Frequently asked questions

Acquired thrombotic thrombocytopenic purpura (aTTP) can also be referred to as iTTP (immune-mediated thrombotic thrombocytopenic purpura). It is a rare, life-threatening blood disorder. You may hear your doctor or some people refer to it as thrombotic thrombocytopenic purpura (TTP). This is a general term. The 2 main types of TTP are inherited and acquired; however, 95% of all TTP cases are acquired/immune-mediated.

Acquired: Describes a condition that is developed later in life. It may be unknown why aTTP/iTTP is developed.

Thrombotic: Refers to the small blood clots that form inside blood vessels.

Thrombocytopenic: Means having a low platelet count.

Purpura: Red- or purple-colored spots on the skin that do not go away when pressure is applied. It may look like a bruise.

aTTP=acquired thrombotic thrombocytopenic purpura; iTTP=immune-mediated thrombotic thrombocytopenic purpura; TTP=thrombotic thrombocytopenic purpura.

There are 2 main types of TTP: acquired or immune-mediated TTP (aTTP/iTTP) and hereditary or congenital TTP (cTTP).

• In congenital TTP, people are born with a faulty gene that causes TTP. cTTP is very rare - it is estimated that only 5% of all cases of TTP are cTTP.
• Most people who have TTP have acquired or immune-mediated TTP. This means they developed TTP later in their lives. Around 95% of all TTP cases are acquired.

aTTP=acquired thrombotic thrombocytopenic purpura; iTTP=immune-mediated thrombotic thrombocytopenic purpura; TTP=thrombotic thrombocytopenic purpura.

aTTP/iTTP affects up to 5 people per million (or fewer than 2000 people) in the United States every year. It usually affects:

• People with 30 to 50 years of age
• Women
• People with weak immune system (e.g., due to cancer or HIV), obesity, women who are pregnant, African American heritage, and people with infection

aTTP=acquired thrombotic thrombocytopenic purpura; iTTP=immune-mediated thrombotic thrombocytopenic purpura.

Everyone's experience with aTTP/iTTP is different. You may have had some of the following symptoms. These can be signs of aTTP/iTTP and could be a warning of having another aTTP/iTTP episode. This is called a recurrence.

Bleeding-related symptoms:

• Bleeding from the gums or nose
• Purple bruises (known as purpura) and/or red or purple dots (known as petechiae) on the skin
• Blood in urine
• Stomach pain

Blood clot-related symptoms:

• Chest pain
• Seizures
• Headaches, confusion, and distorted vision
• Tiredness and jaundice (a yellowing of the skin and eyes)

aTTP=acquired thrombotic thrombocytopenic purpura; iTTP=immune-mediated thrombotic thrombocytopenic purpura.

One of the components in your blood that helps your platelets stick together is called von Willebrand factor (vWF). Platelets are small cells found in the blood and are involved in clotting. Their main job is to form blood clots when bleeding happens. Platelets stick together to seal a cut or injury. This stops bleeding. Normally, vWF is cut up into smaller pieces by an enzyme called ADAMTS13. In people with aTTP/iTTP, the body's immune system makes proteins called autoantibodies that inhibit ADAMTS13 from cutting up vWF. These large molecules of vWF attract platelets. This forms blood clots even when there is no bleeding. These clots:

• Can block the flow of blood to important organs, such as the brain, heart, or kidneys. This can cause serious health problems, such as stroke or seizure.
• Can destroy red blood cells, leading to a condition called hemolytic anemia. Because red blood cells carry oxygen, the body doesn't get as much as it needs. This can make people feel tired and may cause a higher heart rate.

  aTTP=acquired thrombotic thrombocytopenic purpura; iTTP=immune-mediated thrombotic thrombocytopenic purpura.

  Learn more about aTTP/iTTP

It is unknown why someone develops aTTP/iTTP, but the following could be triggers:

• Certain disorders or conditions, including cancer, HIV, lupus, infections, or pregnancy
• Surgery or certain other medical procedures
• Certain drugs or medications, including chemotherapy, hormone therapy, and estrogen-based medicines (such as oral contraceptives)

aTTP=acquired thrombotic thrombocytopenic purpura; iTTP=immune-mediated thrombotic thrombocytopenic purpura.

Diagnosis is normally made from a combination of medical history, physical examination, and laboratory tests.

• Medical history may indicate to the doctor that some of the potential triggers for TTP are present, such as having certain conditions or having taken certain medications.
• Physical examinations may show some of the common symptoms of TTP, such as bruising under the skin, yellowing of the skin, changes in the color of the urine, or neurological symptoms (e.g., headache or confusion).
• Diagnostic tests help the doctor to identify some other signs of TTP, such as low platelet count, low red blood cell levels, and indications that ADAMTS13 enzyme and some organs are not functioning properly.

TTP=thrombotic thrombocytopenic purpura.

People with aTTP/iTTP are usually diagnosed in the hospital because of their signs and symptoms. In addition to asking about your symptoms, your doctor is likely to perform lab tests to check the following:

• Platelet and red blood cell count
• ADAMTS13 enzyme activity levels
• Organ function

aTTP=acquired thrombotic thrombocytopenic purpura; iTTP=immune-mediated thrombotic thrombocytopenic purpura.

Learn more about aTTP/iTTP

Potential aTTP/iTTP treatments are given with 2 major therapeutic goals:

• Treat low platelet count
• Protect from autoimmune response against ADAMTS13 enzymes

Your doctor will decide what treatment is best for you. It is common for aTTP/iTTP to be treated with plasma exchange and immunosuppressive therapy.

aTTP=acquired thrombotic thrombocytopenic purpura; iTTP=immune-mediated thrombotic thrombocytopenic purpura.

An aTTP (also referred to as iTTP) episode is an acute episode. This means it happens suddenly and lasts for a short time with treatment. However, some patients who had one aTTP/iTTP episode will experience another. This is called a recurrence.

aTTP=acquired thrombotic thrombocytopenic purpura; iTTP=immune-mediated thrombotic thrombocytopenic purpura.

It is important to be aware of and watch for any aTTP/iTTP symptoms. If you think that you have signs and symptoms of an aTTP/iTTP episode, talk to your doctor or visit your local emergency room right away.

Symptoms can include:

• Bleeding from the gums or nose
• Purple bruises (known as purpura) and/or red or purple dots (known as petechiae) on the skin
• Blood in urine
• Stomach pain
• Chest pain
• Seizures
• Headaches, confusion, or distorted vision
• Tiredness and jaundice (yellowing of the skin and eyes)

aTTP=acquired thrombotic thrombocytopenic purpura; iTTP=immune-mediated thrombotic thrombocytopenic purpura.

CABLIVI is a treatment for aTTP/iTTP. CABLIVI is used together with a procedure called plasma exchange and medication called immunosuppressive therapy to treat aTTP/iTTP in adults. It is intended to prevent your body from forming dangerous blood clots during an aTTP/iTTP episode.

aTTP=acquired thrombotic thrombocytopenic purpura; iTTP=immune-mediated thrombotic thrombocytopenic purpura.

CABLIVI (caplacizumab-yhdp) is a prescription medicine used for the treatment of adults with aTTP/iTTP, in combination with plasma exchange and immunosuppressive therapy.

aTTP=acquired thrombotic thrombocytopenic purpura; iTTP=immune-mediated thrombotic thrombocytopenic purpura.

CABLIVI is used together with a procedure called plasma exchange and medication called immunosuppressive therapy to treat aTTP/iTTP in adults.

CABLIVI has an active ingredient called caplacizumab-yhdp (cap-luh-siz-uh-mab). It is a small protein that is intended to prevent your body from forming dangerous blood clots during an aTTP/iTTP episode.

aTTP=acquired thrombotic thrombocytopenic purpura; iTTP=immune-mediated thrombotic thrombocytopenic purpura.

CABLIVI treatment starts in the hospital with plasma exchange, then continues at home for at least 30 days after daily plasma exchange ends.

Keep taking CABLIVI until your doctor tells you to stop. Your doctor may decide you need to take CABLIVI for more than 30 days. This additional period may last up to 28 days. Your doctor may order additional blood tests, including a test to measure your ADAMTS13 level, an important marker of aTTP/iTTP. This test may help your doctor decide when you no longer need CABLIVI.

aTTP=acquired thrombotic thrombocytopenic purpura; iTTP=immune-mediated thrombotic thrombocytopenic purpura.

CABLIVI treatment starts in the hospital with plasma exchange. Your first dose of CABLIVI will be a bolus IV injection before you receive your first plasma exchange. Every day after plasma exchange, CABLIVI will be given as an injection into the skin of your stomach.

IV=intravenous.

Taking CABLIVI daily, as directed by your doctor, is key for successful treatment. However, if you miss a dose, do the following:

• During your plasma exchange sessions: Take CABLIVI as soon as possible
• After you're no longer on plasma exchange: Take CABLIVI daily within 12 hours of your scheduled time. After 12 hours, skip the dose and take the next scheduled dose

CABLIVI can cause side effects, although not everyone will experience them. In clinical studies, the most common side effects included nosebleeds, headaches, and bleeding from gums. Because CABLIVI works by preventing the formation of blood clots, it may affect normal blood clotting and increase the risk of severe bleeding. In clinical studies, severe bleeding, nosebleed, bleeding from the gums, bleeding in the stomach or intestines, and bleeding from the uterus were each reported in 1% of subjects.

Contact your doctor immediately if excessive bleeding or bruising happens. Your doctor will decide on next steps. Tell your doctor if you have a medical condition such as a bleeding disorder. You may have a higher risk of bleeding if you have a bleeding disorder (such as hemophilia) or if you take other medicines that increase your risk of bleeding (such as anticoagulants).

CABLIVI should be stopped for 7 days before surgery or any medical or dental procedure. Talk to your doctor before you stop taking CABLIVI.

With CABLIVI Patient Solutions, support is available to cover the cost of CABLIVI's co-pay or co-insurance for qualified, commercially insured patients, up to the program maximum.* CABLIVI will be provided at no cost to eligible underinsured or uninsured patients.^†

*Not valid for CABLIVI prescriptions covered by or submitted for reimbursement under Medicare, Medicaid, VA, DoD, Tricare, or similar federal or state programs including any state pharmaceutical assistance programs. Not valid where prohibited by law. Savings may vary depending on the patient's out-of-pocket costs. Upon registration, the patient will receive all program details.

^†Approval is not guaranteed. Sanofi reserves the right to modify or discontinue the programs at any time without notice.

Every insurance plan is different, so you will need to contact your insurance company to learn about your coverage options.

With CABLIVI Patient Solutions, support is available to cover the cost of CABLIVI's co-pay or co-insurance for qualified, commercially insured patients, up to the program maximum.* CABLIVI will be provided at no cost to eligible underinsured or uninsured patients.^†

*Not valid for CABLIVI prescriptions covered by or submitted for reimbursement under Medicare, Medicaid, VA, DoD, Tricare, or similar federal or state programs including any state pharmaceutical assistance programs. Not valid where prohibited by law. Savings may vary depending on the patient's out-of-pocket costs. Upon registration, the patient will receive all program details.
^†Approval is not guaranteed. Sanofi reserves the right to modify or discontinue the programs at any time without notice.

Fill out our Stay Connected form to learn more about CABLIVI and aTTP/iTTP.

aTTP=acquired thrombotic thrombocytopenic purpura; iTTP=immune-mediated thrombotic thrombocytopenic purpura.

For questions specific to your treatment and how you are doing, talk to your doctor.

For questions about CABLIVI services, CABLIVI Patient Solutions is available Monday through Friday, 8:00 AM to 8:00 PM ET, at 1-855-724-7222.

Taking CABLIVI once a day is key for successful treatment. However, if you miss a dose, do the following:

• During your plasma exchange sessions: Take CABLIVI as soon as possible
• After you're no longer on plasma exchange: Take CABLIVI daily within 12 hours of your scheduled time. After 12 hours, skip the dose and take the next scheduled dose

You should inject only one (1) vial of CABLIVI at a time, as recommended by your doctor. If you have accidentally used more than this, don't inject any more and call your doctor right away.

Talk to your doctor before you stop treatment with CABLIVI. Do not stop treatment, even if you start to feel better, as stopping treatment too early could cause you to have another aTTP/iTTP episode.

aTTP=acquired thrombotic thrombocytopenic purpura; iTTP=immune-mediated thrombotic thrombocytopenic purpura.