Assessment of Macrophage Activation syndromE in STill's Disease (AMETHYST)

Assessment of Macrophage Activation syndromE in STill's Disease: Retrospective Chart Analysis of Patient HistorY, Symptom Resolution and Treatment Characteristics

Assessment of Macrophage activation syndrome in STill's disease: retrospective chart analysis of patient History, Symptom resolution and Treatment characteristics

Study Overview

• Status: Completed
• Conditions: Still's Disease, Adult-Onset; Macrophage Activation Syndrome; Stills Disease, Juvenile-Onset
• Intervention / Treatment: Other: Chart review

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 Type: Observational
• Enrollment (Actual): 75

Contacts and Locations

Study Locations

• Canada
  • Calgary, Canada
    • Swedish Orphan Biovitrum Research site
• France
  • Paris, France
    • Swedish Orphan Biovitrum Research site
• Germany
  • Heidelberg, Germany
    • Swedish Orphan Biovitrum Research site
• Italy
  • Milan, Italy
    • Swedish Orphan Biovitrum Research site
  • Rome, Italy
    • Swedish Orphan Biovitrum Research site
• Netherlands
  • Utrecht, Netherlands
    • Swedish Orphan Biovitrum Research site
• Spain
  • Barcelona, Spain
    • Swedish Orphan Biovitrum Research site
• United States
  • California
    • Los Angeles, California, United States, 90095
      • Swedish Orphan Biovitrum Research site
  • Georgia
    • Atlanta, Georgia, United States, 30329
      • Swedish Orphan Biovitrum Research site
  • Ohio
    • Cincinnati, Ohio, United States, 45229
      • Swedish Orphan Biovitrum Research site
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19104
      • Swedish Orphan Biovitrum Research site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

• 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.

Description

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 Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Overall survival	The proportion of patients alive after 1-year of follow-up	1 year
Time to laboratory value normalization	Time to normalization for the key laboratory values	From Index date up to 26 weeks
Time to Macrophage Activation Syndrome (MAS) laboratory remission	Time to normalization for all key laboratory values	From Index date up to 26 weeks
Time to partial MAS laboratory remission	Time to normalization for at least 3 key laboratory values	From Index date up to 26 weeks
Time to tapering of Glucocorticoids (GCs)	From index date to the last of 7 consecutive days receiving ≤1 mg/kg/day of prednisone (PDN).	from index date to the last of 7 consecutive days
Number of recurrent MAS episodes	Occurring any time after the end of the data collection period for the index MAS episode	through study completion
Administration of organ support care	Proportion of patients receiving hemodialysis, assisted ventilation, cardiac support, or inotropic drugs	from the index date until either the end of hospitalization or 26 weeks, whichever occurs later
Characteristics of MAS treatment	description of treatment patterns of MAS-related treatments for both index and recurrent MAS episodes.	for MAS episodes occurring during the data collection period
Clinical signs	No of MAS symptoms (Fever, Skin rash, Hemorrhagic manifestations, Evidence of CNS involvement) present	At index date and 8 weeks after index date or at the earliest time this data becomes available between 8 and 12 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Time to hospital discharge	Defined as time from the index date to discharge from the hospital	Up to 26 weeks
Time to intensive care unit (ICU) discharge	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	Up to 26 weeks
Duration of Clinical Response	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.	Until week 26

Collaborators and Investigators

• Sponsor: Swedish Orphan Biovitrum
• Investigators: Study Director: Uwe Ullman, MD, Swedish Orphan Biovitrum AG Riehenring 182, 4058 Basel, Switzerland

Study record dates

Study Major Dates

• Study Start (Actual): September 27, 2023
• Primary Completion (Actual): September 26, 2024
• Study Completion (Actual): September 26, 2024

Study Registration Dates

• First Submitted: April 9, 2024
• First Submitted That Met QC Criteria: May 7, 2024
• First Posted (Actual): May 8, 2024

Study Record Updates

• Last Update Posted (Actual): October 21, 2024
• Last Update Submitted That Met QC Criteria: October 18, 2024
• Last Verified: October 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Immune System Diseases; Lymphoproliferative Disorders; Immunoproliferative Disorders; Autoimmune Diseases; Disease; Joint Diseases; Musculoskeletal Diseases; Rheumatic Diseases; Connective Tissue Diseases; Arthritis; Arthritis, Rheumatoid; Syndrome; Arthritis, Juvenile; Still's Disease, Adult-Onset; Macrophage Activation Syndrome
• Other Study ID Numbers: Sobi.Emapalumab-105

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: Yes