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PDGFRA GIST

What is PDGFRA GIST?

Patient portrayal of African-American adult female looking into distance. Slanted blue shape

What may happen if you have PDGFRA GIST?

Gastrointestinal stromal tumor with PDGFRA exon 18 mutation (otherwise known as PDGFRA GIST) is a rare disease that affects the production of cells in the gastrointestinal tract known as interstitial cells of Cajal (ICC).

  • ICCs help move food and liquid through the gut
  • In PDGFRA GIST, a genetic mutation makes ICCs grow out of control
  • This forms a tumor, most often in the stomach or small intestine

What are some of the most common symptoms of PDGFRA GIST?

Icon represnting the intestines

Blood in vomit and stools

Icon representing abdominal pain and swelling

Abdominal pain and swelling

Icon representing difficulty swallowing

Difficulty swallowing

Icon representing a scale

Loss of appetite and weight loss

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Want to learn more about AYVAKIT® (avapritinib)?

GIST FAQs

GISTs are cancers made up of a type of nerve cell found in the walls of the digestive organs that are involved in helping to move food through the gut. GISTs begin in the digestive system, most often in the stomach and small intestine. A majority of GISTs have mutations in a gene known as PDGFRA. Such mutations can cause cells to grow out of control.

No one knows the exact cause of the mutations that result in GISTs. There are rare instances where these mutations are passed down or inherited from parent to child, but otherwise the only known risk factors for GISTs is older age.

The platelet-derived growth factor receptor alpha (PDGFRA) gene makes a protein that controls cell growth and survival of many types of cells throughout the body.

A mutation is an abnormal change in a gene. Some mutations can lead to cancer cell growth. Among people with GIST, about 10% to 15% have a mutation on a gene called platelet-derived growth factor receptor alpha (PDGFRA). When these specific mutations occur, a protein produced by the abnormal gene activates and can cause cells to multiply uncontrollably. These cells build up and form tumors known as GISTs.

KIT is a gene that produces a protein that helps cells grow under control. If the KIT gene has a change or mutation, it can produce an abnormal protein that can cause the cells to grow uncontrollably and form a tumor.

One common symptom of GIST is bleeding which can be seen in vomit (may look brown or gritty) or in bowel movements (black, tar-looking or have visible blood). Bleeding can also lower red blood cell counts (also known as anemia), which may make people feel tired and weak.

Other GIST symptoms can include:

  • Abdominal pain or swelling
  • Nausea and/or vomiting
  • Difficulty swallowing
  • Weight loss, loss of appetite or feeling full after eating only small amounts

Some people with GISTs may not have any symptoms, and the tumors may be discovered during testing or treatment for other conditions.

GISTs are rare, and PDGFRA mutations occur in about 10% to 15% of GIST cases. Some estimate that there are fewer than 3 cases of PDGFRA-mutated GISTs per 1 million people worldwide.

INDICATION

WHAT IS AYVAKIT® (avapritinib)?

AYVAKIT® (avapritinib) is a prescription medicine used to treat adults with a certain type of stomach, bowel, or esophagus cancer called gastrointestinal stromal tumor (GIST) that cannot be treated with surgery or that has spread to other parts of the body (metastatic), and that is caused by certain abnormal platelet-derived growth factor receptor alpha (PDGFRA) genes. Your healthcare provider will perform a test to make sure that you have this abnormal PDGFRA gene and that AYVAKIT is right for you. 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, nausea, vomiting, vision changes, drowsiness, dizziness, confusion, or severe weakness on one or more side of your body. Your healthcare provider will check your platelet counts before and during treatment with AYVAKIT.

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 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 GIST include: fluid retention or swelling, nausea, tiredness or weakness, trouble thinking, vomiting, decreased appetite, diarrhea, increased eye tearing, stomach area (abdominal) pain, constipation, rash, dizziness, hair color changes and changes in certain blood tests.

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.

AYVAKIT is available in 100 mg, 200 mg and 300 mg tablets.

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

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 AYVAKIT and tell your healthcare provider right away if you develop any symptoms such as severe headache, nausea, vomiting, vision changes, drowsiness, dizziness,

INDICATION & IMPORTANT SAFETY INFORMATION

Warning: AYVAKIT® (avapritinib) may cause serious side effects, including bleeding in your brain, cognitive effects, skin sensitivity to sunlight (photosensitivity), harm to your unborn baby.

IMPORTANT SAFETY INFORMATION & INDICATION

Warning: AYVAKIT® (avapritinib) may cause serious side effects, including bleeding in your brain, cognitive effects, skin sensitivity to sunlight (photosensitivity), harm to your unborn baby.

INDICATION

WHAT IS AYVAKIT® (avapritinib)?

AYVAKIT® (avapritinib) is a prescription medicine used to treat adults with a certain type of stomach, bowel, or esophagus cancer called gastrointestinal stromal tumor (GIST) that cannot be treated with surgery or that has spread to other parts of the body (metastatic), and that is caused by certain abnormal platelet-derived growth factor receptor alpha (PDGFRA) genes. Your healthcare provider will perform a test to make sure that you have this abnormal PDGFRA gene and that AYVAKIT is right for you. 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, nausea, vomiting, vision changes, drowsiness, dizziness, confusion, or severe weakness on one or more side of your body.

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 bleeding in your brain
  • have a history of stroke within the last year
  • have low platelet counts
  • have or have had liver problems
  • 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 GIST include: fluid retention or swelling, nausea, tiredness or weakness, trouble thinking, vomiting, decreased appetite, diarrhea, increased eye tearing, stomach area (abdominal) pain, constipation, rash, dizziness, hair color changes and changes in certain blood tests.

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.

AYVAKIT is available in 100 mg, 200 mg and 300 mg tablets.

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