{
  "abstract": "Introduction Recent advances in treatment and care have improved survival rates for children and young adults with severe blood disorders such as sickle cell disease (SCD), transfusion-dependent beta-thalassaemia (TDT) and acute leukaemia. However, their quality of life and reproductive and psychosocial outcomes are not yet well studied. For SCD and TDT, robust survival data are mainly limited to North America. Thus, there is a need to fill these knowledge gaps to guide improvements in care, address unmet clinical needs and rigorously assess the efficacy of emerging novel therapies.Methods and analysis This is an observational population-based mixed-methods study of individuals diagnosed with SCD, TDT or acute leukaemia when under the age of 18 in England, involving a data linkage component and a patient-reported outcomes measures survey. Data linkage-eligible participants will be identified from national and regional databases, including the Hospital Episode Statistics, Yorkshire Specialist Register of Cancer in Children & Young People and the National Congenital Anomaly and Rare Diseases Registration Service. Data linkage will be processed within the NHS England and the University of Leeds’ secure, trusted research environments. Data will be accessed without consent under section 251 and approval by the confidentiality advisory group. It will assess survival rates for SCD and TDT as well as clinical, educational and mental health outcomes for SCD, TDT and acute leukaemia diagnosed in childhood.Survey-eligible participants for SCD, TDT and acute leukaemia cohorts will be checked for their suitability to participate by the North of England clinical care teams. An NHS-approved survey provider will facilitate data checks with the NHS National Data Opt-Out Service. Consent is required for participation in the survey and for subsequent data linkage to existing databases. Surveys are conducted in various formats (online, paper and phone), with reminders sent after 21 days. The survey will assess quality of life and psychosocial and reproductive outcomes. Participants can withdraw at any time, and support is available via telephone helplines.Ethics and dissemination The study has received ethical and information governance approval from the Health Research Authority (Reference 24/YH/0186) and the Confidentiality Advisory Group (CAG 24/CAG/0138) to process identifiable data without consent. Study results will be available to patients, physicians, researchers, stakeholders and others through open-access publishing, results sharing via media platforms and presentations at conferences and meetings.",
  "authors": [
    {
      "affiliations": [
        "School of Medicine and Population Health, University of Sheffield, Sheffield, UK",
        "Leeds Institute for Data Analytics, School of Medicine, University of Leeds, Leeds, UK"
      ],
      "name": "Khalid Ahmed"
    },
    {
      "affiliations": [
        "Leeds Institute for Data Analytics, School of Medicine, University of Leeds, Leeds, UK"
      ],
      "name": "Ivana Holloway"
    },
    {
      "affiliations": [
        "Leeds Institute for Data Analytics, School of Medicine, University of Leeds, Leeds, UK"
      ],
      "name": "Kate Absolom"
    },
    {
      "affiliations": [
        "Leeds Institute for Data Analytics, School of Medicine, University of Leeds, Leeds, UK"
      ],
      "name": "Samantha J Mason"
    },
    {
      "affiliations": [
        "Academic Unit of Heath Economics, Leeds Institute of Health Sciences, School of Medicine, University of Leeds, Leeds, UK"
      ],
      "name": "Ruben Mujica-Mota"
    },
    {
      "affiliations": [
        "Academic Unit of Heath Economics, Leeds Institute of Health Sciences, School of Medicine, University of Leeds, Leeds, UK"
      ],
      "name": "Georgios Gkountouras"
    },
    {
      "affiliations": [
        "Academic Unit of Heath Economics, Leeds Institute of Health Sciences, School of Medicine, University of Leeds, Leeds, UK"
      ],
      "name": "Adam Martin"
    },
    {
      "affiliations": [
        "Leeds Institute for Data Analytics, School of Medicine, University of Leeds, Leeds, UK"
      ],
      "name": "Thuvia Flannery"
    },
    {
      "affiliations": [
        "Department of Haematology, Leeds Teaching Hospitals NHS Trust, Leeds, UK"
      ],
      "name": "Michael Richards"
    },
    {
      "affiliations": [
        "Department of Haematology, Sheffield Children’s Hospital NHS Foundation Trust, Sheffield, UK"
      ],
      "name": "Emma Astwood"
    },
    {
      "affiliations": [
        "Department of Haematology, Bradford Teaching Hospitals NHS Foundation Trust, Bradford, UK"
      ],
      "name": "Sam Ackroyd"
    },
    {
      "affiliations": [
        "Department of Haematology, Royal Victoria Infirmary, Newcastle upon Tyne, UK"
      ],
      "name": "Brigit Greystoke"
    },
    {
      "affiliations": [
        "School of Allied Health Professions, Nursing, and Midwifery, University of Sheffield, Sheffield, UK"
      ],
      "name": "Diana M Greenfield"
    },
    {
      "affiliations": [
        "Department of Haematology, Leeds Teaching Hospitals NHS Trust, Leeds, UK"
      ],
      "name": "Quentin Hill"
    },
    {
      "affiliations": [
        "Department of Haematology, Leeds Teaching Hospitals NHS Trust, Leeds, UK"
      ],
      "name": "Beki James"
    },
    {
      "affiliations": [
        "Leeds Teaching Hospitals NHS Trust, Leeds, UK"
      ],
      "name": "Michelle Kwok- Williams"
    },
    {
      "affiliations": [
        "Department of Endocrinology, Leeds Teaching Hospitals NHS Trust, Leeds, UK"
      ],
      "name": "Robert D Murray"
    },
    {
      "affiliations": [
        "Department of Haematology, Sheffield Teaching Hospitals NHS Foundation Trust, Sheffield, UK"
      ],
      "name": "Clare Samuelson"
    },
    {
      "affiliations": [
        "Department of Haematology, Royal Liverpool University Hospital, Liverpool, UK"
      ],
      "name": "David Simcox"
    },
    {
      "affiliations": [
        "School of Medicine and Population Health, University of Sheffield, Sheffield, UK"
      ],
      "name": "John A Snowden"
    },
    {
      "affiliations": [
        "Department of Haematology, Manchester University NHS Foundation Trust, Manchester, UK"
      ],
      "name": "Joseph Sharif"
    },
    {
      "affiliations": [
        "Department of Haematology, Manchester University NHS Foundation Trust, Manchester, UK"
      ],
      "name": "Nandini Sadasivam"
    },
    {
      "affiliations": [
        "Leeds Institute for Data Analytics, School of Medicine, University of Leeds, Leeds, UK"
      ],
      "name": "Richard Feltbower"
    },
    {
      "affiliations": [
        "Leeds Institute for Data Analytics, School of Medicine, University of Leeds, Leeds, UK"
      ],
      "name": "Adam Glaser"
    }
  ],
  "title": "Adult survivors of sickle cell disease, transfusion-dependent beta-thalassaemia and childhood acute leukaemia in England: protocol for a mixed methods data linkage and health-related quality of life survey study",
  "uid": "5519b0cb-3425-5700-b068-83ccb27c7ff7"
}