05-07
• Study First Posted: 2024-05-08
• Primary Completion: 2024-09-26
• Study Completion: 2024-09-26
• Status Verified: 2024-10
• Last Update Submitted: 2024-10-18
• Last Update Posted: 2024-10-21

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 75

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.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
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
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
Overall survival	1 year	The proportion of patients alive after 1-year of follow-up
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
Time to laboratory value normalization	From Index date up to 26 weeks	Time to normalization for the 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).
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.

Secondary Outcome Measures

Name	Time	Method
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 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
Time to hospital discharge	Up to 26 weeks	Defined as time from the index date to discharge from the hospital

Trial Locations

Locations (1)

Swedish Orphan Biovitrum Research Site; 🇪🇸 Barcelona, Spain