PIDD is a group of more than 450 hereditary immunodeficiency disorders caused by genetic defects that affect the immune system. People with PIDD may be more susceptible to infections, especially those caused by bacteria, viruses, and fungi.
Antibody deficiencies are the most common type of PIDD, accounting for more than 50% of disorders.7
Time to diagnosis often takes many years. Symptoms are heterogeneous and can complicate the diagnosis.4,8
20 years is the average time a person goes undiagnosed8
67% of patients were not diagnosed with PIDD until ≥35 years of age8
44% of patients experienced permanent functional impairment while waiting for diagnosis9
54% of patients reported that the most common functional decline is lung function9
Patients with PIDD are up to 10 times more likely to develop lymphoma10*
*The excess relative risk of lymphoma was 10-fold in men (P<0.001) and 8-fold in women (P<0.001).10
Living with undiagnosed PIDD adversely affects prognosis and increases patient risk for1, 2:
Chronic complications
More frequent and longer hospital stays
Psychosocial burdens
Reduced quality of life
A total of 490 physicians from 192 centers identified 60,364 patients with defined PIDD, of which 23.4% (14,140) required IG replacement therapy.
Before diagnosis
$138,760
After diagnosis and start of IG therapy
$60,297
Annual Savings
$78,166
| Annual cost per patient | |
|---|---|
|
Before diagnosis |
$138,760 |
|
After diagnosis and start of IG therapy |
$60,297 |
|
Annual Savings |
$78,166 |
Most common symptoms to consider for identifying a patient with PIDD12, 13
Recurrent, unusual, or difficult-to-treat ear infections
Multiple courses of antibiotics or IV antibiotics to clear infections
Two or more new sinus infections within 1 year, in the absence of allergy
Recurrent, deep abscesses of the skin or internal organs
Recurrent pneumonia, ear infections, or sinusitis
Persistent thrush or fungal infection on skin or elsewhere
Chronic diarrhea with weight loss
Infection with normally harmless tuberculosis-like bacteria
Recurrent viral infections (colds, herpes, warts, condyloma)
Swollen lymph glands or an enlarged spleen
Autoimmune disease
Family history of PIDD
PIDD can affect multiple systems in the body. Download our guide to learn about signs and symptoms that may indicate that testing is needed.
Tests to confirm a PIDD diagnosis include blood tests to identify specific immune system abnormalities14:
If these tests are inconclusive and clinical suspicion of an antibody deficiency remains, it may be appropriate to conduct further testing and/or seek a second opinion.14
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Xembify Connexions™ can help you and your patients
Indication
XEMBIFY® (immune globulin subcutaneous human–klhw) is a 20% immune globulin indicated for treatment of primary humoral immunodeficiency disease (PIDD) in patients 2 years of age and older. XEMBIFY is for subcutaneous administration only.
Important Safety Information
WARNING: THROMBOSIS
Contraindications
XEMBIFY is contraindicated in patients who have had an anaphylactic or severe systemic reaction to the administration of human immune globulin. It is contraindicated in IgA-deficient patients with antibodies against IgA and a history of hypersensitivity.
Warnings and Precautions
Hypersensitivity. Severe hypersensitivity reactions may occur with immune globulin products, including XEMBIFY. In case of hypersensitivity, discontinue infusion immediately and institute appropriate treatment. XEMBIFY contains IgA. Patients with known antibodies to IgA may have a greater risk of developing potentially severe hypersensitivity and anaphylactic reactions.
Thrombosis. Thrombosis may occur following treatment with immune globulin products, including XEMBIFY. Thrombosis may occur in the absence of known risk factors. In patients at risk, administer at the minimum dose and infusion rate practicable. Ensure adequate hydration before administration. Monitor for signs and symptoms of thrombosis and assess blood viscosity in patients at risk of hyperviscosity.
Aseptic meningitis syndrome (AMS). AMS may occur with human immune globulin treatment, including XEMBIFY. Conduct a thorough neurological exam on patients exhibiting signs and symptoms to rule out other causes of meningitis. Discontinuation of treatment has resulted in remission within several days without sequelae.
Renal dysfunction/failure. Acute renal dysfunction/failure, acute tubular necrosis, proximal tubular nephropathy, osmotic nephrosis, and death may occur with use of human immune globulin products, especially those containing sucrose. XEMBIFY does not contain sucrose. Ensure patients are not volume-depleted prior to starting infusion. In patients at risk due to preexisting renal insufficiency or predisposition to acute renal failure, assess renal function prior to the initial infusion of XEMBIFY and again at appropriate intervals thereafter. If renal function deteriorates, consider discontinuation.
Hemolysis. XEMBIFY may contain blood group antibodies that may cause a positive direct antiglobulin reaction and hemolysis. Monitor patients for clinical signs and symptoms of hemolysis. If signs and symptoms are present after infusion, perform confirmatory lab testing.
Transfusion-related acute lung injury (TRALI). Noncardiogenic pulmonary edema may occur in patients following treatment with immune globulin products, including XEMBIFY. Monitor patients for pulmonary adverse reactions. If TRALI is suspected, perform appropriate tests for the presence of antineutrophil and anti-HLA antibodies in both the product and patient serum. TRALI may be managed using oxygen therapy with adequate ventilatory support.
Transmissible infectious agents. Because XEMBIFY is made from human blood, it may carry a risk of transmitting infectious agents, eg, viruses, the variant Creutzfeldt-Jakob disease (vCJD) agent, and, theoretically, the Creutzfeldt-Jakob disease (CJD) agent. No cases of transmission of viral diseases, vCJD, or CJD have ever been associated with the use of XEMBIFY.
Interference with lab tests. After infusion of XEMBIFY, passively transferred antibodies in the patient’s blood may yield positive serological testing results, with the potential for misleading interpretation.
Adverse Reactions
The most common adverse reactions in ≥ 5% of subjects in the clinical trial were local adverse reactions, including infusion-site erythema (redness), infusion-site pain, infusion-site swelling (puffiness), infusion-site bruising, infusion-site nodule, infusion-site pruritus (itching), infusion-site induration (firmness), infusion-site scab, infusion-site edema, and systemic reactions including cough and diarrhea.
Drug Interactions
Passive transfer of antibodies may transiently interfere with the immune responses to live attenuated virus vaccines (eg, measles, mumps, rubella, and varicella).
Please see accompanying full Prescribing Information for XEMBIFY.
Terms to know
IG, immune globulin; IgA, immunoglobulin A; IgE, immunoglobulin E; IgG, immunoglobulin G; IgM, immunoglobulin M; IV, intravenous; PI, primary immunodeficiency; PIDD, primary immunodeficiency disease.
References