Haemoglobinopathy , a challenge in service design

Prevalence very variableMinority communities

Little political “ muscle “ English not first language

Often low socio-economic status Asylum seekers

Seen as “ not our problem “

Sickle cell disease is the most common genetic condition in England, with higher prevalence than cystic fibrosis

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)

Affects an estimated ~10,000 individuals (audit 2008)

Reported in more than 1 in 2,000 live births

Most common inherited disease of Caucasians (recessively inherited)

Affects an estimated ~8,400 individuals

Reported in more than 1 in 2,500 live births

Sickle Cell Disease & Thalassemia1

Cystic Fibrosis2

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 20072. Source: Specialist Services National Definitions Set (2nd Edition)

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

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%

??

?

?

UK Thalassaemia Register

1999:807 patients / 164

doctors

71 only 1 attending77 2 – 9

patients12 10 – 304 > 50

11 doctors @ 9 sites saw 20 or more

patients

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

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

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

Super-specialist services eg specifically experienced

BMT, fertility clinic

Clinic

Clinic

Centre

Specialist Team

Annual Review

Special investigations

New treatment options

Liaiases with Clinic / GP

Clinic undertakes: regular transfusions and pre-transfusion review, offers ready access, prescribes chelation, regular tests.

Refers / liaises with Centre

Primary care, outreach and community

services

WORK

All need informedfront line / A&E staff

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 .

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 .

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

What exists at present

• Clinical standards • Specialist definition • Some very well developed formal and

informal groups • Commissioner engagement • Some excellent practise • Registry • Goodwill

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

• Design Sickle services for the future– Review workforce requirement (specialist services)– Support national leadership

1

2

3

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

With policy makers

With clinicians and care providers

With commissioners(at both PCT and SCG level

Involving patients and the Sickle cell society

Design Sickle services for the future

Develop best practice care pathways

Improve service delivery

1

2

3

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

What is in process

• Peer review

• Expansion of Registry to include adverse event reporting and annual review

• Specialist commissioning overview

Some themes

• Inequality both within individual networks and between networks .

• Access

• Community/specialist nursing support

• manpower ,psychology particularly poor .

• Second string to cancer

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

Moyamoya

Severe stenosis or occlusion of the terminalinternal carotid artery / proximal middlecerebral artery with collateral vessels Yoon 2000

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 .

Children aged 2 – 6 yearsInitial TCD Study

Normal< 170 cm/s

Conditional170-199 cm/s

Abnormal 200 cm/s

Restudy1 year

Low Conditional170-184 cm/s

High Conditional185 -199 cm/s

Restudy4 months

Restudy2 weeks

Unchanged6 months

Abnormal200 - 219 cm/s

Abnormal 220 cm/s

Restudy2 weeks

Begin transfusion

Unchanged1 year

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.

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 .

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 . .

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

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 .

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 . .

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 .

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 .

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 .

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 .

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

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

MRI T2*MRI T2*Lack of Correlation: Liver and Cardiac IronLack of Correlation: Liver and Cardiac Iron

LiverLiverLiverLiver

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 .

Comparison of Currently Available Iron Chelators

Approved in US, Switzerland, and

30 countries

Licensed outside US/Canada

(approved in 46 countries)

LicensedStatus

FaecalUrinaryUrinary, faecalExcretion

12-16 hr3-4 hr20-30 minHalf-life

Oral

(Once daily)

Oral

(3 times daily)

SC, IV

(8-12 hr, 5 d/wk)Route

20-307525-60Usual dose(mg/kg/d)

DeferasiroxDeferiproneDesferrioxamineProperty

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

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

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 .

There are high concentrations of children with sickle cell disease in key cities – particularly London, Manchester and Birmingham

Milton KeynesCambridge

Luton

London

Sheffield

Nottingham

Leicester

Northampton & Kettering

Newcastle

Leeds

Manchester

Liverpool

Oxford

Portsmouth

Bristol

Birmingham

Reading

Southampton

Plymouth

Royal London

Great Ormond St

Whipps Cross

Central Middlesex

Whittington

University College

Royal Free

North Middlesex

St Mary’s

Ealing

Hillingdon

King’s College

Guy’s & St. Thomas’s

University Hospital, Lewisham

QE Hospital, Woolwich

St George

St Helier

River Thames

London

51-100

21-50

<21

>100

Number children with sickle cell disease

Unknown

BHR

Medway Maritime Hosp

Mayday University Hospital

Source: Sickle Cell Anaemia Survey (May, 2008)

There are a number of work areas which have been identified for short-term action to improve treatment of Haemoglobin disorders

Enhance service provision

Improve training & education

Raise the profile

• 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

• Continue to work on education for doctors, scientists and nurses • Improve education – particularly for non-haematologists:

- GPs/A&E doctors/Anaesthetists

• Roll out a comprehensive, national database for capturing and analysing prevalence and treatment data and outcomes (NCEPOD)Capture key data

Provide better coordination

• Ensure full involvement of key stakeholder groups:- Policymakers, commissioners and patients- Royal Colleges including Paediatricians and Nursing - Regular meetings of clinicians with key stakeholders

• 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