Top Therapies for Immunodeficiency Disorders


Nearly 500,000 Americans have a form of immunodeficiency disorder. If you or a loved one have any of the 200 forms of the disorder, you understand how difficult it can be to manage the condition.  

Thankfully, modern medicine provides those affected by immunodeficiency disorders with a range of therapeutic options to aid their immune systems in fighting against infection. Each of these options offers patients an opportunity to live a full and active life.

Here are some of the most effective forms of immunodeficiency therapy.  

1. Antimicrobial Therapy

Immunodeficiency disorders limit the immune system’s response to infection. Antimicrobial therapies can either kill or slow the growth of fungi, bacteria, and viruses that have invaded the body.

Antimicrobial therapy typically begins with a series of tests conducted by a doctor to assess whether a patient has been infected by any number of microorganisms. Forms of testing include X-rays, CT scans, and the extraction of cultures from the infected area. Once a medical professional identifies the microorganism, they will then test several medications to determine which is the most effective in combating the source of the infection.

Doctors may also prescribe antimicrobial medications to prevent infection. In such cases, patients will be asked to regularly take the medication.  

2. Intravenous Immunoglobulin Therapy

The body’s immune system responds to infection by creating proteins called antibodies. Antibodies fight against bacteria, viruses, and other foreign bodies. Immunodeficiency disorders prevent the body from creating enough antibodies to fight against infection.

Intravenous Immunoglobulin Therapy, or IVIG therapy, provides the body’s immune system with the antibodies it needs to fight against infection. The name of the therapy refers to the substance from which antibodies are extracted. Liquid immunoglobulin is taken from the plasma of blood donors. It is then tested to ensure it is free of blood diseases such as hepatitis and AIDS, and then purified.

Processed immunoglobulin is then infused into the veins of the patient using a needle. The procedure typically takes between two and four hours. Patients should expect to repeat the treatment every three to four weeks. The therapy can be administered in a range of settings, including hospitals, clinics, doctor’s offices, and even at home accompanied by a medical professional.  

3.  Enzyme Replacement Therapy

Adenosine deaminase is a bodily enzyme that plays a key role in eliminating waste within the body’s immune system. Some individuals are born with a condition called adenosine deaminase deficiency (ADA). Roughly 15 percent of babies with ADA contract an acute immunodeficiency disorder called severe combined immunodeficiency disorder (SCID) within their first six months of life.

People who have SCID are virtually incapable of fighting off bacterial, viral, and fungal infections. If left untreated, patients with SCID typically do not live past the age of two. Fortunately, enzyme replacement therapy (ERT) offers individuals with SCID the chance at a normal life.

ERT involves once- or twice-a-week injections into the muscle of a drug called Revcovi, which contains the missing enzyme. Initially, a medical professional administers the injections. Eventually, however, parents are able to administer the injection at home.

The length of the therapy depends on whether the patient undergoes either Gene Therapy or hematopoietic stem cell transplantation. Doctors can advise parents on whether either treatment is appropriate. If the baby is not suited for either of these alternative treatments, the doctor may recommend they continue ERT indefinitely.  

4.  Gene Therapy

Red Blood Cells

Genetic defects are often the root cause of immunodeficiency disorders. They can prevent the immune system from responding forcefully to infection. For patients with immunodeficiency disorders caused by genetic defects, gene therapy offers the potential for relief. Gene therapy uses a type of cell called hematopoietic stem cells. These stem cells are capable of developing into all types of blood cells.

Doctors first remove defective hematopoietic stem cells from the patient. They then correct the cell’s genes either by introducing a new one into the cell using a virus as a vector, or by editing the existing gene. Once the stem cell’s genes have been corrected, it is then reintroduced to the body. If successful, gene therapy corrects the immune system’s response to infection. As mentioned earlier, gene therapy is still a relatively new approach to treating immunodeficiency disorders. But, it potentially represents a long-term solution for those who suffer from these conditions.    

5.  Hematopoietic Stem Cell Transplantation

Unlike Gene Therapy, in which a hematopoietic stem cell is removed from an immunodeficiency disorder patient, corrected, and then returned, hematopoietic stem cell therapy (HSCT) involves obtaining a stem cell from a donor with properly functioning cells.   The first step in the transplantation process is identifying a donor. This can take up to several months. From there, doctors remove healthy stem cells from the donor. Hematopoietic stem cells can be collected from bone marrow, peripheral blood, or umbilical cord blood.

Before the transplantation process begins, patients must undergo a conditioning regime, in which the patient’s immune system and bone marrow cells are destroyed to prepare for the introduction of the new stem cells. The conditioning regime consists of a combination of chemotherapy and serotherapy. Once the conditioning is complete, the healthy stem cells are infused in a manner similar to a blood transfusion.

Patients should expect a hospital stay of between six and eight weeks. Doctors will evaluate the patient to determine if their immune system functionality is fully restored before discharging them. After they return home, the patient will continue to take the medication in isolation until their cell count returns to normal.  

Relief is Available

If you suffer from an immunodeficiency disorder, help is available. Talk with your doctor before making a decision.

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