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PI–General Information

Incidence and prevalence of PI diseases

More than 250,000 people in the United States are diagnosed with primary immunodeficiency (PI) diseases, and thousands more go undetected. PI diseases are intrinsically or genetically occurring defects of the immune system, and comprise a diverse group of illnesses. To date, there are over 130 different diseases that are categorized as primary immunodeficiencies.1

Educational advances have led to a substantial increase in awareness and suspicion of PI diseases in the medical community; however, underdiagnosis remains a concern.

Classifications of PI diseases

Among the subcategories of PI are a group of disorders in which antibody production is reduced or absent (see below).2

Primary immunodeficiency diseases that impair humoral immunity

Defects that exclusively impair humoral immunity

  • X-linked agammaglobulinemia
  • Selective IgA deficiency
  • IgG subclass deficiency

Defects that predominantly impair humoral immunity

  • Common variable immunodeficiency
  • X-linked hyper-lgM syndrome
  • Wiskott-Aldrich syndrome

Defects of both humoral and cell-mediated immunity

  • Severe combined immunodeficiency
  • DiGeorge syndrome

Signs and symptoms of PI

Increased susceptibility to infection is common to all of the PI disorders, with the possible exception of asymptomatic IgA deficiency. However, with the rapid institution of antibiotics, it is rare today to see classic presentations of osteomyelitis, meningitis, or consolidated pneumonia.

More commonly seen are recurrent upper respiratory tract infections, such as otitis media and sinusitis. Less commonly seen are when patients develop mastoiditis, failure to thrive, and chronic/recurrent diarrhea. In some forms of antibody deficiency, such as the hyper-IgM syndrome, autoimmune cytopenias, infection with unusual pathogens such as Pneumocystis carinii or Cryptosporidium parvum infection, or chronic inflammatory disorders (eg, sclerosing cholangitis) may be features of the disease.2

When to suspect PI disease3:

  • Eight or more new ear infections within 1 year
  • Two or more serious sinus infections within 1 year
  • Two or more months of antibiotics with little effect
  • Two or more pneumonias within 1 year
  • Failure of an infant to gain weight or grow normally
  • Recurrent, deep skin or organ abscesses
  • Persistent thrush in mouth or elsewhere on skin, after age 1
  • Need for intravenous antibiotics to clear infections
  • Two or more deep-seated infections such as meningitis, osteomyelitis, cellulitis, or sepsis
  • A family history of PI

PI management guidelines2

Based on information from the Immune Deficiency Foundation Medical Advisory Committee...

PI is a life-long disease that requires an integrated, team approach among the patient, the primary care physician, and an immunologist.

Important Safety Information for GAMUNEX

Gamunex, Immune Globulin Intravenous (Human), 10% Caprylate/Chromatography Purified, is indicated for the treatment of primary humoral immunodeficiency disease (PI), idiopathic thrombocytopenic purpura (ITP), and chronic inflammatory demyelinating polyneuropathy (CIDP).

Immune Globulin Intravenous (Human) products have been reported to be associated with renal dysfunction, acute renal failure, osmotic nephrosis and death. Patients predisposed to acute renal failure include patients with any degree of pre-existing renal insufficiency, diabetes mellitus, age greater than 65, volume depletion, sepsis, paraproteinemia, or patients receiving known nephrotoxic drugs. Especially in such patients, IGIV products should be administered at the minimum concentration available and the minimum rate of infusion practicable. While these reports of renal dysfunction and acute renal failure have been associated with the use of many of the licensed IGIV products, those containing sucrose as a stabilizer accounted for a disproportionate share of the total number. Gamunex does not contain sucrose. Glycine, a natural amino acid, is used as a stabilizer.

Gamunex is contraindicated in individuals with acute severe hypersensitivity reactions to Immune Globulin (Human). It is contraindicated in IgA deficient patients with antibodies against IgA and history of hypersensitivity.

There have been reports of noncardiogenic pulmonary edema [Transfusion-Related Lung Injury (TRALI)], hemolytic anemia, and aseptic meningitis in patients administered with IGIV.

Thrombotic events have been reported in association with IGIV. Patients at risk for thrombotic events may include those with a history of atherosclerosis, multiple cardiovascular risk factors, advanced age, impaired cardiac output, and/or known or suspected hyperviscosity. Hyperproteinemia, increased serum viscosity, and hyponatremia may occur in patients receiving IGIV therapy.

Gamunex is made from human plasma. Because this product is made from human plasma, it may carry a risk of transmitting infectious agents, e.g., viruses, and, theoretically, the Creutzfeldt-Jakob disease (CJD) agent.

In clinical studies, the most common adverse reactions with Gamunex were headache, fever, chills, hypertension, rash, nausea, and asthenia (in CIDP); headache, cough, injection site reaction, nausea, pharyngitis, and urticaria (in PI); and headache, vomiting, fever, nausea, back pain, and rash (in ITP). The most serious adverse reactions were pulmonary embolism (PE) in one subject with a history of PE (in CIDP), an exacerbation of autoimmune pure red cell aplasia in one subject (in PI), and myocarditis in one subject that occurred 50 days post-study drug infusion and was not considered drug related (in ITP).

Please see accompanying GAMUNEX full Prescribing Information for complete prescribing details.

You are encouraged to report negative side effects of prescription drugs to the FDA.
Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.

References:

  1. Immune Deficiency Foundation. Immune Deficiency Foundation applauds legislation to improve patient and provider access to immunoglobulin products. July 28, 2007. http://www.primaryimmune.org/advocacy_center/activities/July_28_2007.pdf. Accessed November 7, 2007.

  2. Gelfand EW, Goldsmith J, Lederman HM. Primary humoral immunodeficiency: optimizing IgG replacement therapy. Immune Deficiency Foundation. http://www.primaryimmune.org/publications/publications.htm. Published October, 2003. Accessed May 28, 2009.

  3. Jeffrey Modell Foundation Medical Advisory Board. Ten warning signs of primary immunodeficiency. http://www.info4pi.org/aboutPI/index.cfm?section=aboutPI&content=warningsigns&CFID=3405760&CFTOKEN=98106331. Accessed May 29, 2009.