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This presentation by Dr. John Booss provides an overview of multiple sclerosis (MS) including its causes, symptoms, diagnosis, types, treatment, and impact on daily living and employment. It explores how MS fits in with the Compassionate Allowance process and suggests an approach to MS in terms of Compassionate Allowances.
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Applying Compassionate Allowances to Multiple Sclerosis Presenter John Booss, M.D., F.A.A.N
Outline • Basic MS background • Causes • Symptoms • Diagnosis • Types of MS • Treatment • New research and potential advances on the horizon • Impact on activities of daily living • Progression and impact on employment • How we think MS fits with the CA process • Challenges that we recognize • Suggested way to approach MS in terms of Compassionate Allowances
What is MS? • A chronic, inflammatory, degenerative, and generally disabling condition affecting the brain, optic nerves, and spinal cord • Peak age of onset in the early 30’s; 70% have onset between 20 and 50; onset can occur at any age • No pathognomonic sign, symptom, or test and so diagnosis is a result of accumulation of clinical and paraclinical evidence • Cause is not known but thought to be autoimmune resulting from genetic predisposition and environmental trigger(s) • More common in females, Caucasians, northern Europeans, and those living farther from equator but also involves other ethnic groups • No cure or prevention at present but many therapies to alter disease course and treat symptoms
myelinated nerve fiber myelinated nerve fiber $721 million cumulative research investment (estimated through 2010) Myelin Nerve Fiber (Quarles RH et al, 2006)
What causes MS? Genetic Predisposition Environmental Trigger(s) Autoimmunity Loss of myelin & nerve fibers
MS interrupts the flow of information between the brain and the body $721 million cumulative research investment (estimated through 2010) Nerve cell with MS damage – may include transection of axon itself Functional nerve cell
MS histopathology – perivascular inflammation (Berger JR, 2008)
Axonal transection in the lesions of MS Transected axon (Trapp, BD et al, 1998)
Self-reported symptoms P<.01 (Minden, SL et al, 2004)
Diagnostic techniques • Clinical signs and symptoms suggestive of CNS demyelination • Magnetic resonance imaging of the brain (more often) and/or spinal cord (less often) • Oligonclonal bands • Evoked potentials (generally visual) • Rule out other possibilities (Polman c, et al, in press)
85% at onset 10-15% $721 million cumulative research investment (estimated through 2010) ~19 yrs ~50% of RRMS develop SPMS 5-10%
Better Prognosis Few attacks early in course Long intervals between attacks Complete recovery from attacks Attacks that are mainly sensory Nearly normal neurological exam after 5 years Young when diagnosed Female Caucasian Worse Prognosis Frequent attacks early in course Incomplete recovery from attacks Early cerebellar and/or brainstem symptoms Gait impairment More lesions on MRI early in course Early development of abnormal neurological exam Prognostic indicators
The Expanded Disability Status Scale Illustrated Zone where disability is very likely (adapted from Kurtzke, J. 1983)
Therapies used in MS • Corticosteroids • Interferon-beta • Copolymer • Chemotherapeutic agents • Monoclonal antibodies • Symptomatic therapies • Rehabilitation
Research and advances on the horizon • Stop the Progression of MS • Development of effective treatments for progressive forms of MS • Development of biomarkers to track progression • Restore Function • Homing in on the most effective rehabilitation strategies • Development of viable neural protection/neural repair therapies • End MS Forever • Enhanced understanding of genetic/environmental contributions to risk • Development of measures to prevent the development of MS in the first place
Needs help with daily activities (Minden, SL et al, 2004)
Need for assistance with ADL’s for RRMS vs PPMS People with PPMS are more than twice as likely to need assistance with ADL’s (Sonya Slifka Longitudinal Study, 2006)
Labor force participation • Approximately 90% of people with MS have a work history • At the time of diagnosis, the labor force participation of people with MS is the same as the general population • Labor force participation declines over time and at present only around 40% of people with MS are employed • The decline in labor force participation soon after diagnosis can be as high as three percentage points per year (Minden, SL et al, 2006) (Sonya Slifka Study, 2006)
Employment status of persons with PPMS vs RRMS drop (Sonya Slifka Study, 2006)
Decrease in percent employed with increasing duration of MS (Sonya Slifka Study, 2006)
Impact of Cognitive Impairment on Daily Functioning(Environmental Status Scale) Work status Social activity Personal assistance Community services Financial status Transportation Personal residence P<0.01 P<0.01 P<0.05 P<0.05 P<0.01 P<0.01 Cognitively intact (n=52) Cognitively impaired (n=48) 0 1 2 3 Mean scale score Worsening (Rao et al., 1991)
How MS fits in with CAL and challenges “The rules for determining disability can be very complicated, but some individuals have such serious medical conditions that their conditions obviously meet our disability standards. To address these individuals’ needs, we strive to provide not only responsive, but also compassionate, public service that ensures the most severely disabled in our society who meet the Act’s requirements are awarded benefits quickly.” Social Security Administration. Compassionate Allowances: Advance notice of proposed rulemaking. Federal Register, Vol 72, No. 146, Tuesday, July 31, 2007. (p. 41649)
How we think MS fits in with CAL and challenges - continued • The fit – A substantial proportion of applicants with MS “obviously meet our disability standards.” • The challenge – Owing to the complexity of MS, there is no single symptom or sign that one can utilize to determine who “obviously meets the standards.” Instead there are several.
Examples of handling MS for CAL $721 million cumulative research investment (estimated through 2010)
Jacqueline du Pré “ …she had little control of her fingering and bowing…” “…there were times when friends attempted to carry her, in the chair, up or down a flight of stairs, and she unceremoniously ended up on the floor…” “…she again had pneumonia, this time there were no options…”
Supplementary slides • Female to male ratio in the MS population • 2010 revision of MS diagnostic criteria • Use of paraclinical evidence in MS diagnosis • Common differential diagnostic conditions • FDA approved drugs for the treatment of MS • FDA approved drugs used to treat the symptoms of MS • Condensed version of the Expanded Disability Status Scale • References
MS is more common among women and the gap seems to be growing Females Total Males (Noonan, CW, et al, 2002)
Diagnostic criteria – revised 2010 (Adapted from Polman, C et al in press)
Paraclinical evidence in MS diagnosis - revised 2010 (Adapted from Polman, C et al in press)
Common differential diagnostic conditions Variant • Neuromyelitis optica (NMO)/Devic’s – recurrent optic neuritis and transverse myelitis extending over 3 or more vertebral segments More Common Mimics • Acute disseminated encephalomyelitis (pediatric cases) • Human T-cell lymphotrophic virus-1 (HTLV-1) • Mild stroke • Neuro Lyme disease Less common mimics • Lupus • Sjogren’s • Myasthenia gravis • Sarcoidosis • Binswanger's • Dural Arteriovenous Fistulas
FDA approved disease modifying therapies • Avonex– weekly IM • Betaseron– every other day SC • Copaxone– daily SC • Extavia– every other day SC • Gilenya– daily oral • Novantrone– every three months IV • Rebif– three times per week SC • Tysabri – every four weeks IV
Examples of FDA approved drugs used to treat the symptoms of MS • Ampyra – approved to treat speed of walking in MS • Baclofen – approved for the treatment of spasticity • Botox – approved to treat upper limb spasticity • Nuedexta – approved to treat pseudobulbar affect • Tizanidine – approved for the treatment of spasticity • Other classes – antibiotics, bladder control medications, antidepressants, pain killers, stool softeners
Expanded Disability Status Scale (abbreviated and paraphrased) 0.0 Normal neurological exam 1.0 No disability, minimal signs in one FS 2.0 Minimal disability in one FS 3.0 Moderate disability in one FS or mild disability in three or four FS 4.0 Fully ambulatory without aid but severe disability in one FS or combination 5.0 Ambulatory without aid for 200 meters; disability impairs full daily activities 6.0 Intermittent or unilateral constant assistance required to walk 100 meters 7.0 Unable to walk more than 5 meters; wheels self in wheelchair 8.0 Restricted to wheelchair or bed; retains some self-care functions 9.0 Confined to bed; needs assistance with all activities; able to communicate and eat 10.0 Death due to MS
References Berger JR. Multiple sclerosis. PowerPoint presentation 2008. Accessed 2/25/2011: http://www.cecentral.com/assets/1487/07_SU_MS_Berger.pdf Kurtzke JF. Rating neurologic impairment in multiple sclerosis: an expanded disability status scale (EDSS). Neurology. 1983; 33:1444-52. Minden SL, Frankel D, Hadden LS, Srinath KP, Perloff JN. Disability in elderly people with multiple sclerosis: An analysis of baseline data from the Sonya Slifka Longidtudinal Multiple Sclerosis Study. Neurorehabilitation 2004; 19:55-67. Minden SL, Frankel D, Hadden L, Perloff J, Srinath KP, Hoaglin DC. The Sonya Slifka Longitudinal Multiple Sclerosis Study: methods and sample characteristics. Multiple Sclerosis 2006; 12:24-38. Noonan CW, Kathman SJ, White MC. Prevalence estimates for MS in the US and evidence of an increasing trend for women. Neurology 2002; 58:136–138. Polman CH, et al. Diagnostic criteria for multiple sclerosis: 2010 revisions to the “McDonald Criteria.” annals of Neurology. (in press).* Quarles RH, Macklin WB, Morrell P. Myelin formation, structure, and biochemistry. In Siegel GJ, Albers RW. (eds.) Basic neurochemistry, Volume I: molecular, cellular, and medical aspects, Seventh Edition. Burlington, MA, 2006, Elsevier Academic Press. (Chapter 4, pp. 51-72). Rao SM, Leo GJ, Ellington L, Nauertz T, Bernardin L, Unverzagt F. Cognitive dysfunction in multiple sclerosis. II. Impact on employment and social functioning. Neurology 1991; 41:692-696. Social Security Administration. Compassionate Allowances: Advance notice of proposed rulemaking. Federal Register, Vol 72, No. 146, Tuesday, July 31, 2007. (p. 41649). Sonya Slifka Longitudinal Multiple Sclerosis Study – findings from the baseline and follow-up interviews, NMSS research contract #HC 0032: 2006. Trapp BD, Peterson J, Ransohoff RM, Rudick R, Mork S, Bo L. Axonal transection in the lesions of multiple sclerosis. New England Journal of Medicine 1998; 338:278-285. *A pocket card summarizing the 2010 revision of the McDonald Criteria is in preparation by the National MS Society.