{
  "abstract": "Objectives To define a clinically meaningful treatment response definition for haemolytic anaemia, that will form part of a novel multidomain outcome measure for SLE clinical trials.Methods An international working group comprising 12 clinicians and 4 patients completed a multistep consensus process. First, the scope of haemolytic anaemia for the SLE clinical trial context was defined. Candidate response measures were then nominated via an online survey and shortlisted for evaluation via a systematic literature review (SLR) focussing on relevant measurement properties and clinical trial utility. Informed by the SLR results, the working group used nominal group technique (NGT) to reach consensus on a response measure, as well as entry, clinically meaningful response and complete response thresholds.Results The domain scope was unanimously agreed on as ‘immune-mediated haemolytic anaemia attributed to active lupus that impacts the patient and is modifiable by therapy to reduce or control disease activity’, including Coombs’ positive autoimmune haemolytic anaemia and rarer subtypes. Six laboratory measures to fit this scope were shortlisted for the SLR, which screened 1519 articles, of which 10 were included. Consensus via NGT was reached on haemoglobin (Hb) improvement to measure haemolytic anaemia treatment response. Thresholds were defined for entry (Hb <100 g/L with laboratory confirmation of haemolysis), minimum clinically meaningful response (Hb improvement >20 g/L or Hb normalised with ongoing haemolysis) and complete response (Hb normalised without haemolysis).Conclusions Hb, with meaningful thresholds for entry and improvement, will be adopted to classify haemolytic anaemia treatment response in a novel multi-domain outcome measure for SLE clinical trials.",
  "authors": [
    {
      "affiliations": [
        "Centre for Inflammatory Diseases, Monash University, Clayton, Victoria, Australia",
        "Rheumatology, Monash Health, Clayton, Victoria, Australia"
      ],
      "name": "Kathryn Connelly"
    },
    {
      "affiliations": [
        "Rheumatology, Monash Health, Clayton, Victoria, Australia",
        "Sub-Faculty of Clinical and Molecular Medicine, Monash University, Clayton, Victoria, Australia"
      ],
      "name": "Eric Morand"
    },
    {
      "affiliations": [
        "Centre for Inflammatory Diseases, Monash University, Clayton, Victoria, Australia"
      ],
      "name": "Rachel Koelmeyer"
    },
    {
      "affiliations": [
        "Centre for Inflammatory Diseases, Monash University, Clayton, Victoria, Australia",
        "Rheumatology, Monash Health, Clayton, Victoria, Australia"
      ],
      "name": "Lavanya Rajagopala"
    },
    {
      "affiliations": [
        "Centre for Inflammatory Diseases, Monash University, Clayton, Victoria, Australia",
        "Rheumatology, Monash Health, Clayton, Victoria, Australia"
      ],
      "name": "Ambika Wakhlu"
    },
    {
      "affiliations": [
        "Centre for Inflammatory Diseases, Monash University, Clayton, Victoria, Australia"
      ],
      "name": "Yanjie Hao"
    },
    {
      "affiliations": [
        "Centre for Inflammatory Diseases, Monash University, Clayton, Victoria, Australia",
        "Rheumatology, Monash Health, Clayton, Victoria, Australia"
      ],
      "name": "Katie Liao"
    },
    {
      "affiliations": [
        "Monash University, Melbourne, Victoria, Australia"
      ],
      "name": "Darshini Ayton"
    },
    {
      "affiliations": [
        "Library, Monash Health, Clayton, Victoria, Australia"
      ],
      "name": "Cassandra Gorton"
    },
    {
      "affiliations": [
        "Haematology, Monash Health, Clayton, Victoria, Australia",
        "School of Clinical Sciences, Monash University, Clayton, Victoria, Australia"
      ],
      "name": "Ashwini Bennett"
    },
    {
      "affiliations": [
        "Epidemiology, UTHealth Houston, Houston, Texas, USA"
      ],
      "name": "Najha Black"
    },
    {
      "affiliations": [
        "Department of Autoimmune Diseases, Hospital Clínic, Institut d’Investigacions Biomèdiques August Pi i Sunyer, University of Barcelona, Barcelona, Spain"
      ],
      "name": "Ricard Cervera"
    },
    {
      "affiliations": [
        "Division of Rheumatology, Inflammation and Immunity, Brigham and Women’s Hospital, Harvard Medical School, Boston, Massachusetts, USA"
      ],
      "name": "Karen Costenbader"
    },
    {
      "affiliations": [
        "Rheumatology and Clinical Immunology Unit, Attikon University Hospital, National and 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 MC University Medical Center Rotterdam, Rotterdam, Netherlands"
      ],
      "name": "S S M Kamphuis"
    },
    {
      "affiliations": [
        "Lupus Europe, Brussels, Belgium"
      ],
      "name": "Zoe Karakikla-Mitsakou"
    },
    {
      "affiliations": [
        "Department of Rheumatology, University of Santo Tomas Hospital, Manila, Philippines"
      ],
      "name": "Sandra Navarra"
    },
    {
      "affiliations": [
        "Pediatrics, Rheumatology, Cincinnati Childrens Hospital Medical Center, Cincinnati, Ohio, USA",
        "Pediatrics, Johns Hopkins University, Baltimore, Maryland, USA"
      ],
      "name": "Ekemini A Ogbu"
    },
    {
      "affiliations": [
        "University Center of Excellence on Nephrologic, Rheumatologic and Rare Diseases, San Giovanni Bosco Hub Hospital, and Department of Clinical and Biological Sciences, University of Turin, Torino, 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, San Francisco, California, USA"
      ],
      "name": "Jinoos Yazdany"
    },
    {
      "affiliations": [
        "LORA Group, Royal Oak, Michigan, 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": "Measurement of haemolytic anaemia in a novel treatment response measure for SLE clinical trials",
  "uid": "24bd296b-0bc2-5751-88c9-cf6162e70956"
}