Assessment of Macrophage Activation syndromE in STill's Disease in Italy

Swedish Orphan Biovitrum32 enrolled

Overview

Assessment of macrophage activation syndrome in Still's disease: retrospective chart analysis of patient history, symptom resolution and treatment characteristics in Italy

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. The study will be conducted in Italy.

Study Type

OBSERVATIONAL

Enrollment

Conditions

Macrophage Activation Syndrome (MAS)Still Disease, Juvenile Onset Still's Disease, Adult-Onset

Eligibility

Sex: ALLMin age: 6 MonthsMax age: 80 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Age \>6 months and ≤80 years at the beginning of the index MAS episode. * Diagnosis of Still's disease (sJIA or AOSD 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. * According to local regulations, waivers of consent will be sought for study participants from the appropriate regulatory authorities and/or the independent ethics committee (IEC)/institutional review board (IRB). o For patients not covered by waivers of consent, signed, and dated informed consent provided by the patient, or the patient's legally authorized representative(s) for patients under the legal age (with patient assent, as applicable) or who have died, should be obtained before any study-related activities are undertaken. Exclusion Criteria: * A diagnosis of primary 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.

Locations (2)

Fondazione IRCCS CA' Granda Ospedale Maggiore Policlinico

Milan, Italy

Ospedale Pediatrico Bambin Gesù

Rome, Italy

Outcomes

Primary Outcomes

Overall survival

defined as the survival time of patients from the index date

Time frame: from 2012 to 2022 (data abdstraction period)

Time to laboratory value normalization

defined as the time to normalization for key laboratory values

Time frame: up to 24 weeks

Time to MAS laboratory remission

defined as the time from index date to the last date of normalization of key laboratory assessments

Time frame: up to 24 weeks

Time to partial MAS laboratory remission

defined as the time from index date to the last date of normalization of at least 3 of the key laboratory assessments

Time frame: up to 24 weeks

Time to tapering of GCs

defined as the time from index date to the last of 7 consecutive days receiving ≤1 mg/kg/day of prednisone (PDN) equivalent dose

Time frame: from index date to the last of 7 consecutive days receiving ≤1 mg/kg/day of prednisone (PDN) equivalent dose

Number of recurrent MAS episodes

defined as the number of MAS episodes occurring any time after the end of the data collection period for the index MAS episode (hospital discharge or 26-weeks from the index date, whichever occurs later) until the end of study data collection

Time frame: from hospital discharge or 26-weeks from the index date, whichever occurs later, until the end of study data collection]

Administration of organ support care

defined as the proportion of patients receiving hemodialysis, assisted ventilation, cardiac support, or inotropic drugs at any time from the index date until either the hospital discharge or 26 weeks, whichever occurs later

Data from ClinicalTrials.gov

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Secondary Outcomes

Time to hospital discharge

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

Time frame: up to 24 weeks

Time to intensive care unit (ICU) discharge

defined as time from index date to ICU discharge

Time frame: up to 24 weeks

Normalization of key laboratory values

Normalization of key laboratory values at Week 8 after index date (allowed time window 6 to 12 weeks)

Time frame: Week 8 after index date (allowed time window 6 to 12 weeks)

MAS laboratory remission

Normalization of Key laboratory values at Week 8 after index date (allowed time window 6 to 12 weeks).

Time frame: at Week 8 after index date (allowed time window 6 to 12 weeks).

Partial MAS laboratory remission

Normalization of at least 3 key laboratory values at Week 8 after index date (allowed time window 6 to 12 weeks)

Time frame: at Week 8 after index date (allowed time window 6 to 12 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.

Time frame: up to 24 weeks