PID Treatments

Research and advances in therapies have resulted in improved health and a longer life for people with PIDs. There are currently six main types of treatment options: 

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, and this may require long term access.

IMMUNOGLOBULIN REPLACEMENT THERAPY (IRT)

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, typically in a hospital setting, or given at home using subcutaneous immunoglobulin (SCIg) injected under the skin.

These products are derived from pooled 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 – INCLUDING BIOLOGICS

There is an increasing number of drugs (medications) used to increase or decrease immune function.

Medications include corticosteroids, biologics such as monoclonal antibodies, small molecule inhibitors and other immunosuppressive drugs.

Medications that specifically target pathways in genetically defined PIDs (known as targeted therapy or precision medicine) can be effective in some PIDs that have failed standard therapy.

However, many of these are not funded for PID indications. Therefore, they are not accessible unless funding for the affected individual can be negotiated through hospital drug committees or through compassionate access from the drug company.

HEREDITARY ANGIOEDEMA (HAE) TREATMENTS

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

Prophylactic treatment is increasingly important in the management of HAE as it restores quality of life to patients who otherwise live with the ever present threat of debilitating attacks.

Prophylactic management is rapidly evolving with a number of newer, more effective agents becoming available. Improved access to effective prophylaxis with be life-changing for many people with HAE.

HAEMATOPOIETIC STEM CELL TRANSPLANTATION (HSCT)

Current standard of care for definitive correction of SCID is HSCT, which must be performed urgently, as outcomes are best when performed at an early age with no active infection.

HSCT should only be undertaken in a specialist centre with suitable expertise and facilities to isolate and manage infants with SCID.

HSCT is also being used for other PID in adults and children and can be curative, such as CGD and some combined immune deficiencies.

However, this form of treatment requires specialist expertise in centres with recognition and experience in PID.

GENE THERAPY

Rather than replacing a person’s immune system using stem cells from a donor (bone marrow transplant), for a small number of PIDs with known gene defects, it is possible to treat patients using gene therapy.

This involves collecting stem cells from the affected individual, modifying them to include a healthy copy of the gene and returning them to the person to develop functioning immune cells.

Gene therapy is already commercially available in Europe for adenosine deaminase (ADA) SCID, with trials ongoing for several other PIDs.

It is important to develop clinical expertise in managing gene therapy, including the collection, chemotherapy and reinfusion elements similar to HSCT.  

This is the model that has already been adopted for chimeric antigen receptor T (CAR-T) cell treatment in leukaemia, which is available in Australia.

 

Content updated November 2020