Stay on top of plexiform neurofibroma growth

Caleb, a 7-year-old living with NF1 PN, playing with his mom

Caleb, age 7, living with NF1 PN, shown here with his mom.
Caleb is not currently taking Koselugo.

What is neurofibromatosis type 1 (NF1)?

NF1 is a rare genetic disorder that affects nearly 1 in 3000 people worldwide. Individuals with NF1 commonly have tumors along nerves in their body. Plexiform neurofibromas (PN) are one type of tumor seen in NF1.

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Some people may refer to plexiform neurofibromas as plexis, plexiforms, or plexiform tumors.

What are plexiform neurofibromas?

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Noncancerous tumors that can grow along nerves in the body.

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Seen in up to 50% of children
with NF1.*

*Using whole-body magnetic resonance
imaging (MRI).
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Plexiform neurofibromas generally continue to grow throughout childhood.

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Before KOSELUGO® (selumetinib), there was no FDA-approved medication proven to shrink plexiform neurofibromas in children and teens with NF1

Where do plexiform neurofibromas occur?

Plexiform neurofibromas can grow on any nerve in the body. Regardless of size, any plexiform neurofibroma may cause symptoms, depending on where it's located in the body.

As plexiform neurofibromas grow, they may cause new or worsening symptoms.

Image of mother and child

Plexiform neurofibroma symptoms to look out for†:

  • Lumps under the skin
  • Unusual coloring on the surface of the skin
  • Increased pain or discomfort
  • Difficulty moving
  • Tingling or numbness
  • Trouble breathing
  • Changes in eyesight
  • Bedwetting or other bowel or bladder problems
  • Fatigue and/or weakness
  • Irritability or change in behavior
  • Eating problems, such as loss of appetite
  • Difficulty falling or staying asleep

†This is not an exhaustive list.
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Start the treatment conversation

Acting early is essential to stay on top of plexiform neurofibroma growth. Ask your child's doctor for a referral to an NF1 specialist or an oncologist to see if Koselugo may be right for them.

Download the guide

Plexiform neurofibromas generally continue to grow over time

The National Cancer Institute (NCI) NF1 Natural History study monitored people with plexiform neurofibromas over time. This study was conducted to help understand how plexiform neurofibromas grow.

Percentage change in target PN volume, Natural History study

Natural History Study chart: percentage change in target PN volume over time
The NCI NF1 Natural History study began in 2008 and is an ongoing study investigating NF1 PN growth. 92 people with NF1-related PN between the ages 3 and 18 years who had at least 2 volumetric scans were included in the analysis as the age- and period-matched external control for the SPRINT study.
  • Most people in the study saw significant growth of their plexiform neurofibromas over time (20% growth or more)
  • No one in this study saw their plexiform neurofibromas shrink more than 20% per year

Early action is key in the treatment of plexiform neurofibromas

Plexiform neurofibroma growth may be unpredictable and should not be ignored. Plexiform neurofibromas typically grow fastest in the first 10 years of life, making it important to have a conversation with your child's doctor as early as possible if your child is experiencing symptoms.

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Koselugo is proven to shrink plexiform neurofibromas

If your child's plexiform neurofibromas cannot be removed by surgery, Koselugo may be able to help. Ask your child's doctor if Koselugo is right for you.

How are plexiform neurofibromas treated?

Which doctors and healthcare specialists treat pediatric plexiform neurofibromas?

Children with plexiform neurofibromas are usually seen by specialists who focus on treating symptoms caused by this disease. The location and symptoms of your child's plexiform neurofibromas will help determine which doctors are best to see for treatment.

By spotting plexiform neurofibromas early and communicating new or worsening symptoms with your child's doctor, you can help your child get the treatment they need.

Why can’t some plexiform neurofibromas be completely removed by surgery?

Surgery for plexiform neurofibromas may not be recommended by your child's doctor if they are closely intertwined with critical nerves and blood vessels, located near major organs, or cannot be completely removed by surgery.

Quentin, a 16-year-old who takes Koselugo for NF1 PN, playing basketball with a friend
Quentin, age 16, living with NF1 PN.
Quentin is taking Koselugo.

Koselugo has over 7 years of established safety‡§

Learn about how Koselugo was studied in children and teens with NF1 plexiform neurofibromas.

See the study results
‡Data cutoff February 2021. Based on the long-term follow-up of the SPRINT Phase 1 and Phase 2 Stratum 1 studies, the evaluation of Koselugo is run by the NCI and is designed to test the efficacy and safety of Koselugo. These data reflect exposure to Koselugo in 74 children and teens with NF1 PN that could not be removed by surgery without risk of issues because of their location or size. These patients received a dosage ranging from 20 mg/m2 to 30 mg/m2 orally twice daily in SPRINT.
§The median duration of Koselugo exposure is 4.4 years (range: 28 days to 7.7 years). Median is defined as the middlemost point in a dataset. In this case, 50% of patients received Koselugo treatment for less than 4.4 years, while the other 50% received Koselugo treatment for more than 4.4 years.

STAY INFORMED ABOUT NF1 PLEXIFORM NEUROFIBROMAS

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important safety information

What are the possible side effects of Koselugo?

Koselugo may cause serious side effects, including:

  • Heart problems. Koselugo can lower the amount of blood pumped by your heart, which is common and can also be severe. Your healthcare provider will do tests before and during treatment to check how well your heart is working. Tell your healthcare provider right away if you get any of the following signs or symptoms: persistent coughing or wheezing, shortness of breath, swelling of your ankles and feet, tiredness, increased heart rate.
  • Eye problems. Koselugo can cause eye problems that can lead to blindness. Your healthcare provider will check your vision before and during treatment. Tell your healthcare provider right away if you get any of the following signs or symptoms: blurred vision, loss of vision, dark spots in your vision (floaters), other changes to your vision.
  • Severe diarrhea. Diarrhea is common with Koselugo and can also be severe. Tell your healthcare provider right away the first time that you get diarrhea during treatment. Your healthcare provider may give you medicine to help control your diarrhea and may tell you to drink more fluids.
  • Skin rash. Skin rashes are common with Koselugo and can also be severe. Tell your healthcare provider if you get any of the following signs or symptoms: rash that covers a large area of your body, peeling skin, blisters.
  • Muscle problems (rhabdomyolysis). Muscle problems are common with Koselugo and can also be severe. Treatment with Koselugo may increase the level of a muscle enzyme in your blood called creatine phosphokinase (CPK) and may be a sign of muscle damage. Your healthcare provider should do a blood test to check your muscle enzyme levels of CPK before you start taking Koselugo and during treatment. Tell your healthcare provider right away if you get any of the following signs or symptoms: muscle aches or pain; muscle spasms and weakness; dark, reddish urine.

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

  • have heart problems.
  • have eye problems.
  • have liver problems.
  • are pregnant or plan to become pregnant. Koselugo can harm your unborn baby. Your healthcare provider should verify if you/your partner are pregnant before beginning treatment. Ensure you/your partner use effective birth control (contraception) during treatment and for 1 week after your last dose if there is possibility pregnancy could occur. Tell your healthcare provider right away if you/your partner think you may be pregnant.
  • are breastfeeding or plan to breastfeed. It is not known if Koselugo passes into your breast milk. Do not breastfeed during treatment and for 1 week after your last dose. 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, over-the-counter medicines, vitamins, or herbal supplements. Especially tell your healthcare provider if you are taking aspirin, blood thinners, or other medicines to treat blood clots. Koselugo contains vitamin E, which may increase risk of bleeding.

What should I avoid while taking Koselugo?

Do not drink grapefruit juice, eat grapefruit, or take supplements with grapefruit or St. John’s Wort during treatment.

Most common side effects include: vomiting, stomach-area pain, nausea, dry skin, muscle and bone pain, feeling of tiredness or lacking energy, fever, sores in your mouth, headache, redness around the fingernails, itching.

These are not all the possible side effects of Koselugo. Call your healthcare provider for medical advice about side effects. Your healthcare provider may change your dose, temporarily stop, or permanently ask you to stop taking Koselugo if you have any of these side effects. You may report side effects to AstraZeneca at 1-800-236-9933 or at https://
us-aereporting.astrazeneca.com or FDA at
1-800-FDA-1088 or www.fda.gov/medwatch.

Indication

What is Koselugo?

Koselugo is a prescription medicine that is used to treat children 2 years of age and older with neurofibromatosis type 1 (NF1) who have plexiform neurofibromas that cannot be completely removed by surgery. It is not known if Koselugo is safe and effective in children under 2 years of age.

Please see Patient Information in the full Prescribing Information for Koselugo (selumetinib).

All families shown in this website have been compensated by Alexion, unless otherwise noted.