Haemoglobinopathy , a challenge in service design

1. Haemoglobinopathy , a challenge in service design Prevalence very variable Minority communities Little political “ muscle “ English not first language Often low socio-economic status Asylum seekers Seen as “ not our problem “

2. Sickle cell disease is the most common genetic condition in England, with higher prevalence than cystic fibrosis Sickle Cell Disease & Thalassemia1 Cystic Fibrosis2 Affects an estimated ~10,000 individuals (audit 2008) Affects an estimated ~8,400 individuals Reported in more than 1 in 2,000 live births Reported in more than 1 in 2,500 live births Highest prevalence of SCD occurring in people of African and Afro-Caribbean origin, with birth prevalence as high as 1 in 300 in some areas whilst Thal more common in Asian and Southern European (recessive) Most common inherited disease of Caucasians (recessivelyinherited) In the UK, sickle cell disease is as prevalent as cystic fibrosis – the only difference is in the ethnic group most likely to be impacted 1. Source: Development of transcranial Doppler screening services in England, Dianne Addei, Nov 2007 2. Source: Specialist Services National Definitions Set (2nd Edition)

3. The Process • Define population at risk demographics, numbers and disease • what exists at present • what do we aspire to ,define standards • what is the gap • how to move from existing services to new clinical units • how to maintain /continually improve services

4. Haemoglobinopathy Forum survey of existing resources • 65% see 5 or less Sickle per year • 88% see 5 or less Thal per year • Of 21 units seeing greater than 20 children with Sickle 17 in London • Response from 88%

5. UK Thalassaemia Register • 1999: • 807 patients / 164 doctors • 71 only 1 attending • 77 2 – 9 patients • 10 – 30 • 4 > 50 • 11 doctors @ 9 sites saw 20 ormore • patients ? ? ? ?

6. Survey ( cont) • of smaller units < 20 per year 76% would treat painful crises but only 40% would treat Acute chest or Priapism • Of larger units 14 would refer on for neurologic episode needing MRI and for ICU facilities • Only 6 units have facility for TCD

7. Conclusions • Services best described as “patchy” • Little organisation about who does what • Particular concern is links or not of small units • Urgent need for standards and agreed networks of care

8. Drivers for change • Screening programme • Standards for adults and children • Treatment/care for long term conditions • NCEPOD report into Haemoglobinopathy mortality • Haemoglobinopathy coming under SSA remit • Patient expectations

9. Clinic Centre Specialist Team Annual Review Special investigations New treatment options Liaiases with Clinic / GP Primary care, outreach and community services Clinic Super-specialist services eg specifically experienced BMT, fertility clinic Clinic undertakes: regular transfusions and pre-transfusion review, offers ready access, prescribes chelation, regular tests. Refers / liaises with Centre WORK All need informed front line / A&E staff

10. What is missing • Any infrastructure , need agreement on core components • Detailed data • Tariffs fairly basic • Understanding of how they will be supported long term • An overview of training and education needs of the networks .

11. Standards • Thalassaemia major completed three years ago • sponsored by Thalassaemia society • multidisciplinary group • backing of DH • main theme is promoting the development of a patient and family centred service .

12. Sickle Standards • Again produced by multidisciplinary group including patient representative group Sickle cell society • Themes very similar to Thal • Define specialist units and relationship to smaller units • Defines good /best practice

13. What exists at present • Clinical standards • Specialist definition • Some very well developed formal and informal groups • Commissioner engagement • Some excellent practise • Registry • Goodwill

14. The national strategy for Haemoglobinopathy services would drive improvements in care and services Develop best practice care pathways From: Screening and diagnosis To: Treatment and prevention of major illnesses Involving: health and social care Resulting in : Reduced A&E burden Support for patients living with HbO Improve service delivery Design best practice service delivery model to improve consistency and equity of service provision (hospital and community) Roll out hub and spoke model of service centres to enhance provision Enhance commissioning Review funding Improve training and education (NCEPOD) Collect and analyse data (NCEPOD) • Make available the right care, advice and treatment • Tackle inequities • Build for the future 1 2 3 • Design Sickle services for the future • Review workforce requirement (specialist services) • Support national leadership

15. In order to address the unmet needs of sickle cell patients, a comprehensive national strategy is urgently required A national strategy for managing sickle cell disease would deliver multiple benefits … Raise the profile of sickle cell disease across England Drive improvements in care and services Develop best practice care pathways With policy makers 1 With commissioners (at both PCT and SCG level Improve service delivery 2 With clinicians and care providers Design Sickle services for the future 3 Involving patients and the Sickle cell society

16. Objectives • Gathering information about individual units on behalf of DH ,up to date picture of secondary and tertiary services • Performance in key areas against standards • Nature of existing clinical networks • Nature of present commissioning arrangements • Perceived gaps in services and how to improve • Inform DH strategy

17. What is in process • Peer review • Expansion of Registry to include adverse event reporting and annual review • Specialist commissioning overview

18. Some themes • Inequality both within individual networks and between networks . • Access • Community/specialist nursing support • manpower ,psychology particularly poor . • Second string to cancer

19. TIAs, Stroke, Coma9y girl HbSS, previously well, ‘Top of class’

20. Moyamoya Severe stenosis or occlusion of the terminal internal carotid artery / proximal middle cerebral artery with collateral vessels Yoon 2000

21. TCD • Great change from 4 years ago • Theoretical coverage now good only a couple of areas without firm plans to get in place this year . • Challenge now to increase uptake , use of one stop clinics , outreach combined with annual review • Training programme tenders now out .

22. Children aged 2 – 6 years Initial TCD Study Normal < 170 cm/s Conditional 170-199 cm/s Abnormal  200 cm/s Restudy 1 year Low Conditional 170-184 cm/s High Conditional 185 -199 cm/s Abnormal 200 - 219 cm/s Abnormal  220 cm/s Restudy 4 months Restudy 2 weeks Restudy 2 weeks Begin transfusion Unchanged 1 year Unchanged 6 months TCD (non-imaging) blood velocity action limits and follow up protocols for sickle cell children aged between 2 and 6 years. (Time-averaged maximum mean blood velocities) The action limits could be 10% lower when using TCDi to allow for the reported differences between imaging and non-imaging techniques.

23. Specialist nursing • Probably biggest area of unmet need • Need for flexibility in low prevalence areas • suggestion that screening has possibly reduced time available from community side .

24. Community support • Vast range of arrangements , some excellent ,some not ! • Good ones typified by leadership and clarity of roles between Acute Trust and community work • Little use of new recommendations about planned care packages in long term conditions . .

25. Specialist nursing/community • Many trusts need to go back and look at what is needed –ask the patients • Define existing resource , formalise agreement about who does what • Look for resource if not available , • Must be seen as a whole package

26. Specialist/community • Too often service artificially limited by PCT boundaries . • Too often operating as separate teams • Need to include patient groups more in design , monitoring problem solving in this area .

27. Access • Huge variation , again some very good arrangements in low prevalence Trusts ,more easily able to individualise treatment • sometimes second string to cancer • Many continue to be less than satisfied that A and E performance often based on audit data . .

28. Access • Open access and use of either dedicated day case or Haematology day case areas seemed to give high level of satisfaction • Use of A and E with some for of alert , fast track , patient held care plan , next • Lowest level of perceived satisfaction (mainly among providers ) was A and E with no special arrangements .

29. High/Low prevalence • Challenges very different • How to keep up interest/expertise /training • Can often be more flexible • Innovative use of components of other services • Again leadership key factor .

30. Transition • All had arrangements in place • Works most easily when adult/Paed service on same site • Surprisingly little use of compatible protocols • Widespread anxiety/dissatisfaction at services available for the young adult .

31. Neonatal screening • All had clear pathways from result to getting result to clinician/family • Not an automatic link to larger centre • Not always clear whose responsibility to ensure patient actually has been seen .

32. Thalassaemia • Distribution very different from Sickle • All comfortable with concept of regular review at a larger centre even if already had high Sickle expertise • Tendency sometimes to get lost in Sickle numbers

33. Transfusion • Little flexibility • Minimal use of patient/family orientated transfusion • Exchange programmes not readily available • Smaller units often able to be more flexible than larger ones

34. MRI T2* Lack of Correlation: Liver and Cardiac Iron Liver Liver

35. Procurement • A variety of different arrangements • Chelation drugs outside tariff from April 09 so likely that individual application to PCT unless your network negotiates a different arrangement .

37. Networks • What should they have in place -- • Regular meetings , morbidity/mortality • Network audits ,performance in key areas • Discussion development of at least compatible policies /protocols • Defined referral pathways • Defined governance responsibilities • A forum for patient engagement • Data collection

38. Networks • Dissemination /development of good/innovative practice • Key link with local commissioners • ? Investigation of adverse events /incidents • Links with a national group • Client group involvement

39. What do commissioners want • To be offered sensible proposals for all aspects of care in Haemoglobinopathy that are affordable ! • To deal with a small number of key groups • Not to see a proliferation of “ specialist “ arrangements . • They will be looking critically at London , they want a pan London implementation group to develop services in conjunction with professional leads .

40. There are high concentrations of children with sickle cell disease in key cities – particularly London, Manchester and Birmingham Number children with sickle cell disease >100 51-100 21-50 Unknown <21 London North Middlesex Whipps Cross Whittington Newcastle BHR Royal Free Central Middlesex Great Ormond St St Mary’s Royal London River Thames Ealing University College Leeds Hillingdon Guy’s & St. Thomas’s QE Hospital, Woolwich Manchester King’s College Sheffield University Hospital, Lewisham Liverpool St George Nottingham St Helier Leicester Birmingham Mayday University Hospital Northampton & Kettering Cambridge Milton Keynes Luton Oxford London Bristol Reading Medway Maritime Hosp Portsmouth Southampton Plymouth Source: Sickle Cell Anaemia Survey (May, 2008)

41. There are a number of work areas which have been identified for short-term action to improve treatment of Haemoglobin disorders Raise the profile • Ensure full involvement of key stakeholder groups: • Policymakers, commissioners and patients • Royal Colleges including Paediatricians and Nursing • Regular meetings of clinicians with key stakeholders • Address gaps in TCD provision initially on Regional basis • Address shortages in nursing (both hospital and community) and trained TCD ultrasonographers • Formulate a plan for direct access to medical advice/care Enhance service provision Provide better coordination • Introduce regional and national roles for coordinating provision of services and best practice sharing via DH then exemplar sites • Involve the SC society who have done much in involving and informing patients Improve training & education • Continue to work on education for doctors, scientists and nurses • Improve education – particularly for non-haematologists: • GPs/A&E doctors/Anaesthetists Capture key data • Roll out a comprehensive, national database for capturing and analysing prevalence and treatment data and outcomes (NCEPOD)