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1.
Expert Rev Neurother ; 18(7): 573-588, 2018 07.
Article in English | MEDLINE | ID: mdl-29932785

ABSTRACT

INTRODUCTION: Myasthenia gravis (MG) is an autoimmune neuromuscular disorder with no cure and conventional treatments limited by significant adverse effects and variable benefit. In the last decade, therapeutic development has expanded based on improved understanding of autoimmunity and financial incentives for drug development in rare disease. Clinical subtypes exist based on age, gender, thymic pathology, autoantibody profile, and other poorly defined factors, such as genetics, complicate development of specific therapies. Areas covered: Clinical presentation and pathology vary considerably among patients with some having weakness limited to the ocular muscles and others having profound generalized weakness leading to respiratory insufficiency. MG is an antibody-mediated disorder dependent on autoreactive B cells which require T-cell support. Treatments focus on elimination of circulating autoantibodies or inhibition of effector mechanisms by a broad spectrum of approaches from plasmapheresis to B-cell elimination to complement inhibition. Expert commentary: Standard therapies and those under development are disease modifying and not curative. As a rare disease, clinical trials are challenged in patient recruitment. The great interest in development of treatments specific for MG is welcome, but decisions will need to be made to focus on those that offer significant benefits to patients.


Subject(s)
Myasthenia Gravis/therapy , Autoantibodies/blood , Autoimmunity , Humans , Muscle Weakness/physiopathology , Myasthenia Gravis/diagnosis , Myasthenia Gravis/immunology , Myasthenia Gravis/physiopathology
2.
Muscle Nerve ; 45(3): 363-6, 2012 Mar.
Article in English | MEDLINE | ID: mdl-22334170

ABSTRACT

INTRODUCTION: In this study we estimated the costs paid by U.S. health plans for treating myasthenia gravis (MG) in 2009 and determined the major cost drivers. METHODS: One hundred thirteen MG patients were matched by propensity scores with 339 non-MG patients from a comprehensive health-care insurance database. The mean annual costs paid by the health plan for treating MG, costs by place of service, and costs for intravenous immunoglobulin (IVIg) and plasma exchange were determined. RESULTS: Mean annual costs paid by the health plan per MG patient were $20,190 (SEM $4,763) and costs attributable to treating MG were $15,675. Home health services accounted for 23% of MG patient costs and represented almost exclusively IVIg infusion costs. Six MG patients had a total of 136 outpatient IVIg infusions at an average annual cost of $109,463 ± $57,303. CONCLUSIONS: The estimated annual health plan paid costs for treating MG were $15,675. Home health services represented 23% of MG patient costs, largely driven by IVIg administration.


Subject(s)
Health Care Costs , Insurance Claim Review/economics , Myasthenia Gravis/economics , Myasthenia Gravis/therapy , Female , Humans , Male , Middle Aged , Myasthenia Gravis/epidemiology , Statistics, Nonparametric , United States/epidemiology
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