Evaluación del síndrome de activación de macrófagos en la enfermedad de Still (AMETHYST)
Evaluación del síndrome de activación de macrófagos en la enfermedad de Still: análisis gráfico retrospectivo del historial del paciente, resolución de los síntomas y características del tratamiento
Assessment of Macrophage activation syndrome in STill's disease: retrospective chart analysis of patient History, Symptom resolution and Treatment characteristics
Descripción general del estudio
Estado
Condiciones
Intervención / Tratamiento
Descripción detallada
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.
Tipo de estudio
Inscripción (Actual)
Contactos y Ubicaciones
Ubicaciones de estudio
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Heidelberg, Alemania
- Swedish Orphan Biovitrum Research Site
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Calgary, Canadá
- Swedish Orphan Biovitrum Research Site
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Barcelona, España
- Swedish Orphan Biovitrum Research Site
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California
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Los Angeles, California, Estados Unidos, 90095
- Swedish Orphan Biovitrum Research Site
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Georgia
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Atlanta, Georgia, Estados Unidos, 30329
- Swedish Orphan Biovitrum Research Site
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Ohio
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Cincinnati, Ohio, Estados Unidos, 45229
- Swedish Orphan Biovitrum Research Site
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Pennsylvania
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Philadelphia, Pennsylvania, Estados Unidos, 19104
- Swedish Orphan Biovitrum Research Site
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Paris, Francia
- Swedish Orphan Biovitrum Research Site
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Milan, Italia
- Swedish Orphan Biovitrum Research Site
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Rome, Italia
- Swedish Orphan Biovitrum Research Site
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Utrecht, Países Bajos
- Swedish Orphan Biovitrum Research Site
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Criterios de participación
Criterio de elegibilidad
Edades elegibles para estudiar
Acepta Voluntarios Saludables
Géneros elegibles para el estudio
Método de muestreo
Población de estudio
The study population will include patients with Still's disease who were diagnosed with MAS that was refractory to GC treatment.
Descripción
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.
Plan de estudios
¿Cómo está diseñado el estudio?
Detalles de diseño
- Modelos observacionales: OTHER
- Perspectivas temporales: RETROSPECTIVE
¿Qué mide el estudio?
Medidas de resultado primarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
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Overall survival
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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)
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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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Medidas de resultado secundarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
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Time to hospital discharge
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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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Colaboradores e Investigadores
Patrocinador
Investigadores
- Director de estudio: Uwe Ullman, MD, Swedish Orphan Biovitrum AG Riehenring 182, 4058 Basel, Switzerland
Fechas de registro del estudio
Fechas importantes del estudio
Inicio del estudio (Actual)
Finalización primaria (Actual)
Finalización del estudio (Actual)
Fechas de registro del estudio
Enviado por primera vez
Primero enviado que cumplió con los criterios de control de calidad
Publicado por primera vez (Actual)
Actualizaciones de registros de estudio
Última actualización publicada (Actual)
Última actualización enviada que cumplió con los criterios de control de calidad
Última verificación
Más información
Términos relacionados con este estudio
Términos MeSH relevantes adicionales
- Enfermedades musculoesqueléticas
- Artritis
- Enfermedades Articulares
- Enfermedades reumáticas
- Enfermedades del tejido conectivo
- Enfermedades autoinmunes
- Enfermedades del sistema inmunológico
- Trastornos linfoproliferativos
- Trastornos inmunoproliferativos
- Artritis Reumatoide
- Enfermedades de la piel y del tejido conectivo
- Artritis Juvenil
- Síndrome de activación de macrófagos
- Enfermedad de Still, inicio en adultos
Otros números de identificación del estudio
- Sobi.Emapalumab-105
Información sobre medicamentos y dispositivos, documentos del estudio
Estudia un producto farmacéutico regulado por la FDA de EE. UU.
Estudia un producto de dispositivo regulado por la FDA de EE. UU.
producto fabricado y exportado desde los EE. UU.
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