Annette Gilmore RN, MSc Haematology Department, Central Middlesex Hospital, London.

1. Process and Outcomes of a Multicentre Sickle Cell Disease Registry for Patient Management and Research4th July 2006 Annette Gilmore RN, MScHaematology Department, Central Middlesex Hospital, London. www.europeanhbregistry.org.uk or www.hbregistry.org.uk

2. Introduction • The European Haemoglobinopathy Registry (EHR) is based in the Haematology Department, Central Middlesex Hospital, London, UK. • It collects longitudinal clinical and demographic information on sickle cell disease (SCD) patients to assist with their clinical management and for research. • Two national reports1 recommended the establishment of population based information systems as vital for improvement of services for SCD.1 National review of Haemoglobinopathy services: SMAC Report (1993) and Fair Shares for London (1997).

3. What is Sickle Cell Disease (SCD) ? • Inherited disease of red blood cells. • Abnormal haemoglobin causes red blood cells to become stiff and malformed, causing blockages insmall blood vessels. • Approximately 10,000 people with SCD in UK. • Disease severity varies among individuals and during life cycle. • Symptoms – pain episodes, chronic anaemia, severe infections and damage to vital organs. • Treatment aimed at symptom prevention & control. • Minority cured by bone marrow transplant. • Reduced life expectancy.

4. Aims • Study the natural history of the Haemoglobinopathies. • Evaluation of treatments – Hydroxyurea, Blood Tx, Chelating Agents (Desferal & L1). • Management tool for improving patient care. • Accurate information for clinical practice, clinical governance & audit, and planning services. • Collaboration and information sharing. • Impact on future research.

5. EHR Research Ethics Process • Data Protection Act Registration[Registration No. Z5730583]. • MREC [Approval - MREC/99/2/4]. • LREC [Approval - BEC 612]. • Patient Information and Consent Forms. • Secure Access to Registry (PATS): • Password Access. • Encrypted Data Communications.

6. Sampling Method • Comprehensive list of all eligible patients created. • Inclusion criteria: • All patients with SCD diagnosis (e.g. SS, SC, SBthal). • Attends participating hospital as an outpatient or inpatient. • Exclusion criteria: • Failure to obtain consent. • All consecutive cases selected since November 1999.

7. Data Collection and Reporting • Initial registration requires retrospective review of patients‘ medical records. • Follow-up proformas are completed annually. • Additional proformas are completed as required (e.g. Adverse Events, Hydroxyurea and Transfusion Therapy). • Automated data validation, processing and clinical reporting. • Web site for general public and members. • Data collection tools and reports available to members via secure website.

14. Achieving Reliability and Validity The quality of a database is assessed by its “coverage” and “accuracy”. • Robust sampling method. • Data abstractors blinded to hypotheses. • Standardized proformas. • Glossary with definitions of clinical events. • Guidelines for proforma completion. • Consensus management of conflicting and 'difficult to interpret' data. • Abstractors are health professionals or experienced heathcare employees. • Abstractors receive training, ongoing supervision and support. • Sample of data source (patient medical records) are second checked. • Automated data validation and processing. • Draft patient reports returned to abstractors for verification and completion of missing and/or conflicting information. • Intraobserver and interobserver reliability study of abstractors in progress. • Assessing completeness of variables.

15. Results • 626 patients out of 927 consented. • 474 patients (216 males, 258 females) have data collected, totalling 4,515 patient years (yrs) of data. • Mean length of follow-up is 9.5 yrs(SD 9 yrs, range 0 mths - 42 yrs). • Mean age is 25.8 yrs(SD 15.8 yrs, range 0 yr - 74 yrs). • Patients on Hydroxyurea (HU) Treatment: • 258 patient yrs of HU data has been collected on 70 patients. • Mean length of follow-up is 3.7 yrs(SD 3 yrs, range 0 mths - 11 yrs).

16. North West London & RegistryPopulation by Age Group

17. North West London & RegistryPopulation by Haemoglobinopathy Type

18. Funding Structure • The registry has no formal long-term funding. • Funding to set-up and run the central registry at CMH and to set-up and collect data in the four local participating hospitals is approx. £575,000 over 6½ years. Cost per patient year of data is approx. £115. • Funded by: • Research grants (DoH x 2, European sources). • 3 year development project grant from local PCTs. • Brent PCT funding – 3 years. • Charitable monies.

19. How can you contact us? • Email: annette.gilmore@nhs.net • By post:Annette Gilmore Haematology Department Central Middlesex Hospital Acton Lane London NW10 7NS • Web site: www.europeanhbregistry.org.uk or www.hbregistry.org.uk • Telephone: +44-(0)20-8453 2135 +44-(0)20-8453 2112/2113 (Haematology Office) • Fax: +44-(0)20-8965 1115