{
  "abstract": "Objectives Systemic lupus erythematosus (SLE) is a chronic, immune-mediated disease that can affect multiple organ systems, causing debilitating symptoms, organ damage and impaired quality of life. Patients with active SLE, including lupus nephritis (LN), frequently experience inadequate responses to glucocorticoids (GC) and immunosuppressants (IS), leading to refractory disease and poor outcomes. There are limited prospective data on the efficacy and safety of contemporaneous standard of care (SoC) in these populations, especially using comprehensive outcome measures utilized in clinical trials.This global, multicenter, single-arm, prospective, observational study (Benchmark-SLE, CA0611025, NCT07175285) will evaluate the efficacy of SoC in achieving remission and improving clinical outcomes in patients with active SLE (including LN) refractory to GC and at least 2 IS, and to report safety and patient-reported outcomes (PROs).Methods The study will enroll 223 adults (at least 16 years) with active SLE meeting EULAR/ACR 2019 criteria and inadequate response to therapeutic doses of GC and at least 2 IS (for at least 3 months each). Approximately 50–75% will have LN. The primary endpoint is DORIS remission at 6 months: clinical SLEDAI = 0, PhGA < 0.5 (VAS 0–3), with permitted low-dose GCs, antimalarials, and/or stable IS/biologics. Secondary endpoints include drug-free DORIS remission, complete renal response (CRR) for subjects with baseline LN, maintenance/duration of response, SRI-4, LLDAS, SLICC/ACR damage index, incidence/severity of flares, changes in proteinuria/eGFR, and change in FACIT-Fatigue (PRO). An optional renal biopsy substudy will assess ongoing activity/damage in LN patients with persistent proteinuria at 6 months. AEs/SAEs will be monitored as well. Data will be summarized descriptively.Results Enrollment is ongoing. The study will provide up-to-date real-world rates of remission, response, and safety in this refractory SLE population using standardized trial endpoints and may be used to contextualize the data from other ongoing SLE clinical trials evaluating novel investigational agents.Conclusions Benchmark-SLE will generate robust real-world evidence on the efficacy and safety of current standard-of-care therapies in patients with refractory SLE and LN. These findings will inform future therapeutic strategies and trial design while addressing unmet needs in this complex population.",
  "authors": [
    {
      "affiliations": [
        "University of Toronto and Krembil Research Institute, Toronto, ON, Canada"
      ],
      "name": "Zahi Touma"
    },
    {
      "affiliations": [
        "Oklahoma Medical Research Foundation, Oklahoma City, OK, USA"
      ],
      "name": "Cristina Arriens"
    },
    {
      "affiliations": [
        "Linköping University and Bristol Myers Squibb, Linköping, Sweden"
      ],
      "name": "Christopher Sjöwall"
    },
    {
      "affiliations": [
        "Bristol Myers Squibb, Princeton, NJ, USA"
      ],
      "name": "Lu Gao"
    },
    {
      "affiliations": [
        "Bristol Myers Squibb, Princeton, NJ, USA"
      ],
      "name": "Massimiliano Cristofanilli"
    },
    {
      "affiliations": [
        "University of Occupational and Environmental Health, Kitakyushu, Japan"
      ],
      "name": "Yoshiya Tanaka"
    }
  ],
  "title": "PO:07:179 Benchmark-SLE: prospective observational study of standard of care efficacy and safety in active SLE with inadequate response to glucocorticoids and at least 2 immunosuppressants",
  "uid": "3a88ffaf-d097-534b-9426-da0346c5b519"
}