Bewertung des Makrophagenaktivierungssyndroms bei Morbus STill (AMETHYST)
Beurteilung des Makrophagenaktivierungssyndroms bei Morbus STill: Retrospektive Diagrammanalyse der Patientengeschichte, Symptomauflösung und Behandlungsmerkmale
Assessment of Macrophage activation syndrome in STill's disease: retrospective chart analysis of patient History, Symptom resolution and Treatment characteristics
Studienübersicht
Status
Bedingungen
Intervention / Behandlung
Detaillierte Beschreibung
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.
Studientyp
Einschreibung (Tatsächlich)
Kontakte und Standorte
Studienorte
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Heidelberg, Deutschland
- Swedish Orphan Biovitrum Research Site
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Paris, Frankreich
- Swedish Orphan Biovitrum Research Site
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Milan, Italien
- Swedish Orphan Biovitrum Research Site
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Rome, Italien
- Swedish Orphan Biovitrum Research Site
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Calgary, Kanada
- Swedish Orphan Biovitrum Research Site
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Utrecht, Niederlande
- Swedish Orphan Biovitrum Research Site
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Barcelona, Spanien
- Swedish Orphan Biovitrum Research Site
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California
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Los Angeles, California, Vereinigte Staaten, 90095
- Swedish Orphan Biovitrum Research Site
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Georgia
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Atlanta, Georgia, Vereinigte Staaten, 30329
- Swedish Orphan Biovitrum Research Site
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Ohio
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Cincinnati, Ohio, Vereinigte Staaten, 45229
- Swedish Orphan Biovitrum Research Site
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Pennsylvania
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Philadelphia, Pennsylvania, Vereinigte Staaten, 19104
- Swedish Orphan Biovitrum Research Site
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Teilnahmekriterien
Zulassungskriterien
Studienberechtigtes Alter
Akzeptiert gesunde Freiwillige
Studienberechtigte Geschlechter
Probenahmeverfahren
Studienpopulation
The study population will include patients with Still's disease who were diagnosed with MAS that was refractory to GC treatment.
Beschreibung
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.
Studienplan
Wie ist die Studie aufgebaut?
Designdetails
- Beobachtungsmodelle: OTHER
- Zeitperspektiven: RETROSPECTIVE
Was misst die Studie?
Primäre Ergebnismessungen
Ergebnis Maßnahme |
Maßnahmenbeschreibung |
Zeitfenster |
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Overall survival
Zeitfenster: 1 year
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The proportion of patients alive after 1-year of follow-up |
1 year
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Time to laboratory value normalization
Zeitfenster: From Index date up to 26 weeks
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Time to normalization for the key laboratory values |
From Index date up to 26 weeks
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Time to Macrophage Activation Syndrome (MAS) laboratory remission
Zeitfenster: From Index date up to 26 weeks
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Time to normalization for all key laboratory values |
From Index date up to 26 weeks
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Time to partial MAS laboratory remission
Zeitfenster: From Index date up to 26 weeks
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Time to normalization for at least 3 key laboratory values |
From Index date up to 26 weeks
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Time to tapering of Glucocorticoids (GCs)
Zeitfenster: from index date to the last of 7 consecutive days
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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
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Number of recurrent MAS episodes
Zeitfenster: through study completion
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Occurring any time after the end of the data collection period for the index MAS episode |
through study completion
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Administration of organ support care
Zeitfenster: from the index date until either the end of hospitalization or 26 weeks, whichever occurs later
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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
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Characteristics of MAS treatment
Zeitfenster: for MAS episodes occurring during the data collection period
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description of treatment patterns of MAS-related treatments for both index and recurrent MAS episodes. |
for MAS episodes occurring during the data collection period
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Clinical signs
Zeitfenster: At index date and 8 weeks after index date or at the earliest time this data becomes available between 8 and 12 weeks
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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
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Sekundäre Ergebnismessungen
Ergebnis Maßnahme |
Maßnahmenbeschreibung |
Zeitfenster |
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Time to hospital discharge
Zeitfenster: Up to 26 weeks
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Defined as time from the index date to discharge from the hospital |
Up to 26 weeks
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Time to intensive care unit (ICU) discharge
Zeitfenster: Up to 26 weeks
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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
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Duration of Clinical Response
Zeitfenster: Until week 26
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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
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Mitarbeiter und Ermittler
Sponsor
Ermittler
- Studienleiter: Uwe Ullman, MD, Swedish Orphan Biovitrum AG Riehenring 182, 4058 Basel, Switzerland
Studienaufzeichnungsdaten
Haupttermine studieren
Studienbeginn (Tatsächlich)
Primärer Abschluss (Tatsächlich)
Studienabschluss (Tatsächlich)
Studienanmeldedaten
Zuerst eingereicht
Zuerst eingereicht, das die QC-Kriterien erfüllt hat
Zuerst gepostet (Tatsächlich)
Studienaufzeichnungsaktualisierungen
Letztes Update gepostet (Tatsächlich)
Letztes eingereichtes Update, das die QC-Kriterien erfüllt
Zuletzt verifiziert
Mehr Informationen
Begriffe im Zusammenhang mit dieser Studie
Zusätzliche relevante MeSH-Bedingungen
- Erkrankungen des Bewegungsapparates
- Arthritis
- Gelenkerkrankungen
- Rheumatische Erkrankungen
- Bindegewebserkrankungen
- Autoimmunerkrankungen
- Erkrankungen des Immunsystems
- Lymphoproliferative Erkrankungen
- Immunproliferative Erkrankungen
- Arthritis, Rheuma
- Haut- und Bindegewebserkrankungen
- Arthritis, juvenil
- Makrophagenaktivierungssyndrom
- Morbus Still, Beginn im Erwachsenenalter
Andere Studien-ID-Nummern
- Sobi.Emapalumab-105
Arzneimittel- und Geräteinformationen, Studienunterlagen
Studiert ein von der US-amerikanischen FDA reguliertes Arzneimittelprodukt
Studiert ein von der US-amerikanischen FDA reguliertes Geräteprodukt
Produkt, das in den USA hergestellt und aus den USA exportiert wird
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