A Treatment Effectiveness Study Among SLE Patients Receiving Anifrolumab in Routine Clinical Practice

Study Overview
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Brief Summary
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Anifrolumab Study of Treatment Effectiveness in the Real World (ASTER) study will collect real world data to obtain a good understanding of the (sustained) clinical effect and patient quality of life outcomes among diagnosed SLE patients who initiate anifrolumab treatment. ASTER will generate critical real-world evidence on the benefits of adding anifrolumab to standard of care treatment for SLE in routine clinical practice, to inform physicians, payers and patients.

Detailed Description
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ASTER is a multi-country, single-arm, prospective, observational study. The study will be initiated on a country-by country basis following the commercial launch of anifrolumab. ASTER is a cohort study, with 1-year retrospective baseline data and 3 years of follow-up data. The minimum enrolment period is anticipated to be 18 months per country and will be extended if necessary to reach the overall study target. In general, patients will enter the study between the first anifrolumab prescription and infusion (index), with follow-up until death, loss to follow-up, patient discontinuing the study, or end of study period (whichever occurs first). Relevant clinical and patient-reported outcome (PRO) data collection for the entire follow-up will continue for patients who have discontinued anifrolumab during the study, unless the patients have withdrawn their consent for participation in the study. The study will use clinical assessments that are relevant for SLE-treating physicians in routine clinical practice, as well as introduce a specific measure for skin manifestations to affirm the potency of anifrolumab in treating SLE-related skin manifestations. The eCRFs will be accessed through secure web-based portals and will be used to ensure consistent data collection for each healthcare provider involved in this study. Electronic data collection will be the only method of data collection in this study.

Official Title
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ASTER: Anifrolumab Study for Treatment Effectiveness in the Real World Multi-National, Observational, Post-Launch Effectiveness Study Among SLE Patients Receiving Anifrolumab in Routine Clinical Practice

Conditions
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Systemic Lupus Erythematosus

Intervention / Treatment
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* Other: None (Observational study)


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Fulfilled the 2019 EULAR (European League Against Rheumatism)/ACR (American College of Rheumatology) criteria for SLE at the time of study entry. Prescribed anifrolumab for their SLE treatment for the first time, according to approved country-specific label. It is important to note that a physician decision to prescribe anifrolumab will need to occur prior to any study-related discussion. In countries where prescription reimbursements are authorized on a case-by-case basis, authorization (i.e. patient access to treatment) will be required for study entry. Exclusion Criteria: Currently participating in an anifrolumab early access/compassionate use program or an interventional clinical trial with an investigational product. Previous exposure to anifrolumab as part of a clinical trial or early access program. Documented diagnosis of severe or rapidly progressive Class III or IV glomerulonephritis requiring induction therapy (mycophenolate mofetil [MMF]/cyclophosphamide [CYC] + high dose steroids), isolated Class V lupus nephritis, or active severe or unstable neuropsychiatric lupus. Any other condition which the investigator deems to limit a patient's ability to understand the informed consent or complete the PROs.

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 130 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Prospective Cohort<br>All patients aged ≥18 years diagnosed with SLE who initiate anifrolumab as prescribed by their healthcare provider (HCP) per the approved country-specific label and are treated at the study sites will be eligible for inclusion. | Other: None (Observational study)<br>* Not Applicable since Observational Study<br>* Other names: Observational Study;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Disease activity assessed by the Physician Global Assessment (PGA) | The PGA is a single-item visual analogue scale that describes the physician assessment of a patient's disease and its impact on daily functioning at the time of assessment. The scale ranges from 0 (asymptomatic disease and no limitation of normal activities) to 3 (severe disease and limitation of normal activities). | From Baseline (1 year pre-anifrolumab) until follow-up 3 years post-anifrolumab initiation | 
| Disease activity assessed by Systemic Lupus Erythematosus Disease Activity Index 2000 (SLEDAI-2K) | The SLEDAI-2K is a global index and includes 24 clinical symptoms and laboratory variables that are weighted by the type of manifestation, but not by severity or dynamic of the individual item. The SLEDAI-2K includes scoring for antibodies and low complement, as well as some renal and hematologic parameters. The total score ranges between 0 and 105, with higher scores representing increased disease activity. | From Baseline (1 year pre-anifrolumab) until follow-up 3 years post-anifrolumab initiation | 
| Proportion of patients attaining the composite endpoint of lupus low disease activity (LLDAS) | The proportion of patients attaining the composite endpoint of LLDAS will be assesed. | From Baseline (1 year pre-anifrolumab) until follow-up 3 years post-anifrolumab initiation | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Clinical SLE Flares assessment by modified revised Safety of Estrogens in Lupus National Assessment-Systemic Lupus Erythematosus Disease Activity Index (SELENA-SLEDAI) flare index (rSFI) | Flare will be defined as any one criterion present either the Mild/Moderate Flare or Severe Flare categories. New or worsened manifestation will only be reported for manifestation of SLE. | From Baseline (1 year pre-anifrolumab) until follow-up 3 years post-anifrolumab initiation | 
| Proportion of patients with irreversible organ damage, using the Systemic Lupus International Collaborating Clinics/American College of Rheumatology Damage Index (SDI). | The proportion of patients with irreversible organ damage, using the Systemic Lupus International Collaborating Clinics/American College of Rheumatology Damage Index will be assessed. The irreversible, accumulated organ damage from either the disease process or disease treatment, which has been present for at least 6 months, in 12 organ systems will be measured. | From Baseline (1 year pre-anifrolumab) until follow-up 3 years post-anifrolumab initiation | 
| SLE treatment patterns prior to, concomitant with and after anifrolumab | SLE treatment patterns will be analyzed through prevalence and incidence in respect to time to discontinuation. | From Baseline (1 year pre-anifrolumab) until follow-up 3 years post-anifrolumab initiation | 
| Functional Assessment of Chronic Illness Therapy - Fatigue Scale (FACIT-Fatigue) | The FACIT-Fatigue is a 13-item questionnaire formatted for self-administration that assesses patient-reported fatigue and its impact upon daily activities and function over the past 7 days. Patients will be asked to answer each question using a 5-point verbal rating scale, with total scores ranging from 0 (most fatigued) to 52 (least fatigued). | From Baseline (1 year pre-anifrolumab) until follow-up 3 years post-anifrolumab initiation | 
| Lupus Quality of Life (LupusQoL) | The LupusQoL is a validated SLE-specific HRQoL (health-related quality of life) instrument consisting of 34 items across 8 domains (Physical health, Emotional health, Body image, Pain, Planning, Fatigue, Intimate relationships, and Burden to others). The LupusQoL has a 5-point verbal rating scale, and uses a 4-week recall period. The mean raw domain score is transformed to scores ranging from 25 (worst HRQoL) to 125 (best HRQoL) by dividing by 4 and then multiplying by 100. | From Baseline (1 year pre-anifrolumab) until follow-up 3 years post-anifrolumab initiation | 
| Patient Global Assessment (PtGA) | The PtGA is a single-item question that takes into account all the ways in which illness and health conditions may affect the patient at this time. The patient should consider the previous week when answering this question. Responses range from 0 (Very Well) to 100 (Very Poorly) on a visual analogue scale (VAS). | From Baseline (1 year pre-anifrolumab) until follow-up 3 years post-anifrolumab initiation | 
| EuroQol 5-Dimension Health Questionnaire 5 Level (EQ-5D-5L) | The EQ-5D-5L is a general health status measure comprising a descriptive system and the EQ VAS. The descriptive system comprises 5 dimensions: Mobility, Self-care, Usual activities, Pain/discomfort and Anxiety/depression. Patients are asked to rate their current health and functional status on a 5-point verbal rating scale for each of the 5 domains. Responses are converted into an overall quality of life score via a preference-based statistical mapping algorithm. The scores on these 5 dimensions can be presented as a health profile or can be converted to a single summary index number (utility) reflecting preferability compared to other health profiles. Additionally, patients are asked to indicate how they rate their current overall health on a visual analog scale (EQ-VAS) ranges from 100 for best imaginable health state to 0 for worst imaginable health state. | From Baseline (1 year pre-anifrolumab) until follow-up 3 years post-anifrolumab initiation | 
| Work Productivity and Activity Impairment - Lupus (WPAI:lupus) | The WPAI:Lupus is an SLE-specific, self-administered questionnaire, that assesses the impact of disease on productivity. The WPAI:Lupus consists of 6 items and has a recall period of the past 7 days. The WPAI:Lupus is divided into 4 domains: Absenteeism (work time missed), Presenteeism (VAS [scored from 0 to 10] rating of impairment while working), Working Productivity Loss (overall work impairment/absenteeism plus presenteeism), and Activity Impairment (VAS [scored from 0 to 10] rating of daily activity, other than work at a job). Scores for each domain are expressed as impairment percentages, with higher scores indicating greater productivity impairment. | From Baseline (1 year pre-anifrolumab) until follow-up 3 years post-anifrolumab initiation | 
| Pain Numerical Rating Scale (NRS) | The pain NRS will measure the pain severity in the past seven days on a scale of 0-10 (0: no pain; 10: worst pain imaginable). | From Baseline (1 year pre-anifrolumab) until follow-up 3 years post-anifrolumab initiation | 
| Number of outpatient hospital and emergency room visits and procedures | The healthcare resource utilization (HCRU) for SLE after anifrolumab initiation as recorded in medical records will be assessed. The number of outpatient hospital and emergency room visits and procedures. | From Baseline (1 year pre-anifrolumab) until follow-up 3 years post-anifrolumab initiation | 
| Number of hospital admissions and inpatient hospital procedures | The HCRU for SLE after anifrolumab initiation as recorded in medical records will be assessed. The number of hospital admissions and inpatient hospital procedures (including duration of hospital stay and reason for hospitalization, stratified by admission to an intensive care unit [ICU] vs non-ICU) will be assessed. | From Baseline (1 year pre-anifrolumab) until follow-up 3 years post-anifrolumab initiation | 
| Number of rheumatologist visits and procedures | The HCRU for SLE after anifrolumab initiation as recorded in medical records will be assessed. The number of rheumatologist visits and procedures (including SLE-related laboratory tests) will be assessed. | From Baseline (1 year pre-anifrolumab) until follow-up 3 years post-anifrolumab initiation | 
| Number of dialysis appointments | The HCRU for SLE after anifrolumab initiation as recorded in medical records will be assessed. The number of dialysis appointments will be assessed. | From Baseline (1 year pre-anifrolumab) until follow-up 3 years post-anifrolumab initiation | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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chronic autoimmune disease, immunosuppressants, corticosteroids, human monoclonal antibody (IgG1ƙ mAb), Real World