What causes thalassemia?

What happens in thalassemia?

The bone marrow creates the building blocks that develop into new red blood cells (RBCs). In people who don’t have thalassemia, developing RBCs mature into healthy RBCs that carry oxygen throughout the body.

In people with thalassemia, the RBCs aren’t made well:

Image showing thalassemic red blood cells breaking down early, before leaving the bone marrow

They don't develop properly

Some developing RBCs break down before they leave the bone marrow. This means they can’t deliver oxygen to the body.

Image showing thalassemic RBCs in the blood stream breaking down early

They break down early

Some RBCs that leave the bone marrow and fully develop break down early. This means they can deliver oxygen, but not for as long as healthy RBCs.

These thalassemic RBCs don't have enough energy to withstand damage, and don't develop properly and break down early

Image showing single thalassemic RBC breaking down early

Without enough energy to withstand damage, fragile RBCs have a shorter lifespan and can’t deliver as much oxygen throughout the body.

In people with thalassemia, RBCs live for about 17-33 days, while healthy RBCs live for about 120 days.

This results in chronic anemia and can be associated with complications over time.

Thalassemia can have many different symptoms

LEARN ABOUT SYMPTOMS

Learn about an anemia treatment for people with thalassemia

DISCOVER THE SCIENCE

IMPORTANT SAFETY INFORMATION

AQVESME may cause serious side effects, including:

  • Liver injury. AQVESME can cause serious liver injury. Liver injury has happened in people with thalassemia within the first 6 months of treatment with AQVESME. Your healthcare provider will do blood tests to check your liver before you start treatment with AQVESME, every 4 weeks for the first 24 weeks of treatment, and as needed. Your healthcare provider may temporarily or permanently stop your treatment with AQVESME if you have abnormal liver blood tests.

    Tell your healthcare provider right away if you develop any new or worsening signs or symptoms of liver problems, including:

    • loss of appetite
    • nausea
    • pain in the upper right side of your stomach area
    • vomiting
    • yellowing of the skin and white part of your eyes (jaundice)
    • dark-colored urine

In clinical studies of AQVESME, 2 of 301 people (0.66%) treated with AQVESME experienced adverse reactions suggestive of liver injury. Three additional people experienced adverse reactions suggestive of liver injury during the open-label extension periods, after switching from placebo to AQVESME. Of the 5 people, two had serious liver injury requiring hospitalization, including 1 who developed jaundice. Another developed jaundice without requiring hospitalization. All 5 people discontinued treatment with AQVESME, and these reactions improved upon treatment discontinuation.

Because of the risk for liver injury, AQVESME is only available through a restricted access program called the AQVESME Risk Evaluation and Mitigation Strategy (REMS).

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

  • have liver problems, such as cirrhosis
  • are pregnant or plan to become pregnant. It is not known if AQVESME will harm your unborn baby. Tell your healthcare provider right away if you become pregnant or think that you are pregnant during treatment with AQVESME.
  • are breastfeeding or plan to breastfeed. It is not known if AQVESME passes into your breast milk. Talk to your healthcare provider about the best way to feed your baby during treatment with AQVESME.

Tell your healthcare provider about all the medicines that you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Make sure to tell your healthcare provider if you take or use hormonal birth control (contraceptives). If you take or use hormonal birth control (except for intrauterine systems containing levonorgestrel), it may not work as well during treatment with AQVESME. Use a different type of birth control or use an additional nonhormonal birth control method (such as condoms) during treatment with AQVESME and for 28 days after stopping treatment with AQVESME. AQVESME and certain other medicines may affect each other and cause side effects. AQVESME may affect the way other medicines work, and other medicines may affect the way AQVESME works.

The most common side effects of AQVESME in patients:

  • headache
  • insomnia

These are not all of the possible side effects of AQVESME. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

What is AQVESME?

AQVESME is a prescription medicine used to treat anemia (low red blood cells) in adults with alpha- or beta-thalassemia. It is not known if AQVESME is safe and effective in children.

Please see full Prescribing Information, including Medication Guide.

IMPORTANT SAFETY INFORMATION

AQVESME may cause serious side effects, including:

  • Liver injury. AQVESME can cause serious liver injury. Liver injury has happened in people with thalassemia within the first 6 months of treatment with AQVESME. Your healthcare provider will do blood tests to check your liver before you start treatment with AQVESME, every 4 weeks for the first 24 weeks of treatment, and as needed. Your healthcare provider may temporarily or permanently stop your treatment with AQVESME if you have abnormal liver blood tests.

    Tell your healthcare provider right away if you develop any new or worsening signs or symptoms of liver problems, including:

    • loss of appetite
    • nausea
    • pain in the upper right side of your stomach area
    • vomiting
    • yellowing of the skin and white part of your eyes (jaundice)
    • dark-colored urine

In clinical studies of AQVESME, 2 of 301 people (0.66%) treated with AQVESME experienced adverse reactions suggestive of liver injury. Three additional people experienced adverse reactions suggestive of liver injury during the open-label extension periods, after switching from placebo to AQVESME. Of the 5 people, two had serious liver injury requiring hospitalization, including 1 who developed jaundice. Another developed jaundice without requiring hospitalization. All 5 people discontinued treatment with AQVESME, and these reactions improved upon treatment discontinuation.

Because of the risk for liver injury, AQVESME is only available through a restricted access program called the AQVESME Risk Evaluation and Mitigation Strategy (REMS).

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

  • have liver problems, such as cirrhosis
  • are pregnant or plan to become pregnant. It is not known if AQVESME will harm your unborn baby. Tell your healthcare provider right away if you become pregnant or think that you are pregnant during treatment with AQVESME.
  • are breastfeeding or plan to breastfeed. It is not known if AQVESME passes into your breast milk. Talk to your healthcare provider about the best way to feed your baby during treatment with AQVESME.

Tell your healthcare provider about all the medicines that you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Make sure to tell your healthcare provider if you take or use hormonal birth control (contraceptives). If you take or use hormonal birth control (except for intrauterine systems containing levonorgestrel), it may not work as well during treatment with AQVESME. Use a different type of birth control or use an additional nonhormonal birth control method (such as condoms) during treatment with AQVESME and for 28 days after stopping treatment with AQVESME. AQVESME and certain other medicines may affect each other and cause side effects. AQVESME may affect the way other medicines work, and other medicines may affect the way AQVESME works.

The most common side effects of AQVESME in patients:

  • headache
  • insomnia

These are not all of the possible side effects of AQVESME. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

What is AQVESME?

AQVESME is a prescription medicine used to treat anemia (low red blood cells) in adults with alpha- or beta-thalassemia. It is not known if AQVESME is safe and effective in children.

Please see full Prescribing Information, including Medication Guide.