{
  "abstract": "Objectives To define a fit-for-purpose clinical outcome assessment, and thresholds for entry and improvement, of haemolytic anaemia, for inclusion as part of a novel multi-domain outcome measure for SLE clinical trials, Treatment Response Measure for Systemic Lupus Erythematosus (TRM-SLE).Methods An international working group comprising 12 clinicians (including adult and paediatric rheumatologists and a haematologist) and 4 patients with lived experience of haematological lupus activity completed a multi-step consensus process. First, the domain was defined and the scope of haemolytic anaemia for the SLE clinical trial context was enumerated. Candidate response measures were then nominated via an online survey, discussed and shortlisted. Shortlisted measures were evaluated via a systematic literature review (SLR) guided by COSMIN methodology focussing on relevant measurement properties and clinical trial utility. Informed by the SLR, the working group used nominal group technique (NGT) to reach consensus on a response measure, as well as entry, minimum and complete response thresholds.Results The domain was defined with 100% consensus as ‘immune-mediated haemolytic anaemia attributed to active lupus that impacts the patient and is modifiable by therapy to reduce or control disease activity’, and domain scope was defined to include Coombs’ positive autoimmune haemolytic anaemia and rarer subtypes ( figure 1). Six laboratory measures to fit this scope were shortlisted for the SLR, which screened 1,519 articles, of which 10 were included, including studies in non-SLE patient populations with autoimmune haemolytic anaemia. Consensus via NGT was reached on haemoglobin (Hb) improvement to measure haemolytic anaemia treatment response. Thresholds were defined for entry (Hb <10g/dL with laboratory confirmation of haemolysis), minimum clinically meaningful response (Hb improvement >2g/dL or Hb normalised with ongoing haemolysis) and complete response (Hb normalised and no laboratory evidence of haemolysis). These improvement thresholds correspond with previously published consensus response criteria for primary autoimmune haemolytic anaemia.Abstract PO:05:140 Figure 1Conclusions Consensus was reached to use Hb, with clinically meaningful thresholds for entry and improvement, to classify haemolytic anaemia treatment response in a novel multi-domain outcome measure for SLE clinical trials. This will improve the study of haemolytic anaemia and its treatment in SLE, and ensure that meaningful improvement contributes to the classification of treatment response.",
  "authors": [
    {
      "affiliations": [
        "Centre for Inflammatory Diseases, Monash University, Clayton, Australia",
        "Department of Rheumatology, Monash Health, Clayton, Australia"
      ],
      "name": "Kathryn Connelly"
    },
    {
      "affiliations": [
        "Centre for Inflammatory Diseases, Monash University, Clayton, Australia",
        "Department of Rheumatology, Monash Health, Clayton, Australia"
      ],
      "name": "Eric F Morand"
    },
    {
      "affiliations": [
        "Centre for Inflammatory Diseases, Monash University, Clayton, Australia"
      ],
      "name": "Rachel Koelmeyer"
    },
    {
      "affiliations": [
        "Centre for Inflammatory Diseases, Monash University, Clayton, Australia",
        "Department of Rheumatology, Monash Health, Clayton, Australia"
      ],
      "name": "Lavanya Rajagopala"
    },
    {
      "affiliations": [
        "Centre for Inflammatory Diseases, Monash University, Clayton, Australia",
        "Department of Rheumatology, Monash Health, Clayton, Australia"
      ],
      "name": "Ambika Wakhlu"
    },
    {
      "affiliations": [
        "Centre for Inflammatory Diseases, Monash University, Clayton, Australia"
      ],
      "name": "Yanjie Hao"
    },
    {
      "affiliations": [
        "Centre for Inflammatory Diseases, Monash University, Clayton, Australia",
        "Department of Rheumatology, Monash Health, Clayton, Australia"
      ],
      "name": "Katie Liao"
    },
    {
      "affiliations": [
        "School of Public Health and Preventive Medicine, Monash University, Melbourne, Australia"
      ],
      "name": "Darshini Ayton"
    },
    {
      "affiliations": [
        "Library, Monash Health, Clayton, Australia"
      ],
      "name": "Cassandra Gorton"
    },
    {
      "affiliations": [
        "Department of Haematology, Monash Health, Clayton, Australia",
        "School of Clinical Sciences at Monash Health, Monash University, Clayton, Australia"
      ],
      "name": "Ashwini Bennett"
    },
    {
      "affiliations": [
        "Department of Autoimmune Diseases, Hospital Clínic, Institut d’Investigacions Biomèdiques August Pi i Sunyer, Barcelona, Spain"
      ],
      "name": "Ricard Cervera"
    },
    {
      "affiliations": [
        "Division of Rheumatology, Inflammation and Immunity, Brigham and Women’s Hospital, Harvard Medical School, Boston, USA"
      ],
      "name": "Karen H Costenbader"
    },
    {
      "affiliations": [
        "Rheumatology and Clinical Immunology Unit, Attikon University Hospital, National Kapodistrian University of Athens, Athens, Greece"
      ],
      "name": "Antonis Fanouriakis"
    },
    {
      "affiliations": [
        "Lupus Europe, Brussels, BELGIUM"
      ],
      "name": "Susanne Gydesen"
    },
    {
      "affiliations": [
        "Lupus Europe, Brussels, BELGIUM"
      ],
      "name": "Cathrine Hjelmeset"
    },
    {
      "affiliations": [
        "Division of Pediatric Rheumatology, Sophia Children’s Hospital, Erasmus University Medical Center, Rotterdam, The Netherlands"
      ],
      "name": "Sylvia Kamphuis"
    },
    {
      "affiliations": [
        "Lupus Europe, Brussels, BELGIUM"
      ],
      "name": "Zoe Karakikla-Mitsakou"
    },
    {
      "affiliations": [
        "Department of Epidemiology, UTHealth Houston, Houston, USA"
      ],
      "name": "Najha Black"
    },
    {
      "affiliations": [
        "Department of Rheumatology, University of Santo Tomas Hospital, Manila, Philippines"
      ],
      "name": "Sandra Navarra"
    },
    {
      "affiliations": [
        "Department of Pediatrics, Cincinnati Children’s Hospital Medical Center, University of Cincinnati College of Medicine, Cincinnati, USA"
      ],
      "name": "Ekemini A Ogbu"
    },
    {
      "affiliations": [
        "University Center of Excellence on Nephrologic, Rheumatologic and Rare Diseases, San Giovanni Bosco Hub Hospital, Turin, Italy"
      ],
      "name": "Savino Sciascia"
    },
    {
      "affiliations": [
        "Department of Clinical and Academic Rheumatology, King’s College Hospital, London, UK"
      ],
      "name": "Chris Wincup"
    },
    {
      "affiliations": [
        "Division of Rheumatology, Department of Medicine, University of California, San Francisco, USA"
      ],
      "name": "Jinoos Yazdany"
    },
    {
      "affiliations": [
        "LORA Group, Royal Oak, USA"
      ],
      "name": "Laurie Burke"
    },
    {
      "affiliations": [
        "Department of Clinical and Experimental Medicine, University of Pisa, Pisa, Italy"
      ],
      "name": "Marta Mosca"
    },
    {
      "affiliations": [
        "Grupo Oroño-Centro Regional de Enfermedades Autoinmunes y Reumáticas (GO-CREAR), Rosario, Argentina"
      ],
      "name": "Guillermo J Pons-Estel"
    }
  ],
  "title": "PO:05:140 Measurement of haemolytic anaemia in a novel treatment response measure for systemic lupus erythematosus clinical trials",
  "uid": "3e6faee2-0f48-5619-a7ad-4665dc6457e9"
}