Anifrolumab Study for Treatment Effectiveness in the Real World (ASTER) among patients with systemic lupus erythematosus: protocol for an international observational effectiveness study

Abstract

Introduction: Systemic lupus erythematosus (SLE) is a chronic autoimmune disease with a diverse clinical presentation that involves multiple organ systems and may lead to organ damage and increased risk of mortality. SLE is associated with a high burden of disease that can include loss of productivity and employment and reduced health-related quality of life. The current standard of care for SLE is primarily based on immunosuppression and glucocorticoids and is associated with risk of toxicities and poor tolerability. Anifrolumab, a human monoclonal antibody to type I interferon receptor subunit 1, was recently approved as a new treatment for patients with moderate-to-severe SLE.

Methods and analysis: Here, we report the study design of the ongoing, multinational Anifrolumab Study for Treatment Effectiveness in the Real World (ASTER) that includes 3-years of follow-up beginning with the first infusion of anifrolumab and 1 year of retrospective baseline data. ASTER aims to enrol 500 adult patients receiving anifrolumab for SLE in Europe and Canada. The key study objective is to describe the real-world effectiveness of anifrolumab in routine clinical practice, including clinician-reported disease activity and patient-reported outcomes collected via mobile application. This mobile application also includes a medication diary, where patients report their prescription and non-prescription medication use for SLE on a weekly basis; these data will lend insights on treatment patterns for the study population.

Ethics and dissemination: The design of the ASTER study was informed through consultations with patients with SLE who provided important insights to help maximise patient engagement, retention and the collection of key, patient-relevant endpoints. ASTER enrolment began in February 2023 and the study is expected to finish in 2029.

Trial registration number: NCT05637112.

Keywords: Chronic Disease; Immunology; Observational Study; Patient Reported Outcome Measures; Patients.

Figure 1. Anifrolumab Study for Treatment Effectiveness in the Real World study design and outcomes. ^aAnifrolumab is administered according to country-specific labelling; the first dose defines the study index date for each patient. If anifrolumab is discontinued, data collection continues through year 3, unless the patient withdraws consent. ^bBaseline data are obtained from medical records covering 12 months prior to the index date and from clinician- and patient-reported disease assessments at the final standard-of-care visit prior to the index date. ^cClinical laboratory tests performed during routine clinical management may include haematology, clinical chemistry, urinalysis assessments, anti-dsDNA antibodies, hs-CRP and serum component (C3 or C4). ^dPatient-reported data are collected at the final standard-of-care visit before the index date, after which patient reporting occurs via the mobile app. ^eMedical events of interest include anaphylaxis following anifrolumab treatment, herpes zoster (and vaccination), serious infections, COVID-19 infection (and date(s) and type(s) of vaccination), major adverse cardiovascular events (including (cardiovascular) death, non-fatal myocardial infarction, non-fatal stroke, hospitalisation for unstable angina, hospitalisation for heart failure, transient ischaemic attack, coronary revascularisation procedures, urgent cerebrovascular revascularisation, arrhythmia (not associated with ischaemia), peripheral arterial event, venous thromboembolic event and other non-fatal cardiovascular events), malignancies and pregnancy. ADR, adverse drug reaction; CLASI, Cutaneous Lupus Erythematosus Disease Area and Severity Index; eCRF, electronic case report form; EQ-5D-5L, EuroQol 5-Dimension Health Questionnaire 5 Level; FACIT-Fatigue, Functional Assessment of Chronic Illness Therapy–Fatigue Scale; hs-CRP, high-sensitivity C reactive protein; LupusQoL, Lupus Quality of Life; NRS, Numerical Rating Scale; PGA, Physician Global Assessment; PtGA, Patient Global Assessment; rSFI, revised Safety of Estrogens in Lupus National Assessment-SLEDAI flare index; SAE, serious adverse event; SDI, Systemic Lupus International Collaborating Clinics/American College of Rheumatology Damage Index; SLE, systemic lupus erythematosus; SLEDAI-2K, SLE Disease Activity Index 2000; WPAI:Lupus, Work Productivity and Activity Impairment–Lupus.