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How can Primary Immunodeficiencies be treated?

With proper treatment, many people with PI experience a dramatic improvement in their health, well-being and quality of life.

Immunoglobulin Replacement Therapy

The most common treatment option for PI patients is Immunoglobulin (Ig) replacement therapy. Immunoglobulin refers to the liquid plasma part of blood that contains immunoglobulins or antibodies. These replacement antibodies help fight infections that your body cannot.

Health Canada licenses several Ig products for patients with PI, all produced outside Canada. Canadian Blood Services distributes the Ig products at no cost to the patient. 

Ig replacement therapy can be administered in two ways:

  • Intravenous (IVIg) – through an IV needle and pump, usually given in a hospital or clinic setting once a month or;
  • Subcutaneous (SCIg or SubQ ) – through the fat tissues (stomach, thighs or upper arms), usually administered at home weekly.

Both formulations are considered equally effective; deciding which treatment is right for you will be up to you and your healthcare provider. The important thing to remember is that each method is safe and will help keep you healthy if properly administered.

Hematopoeitic Stem Cell Transplantation (HSCT)

Hematopoietic Stem Cell Transplantation (HSCT) is typically used for patients with severe forms of primary immunodeficiency that are likely to cause significant harm or be fatal if not corrected. The procedure has a much higher success rate when performed as early as possible, ideally within the first 3.5 months of life.

HSCT and BMT procedures have excellent outcomes when performed with a matched donor with identical Human Leukocyte Antigens (HLAs). Siblings are the best option for a potential donor, with a 1 in 4 chance (25%) of being an HLA match.

Newborn screening is critical for the early detection of severe primary immunodeficiency and successful treatment via stem cell transplantation.

Bone Marrow Transplants

Stem cells have traditionally been taken from bone marrow, but today they can be collected from peripheral blood or umbilical cord blood. 

Gene Therapy

Gene therapy is a rapidly growing experimental approach for treating genetic diseases. With gene therapy, a faulty gene is repaired or replaced by a new, normal copy of the gene. 

The procedure commonly uses a virus to help carry the new gene into a cell’s genome, though researchers are testing new delivery methods (called vectors). The virus’ own genes are removed and then replaced by the new gene. When the virus enters the body’s cell, it inserts the genetic material, and if successful, the cell will now carry a new gene that corrects the faulty gene and the problems it causes.

Although it is not an approved treatment option in Canada at this time, gene therapy may present a life-saving option for patients with severe primary immunodeficiency who do not have a matched donor. For this reason, some patients travel to the US and Europe to undergo the therapy.

Antibiotics & Antifungals

Antibiotics are medicines that help fight bacterial infection. They are commonly prescribed to help treat and prevent diseases by killing or inhibiting the growth of bacteria.

Patients with primary immunodeficiencies are more likely to get infections, and most will be treated with antibiotics. But there is a danger in the overuse of antibiotics, especially when they are prescribed to treat viral infections rather than bacterial ones.

Occasionally, a doctor may prescribe long-term use of antibiotics for a patient with PI, either as a treatment or for prevention (prophylaxis). This may help guard against bacterial infections for patients with a history of sinus and pulmonary issues. However, the use of antibiotics prophylactically is debated in the healthcare community due to the potential for developing antibiotic resistance.

Please consult a healthcare professional if you suspect that an infection requires antibiotics. You should discuss all decisions with your healthcare provider.

We’re sick and tired, but we’re not alone.

ImmUnity Canada is a national charity with five provincial chapters across the country.

We empower Canadians impacted by immunodeficiency disorders to live well through education, support, advocacy, community-building, and research.

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