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DoH IVIG Workshop Update, neurology usage and outcomes measurement Dr Michael Lunn National Hospital for Neurology and Neurosurgery Queen Square, London WC1N 3BG 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? IVIG in Neurology Usage update Neurology Infusions by Diagnosis 01/04/2010 - 31/03/2011 Diagnosis Grams Used % Of Total Chronic inflammatory demyelinating polyradiculoneuropathy 473649.1 48.33% Multifocal motor neuropathy 224303.7 22.89% Guillain–Barré syndrome 97814.5 9.98% Myasthenia gravis 69221.5 7.06% Other (Neurology) 45410 4.63% Other (Other) 11697.5 1.19% Paraprotein-associated demyelinating neuropathy (IgM) 11642.5 1.19% Stiff person syndrome 10255 Paraprotein-associated demyelinating neuropathy (IgG or IgA) 7969 94.89% 1.05% 0.81% Paraneoplastic disorders 5196.5 0.53% Acute disseminated encephalomyelitis 3902.5 0.40% Polymyositis 3580 0.37% Lambert Eaton myasthenic syndrome 3215 0.33% Vasculitic neuropathy 2735 0.28% Rasmussen syndrome 2215 0.23% Neuromyotonia 1380 0.14% Multiple sclerosis 1330 0.14% Critical illness neuropathy 1145 0.12% CNS vasculitis 730 0.07% Inclusion body myositis 600 0.06% Bickerstaff's brain stem encephalitis 510 0.05% 442.5 0.05% Dermatomyositis 370 0.04% Autism 360 0.04% Acute idiopathic dysautonomia 175 0.02% Autoimmune diabetic proximal neuropathy 110 0.01% Chronic fatigue syndrome Neurology Patients by Diagnosis 01/04/2010 - 31/03/2011 Diagnosis Patients % Of Total Chronic inflammatory demyelinating polyradiculoneuropathy 854 34.24% Guillain–Barré syndrome 633 25.38% Multifocal motor neuropathy 353 14.15% Myasthenia gravis 296 11.87% Other (Neurology) 93 3.73% Other (Other) 62 2.49% Paraneoplastic disorders 27 1.08% Paraprotein-associated demyelinating neuropathy (IgM) 27 1.08% Stiff person syndrome Acute disseminated encephalomyelitis 27 21 1.08% 0.84% Paraprotein-associated demyelinating neuropathy (IgG or IgA) 16 0.64% Polymyositis 15 0.60% Rasmussen syndrome 10 0.40% Vasculitic neuropathy 10 0.40% Lambert Eaton myasthenic syndrome 9 0.36% Multiple sclerosis 8 0.32% Neuromyotonia 6 0.24% CNS vasculitis 6 0.24% Critical illness neuropathy 5 0.20% Bickerstaff's brain stem encephalitis 5 0.20% Chronic fatigue syndrome 3 0.12% Inclusion body myositis 3 0.12% Intractable childhood epilepsy 1 0.04% Dermatomyositis 1 0.04% Acute idiopathic dysautonomia 1 0.04% Autism 1 0.04% Autoimmune diabetic proximal neuropathy 1 0.04% 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 90.7% capture • In 2009 260 GBS pts in database • Thus ?48 – 60% capture • In 2010 – 633 cases ?almost complete – 90%? Guidelines update • 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. Reassignment of existing indications /inclusion of new indications. Awaiting panel decision Not approved (rejected) 8 Exceptionality 2 1 1 1 3 1 Paraneoplastic disorders 1 1 Polymyositis 2 2 2 Vasculitic neuropathy Other (Neurology) Grey: short term Acute disseminated encephalomyelitis Acute idiopathic dysautonomia Bickerstaff's brain stem encephalitis Intractable childhood epilepsy Grey: long term Blue: short term Blue: long term Red Diagnosis Panel decision not recorded ‘Grey’ diagnosis usage 2009 7 20 4 1 4 10 5 1 1 14 23 1 6 2 1 13 2 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’ 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 Grey indications - changes • Immune-mediated disorders with limited evidence of immunoglobulin efficacy • Presumed immune-mediated disorders with little or no evidence of efficacy 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 • Opsoclonus Myoclonus 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 • Opsoclonus Myoclonus • 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 Abmediated encephalitis since 2009 • 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. 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 There remain rare disorders…. Efficacy outcomes to assess treatment success 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.’