ISM

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What to Know About Indolent Systemic Mastocytosis (ISM)

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Patient Portrayal

What is Indolent Systemic Mastocytosis?

ISM is a rare disease that affects the production and accumulation of mast cells, a type of white blood cell.

Normally, your body makes mast cells as part of your immune response, but a buildup of mast cells in different parts of the body can lead to symptoms similar to severe allergic reactions.

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What causes ISM?

In about 95% of cases, ISM is thought to be caused by a gene mutation called KIT D816V.

How many people have ISM?

Systemic mastocytosis (SM) is a rare condition that affects around 1 in 10,000 patients. The most common form of SM is Indolent Systemic Mastocytosis (ISM), which accounts for around 75-90% of cases of SM.

What are some of the common symptoms of ISM?

Skin lesions and rash

Potentially severe allergic reactions including anaphylaxis

Nausea, vomiting and diarrhea

Other symptoms:

Each patient with ISM may experience different symptoms in different ways. Several symptoms are associated with ISM, including:

Brain fog
Dizziness
Headache
Fatigue
Flushing
Bone pain
Itching
Spots on the skin
Abdominal pain
Diarrhea
Nausea

Talk to your doctor about your symptoms and for medical advice.

How is ISM diagnosed?

ISM is diagnosed by a doctor who may:

Take biopsies (bone and/or organ)
Measure signs of excess mast cells
Test for KIT D816V gene mutation
Test for serum tryptase

KIT D816V testing

A KIT D816V blood test may be helpful to aid in the diagnosis of patients who are being evaluated for ISM. Since the gene mutation is found in approximately 95% of ISM cases, measurement of this genetic marker can help evaluate for ISM. There are different types of KIT tests available that vary in sensitivity. High-sensitivity tests may be required to detect the mutation.

Discover what ISM symptom relief may look like

Learn about AYVAKIT

Symptom Triggers

ISM symptoms may occur in response to certain triggers. These can differ from person to person, but some common ISM symptom triggers may include:

Alcohol
Certain foods
Skin irritation
Exercise

Changes in temperature
Anxiety and stress
Certain medications
Insect stings

Symptom relief from ISM is possible. Find out more about AYVAKIT.

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INDICATION

WHAT IS AYVAKIT^® (avapritinib)?

AYVAKIT^® (avapritinib) is a prescription medicine used to treat adults with indolent systemic mastocytosis (ISM). AYVAKIT is not recommended in people with low platelet counts (less than 50 X 10⁹/L).

It is not known if AYVAKIT is safe and effective in children.

IMPORTANT SAFETY INFORMATION

AYVAKIT^® (avapritinib) may cause serious side effects, including:

Bleeding in your brain. Serious bleeding in the brain may happen during treatment with AYVAKIT and may lead to death. Stop taking AYVAKIT and tell your healthcare provider right away if you develop any symptoms such as severe headache, vomiting, drowsiness, dizziness, confusion, or severe weakness on one or more side of your body. Bleeding in the brain has not been seen in people treated with AYVAKIT for ISM.

Cognitive effects. Cognitive side effects can happen during treatment with AYVAKIT and can be severe. Tell your healthcare provider if you develop any new or worsening cognitive symptoms including forgetfulness, confusion, getting lost, trouble thinking, drowsiness, trouble staying awake (somnolence), word finding problems, seeing objects or hearing things that are not there (hallucinations), or a change in mood or behavior.

Skin sensitivity to sunlight (photosensitivity). Your skin may be sensitive to the sun or other forms of light (photosensitivity) during treatment with AYVAKIT. Avoid or limit exposure to direct sunlight, sunlamps, and other sources of ultraviolet radiation during treatment and for 1 week after stopping treatment with AYVAKIT. Use sunscreen or wear clothes that cover your skin if you need to be out in the sun.

Before taking AYVAKIT, tell your healthcare provider about all of your medical conditions, including if you:

have a history of bulging or weakening of a blood vessel wall (aneurysm) or history of bleeding in your brain
have a history of stroke within the last year
have low platelet counts
have or have had liver problems
are pregnant or plan to become pregnant. AYVAKIT can cause harm to your unborn baby
Females who are able to become pregnant: Your healthcare provider should do a pregnancy test before you start AYVAKIT. You should use effective birth control (contraception) during treatment with AYVAKIT and for 6 weeks after the final dose of AYVAKIT. Talk to your healthcare provider about birth control methods that may be right for you. Tell your healthcare provider right away if you become pregnant or think you may be pregnant during treatment with AYVAKIT.
Males with female partners who are able to become pregnant should use effective birth control (contraception) during treatment and for 6 weeks after the final dose of AYVAKIT.
are breastfeeding or plan to breastfeed. It is not known if AYVAKIT passes into your breast milk. Do not breastfeed during treatment with AYVAKIT and for at least 2 weeks after the final dose of AYVAKIT. Talk to your healthcare provider about the best way to feed your baby during this time

Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. AYVAKIT may affect the way other medicines work, and certain other medicines may affect how AYVAKIT works. Especially tell your healthcare provider if you take medicines that prevent blood clots.

Do not drive or operate hazardous machinery if you have confusion or trouble thinking during treatment with AYVAKIT.

The most common side effects of AYVAKIT in people with ISM include: swelling around your eyes, dizziness, swelling of your arms and legs, and flushing.

Your healthcare provider may change your dose, temporarily stop, or permanently stop treatment with AYVAKIT if you develop certain side effects. AYVAKIT may cause fertility problems in females and males. Talk to your healthcare provider if this is a concern for you.

These are not all of the possible side effects of AYVAKIT. Call your doctor for medical advice about side effects. You are encouraged to report side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088.

Please see the full Prescribing Information and Patient Information for AYVAKIT.

IMPORTANT SAFETY INFORMATION

AYVAKIT^® (avapritinib) may cause serious side effects, including:

Bleeding in your brain. Serious bleeding in the brain may happen during treatment with AYVAKIT and may lead to death. Stop taking AYVAKIT and tell your healthcare provider right away if you develop any symptoms such as severe headache, vomiting, drowsiness, dizziness, confusion, or severe weakness on one or more side of your body. Bleeding in the brain has not been seen in people treated with AYVAKIT for ISM.

Cognitive effects. Cognitive side effects can happen during treatment with AYVAKIT and can be severe. Tell your healthcare provider if you develop any new or worsening cognitive symptoms including forgetfulness, confusion, getting lost, trouble thinking, drowsiness, trouble staying awake (somnolence), word finding problems, seeing objects or hearing things that are not there (hallucinations), or a change in mood or behavior.

INDICATION

WHAT IS AYVAKIT^® (avapritinib)?

AYVAKIT^® (avapritinib) is a prescription medicine used to treat adults with indolent systemic mastocytosis (ISM). AYVAKIT is not recommended in people with low platelet counts (less than 50 X 10⁹/L).

It is not known if AYVAKIT is safe and effective in children.

INDICATION & IMPORTANT SAFETY INFORMATION

AYVAKIT^® (avapritinib) may cause serious side effects, including:

Bleeding in your brain. Serious bleeding in the brain may happen during treatment with AYVAKIT and may lead to death. Stop taking

INDICATION

WHAT IS AYVAKIT^® (avapritinib)?

AYVAKIT^® (avapritinib) is a prescription medicine used to treat adults with indolent systemic mastocytosis (ISM). AYVAKIT is not recommended in people with low platelet counts (less than 50 X 10⁹/L).

It is not known if AYVAKIT is safe and effective in children.

IMPORTANT SAFETY INFORMATION

AYVAKIT^® (avapritinib) may cause serious side effects, including:

Bleeding in your brain. Serious bleeding in the brain may happen during treatment with AYVAKIT and may lead to death. Stop taking AYVAKIT and tell your healthcare provider right away if you develop any symptoms such as severe headache, vomiting, drowsiness, dizziness, confusion, or severe weakness on one or more side of your body. Bleeding in the brain has not been seen in people treated with AYVAKIT for ISM.

Cognitive effects. Cognitive side effects can happen during treatment with AYVAKIT and can be severe. Tell your healthcare provider if you develop any new or worsening cognitive symptoms including forgetfulness, confusion, getting lost, trouble thinking, drowsiness, trouble staying awake (somnolence), word finding problems, seeing objects or hearing things that are not there (hallucinations), or a change in mood or behavior.

Skin sensitivity to sunlight (photosensitivity). Your skin may be sensitive to the sun or other forms of light (photosensitivity) during treatment with AYVAKIT. Avoid or limit exposure to direct sunlight, sunlamps, and other sources of ultraviolet radiation during treatment and for 1 week after stopping treatment with AYVAKIT. Use sunscreen or wear clothes that cover your skin if you need to be out in the sun.

Before taking AYVAKIT, tell your healthcare provider about all of your medical conditions, including if you:

have a history of bulging or weakening of a blood vessel wall (aneurysm) or history of bleeding in your brain
have a history of stroke within the last year
have low platelet counts
have or have had liver problems
are pregnant or plan to become pregnant. AYVAKIT can cause harm to your unborn baby
Females who are able to become pregnant: Your healthcare provider should do a pregnancy test before you start AYVAKIT. You should use effective birth control (contraception) during treatment with AYVAKIT and for 6 weeks after the final dose of AYVAKIT. Talk to your healthcare provider about birth control methods that may be right for you. Tell your healthcare provider right away if you become pregnant or think you may be pregnant during treatment with AYVAKIT.
Males with female partners who are able to become pregnant should use effective birth control (contraception) during treatment and for 6 weeks after the final dose of AYVAKIT.
are breastfeeding or plan to breastfeed. It is not known if AYVAKIT passes into your breast milk. Do not breastfeed during treatment with AYVAKIT and for at least 2 weeks after the final dose of AYVAKIT. Talk to your healthcare provider about the best way to feed your baby during this time

Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. AYVAKIT may affect the way other medicines work, and certain other medicines may affect how AYVAKIT works. Especially tell your healthcare provider if you take medicines that prevent blood clots.

Do not drive or operate hazardous machinery if you have confusion or trouble thinking during treatment with AYVAKIT.

The most common side effects of AYVAKIT in people with ISM include: swelling around your eyes, dizziness, swelling of your arms and legs, and flushing.

Your healthcare provider may change your dose, temporarily stop, or permanently stop treatment with AYVAKIT if you develop certain side effects. AYVAKIT may cause fertility problems in females and males. Talk to your healthcare provider if this is a concern for you.

These are not all of the possible side effects of AYVAKIT. Call your doctor for medical advice about side effects. You are encouraged to report side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088.

Please see the full Prescribing Information and Patient Information for AYVAKIT.