1. DoH IVIG WorkshopUpdate, neurology usage and outcomes measurement Dr Michael Lunn National Hospital for Neurology and Neurosurgery Queen Square, London WC1N 3BG

2. Outline • Neurology IVIG usage update • Neurology in 2011 Guidelines Update • Measuring outcomes • Update guidelines • Measuring outcomes in clinical practice • What’s wrong with what we are using • How can we make it better?

3. IVIG in Neurology Usage update

6. 94.89%

8. How inclusive is the database data now? • In 2009 PASA estimated only 60% data capture • GBS cases • 1.2-1.5 per 100000 • 720-900 cases • 60% require Rx • In 2009 260 GBS pts in database • Thus ?48 – 60% capture • In 2010 – 633 cases ?almost complete – 90%? 90.7% capture

9. Guidelines update

10. This update did not review all of the Second Edition Guidelines content, but limited its focus to three key areas • defining selection criteria for appropriate use; • efficacy outcomes to assess treatment success; • reassignment of existing indications /inclusion of new indications.

11. Reassignment of existing indications /inclusion of new indications.

12. ‘Grey’ diagnosis usage 2009

13. 2009 • 24 patients with polymyositis • Some life threatening • >50% approved • Polymyositis likely to be immune mediated • IBM (previously black) • 2 cases only approved as exceptionality • Not infrequently ‘inflammatory’

14. Myositis criteria • Diagnosis of myositis by a neurologist, rheumatologist, immunologist of: • Patients with PM or DM who have significant muscle weakness; • OR Dysphagia and have not responded to corticosteroids and other immunosuppressive agents; • OR Patients with IBM who have dysphagia affecting nutrition (NOT patients with rapidly progressive IBM) • Outcomes and test dosage schedule suggested

15. Grey indications - changes • Immune-mediated disorders with limited evidence of immunoglobulin efficacy • Presumed immune-mediated disorders with little or no evidence of efficacy

16. Immune-mediated disorders with limited evidence of immunoglobulin efficacy • Acute disseminated encephalomyelitis • Autoimmune encephalitis (including NMDA and VGKC antibodies, among others) • Cerebral infarction with antiphospholipid antibodies • Chronic regional pain syndrome • CNS vasculitis • Intractable childhood epilepsy • Neuromyotonia • OpsoclonusMyoclonus

17. Immune-mediated disorders with limited evidence of immunoglobulin efficacy • Acute disseminated encephalomyelitis • Autoimmune encephalitis (including NMDA and VGKC antibodies, among others) • Cerebral infarction with antiphospholipid antibodies • Chronic regional pain syndrome • CNS vasculitis • Intractable childhood epilepsy • Neuromyotonia • OpsoclonusMyoclonus

18. Autoimmune encephalitis (including NMDA and VGKC antibodies, among others) and neuromyotonia • Granerod J et al 2009 – Lancet Infectious Disease • 203 patients with encephalitis in UK in 2006-2008 • 42% infectious, 21% autoimmune, 37% unknown • 16 case reports and small series of IVIG responsive Ab-mediated encephalitis since 2009

19. Complex Regional Pain Syndrome • Goebel A, Baranowski A, Maurer K, Ghiai A, McCabe C, Ambler G. • Intravenous immunoglobulin treatment of the complex regional pain syndrome: a randomized trial. • Ann Intern Med. 2010 Feb 2;152(3):152-8.

20. Presumed immune-mediated disorders with little or no evidence of efficacy • Acute idiopathic dysautonomia • Diabetic proximal neuropathy • PANDAS • Paraneoplastic disorders that are known not to be B- or T-cell mediated • POEMS

21. There remain rare disorders….

22. Efficacy outcomes to assess treatment success

23. Measuring outcomes:Current practice, potential and future possibilities ‘This update provides efficacy outcomes to be measured in all indications…. Efficacy outcomes are expected to play an important role in the IAP decision-making process for patients……This change reflects the wider change of focus in the NHS to patient outcomes, as presented in The NHS Outcomes Framework.’