Immunology/Neurology

IMMUNODEFICIENCIES

When one of the parts of the immune system is missing or does not work well, we say that the immune system is deficient.  Most often this involves missing or defective T- or B-lymphocytes or inadequate production of antibodies.  The result is that the body is vulnerable to infections that might otherwise be easily defeated.

Immunodeficiencies can be “Primary”, i.e., present at birth and usually genetic, or “Secondary”. Secondary Immunodeficiencies have many causes, including disease, malnutrition, aging, certain medications, chemo- and radiation therapy, and stress.  Probably the most well-known cause of immunodeficiency, though not the most common, is the Human Immunodeficiency Virus (HIV), which can cause AIDS (Acquired Immune Deficiency Syndrome).

There are some 185 Primary Immunodeficiencies recognized by the World Health Organization. Most common are those involving the production of antibodies and are called Primary Antibody Deficiencies Diseases (PADs).  These disorders vary greatly in their underlying defects, but many of them can be managed and their symptoms mitigated by regular infusions of immunoglobulin.

Kedrion’s ongoing research and development has resulted in several immunoglobulin therapies for these conditions. Replacement therapy with immunoglobulin in primary antibody deficiencies increases life expectancy and reduces infection frequency and severity (1).

Reference:

1. Orange, Jordan S. et al.,”Use of intravenous immunoglobulin in human disease: A review of evidence by members of the primary immunodeficiency committee of the American Academy of Allergy, Asthma and Immunology”. Journal of Allergy and Clinical Immunology 117.4 (April 2006): S525-53.

Neurology

Chronic inflammatory demyelinating polyneuropathy (CIDP).  CIDP can be thought of as a chronic form of the autoimmune disorder Guillain-Barré syndrome caused by demyelination of peripheral nerves, resulting in loss of sensation, motor weakness, and sensory symptoms (1).

Its estimated prevalence ranges from 0.8 to 8.4 per 100,000 people. CIDP is often disabling with over 50% of patients having temporary disability and about 10% eventually becoming persistently disabled or dying because of the disease.  The cause of CIDP remains unknown, but there are data supporting an immune pathogenesis. Plasmapheresis (plasma exchange) (4), corticosteroids (3,5) and intravenous immunoglobulin (IVIg) (2,6) therapy are effective treatments, but should be started early to avoid permanent nerve damage.

Reference:

1.    Köller H et al.; Chronic Inflammatory Demyelinating Polyneuropathy. N Engl J Med. 2005 Mar 31;352(13):1343-56.
2.    Mahdi-Rogers M et al.; Overview of the Pathogenesis and Treatment of Chronic Inflammatory Demyelinating Polyneuropathy with Intravenous Immunoglobulins. Biologics. 2010 Mar 24;4:45-9.
3.    E. Nobile Orazio. Intravenous Immunoglobulin Versus Intravenous Methylprednisolone for Chronic Inflammatory Demyelinating Polyradiculoneuropathy: A Randomised Controlled Trial. Lancet Neurol 2012; 11 (6): 493-502.
4.  Dyck PJ, et al. A plasma exchange versus immune globulin infusion trial in chronic inflammatory demyelinating polyradiculoneuropathy. Ann Neurol. 1994;36(6):838-845.
5. Dyck P.J., O’Brien P.C., Oviatt K.F., Dinapoli R.P., Daube J.R., Bartleson J.D., et al. (1982) Prednisone improves chronic inflammatory demyelinating polyradiculoneuropathy more than no treatment. Ann Neurol 11: 136–141.
6. Hughes RAC, et al., “Intravenous immunoglobulin (10% caprylate-chromatography purified) for the treatment of chronic inflammatory demyelinating polyradiculoneuropathy (ICE study): a randomised placebo-controlled trial”. Lancet Neurology.; 7.2 (2008): 136-44.