Hemophilia is a rare genetic condition that prevents your blood from clotting and can lead to bleeding problems. People with hemophilia can have bleeding episodes that happen without trauma and can also bleed longer after an injury. Some people with hemophilia receive treatment to prevent bleeding, and other people with hemophilia are treated only when they are bleeding.
People with hemophilia have low levels of proteins that help your blood clot. These are known as clotting factors. People with hemophilia A have low levels of factor VIII, and people with hemophilia B have low levels of factor IX. The body uses these clotting factors to make thrombin. Thrombin is another substance that helps clot the blood. Replacement of the missing clotting factors with manufactured clotting concentrates has been the standard hemophilia treatment for many years, but treatments that work in new ways are becoming more common.
Qfitlia (fitusiran) is a new hemophilia treatment to prevent or decrease the frequency of bleeding in adults and children 12 and older with certain types of hemophilia. Because people with hemophilia have low levels of clotting factors, they make less thrombin, making it harder for the blood to clot. Another substance in the body, called antithrombin, keeps the body from making more thrombin. Qfitlia is the first medicine to treat hemophilia by lowering the amount of antithrombin in your blood allowing more thrombin production.
How Do I Know if Qfitlia Is Right for Me?
Your health care provider may consider treatment with Qfitlia if you have hemophilia A or B. Qfitlia can be used even if you have developed antibodies (also called inhibitors) to clotting factor replacement products. Your health care provider will do a blood test to measure the amount of antithrombin in your blood before starting Qfitlia treatment. Your health care provider will also check your liver function before and during your treatment with Qfitlia.
How Do I Use Qfitlia?
You or your caregiver will inject Qfitlia under the skin (subcutaneously) in your stomach, thigh, or in your upper arm. You should be careful to avoid areas that are scarred, bruised, or tender. Qfitlia is supplied as a prefilled pen if you are prescribed the 50-milligram dose. Qfitlia is also supplied in a vial and syringe if you need a dose other than 50 milligrams.
If you are already using treatments for hemophilia, you will need to make changes to your other treatments. In some cases, you may need to stop your other treatments after you have started Qfitlia. If this is the case for you, your health care provider will walk you through when and how to stop your other medicines.
Your health care provider will decide how much Qfitlia you need and how often you should receive a Qfitlia injection. You may need as few as six injections each year.
Do not stop using Qfitlia without talking to your health care provider. If you stop taking Qfitlia or miss doses of Qfitlia, you are at increased risk of bleeding. If you miss a dose of Qfitlia, you should give the dose as soon as possible then resume your usual dosing schedule as directed by your health care provider.
How Was Qfitlia Studied for Hemophilia?
Qfitlia was studied in a clinical trial program known as ATLAS. This program included four studies. Two studies (ATLAS-INH and ATLAS A/B) looked at how well Qfitlia works for people with hemophilia who were not already using medicine to prevent bleeding. About three-fourths (78%) of the people had hemophilia A and about one-fourth (22%) had hemophilia B. The studies included 177 males who were at least 12 years old (average age 28-33). About 63% of people were Asian, 34% White, less than 1% Black, and less than 1% Hispanic/Latino.
Some of the people in this study received treatment with Qfitlia. The rest of the people in this study didn't use any medicines to prevent bleeding. Everyone in the study was able to use on-demand treatments when needed to treat active bleeding.
Another study (ATLAS-PPX) included 80 males who were at least 12 years old and were currently using other treatments. About three-fourths (75%) of the people in the study had hemophilia A and about one-fourths (23%) had hemophilia B. Sixty-five percent of people were White, 31% were Asian, 1.5% were Black, and 3% were reported as other. People enrolled in this study switched from their current treatment to prevention with Qfitlia.
What Benefits of Qfitlia Were Seen in the Studies?
People without inhibitors had an average of nine bleeding episodes each year while using Qfitlia, compared to an average of 31 bleeding episodes each year in the people who did not use any medicines to prevent bleeding. This means that people without inhibitors using Qfitlia had 71% fewer bleeds, on average, than people not using a medicine to prevent bleeds.
People with inhibitors had an average of five bleeding episodes each year while using Qfitlia, compared to an average of 19 bleeding episodes each year in the people who did not use any medicines to prevent bleeds. This means that people with inhibitors using Qfitlia had 73% fewer bleeds, on average, than people not using a medicine to prevent bleeds.
In the ATLAS-PPX study, when people without inhibitors switched from clotting factor prevention to Qfitlia, bleeding rates remained similar with an average of six episodes per year during clotting factor use and seven bleeding episodes per year during Qfitlia use.
In the ATLAS-PPX study, when people with inhibitors switched from bypassing agents to Qfitlia, bleeding rates were lowered with Qfitlia from an average of 11 episodes per year during bypassing agent use to three bleeding episodes per year with Qfitlia use.
Your results may differ from what was seen in clinical studies.
What Can I Do to Prevent Side Effects With Qfitlia?
Qfitlia may cause blood clots. Your risk of blood clots is lowered when you use the right dose of Qfitlia for you based on your antithrombin level. You should follow the schedule for monitoring your antithrombin level. Your health care provider will usually test your antithrombin levels at one, three, five, and six months after you start Qfitlia. Your health care provider will adjust your Qfitlia dose based on your antithrombin level. The monitoring schedule will restart if you need a dose adjustment.
Hormonal contraceptives increase your risk of blood clots. Talk with your health care provider about alternative birth control methods.
Your current hemophilia treatment may be changed when you begin Qfitlia. You should follow instructions from your health care provider regarding the management of acute bleeding episodes while you are treated with Qfitlia.
Qfitlia may cause an increase in your blood liver enzymes. Your health care provider will monitor your blood liver enzymes before you start Qfitlia and during your treatment. Follow your health care provider’s instructions for blood tests to monitor your liver function.
Qfitlia can cause gallstones and gallbladder inflammation. Tell your health care provider if you have a history of gallbladder disease before starting Qfitlia. You should contact your health care provider right away if you have stomach pain, upset stomach, nausea, or vomiting.
Qfitlia may increase your risk of infections. You can reduce your chance of infection with frequent hand washing and avoiding sick people.
Is There Any Cost Assistance Available?
You may be eligible for financial assistance to help with the cost of Qfitlia. You can learn more about the assistance program at this website.