{
  "abstract": "Objectives To define a fit-for-purpose clinical outcome assessment for thrombocytopaenia, as part of a novel multi-domain outcome measure for systemic lupus erythematosus (SLE) clinical trials, the Treatment Response Measure for Systemic Lupus Erythematosus (TRM-SLE).Methods An international working group comprising 14 expert clinicians (adult and paediatric rheumatologists and a clinical and laboratory haematologist), and 4 patients with lived experience of haematologic SLE participated in a multi-step consensus process. First, via structured discussion, the scope of thrombocytopaenia was defined for the SLE clinical trial context. Working group members then nominated and shortlisted potential response measures which were evaluated in a systematic literature review (SLR) focusing on relevant measurement properties and clinical trial utility. Informed by the SLR, nominal group technique (NGT) was used to reach consensus on the response measure, and thresholds for entry, minimum clinically meaningful response and complete response.Results The thrombocytopaenia domain was defined with 100% consensus as ‘immune-mediated thrombocytopaenia attributed to active lupus that impacts the patient and is modifiable by therapy to reduce or control disease activity’, irrespective of antiphospholipid syndrome/antibody status, and excluding other causes of thrombocytopenia (e.g. gestational, drugs, cancer, infection). 31 candidate measures (12 unique) were nominated, and after structured discussion 2 were shortlisted for evaluation via SLR: platelet count and blood film schistocytes. 4,755 studies were screened with 33 studies included in the SLR. The NGT process selected platelet count (Plt) as the consensus treatment response measure with the following thresholds: entry (Plt <50 x109/L), minimum clinically meaningful response (Plt &#8805;50 x109/L and at least 2x the baseline value), and complete response (Plt &#8805;100 x109/L). ( Figure 1)Abstract PO:05:138 Figure 1Conclusions Consensus was reached to use platelet count, with thresholds defined for entry and improvement, to classify thrombocytopaenia treatment response as part of the TRM-SLE multi-domain instrument for use in SLE clinical trials.",
  "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": [
        "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, 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 and University of Cincinnati College of Medicine, Cincinnati, USA",
        "Department of Pediatrics, Johns Hopkins University, Baltimore, 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": [
        "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:05:138 Measurement of thrombocytopaenia in a novel treatment response measure for systemic lupus erythematosus clinical trials",
  "uid": "97fcdbe4-c60f-5271-86a2-d3fc9500af78"
}