For US adults only
These are not all the symptoms of thalassemia.
-Andreas, taking AQVESME
People with thalassemia are at risk for serious health issues—regardless of whether they require transfusions. Some possible serious complications include:
Several things that happen in thalassemia can lead to a dangerous blood clot that lodges in a blood vessel, where it can cause a blockage in the heart or a stroke in the brain. For people who do not receive regular transfusions, this can be more common.
Most blood cells are made in the bone marrow (the spongy material inside bones). Thalassemia causes an increased demand for red blood cells, which causes your bones to widen. This can make the bones thin and brittle, increasing the risk of broken bones.
Organ damage is a thalassemia complication that can put your health at serious risk and should be monitored and managed. Some organs that can be affected are the liver and heart.
People with thalassemia may have too much iron. This can be due to the disease or blood transfusions. This means a lot of iron is moving around in the blood.
When too much iron builds up, it collects in places like the heart, liver, and other organs. This can make it hard for these organs to work the way they should, leading to complications.
Hormones are chemicals made in the body. They affect almost every organ and how the organ works. Thalassemia can cause abnormal amounts of certain hormones. This can be associated with several issues, such as growth delays, delayed puberty, thyroid problems, and diabetes.
This can happen in all types of thalassemia when red blood cells are not made properly in the bone marrow. This can lead to some organs getting too big, such as the spleen and liver.
High blood pressure in the lungs is called pulmonary hypertension. It can be caused by the breakdown of red blood cells (hemolysis), as well as iron overload.
Thalassemia results in chronic anemia that can last throughout life. Even if you don't experience a lot of symptoms, chronic anemia can lead to serious complications, including organ damage, and can be life-threatening if not properly monitored and managed.
Each 1 g/dL increase in hemoglobin is independently associated with a decreased risk of complications
*Study design: Data was analyzed from 150 people with beta-NTD thalassemia across 2 chronic care centers in Lebanon and Italy (published 2021). Starting hemoglobin levels (over an average of 6 months) were collected and the development of new complications (like heart, liver and hormone-related) were tracked over a 10 year period.
The risk of certain clinical complications can increase, despite regular transfusions
A recent study showed the risk of certain complications can increase with transfusion burden. This study looked at complications reported in medical claims for people receiving an average of 12 or more red blood cell (RBC) units over a 12-week period compared with those receiving none. Those who received 12 or more RBC units in 12 weeks experienced higher rates of complications.
Heart-related
complications*
▲ 2.9x
Risk of
liver disease*
▲ 1.9x
Complications associated
with hormone production*
▲ 3.2x
*Study design: These estimates come from a study in Taiwan that looked at the medical records of 2984 people with beta-thalassemia from 1/1/01 to 12/31/16. The study compared 2 groups of people to see how many people developed heart, liver, and hormone (endocrine) complications throughout the study period. It looked at people who received a high number of blood transfusions (averaging 12 or more RBC units every 12 weeks) and compared them directly to people who received no blood transfusions at all.
NTD=non–transfusion dependent; TD=transfusion dependent.
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:
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:
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:
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.
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.
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:
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:
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:
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.
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.