Does Belimumab Modify the Natural History of SLE? A Propensity Score-matched, Real-world Study
Trial Snapshot
- Phase
- Not Applicable
- Status
- Recruiting
- Sponsor
- Enrollment
- 684
- Locations
- 1
- Primary Endpoint
- Bilag A flare
Study Overview
Brief Summary
This will be a combined retrospective and prospective cohort study, that will evaluate the incidence of de novo Systemic Lupus Erythematosus major organ manifestations (defined as BILAG A flares) in patients receiving belimumab (Arm A) and compare it to 2 standard-of-care groups (SoC) (Arm B: patients on SoC; Arm C: patients on SoC followed-up up to May 1st 2014, the first date where belimumab was available in Greece).
The investigators will utilize survival analysis methods (Kaplan-Meier survival curves and Cox regression) and mixed effects longitudinal analyses. Additionally, the investigators will employ propensity score matching and/or inverse probability of treatment weighting, to create balanced cohorts and reduce bias.
Detailed Description
Belimumab (BEL) has been used for the treatment of SLE for over 10 years, with clear evidence for a beneficial effect in reduction of disease activity and frequency of flares, as well as prevention of damage accrual.
Whether BEL may also result in prevention of incident, major organ involvement in patients with SLE is less clear. A recent post-hoc analysis of the BLISS trials suggested that BEL (although at the low, rather than standard-dose) reduced the rate of de novo renal flares (defined by BILAG) compared to standard-of-care but real-life data are scarce. Importantly, data on the ability of belimumab to decrease major neuropsychiatric events in SLE such as strokes, seizures and cognitive dysfunction or other severe manifestations such as severe hematologic or cardiopulmonary disease is not known.
To this end, the investigators propose a propensity score-matched, real-world, with the use of longitudinal data from large patient cohorts and time-to-event analyses. Given the non-experimental setting of this study, the investigators will also employ propensity score matching (PSM), to create balanced cohorts of BEL-treated and non-BEL-treated patients and reduce bias in estimating treatment efficacy.
The study will consist of 3 arms, as follows: Arm A: patients on treatment with belimumab; Arm B: patients on standard-of-care (SoC) treatment from May 1st 2014 onwards; Arm C: a historical cohort of patients on SoC followed-up up to May 1st 2014, the first date where belimumab was available in Greece.
Study Design
- Study Type
- Observational
- Observational Model
- Cohort
- Time Perspective
- Other
Eligibility Criteria
- Ages
- 18 Years to — (Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •Systemic lupus erythematosus classification according to the Systemic Lupus International Collaborating Clinics (SLICC) criteria
- •Age ≥ 18 years old
- •Time of follow-up ≥ 6 months
- •Systemic Lupus Erythematosus Disease Activity Index 2000 (SLEDAI-2K) ≥ 4
- •Physician Global Assessment (PGA) visual analogue score > 1
Exclusion Criteria
- •Patients with incomplete medical records or missing key variables
- •Patients with concomitant autoimmune disorders (excluding thyroid disease)
Outcomes
Primary Outcomes
Bilag A flare
Time Frame: From enrollment to the end of treatment (expected follow-up duration 1-4 years).
Major organ systemic lupus erythematosus manifestations as defined by the BILAG (British Isles Lupus Assessment Group) index (BILAG A flare)
Secondary Outcomes
- SLICC/ACR Damage Index(From enrollment to the end of treatment (expected follow-up duration 1-4 years).)
Investigators
DIMITRIOS BOUMPAS
Professor in Medicine
Biomedical Research Foundation, Academy of Athens