We understand that a diagnosis of primary HLH can be overwhelming, but we hope you can take some comfort in the fact that treatment options are available, and that Gamifant was specifically designed to treat primary HLH.
By working with your healthcare team and learning all you can about Gamifant, you and your loved ones can be prepared for the journey ahead.
Gamifant is the first and only FDA-approved treatment made specifically for people with primary HLH who have refractory, recurrent, or progressive disease, or intolerance to conventional therapy.
If your doctor decides that Gamifant is right for you or your child, learning all you can will help you prepare for treatment.
There are many different cytokines involved in primary HLH. These may include interleukin-1 (IL-1), interleukin-6 (IL-6), and tumor-necrosis factor alpha (TNF-α). However, one cytokine called interferon gamma (IFNγ) plays an important role.
IFNy connects to macrophages, directly telling them to begin the process of making more cytokines. This continued production of cytokines contributes to the symptoms and inflammation that can happen with primary HLH.
Gamifant demonstrated safety and efficacy for most people in a clinical trial and was approved by the FDA in 2018. In the trial, investigators looked at:
For those who did not respond to conventional therapy, couldn’t tolerate it, whose symptoms came back or did not improve, or who experienced rapidly progressive disease, Gamifant helped:
63% of people had positive improvements in the signs and symptoms of primary HLH
70% of people with primary HLH proceeded to transplant
When considering Gamifant, it is important to discuss any potential side effects with your doctor.
Because Gamifant affects the immune system, it may lower a patient’s ability to fight infections. Gamifant may increase the risk of serious infections, including:
Doctors will test people for TB before starting Gamifant, and they may prescribe a treatment for TB if a person is at risk or has a positive TB test. Some people may also be considered for therapies that can help prevent certain infections before they receive Gamifant.
There are some serious side effects that can happen during treatment with Gamifant, including fever, high blood pressure (hypertension), and infusion reactions.*
*Infusion reactions such as rash and fever are common with Gamifant and usually happen during or shortly after treatment. Your doctor may temporarily stop the infusion and treat symptoms before continuing therapy if you or your child experiences severe reactions.
If your doctor prescribes Gamifant, you or your child will most likely receive treatment in the hospital.
Gamifant is given by intravenous (IV, or in a vein) infusion. The infusion will take about an hour. Your doctor or infusion nurse will watch for side effects during the infusion.
Gamifant is given 2 times a week (every 3 to 4 days) until you or your child begins conditioning for transplant or no longer needs treatment.
Gamifant is used with a steroid called dexamethasone, which is often given on a daily schedule. Over time, your doctor may reduce the amount of dexamethasone.
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