What is RYTELO?

RYTELO (imetelstat) is a prescription medicine used to treat a condition called low- to intermediate-1 risk myelodysplastic syndromes (MDS) in adults: See more

  • with anemia (low red blood cell counts) who need blood transfusions of 4 or more red blood cell units over 8 weeks

and

  • who have not responded to, have stopped responding to, or cannot be treated with medicines called erythropoiesis-stimulating agents (ESAs).

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

Understanding lower-risk MDS
and why anemia can
be a challenge

These frequently asked questions can help you learn about lower-risk MDS, anemia, and the burden of frequent transfusions.

These frequently asked questions can help you learn about lower-risk MDS, anemia, and the burden of frequent transfusions.

Illustration of blood cells in the bone marrow

What are myelodysplastic syndromes (MDS)?

MDS are a group of blood cancers that start in the bone marrow, where blood cells are made.

In MDS, the bone marrow does not make healthy blood cells the way it should. Many of the cells it produces are abnormal or don’t fully develop, so there may not be enough healthy blood cells in the body.

What is low- to intermediate-1 risk MDS?

Healthcare providers group MDS into risk levels to understand how serious these diseases may be and how quickly they might progress. The risk levels can also be helpful when trying to determine the best treatment options.

When MDS is labeled as low-risk or intermediate-1 risk, it generally means:

  • The disease is growing more slowly than in higher-risk MDS
  • It can often be treated less intensely than higher-risk MDS
  • Many people live with the condition for years

Low- and intermediate-risk MDS may also be called lower-risk MDS (LR-MDS).

An older man consults with his doctor, who is holding a clipboard.

What is anemia?

Anemia happens when your body isn’t making enough red blood cells. These cells carry oxygen throughout the body. Up to 90% of patients with MDS develop anemia.

Symptoms of anemia

  • Fatigue or feeling
very tired

  • Chest pain

  • Dizziness or lightheadedness

  • Shortness of breath

  • Pale skin, nail beds, mouth, or gums

  • Swelling in the hands or feet

  • Fast heartbeat

Downward arrow icon with red blood cells

Why do some people who have LR-MDS with anemia need blood transfusions?

One of the most common issues in LR-MDS is anemia.  

Due to symptoms of anemia, healthcare providers may recommend a red blood cell transfusion. During a transfusion, healthy red blood cells from a donor are given through an IV to help raise the number of red blood cells in the body.

Some people with LR-MDS may only need transfusions occasionally, while others may need them regularly, depending on symptoms and on how low their red blood cell levels are.

What are the risks of frequent transfusions?

Blood transfusions can be helpful, but needing many transfusions over time can cause other health problems. One of the most common concerns is iron overload.

Red blood cells contain iron. When someone receives many transfusions, extra iron can build up in the body. Over time, too much iron may increase your risk of infection and damage organs such as the: 

  • heart
  • liver
  • endocrine glands (hormone-producing organs)

Healthcare providers may monitor iron levels and sometimes prescribe medicines to help remove excess iron from the body.

Repeated transfusions can also mean:

  • Frequent hospital or clinic visits

  • Time spent receiving treatment

  • Feeling limited in daily life

Goals of treatment for LR-MDS with anemia include:

  • Reducing the need for blood transfusions

  • Improving blood
cell counts

  • Feeling less limited
in daily life

  • Slowing the progression of MDS

Expand

What is RYTELO?

RYTELO (imetelstat) is a prescription medicine used to treat a condition called low- to intermediate-1 risk myelodysplastic syndromes (MDS) in adults:

  • with anemia (low red blood cell counts) who need blood transfusions of 4 or more red blood cell units over 8 weeks

and

  • who have not responded to, have stopped responding to, or cannot be treated with medicines called erythropoiesis-stimulating agents (ESAs).

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

Please see RYTELO (imetelstat) full Prescribing Information, including Medication Guide.

Important Safety Information

Before you receive RYTELO, tell your healthcare provider about all of your medical conditions, including if you:

  • are pregnant or plan to become pregnant. RYTELO may harm your unborn baby and may cause loss of pregnancy (miscarriage). Tell your healthcare provider right away if you become pregnant or think you may be pregnant during treatment with RYTELO.

Females who are able to become pregnant:

  • Your healthcare provider will perform a pregnancy test before you are given RYTELO.
  • You should use effective birth control (contraception) during treatment with RYTELO and for 1 week after your last dose.
  • are breastfeeding or plan to breastfeed. It is not known if RYTELO passes into your breastmilk. Do not breastfeed during treatment with RYTELO and for 1 week after your last dose.

Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

What is the most important information I should know about RYTELO?

RYTELO may cause serious side effects, including:

  • Low platelet counts (thrombocytopenia). Low platelet counts are common during treatment with RYTELO and can also be severe. Low platelet counts can increase your risk for bleeding. Your healthcare provider may give you platelet transfusions to reduce the risk of bleeding if you develop a low platelet count during treatment with RYTELO. Tell your healthcare provider right away if you develop any signs or symptoms of bleeding, including:
    • unusual bleeding or bruising
    • bleeding that lasts a long time
    • nosebleeds
    • vomiting blood
    • blood in your stool or black tarry stool
  • Low neutrophil counts (neutropenia). Low counts of a type of white blood cell called neutrophils are common during treatment with RYTELO and can also be severe. Low neutrophil counts can increase your risk for infections, including serious infections and sepsis. Your healthcare provider may give you medicines before you start treatment to help prevent neutropenia and infections and may treat you with medicines if you develop these problems during treatment with RYTELO. Tell your healthcare provider right away if you develop any signs or symptoms of infection during treatment with RYTELO, including:
    • fever
    • shortness of breath or trouble breathing
    • cough
    • chills
    • pain or burning when you urinate

Your healthcare provider will do blood tests to check your platelet and neutrophil counts before starting treatment with RYTELO, weekly for the first 2 cycles of treatment, before you receive each additional cycle, and as needed during your treatment.

Your healthcare provider may delay your next treatment, decrease your dose, or stop treatment with RYTELO if you develop thrombocytopenia or neutropenia during treatment.

What are the possible side effects of RYTELO?

RYTELO may cause serious side effects, including:

  • See “What is the most important information I should know about RYTELO?”
  • Infusion-related reactions. RYTELO can cause infusion-related reactions during or after your infusion that can be severe, including a severe sudden increase in blood pressure called hypertensive crisis. Your healthcare provider will give you medicines before each RYTELO infusion to help prevent or lessen infusion-related reactions and will watch you for at least 1 hour after your infusion. If you develop infusion-related reactions, your healthcare provider may infuse RYTELO more slowly, temporarily stop, or permanently stop your treatment. Tell your healthcare provider if you develop any signs or symptoms of infusion-related reactions, including:
    • stomach pain
    • joint pain
    • weakness and tiredness
    • back and bone pain
    • diarrhea
    • redness
    • headache
    • high blood pressure
    • not feeling well
    • chest pain that is not related to your heart
    • itching
    • hives

The most common side effects of RYTELO include:

  • decreased platelet counts
  • decreased white blood cell counts
  • decreased neutrophil counts
  • increased liver enzymes (AST, alkaline phosphatase, and ALT)
  • tiredness
  • longer than usual blood clotting times
  • joint, bone and muscle pain
  • Covid-19 infections
  • headache

These are not all of the possible side effects of RYTELO. Call your doctor for more information and medical advice about side effects. 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 RYTELO (imetelstat) full Prescribing Information, including Medication Guide.

What is RYTELO?

RYTELO (imetelstat) is a prescription medicine used to treat a condition called low- to intermediate-1 risk myelodysplastic syndromes (MDS) in adults:

  • with anemia (low red blood cell counts) who need blood transfusions of 4 or more red blood cell units over 8 weeks

and

  • who have not responded to, have stopped responding to, or cannot be treated with medicines called erythropoiesis-stimulating agents (ESAs).

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

Please see RYTELO (imetelstat) full Prescribing Information, including Medication Guide.

Important Safety Information

Before you receive RYTELO, tell your healthcare provider about all of your medical conditions, including if you:

  • are pregnant or plan to become pregnant. RYTELO may harm your unborn baby and may cause loss of pregnancy (miscarriage). Tell your healthcare provider right away if you become pregnant or think you may be pregnant during treatment with RYTELO.

Females who are able to become pregnant:

  • Your healthcare provider will perform a pregnancy test before you are given RYTELO.
  • You should use effective birth control (contraception) during treatment with RYTELO and for 1 week after your last dose.
  • are breastfeeding or plan to breastfeed. It is not known if RYTELO passes into your breastmilk. Do not breastfeed during treatment with RYTELO and for 1 week after your last dose.

Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

What is the most important information I should know about RYTELO?

RYTELO may cause serious side effects, including:

  • Low platelet counts (thrombocytopenia). Low platelet counts are common during treatment with RYTELO and can also be severe. Low platelet counts can increase your risk for bleeding. Your healthcare provider may give you platelet transfusions to reduce the risk of bleeding if you develop a low platelet count during treatment with RYTELO. Tell your healthcare provider right away if you develop any signs or symptoms of bleeding, including:
    • unusual bleeding or bruising
    • bleeding that lasts a long time
    • nosebleeds
    • vomiting blood
    • blood in your stool or black tarry stool
  • Low neutrophil counts (neutropenia). Low counts of a type of white blood cell called neutrophils are common during treatment with RYTELO and can also be severe. Low neutrophil counts can increase your risk for infections, including serious infections and sepsis. Your healthcare provider may give you medicines before you start treatment to help prevent neutropenia and infections and may treat you with medicines if you develop these problems during treatment with RYTELO. Tell your healthcare provider right away if you develop any signs or symptoms of infection during treatment with RYTELO, including:
    • fever
    • shortness of breath or trouble breathing
    • cough
    • chills
    • pain or burning when you urinate

Your healthcare provider will do blood tests to check your platelet and neutrophil counts before starting treatment with RYTELO, weekly for the first 2 cycles of treatment, before you receive each additional cycle, and as needed during your treatment.

Your healthcare provider may delay your next treatment, decrease your dose, or stop treatment with RYTELO if you develop thrombocytopenia or neutropenia during treatment.

What are the possible side effects of RYTELO?

RYTELO may cause serious side effects, including:

  • See “What is the most important information I should know about RYTELO?”
  • Infusion-related reactions. RYTELO can cause infusion-related reactions during or after your infusion that can be severe, including a severe sudden increase in blood pressure called hypertensive crisis. Your healthcare provider will give you medicines before each RYTELO infusion to help prevent or lessen infusion-related reactions and will watch you for at least 1 hour after your infusion. If you develop infusion-related reactions, your healthcare provider may infuse RYTELO more slowly, temporarily stop, or permanently stop your treatment. Tell your healthcare provider if you develop any signs or symptoms of infusion-related reactions, including:
    • stomach pain
    • joint pain
    • weakness and tiredness
    • back and bone pain
    • diarrhea
    • redness
    • headache
    • high blood pressure
    • not feeling well
    • chest pain that is not related to your heart
    • itching
    • hives

The most common side effects of RYTELO include:

  • decreased platelet counts
  • decreased white blood cell counts
  • decreased neutrophil counts
  • increased liver enzymes (AST, alkaline phosphatase, and ALT)
  • tiredness
  • longer than usual blood clotting times
  • joint, bone and muscle pain
  • Covid-19 infections
  • headache

These are not all of the possible side effects of RYTELO. Call your doctor for more information and medical advice about side effects. 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 RYTELO (imetelstat) full Prescribing Information, including Medication Guide.