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PID Treatments

Research and advances in therapies have resulted in improved health and a longer life for people with primary immunodeficiencies (PIDs). There are currently five main types of treatment options for PIDs.

Antibiotics

Infections should be treated early, and antibiotics are often required. Some people may be prescribed long term antibiotics (prophylaxis) to reduce infections. Medications against fungi, viruses and parasites may be needed to treat some conditions.

Immunoglobulin Replacement Therapy (IRT)

Immunoglobulin replacement therapy (IRT) is one of the most effective and commonly used treatments for some PIDs. IRT can be given using intravenous immunoglobulin (IVIG), that is injected into the vein, or given at home using subcutaneous immunoglobulin (SCIG), that is injected under the skin.

These products are derived from blood (plasma), are in limited supply, and access is restricted. Doctors must follow specific guidelines to ensure that the product goes to people most in need.

Immunomodulation

There is an increasing number of medications used to increase or decrease immune function. Medications may include corticosteroids, biologics such as monoclonal antibodies, and other immunosuppressive drugs.

Haematopoietic Stem Cell Transplant (HSCT)

For some people with PIDs, a haematopoietic stem cell transplant (HSCT) may be recommended, due to the severe nature of their condition. HSCT provides a new source of stem cells, that are obtained from donated bone marrow or blood.  Stem cells are able to develop into all types of blood cells, including T cells and B cells, which produce antibodies.  HSCT usually cures the underlying PID, but has risks, and must be performed in specialist centres.

For infants with severe combined immunodeficiency (SCID), HSCT is required to survive. Newborn screening for SCID is routinely performed in New Zealand, the United States and in some European countries. Early diagnosis by newborn screening for SCID allows for HSCT to be undertaken before infections cause complications, that may be life threatening.

Hereditary Angioedema (HAE) Treatments

There are two main treatments for severe, acute HAE attacks:

  • Purified C1 esterase inhibitor for intravenous use in hospitals or at home.
  • Icatibant for emergency treatment, which can be self-administered at home.

Tranexamic acid, danazol or C1 esterase inhibitor are sometimes given regularly to prevent attacks (as prophylaxis) to people with frequent HAE attacks.

For more information go to www.immunodeficiencies.org.au 

Content created March 2019


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