How Nplate^® (romiplostim) Works: Video Transcript

Indication

Nplate^® (romiplostim) is a man-made protein medicine used to treat low blood platelet counts in adults with chronic immune thrombocytopenia (ITP), when certain other medicines, or surgery to remove your spleen, have not worked well enough.

Nplate^® is not for use in people with a precancerous condition called myelodysplastic syndrome (MDS) or low platelet count caused by any condition other than chronic (lasting a long time) immune thrombocytopenia (ITP). Nplate^® is only used if your low platelet count and medical condition increase your risk of bleeding. Nplate^® is used to try to keep your platelet count about 50,000 per microliter in order to lower the risk for bleeding. Nplate^® is not used to make your platelet count normal.

Not all patients will respond to Nplate^®. Individual patient results may vary.

Hello! This video is designed to help you understand:

• How platelets are naturally made in the body.
• How immune thrombocytopenia (or ITP) causes low platelet counts.
• How Nplate^® (romiplostim) works to help increase the number of platelets in adults with chronic ITP.
• Nplate^® Important Safety Information.

At any given time, trillions of blood cells are circulating in your body.

There are different types of cells in your blood, including red blood cells, white blood cells and platelets.

ITP is a condition that specifically affects your platelets. Let's take a look inside the body to learn more about platelets.

Platelets are small cells that are needed to help your blood form clots.

They are made by special cells in your bones, in the spongy core called bone marrow.

Platelets are normally destroyed after 9 or 10 days and replaced with new platelets.

A protein called thrombopoietin, or TPO, controls how many platelets are made.

In the bone marrow, TPO attaches to the cells that make platelets. This triggers the cells to make platelets and release them into the blood. If the level of TPO in the blood is low, fewer platelets are made. If the level of TPO in the blood is high, more platelets are made.

Normally when there are adequate or high numbers of platelets in circulation, TPO levels are low. However, when platelet levels drop, TPO levels increase to help make more platelets.

Now let's discuss what happens to platelets in adults with chronic ITP.

In adults with chronic ITP, platelets are destroyed by antibodies.

In ITP, antibodies cause the destruction of TPO, the protein needed for platelet production. Since TPO naturally binds to platelets, it is destroyed along with the platelets.

Because TPO levels are low, there is not a strong enough signal for normal platelet production, and therefore, not enough platelets are being produced.

This means ITP involves both increased platelet destruction and lower-than-expected platelet production, which results in low platelet counts.

People with ITP who have lower-than-normal platelet counts have a condition called thrombocytopenia, which can cause bleeding, bruising, and other symptoms.

Now that we've discussed how platelets are formed, and what happens in adults with chronic ITP, let's discuss how Nplate works to help increase platelet counts in adults with chronic ITP.

Nplate is a type of medicine called a platelet booster.

Like TPO, Nplate^® attaches to special cells in the bone marrow, triggering platelet production.

Nplate^® (romiplostim) increases platelet production at a rate that outpaces platelet destruction, which helps increase platelet counts in adults with chronic ITP.

It's important to know that not all patients will respond to Nplate^® (romiplostim), and individual patient results may vary.

It's important to understand that Nplate^® does not bring platelet counts to normal levels, but it can help raise them to a level high enough to help reduce symptoms of ITP.

Important Safety Information

What is the most important information I should know about Nplate^® (romiplostim)?

Nplate^® (romiplostim) can cause serious side effects:

• Worsening of a precancerous blood condition to a blood cancer (leukemia): Nplate^® (romiplostim) is not for use in people with a precancerous condition called myelodysplastic syndromes (MDS) or for any condition other than chronic (lasting a long time) immune thrombocytopenia (ITP). If you have MDS and receive Nplate^® (romiplostim), your MDS condition may worsen and become an acute leukemia. If MDS worsens to become acute leukemia you may die sooner from the acute leukemia.
• Higher risk for blood clots:
  • You may have a higher risk of getting a blood clot if your platelet count becomes high during treatment with Nplate^® (romiplostim). You may have severe complications or die from some forms of blood clots, such as clots that spread to the lungs or that cause heart attacks or strokes. Your healthcare provider will check your blood platelet counts and change your dose or stop Nplate^® (romiplostim) if your platelet counts get high.
  • If you have a chronic liver disease, you may get blood clots in the veins of your liver. This may affect your liver function.
• Bone marrow changes (increased reticulin and possible bone marrow fibrosis): Nplate^® (romiplostim) may cause changes in your bone marrow, but these changes may improve if you stop taking Nplate^® (romiplostim). These changes may lead to abnormal blood cells or your body making less blood cells. The mild form of these bone marrow changes is called "increased reticulin." It is not known if this may progress to a more severe form called "fibrosis." The mild form may cause no problems while the severe form may cause life-threatening blood problems. Signs of bone marrow changes may show up as abnormalities in your blood tests. Your healthcare provider will decide if abnormal blood tests mean that you should have bone marrow tests or if you should stop taking Nplate^® (romiplostim).
• Worsening low blood platelet count (thrombocytopenia) and risk of bleeding shortly after stopping Nplate^® (romiplostim): When you stop receiving Nplate^® (romiplostim), your low blood platelet count (thrombocytopenia) may become worse than before you started receiving Nplate^® (romiplostim). These effects are most likely to happen shortly after stopping Nplate^® (romiplostim) and may last about 2 weeks. The lower platelet counts during this time period may increase your risk of bleeding, especially if you are taking a blood thinner or other medicine that affects platelets. Your healthcare provider will check your blood platelet counts for at least two weeks after you stop taking Nplate^® (romiplostim). Call your healthcare provider right away to report any bruising or bleeding.
• Lack or loss of response: If you do not experience results from Nplate^® (romiplostim), your body may have created cells that are counteractive to Nplate^® (romiplostim). Your healthcare provider will monitor your platelet counts and test your blood regularly to determine if this is an issue.
• Blood test monitoring: Your healthcare provider will check your platelet count every week and change your dose of Nplate^® (romiplostim) as needed. This will continue until your healthcare provider decides that your dose of Nplate^® (romiplostim) can stay the same. After that, you will need to have blood tests every month. When you stop receiving Nplate^® (romiplostim), you will need blood tests for at least 2 weeks to check if your platelet count drops too low.
• Medication Errors: Medication errors have been reported in patients receiving Nplate^® (romiplostim). Injection of too much Nplate^® (romiplostim) may cause a dangerous increase in your platelet count and serious side effects. Injection of too little Nplate^® (romiplostim) may cause a lower than expected platelet count and increase your risk of bleeding.
• What are the possible side effects of Nplate^® (romiplostim)?
  • Nplate^® (romiplostim) may cause serious side effects. See "What is the most important information I should know about Nplate^® (romiplostim)?"
  • The most common side effects of Nplate^® (romiplostim) are:
    • Headache
    • Joint pain
    • Dizziness
    • Trouble sleeping
    • Muscle tenderness or weakness
    • Pain in arms and legs
    • Abdominal pain
    • Shoulder pain
    • Indigestion
    • Tingling or numbness in hands and feet
  • These are not all the possible side effects of Nplate^® (romiplostim). Tell your healthcare provider if you have any side effect that bothers you or that does not go away. For more information, ask your healthcare provider or pharmacist.
  • If you have any questions about this information, be sure to discuss them with your doctor. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.

Please see Prescribing Information and Medication Guide for more information about Nplate^® (romiplostim).

For more information on Nplate^® (romiplostim), please review the information on this web site and talk to your healthcare provider. To find a doctor, please use the physician locator tool located on this web site.

References:

1. Nplate^® (romiplostim) prescribing information, Amgen.
2. Cines DB, Blanchette VS. Immune thrombocytopenic purpura. N Engl J Med. 2002;346:995—1008.
3. Kaushansky K. The molecular mechanisms that control thrombopoiesis. J Clin Invest. 2005;115:3339—3347.
4. Nichol JL. Endogenous TPO (eTPO) levels in health and disease: possible clues for therapeutic intervention. Stem Cells. 1998;16(suppl 2):165—175.
5. Gernsheimer T. Pathophysiology and thrombokinetics in autoimmune thrombocytopenia. Blood Rev. 2002;16:7-8.