Getting a bone marrow transplant and chronic granulomatous disease (CGD)

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My son Kal was born with CGD, and he was diagnosed at six months old. And then he had a bone marrow transplant when he was nine months old to cure him of that.

Choosing to go the bone marrow transplant route is one of the hardest decisions that you would ever have make.

His immunologist said if you go through the bone marrow transplant process, then they’re gonna give him chemotherapy. They talked to me about the risk of infertility, possible hearing loss, vision impairments, developmental impairment. They’re gonna bring his immune system down so low that even a common cold could kill him.

Once you make the decision to go through the bone marrow transplant process, they have to make sure that the patient is healthy enough up to the point of transplant.

In addition to that, you need to figure out where are those stem cells going to come from. You can get stem cells either from an adult donor, a sibling donor, or an umbilical cord stem cells.

They talked about things that we needed to do to prevent infection afterward. They said you need to sterilize all the toys, wash the walls, make sure that he doesn’t leave the house for an entire year.

He had his bone marrow transplant in the end of July of 2014. We were in the hospital for six weeks. Once he had approval to go home, they watched him for 100 days. He was constantly sick throughout the next four months.

After one full year of Kal not being able to leave the house at all, they told us he’s good. His cells are good. He is basically free to live a normal life.

A bone marrow transplant (BMT) is currently the only proven cure for CGD, although research in this area is ongoing and it may not work every time. The process of performing a BMT involves taking healthy immune cells from one person and putting them into the body of a person with CGD. The person getting the transplant is called the “host” and the person giving the immune cells is called the “donor.” When successful, the immune system of the person with CGD becomes an immune system that can fight off infections.

There are 2 challenges to getting a BMT:

  1. Transplant rejection. When the healthy immune cells are put into the host, the host body may think the “new” cells from the donor are something foreign. When this happens, the host’s body attacks the cells or rejects them.
  2. Risk of graft-versus-host disease (GvHD). In this situation, the donor cells turn against the body of the host. This happens because the new cells from the donor see the tissues and organs of the person with CGD as something foreign and attack them.

To help lower the chances of transplant rejection or getting GvHD, it’s best to have a donor who is related to the person with CGD. Otherwise, the chances of a BMT failure can be higher.

Every person with CGD is different. While a BMT might be right for one person, another person may benefit from medicines to manage his or her CGD. That’s why it’s important to talk to your doctor about the pros and cons of a BMT for your situation. Depending on how serious your CGD is, your doctor may recommend taking an antifungal, an antibiotic, and ACTIMMUNE® (Interferon gamma-1b) to help manage your CGD. You can learn more about the types of BMT and selecting a donor at Living with CGD.

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Important Safety Information

What is ACTIMMUNE® (Interferon gamma-1b) used for?

ACTIMMUNE® is part of a drug regimen used to treat Chronic Granulomatous Disease, or CGD. CGD is a genetic disorder, usually diagnosed in childhood, that affects some cells of the immune system and the body’s ability to fight infections effectively. CGD is often treated (though not cured) with antibiotics, antifungals, and ACTIMMUNE.

ACTIMMUNE is also used to slow the worsening of severe, malignant osteopetrosis (SMO). SMO is a genetic disorder that affects normal bone formation and is usually diagnosed in the first few months after birth.

When should I not take ACTIMMUNE?

Don’t use ACTIMMUNE if you are allergic to interferon-gamma, E coli-derived products, or any ingredients contained in the product.

What warnings should I know about ACTIMMUNE?

At high doses, ACTIMMUNE can cause (flu-like) symptoms, which may worsen some pre-existing heart conditions.

ACTIMMUNE may cause decreased mental status, walking disturbances, and dizziness, particularly at very high doses. These symptoms are usually reversible within a few days upon dose reduction or discontinuation of therapy.

Bone marrow function may be suppressed with ACTIMMUNE, and decreased production of cells important to the body may occur. This effect, which can be severe, is usually reversible when the drug is discontinued or the dose is reduced.

Taking ACTIMMUNE may cause reversible changes to your liver function, particularly in patients less than 1 year old. Your doctor should monitor your liver function every 3 months, and monthly in children under 1 year.

In rare cases, ACTIMMUNE can cause severe allergic reactions and/or rash. If you experience a serious reaction to ACTIMMUNE, discontinue it immediately and contact your doctor or seek medical help.

What should I tell my healthcare provider?

Be sure to tell your doctor about all the medications you are taking.

Tell your doctor if you:

What are the side effects of ACTIMMUNE?

The most common side effects with ACTIMMUNE are “flu-like” symptoms such as fever, headache, chills, muscle pain, or fatigue, which may decrease in severity as treatment continues. Bedtime administration of ACTIMMUNE may help reduce some of these symptoms. Acetaminophen may be helpful in preventing fever and headache.

What other medications might interact with ACTIMMUNE?

Some drugs may interact with ACTIMMUNE to potentially increase the risk of damage to your heart or nervous system, such as certain chemotherapy drugs. Tell your doctor about all other medications you are taking.

Avoid taking ACTIMMUNE at the same time as a vaccination.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.

The risk information provided here is not comprehensive. To learn more, talk about ACTIMMUNE with your healthcare provider or pharmacist. The FDA-approved product labeling can be found at http://www.ACTIMMUNE.com or 1-866-479-6742.