DoH IVIG Workshop Update, neurology usage and outcomes measurement Dr Michael Lunn National Hospital for Neurology and Neurosurgery Queen Square, London.

DoH IVIG WorkshopUpdate, neurology usage and

outcomes measurement

Dr Michael LunnNational 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

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 1.05%

Paraprotein-associated demyelinating neuropathy (IgG or IgA) 7969 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%

Chronic fatigue syndrome 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%

Neurology Infusions by Diagnosis 01/04/2010 - 31/03/2011

94.89%

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 27 1.08%Acute disseminated encephalomyelitis 21 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%

Neurology Patients by Diagnosis 01/04/2010 - 31/03/2011

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

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.

‘Grey’ diagnosis usage 2009Diagnosis

RedBlue: long term

Blue: short term

Grey:

long term

Grey:

short

term

Exceptionalit

y

Not appro

ved (reject

ed)

Awaiting

panel decisi

on

Panel decision

not recorde

d

Acute disseminated encephalomyelitis

2 8 1

Acute idiopathic dysautonomia

1

Bickerstaff's brain stem encephalitis

1 3

Intractable childhood epilepsy 1

Paraneoplastic disorders 1 1 1 4

Polymyositis 2 2 2 10 5 1 2

Vasculitic neuropathy 1 1

Other (Neurology) 7 20 4 14 23 6 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 Ab-mediated 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.’