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Assessment of Macrophage activation syndrome in STill's disease: retrospective chart analysis of patient History, Symptom resolution and Treatment characteristics
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.
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Chart review | Other | Retrospective Chart Review |
| 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 |
| 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 |
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Inclusion Criteria:
Exclusion Criteria:
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The study population will include patients with Still's disease who were diagnosed with MAS that was refractory to GC treatment.
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| Name | Affiliation | Role |
|---|---|---|
| Uwe Ullman, MD | Swedish Orphan Biovitrum AG Riehenring 182, 4058 Basel, Switzerland | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Swedish Orphan Biovitrum Research Site | Los Angeles | California | 90095 | United States | ||
| Swedish Orphan Biovitrum Research Site |
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| ID | Term |
|---|---|
| D055501 | Macrophage Activation Syndrome |
| D016706 | Still's Disease, Adult-Onset |
| D001171 | Arthritis, Juvenile |
| ID | Term |
|---|---|
| D008232 | Lymphoproliferative Disorders |
| D007160 | Immunoproliferative Disorders |
| D007154 | Immune System Diseases |
| D001172 | Arthritis, Rheumatoid |
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| 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 |
| 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 |
| Atlanta |
| Georgia |
| 30329 |
| United States |
| Swedish Orphan Biovitrum Research Site | Cincinnati | Ohio | 45229 | United States |
| Swedish Orphan Biovitrum Research Site | Philadelphia | Pennsylvania | 19104 | United States |
| Swedish Orphan Biovitrum Research Site | Calgary | Canada |
| Swedish Orphan Biovitrum Research Site | Paris | France |
| Swedish Orphan Biovitrum Research Site | Heidelberg | Germany |
| Swedish Orphan Biovitrum Research Site | Milan | Italy |
| Swedish Orphan Biovitrum Research Site | Rome | Italy |
| Swedish Orphan Biovitrum Research Site | Utrecht | Netherlands |
| Swedish Orphan Biovitrum Research Site | Barcelona | Spain |
| D001168 | Arthritis |
| D007592 | Joint Diseases |
| D009140 | Musculoskeletal Diseases |
| D012216 | Rheumatic Diseases |
| D003240 | Connective Tissue Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D001327 | Autoimmune Diseases |