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Completed

NCT Number: NCT06405152

Assessment of Macrophage Activation syndromE in STill's Disease

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

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Key information

Conditions

Macrophage Activation Syndrome Arthritis Arthritis, Juvenile Arthritis, Rheumatoid Autoimmune Diseases Connective Tissue Diseases Immune System Diseases Immunoproliferative Disorders Joint Diseases Lymphoproliferative Disorders Musculoskeletal Diseases Rheumatic Diseases Skin and Connective Tissue Diseases Still's Disease, Adult-Onset Stills Disease, Juvenile-Onset

Age range

6 month–80 year

Sex eligibility

All sexes

Study type

Observational

Primary location

Swedish Orphan Biovitrum Research Site, Calgary, Canada

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About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Chart review

Other

Retrospective Chart Review

Primary outcomes

  1. Overall survival

    Time frame: 1 year

    The proportion of patients alive after 1-year of follow-up

  2. Time to laboratory value normalization

    Time frame: From Index date up to 26 weeks

    Time to normalization for the key laboratory values

  3. Time to Macrophage Activation Syndrome (MAS) laboratory remission

    Time frame: From Index date up to 26 weeks

    Time to normalization for all key laboratory values

  4. Time to partial MAS laboratory remission

    Time frame: From Index date up to 26 weeks

    Time to normalization for at least 3 key laboratory values

  5. Time to tapering of Glucocorticoids (GCs)

    Time frame: 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).

  6. Number of recurrent MAS episodes

    Time frame: through study completion

    Occurring any time after the end of the data collection period for the index MAS episode

  7. Administration of organ support care

    Time frame: 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

  8. Characteristics of MAS treatment

    Time frame: for MAS episodes occurring during the data collection period

    description of treatment patterns of MAS-related treatments for both index and recurrent MAS episodes.

  9. Clinical signs

    Time frame: 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

Secondary outcomes

  1. Time to hospital discharge

    Time frame: Up to 26 weeks

    Defined as time from the index date to discharge from the hospital

  2. Time to intensive care unit (ICU) discharge

    Time frame: 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

  3. Duration of Clinical Response

    Time frame: 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.

Sponsors and collaborators

Lead sponsor

Swedish Orphan Biovitrum

Industry

Registry information

Official study title

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

Acronym: AMETHYST

Important dates

Study start
2023
Primary completion
2024
Study completion
2024
First posted
May 8, 2024
Registry last updated
Oct 21, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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